A GBT021601 ADME Microtracer Study in Healthy Volunteers

A Phase 1 study testing GBT021601 in people with Sickle Cell Disease, run by Pfizer at 1 site in Netherlands.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial summary

An Open-label Study of GBT021601 in 8 to 10 healthy male or female participants to evaluate the absorption, distribution, metabolism, and excretion (ADME) of GBT021601. Read more about this study

Who is it for?: Ages 18 to 55 yearsHealthy volunteers can take part
Conditions the study is for: Sickle Cell Disease
What is being tested?: GBT021601
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 9 participants
Where: 1 siteNetherlands
Study dates: 22 Dec 2022to 10 Aug 2023
Phase: Phase 1
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 55 years
All sexes
Healthy volunteers accepted
Adult

Who may be able to join

Body mass index (BMI): 18.0 to 27.0 kg/m2, inclusive, at screening.
Body weight ≥ 50 kg at screening
Females must be nonlactating and nonpregnant (as confirmed by a negative serum pregnancy test at screening and admission for all females), or of nonchildbearing potential (ie, either surgically sterilized or physiologically incapable of becoming pregnant, or at least 1 year postmenopausal [defined as at least 12 months no menses, and confirmed by a follicle-stimulating hormone test, at screening]).
Creatinine clearance (glomerular filtration rate) as estimated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula: ≥90 mL/min, at screening.

Who cannot join

History or presence of conditions which, in the opinion of the Investigator, are known to interfere with the ADME of drugs, such as previous surgery on the gastrointestinal tract (including removal of parts of the stomach, bowel, liver, gall bladder, or pancreas). Participants who have a history of appendectomy are eligible for enrollment.
History of chronic constipation, or recent complaints of an irregular defecation
Significant and/or acute illness at screening or within 5 days prior to study drug administration that may impact safety assessments, in the opinion of the Investigator.
Known personal or family history of congenital long QT syndrome or known family history of sudden death.
Participation in another ADME study with a radiation burden >0.1 mSv in the period of 1 year prior to screening.

Trial locations and contacts

1 site in 1 country.

Netherlands1 site

ICON Early Phase Clinic, LLC
Groningen, 9728

Other trials in Netherlands

About this study

Official title: A PHASE 1 STUDY TO ASSESS THE MASS BALANCE, EXCRETION, AND PHARMACOKINETICS OF [14C]-GBT021601, AN ORAL HEMOGLOBIN SPOLYMERIZATION INHIBITOR, IN HEALTHY PARTICIPANTS

An Open-label Study of GBT021601 in 8 to 10 healthy male or female participants to evaluate the absorption, distribution, metabolism, and excretion (ADME) of GBT021601.

Read the detailed description

This is an open-label Study administering GBT021601 as a single oral dose of 200 mg in healthy participants.

Conditions: Sickle Cell Disease
Being tested: GBT021601
Medical subject headings: Anemia, Sickle Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

GBT021601Drug
Single oral dose of 200 mg GBT021601, containing ~74 kBq (~2 µCi) of [14C]-GBT021601

Study groups in NCT05718687
Study group	Role	What they receive
Treatment	Experimental	open-label GBT021601 Receives: Drug: GBT021601

What is being measured

The primary outcome is the main question the study is designed to answer.

GBT021601 Concentration in Whole Blood and Plasma at Predose
Measured: Predose (within 60 minutes prior to GBT021601 dosing)
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at Predose
Data reported in microgram equivalent per milliliter (mcgEq/mL).
Measured: Predose (within 60 minutes prior to 14C-GBT021601 dosing)
GBT021601 Concentration in Whole Blood and Plasma at 0.25 Hours
Measured: 0.25 hours post dosing on Day 1
14C-GBT021601 Total Radioactivity Concentrations in Whole Blood and Plasma at 0.25 Hours
Measured: 0.25 hours post dosing on Day 1
GBT021601 Concentration in Whole Blood and Plasma at 0.50 Hours
Measured: 0.50 hours post dosing on Day 1
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 0.50 Hours
Measured: 0.50 hours post dosing on Day 1
GBT021601 Concentration in Whole Blood and Plasma at 1 Hours
Measured: 1 hours post dosing on Day 1
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 1 Hours
Measured: 1 hours post dosing on Day 1
GBT021601 Concentration in Whole Blood and Plasma at 2 Hours
Measured: 2 hours post dosing on Day 1
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 2 Hours
Measured: 2 hours post dosing on Day 1
GBT021601 Concentration in Whole Blood and Plasma at 3 Hours
Measured: 3 hours post dosing on Day 1
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 3 Hours
Measured: 3 hours post dosing on Day 1
GBT021601 Concentration in Whole Blood and Plasma at 4 Hours
Measured: 4 hours post dosing on Day 1
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 4 Hours
Measured: 4 hours post dosing on Day 1
GBT021601 Concentration in Whole Blood and Plasma at 6 Hours
Measured: 6 hours post dosing on Day 1
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 6 Hours
Measured: 6 hours post dosing on Day 1
GBT021601 Concentration in Whole Blood and Plasma at 8 Hours
Measured: 8 hours post dosing on Day 1
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 8 Hours
Measured: 8 hours post dosing on Day 1
GBT021601 Concentration in Whole Blood and Plasma at 12 Hours
Measured: 12 hours post dosing on Day 1
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 12 Hours
Measured: 12 hours post dosing on Day 1
GBT021601 Concentration in Whole Blood and Plasma at 24 Hours
Measured: 24 hours post dosing on Day 1
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 24 Hours
Measured: 24 hours post dosing on Day 1
GBT021601 Concentration in Whole Blood and Plasma at 48 Hours
Measured: 48 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 48 Hours
Measured: 48 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 72 Hours
Measured: 72 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 72 Hours
Measured: 72 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 96 Hours
Measured: 96 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 96 Hours
Measured: 96 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 144 Hours
Measured: 144 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 144 Hours
Measured: 144 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 168 Hours
Measured: 168 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 168 Hours
Measured: 168 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 312 Hours
Measured: 312 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 312 Hours
Measured: 312 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 480 Hours
Measured: 480 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 480 Hours
Measured: 480 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 648 Hours
Measured: 648 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 648 Hours
Measured: 648 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 816 Hours
Measured: 816 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 816 Hours
Measured: 816 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 984 Hours
Measured: 984 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 984 Hours
Measured: 984 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 1320 Hours
Measured: 1320 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 1320 Hours
Measured: 1320 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 1656 Hours
Measured: 1656 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 1656 Hours
Measured: 1656 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 1992 Hours
Measured: 1992 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 1992 Hours
Measured: 1992 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 2328 Hours
Measured: 2328 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 2328 Hours
Measured: 2328 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 2664 Hours
Measured: 2664 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 2664 Hours
Measured: 2664 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 3240 Hours
Measured: 3240 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 3240 Hours
Measured: 3240 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 3576 Hours
Measured: 3576 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 3576 Hours
Measured: 3576 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 4248 Hours
Measured: 4248 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 4248 Hours
Measured: 4248 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 4920 Hours
Measured: 4920 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 4920 Hours
Measured: 4920 hours post dosing
Cumulative Amount of [14C]-GBT021601 Total Radioactivity Excreted in Urine and Feces
Data are reported as aggregate of all scheduled time points.
Measured: Day 1 (prior to dosing), after dosing from 0-6 hours, 6-12 hrs, 12-24 hrs postdose for urine, 24-hr intervals up to Day 29; 816 hrs , 984 hrs, 1320 hrs, 1656 hrs, 1992 hrs, 2328 hrs, 2664 hrs, 3240 hrs, 3576 hrs, 4248 hrs, and 4920 hrs post-dose
Cumulative Fraction of [14C]-GBT021601 Total Radioactivity Excreted in Urine and Feces
Data are reported as aggregate of all scheduled time points.
Measured: Day 1 (prior to dosing), after dosing from 0-6 hours, 6-12 hrs, 12-24 hrs postdose for urine, 24-hr intervals up to Day 29; 816 hrs , 984 hrs, 1320 hrs, 1656 hrs, 1992 hrs, 2328 hrs, 2664 hrs, 3240 hrs, 3576 hrs, 4248 hrs, and 4920 hrs post-dose
Area Under the Plasma Concentration-Time Curve From Time Zero (0) to 24 Hours (AUC0-24) of GBT021601 in Whole Blood and Plasma
Area under the plasma concentration-time curve up to 24 hours post dose. Data reported in Hour*microgram/millimeter (h*mcg/mL).
Measured: Day 1 (prior to dosing) at 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24 hours
Area Under the Plasma Concentration-Time Curve From Time Zero (0) to 24 Hours (AUC0-24) of 14C-GBT021601 in Whole Blood and Plasma
Area under the plasma concentration-time curve up to 24 hours postdose. Data reported in hour*microgram equivalent/milliliter (h*mcgEq/mL).
Measured: Day 1 (prior to dosing) at 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24 hours
Area Under the Concentration-Time From Time Zero to the Last Measurable Concentration (AUC0-last) of GBT021601 in Whole Blood and Plasma
Measured: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Area Under the Concentration-Time From Time Zero to the Last Measurable Concentration (AUC0-last) of 14C-GBT021601 in Whole Blood and Plasma
Measured: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Area Under the Plasma Concentration-Time Curve From Time Zero to Infinity (AUC0-inf) of GBT021601 in Whole Blood and Plasma
Area under the plasma concentration-time curve from time 0 to infinity calculated as: AUC0-inf=AUC0-t+Clast/kel, where Clast is the last measurable plasma concentration.
Measured: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Area Under the Plasma Concentration-Time Curve From Time Zero to Infinity (AUC0-inf) of 14C-GBT021601 in Whole Blood and Plasma
Area under the plasma concentration-time curve from time 0 to infinity calculated as: AUC0-inf=AUC0-t+Clast/kel, where Clast is the last measurable plasma concentration and Kel is apparent terminal elimination rate constant.
Measured: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Maximum Observed Concentration (Cmax) of GBT021601 in Whole Blood and Plasma
Measured: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Maximum Observed Concentration (Cmax) of 14C-GBT021601 in Whole Blood and Plasma
Measured: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Time to Attain Maximum Observed Concentration (Tmax) of GBT021601 in Whole Blood and Plasma
Measured: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Time to Attain Maximum Observed Concentration (Tmax) of 14C-GBT021601 in Whole Blood and Plasma
Measured: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Apparent Terminal Elimination Half-Life (t1/2) of GBT021601 in Whole Blood and Plasma
Apparent terminal elimination half-life, calculated as 0.693/kel where kel is Apparent terminal elimination rate constant.
Measured: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Apparent Terminal Elimination Half-Life (t1/2) of 14C-GBT021601 in Whole Blood and Plasma
Apparent terminal elimination half-life, calculated as 0.693/kel where kel is Apparent terminal elimination rate constant.
Measured: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Renal Clearance (CLR) of GBT021601 and 14C-GBT021601 in Urine
Renal clearance was calculated as CLR = Ae0-inf/AUC0-inf. Where Ae indicates Cumulative amount of drug excreted and AUC0-inf indicates Area under the plasma concentration-time curve from time 0 to infinity.
Measured: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Apparent Volume of Distribution at Terminal Phase (Vz/F) of GBT021601 in Whole Blood and Plasma
Apparent volume of distribution at terminal phase, calculated as: (CL/F) Apparent total clearance, calculated as dose/AUC0-inf/Kel.
Measured: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Apparent Volume of Distribution at Terminal Phase (Vz/F) of 14C-GBT021601 in Whole Blood and Plasma
Apparent volume of distribution at terminal phase, calculated as: (CL/F) Apparent total clearance, calculated as dose/AUC0-inf/Kel.
Measured: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Apparent Total Clearance (CL/F) of GBT021601 in Whole Blood and Plasma
Apparent total clearance, calculated as dose/AUC0-inf.
Measured: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Apparent Total Clearance (CL/F) of 14C-GBT021601 in Whole Blood and Plasma
Apparent total clearance, calculated as dose/AUC0-inf.
Measured: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 0-6 Hours
Measured: 0-6 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 6-12 Hours
Measured: 6-12 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 12-24 Hours
Measured: 12-24 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 0-24 Hours
Measured: 0-24 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 24 Hours
Measured: 24 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 48 Hours
Measured: 48 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 72 Hours
Measured: 72 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 96 Hours
Measured: 96 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 120 Hours
Measured: 120 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 144 Hours
Measured: 144 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 168 Hours
Measured: 168 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 192 Hours
Measured: 192 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 216 Hours
Measured: 216 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 240 Hours
Measured: 240 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 264 Hours
Measured: 264 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 288 Hours
Measured: 288 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 312 Hours
Measured: 312 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 336 Hours
Measured: 336 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 360 Hours
Measured: 360 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 384 Hours
Measured: 384 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 408 Hours
Measured: 408 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 432 Hours
Measured: 432 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 456 Hours
Measured: 456 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 480 Hours
Measured: 480 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 504 Hours
Measured: 504 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 528 Hours
Measured: 528 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 552 Hours
Measured: 552 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 576 Hours
Measured: 576 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 600 Hours
Measured: 600 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 624 Hours
Measured: 624 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 648 Hours
Measured: 648 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 816 Hours
Measured: 816 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 984 Hours
Measured: 984 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 1320 Hours
Measured: 1320 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 1656 Hours
Measured: 1656 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 1992 Hours
Measured: 1992 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 2328 Hours
Measured: 2328 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 2664 Hours
Measured: 2664 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 3240 Hours
Measured: 3240 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 3576 Hours
Measured: 3576 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 4248 Hours
Measured: 4248 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 4920 Hours
Measured: 4920 hours post dose

Secondary outcomes (5)

Number of Participants With Treatment Emergent Adverse Events (TEAEs)
An Adverse event (AE) was any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medical treatment or procedure that may or may not be considered related to the medical treatment or procedure. TEAEs were those which occurred (or worsened) after the dose of study drug during the on-treatment period, defined as the period after the dose of study drug and through the minimum of (Study Day 57, study discontinuation).
Measured: From Day 1 up to maximum of Day 57
Number of Participants With Clinically Significant Laboratory Values
Blood and urine samples was collected for clinical laboratory assessments. Clinical Laboratory Values included Clinical chemistry: alanine aminotransferase, albumin, alkaline phosphatase, aspartate aminotransferase, bicarbonate, bilirubin (total, direct, and indirect), blood urea nitrogen, calcium, chloride, creatinine, gamma glutamyl transferase, glucose, lactate dehydrogenase, magnesium, phosphorus, potassium, sodium, total protein, and uric acid. Hematology: leukocytes, erythrocytes, hematocrit, Hb, mean corpuscular volume, mean corpuscular hemoglobin, mean corpuscular hemoglobin concentration, platelet count (ie, thrombocytes), RBC count, and white blood cell count etc. Urinalysis: determination of pH and specific gravity, and presence of bilirubin, blood, glucose, ketones, nitrite, protein, leukocytes, and urobilinogen etc. Coagulation, Serology, Drug and alcohol screen and Pregnancy test. Clinical significance was judged by investigator.
Measured: Day 1 of dosing to Day 207
Number of Participants With Clinically Significant Vital Signs Values
Systolic and diastolic blood pressure and pulse was recorded after the participant had rested for at least 5 minutes in the supine position. Body temperature and respiratory rate was also measured. Clinical significance was judged by investigator.
Measured: Day 1 of dosing to Day 207
Number of Participants With Clinically Significant ECG Values
Standard 12-lead ECGs were performed after the participant had rested quietly for more than 5 minutes in a supine position utilizing an ECG machine that was equipped with computer-based interval measurements. The following measurement was recorded: the heart rate (of <45 or >100 beats per minute (BPM) , PR interval, QT interval, QT interval corrected using Fridericia's formula (QTcF) of >450 msec for males and >470 msec for females, and QRS duration. Any cardiac rhythm other than sinus rhythm that was interpreted by the Investigator was clinically significant.
Measured: Day 1
Number of Participants With Clinically Significant Physical Examination Values
Physical Examination test included body weight and height. physical examination was conducted at screening. Clinical significance was judged by investigator.
Measured: Screening (-28 Day to -2 Day, day with respect to dosing)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

22 Dec 202210 Aug 2023

First posted8 Feb 2023
Study start22 Dec 2022
Primary completion10 Aug 2023
Study completion10 Aug 2023

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
More trials run by PfizerThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1
Primary purpose: Other
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 9 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05718687
Sponsor's protocol ID: GBT021601-013
Other IDs: C5351003, 2022-003108-34
Status last verified: Nov 2024
First submitted: 30 Jan 2023
Last updated: 8 Jan 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.

Publications

Zimmerman EI, Callegari E, Sharma R, Adenola A, Volpe J, Seetharaman K, Zhang C, Duchin K, Walker GS. A Phase 1 Microtracer Study Evaluating the Mass Balance, Excretion, and Pharmacokinetics of Osivelotor in Healthy Participants. Clin Transl Sci. 2026 Jun;19(6):e70601. doi: 10.1111/cts.70601.
PubMed 42183775

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The sponsor has not listed a contact. The official record may have more details.

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Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
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Will it cost me anything? Are travel or time paid for?
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All Sickle Cell Disease trials

Study at a glance

From the registry record, last updated by the sponsor on 8 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A PHASE 1 STUDY TO ASSESS THE MASS BALANCE, EXCRETION, AND PHARMACOKINETICS OF [14C]-GBT021601, AN ORAL HEMOGLOBIN SPOLYMERIZATION INHIBITOR, IN HEALTHY PARTICIPANTS
ClinicalTrials.gov ID: NCT05718687
Acronym / sponsor ID: GBT021601-013
Conditions: Sickle Cell Disease
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 9 participants (actual)
Lead sponsor: PfizerIndustry (pharma, biotech, devices)
Study start: 22 Dec 2022 (actual)
Primary completion: 10 Aug 2023 (actual)
Study completion: 10 Aug 2023 (actual)
First posted: 8 Feb 2023 (actual)
Results: Posted 8 Jan 2025

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

An Open-label Study of GBT021601 in 8 to 10 healthy male or female participants to evaluate the absorption, distribution, metabolism, and excretion (ADME) of GBT021601.

Detailed description

This is an open-label Study administering GBT021601 as a single oral dose of 200 mg in healthy participants.

Study design

Primary purpose: Other
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Treatment	Experimental	Drug: GBT021601open-label GBT021601

Who may qualify

Ages: 18 to 55 years
Sex: All
Healthy volunteers: Accepted
Age groups: Adult

Key inclusion criteria (4 listed)

Body mass index (BMI): 18.0 to 27.0 kg/m2, inclusive, at screening.
Body weight ≥ 50 kg at screening
Females must be nonlactating and nonpregnant (as confirmed by a negative serum pregnancy test at screening and admission for all females), or of nonchildbearing potential (ie, either surgically sterilized or physiologically incapable of becoming pregnant, or at least 1 year postmenopausal [defined as at least 12 months no menses, and confirmed by a follicle-stimulating hormone test, at screening]).
Creatinine clearance (glomerular filtration rate) as estimated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula: ≥90 mL/min, at screening.

Key exclusion criteria (5 listed)

History or presence of conditions which, in the opinion of the Investigator, are known to interfere with the ADME of drugs, such as previous surgery on the gastrointestinal tract (including removal of parts of the stomach, bowel, liver, gall bladder, or pancreas). Participants who have a history of appendectomy are eligible for enrollment.
History of chronic constipation, or recent complaints of an irregular defecation
Significant and/or acute illness at screening or within 5 days prior to study drug administration that may impact safety assessments, in the opinion of the Investigator.
Known personal or family history of congenital long QT syndrome or known family history of sudden death.
Participation in another ADME study with a radiation burden >0.1 mSv in the period of 1 year prior to screening.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
GBT021601Single oral dose of 200 mg GBT021601, containing ~74 kBq (~2 µCi) of [14C]-GBT021601	Drug	Treatment

Primary outcome

GBT021601 Concentration in Whole Blood and Plasma at Predose
Time frame: Predose (within 60 minutes prior to GBT021601 dosing)
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at Predose
Time frame: Predose (within 60 minutes prior to 14C-GBT021601 dosing)
Data reported in microgram equivalent per milliliter (mcgEq/mL).
GBT021601 Concentration in Whole Blood and Plasma at 0.25 Hours
Time frame: 0.25 hours post dosing on Day 1
14C-GBT021601 Total Radioactivity Concentrations in Whole Blood and Plasma at 0.25 Hours
Time frame: 0.25 hours post dosing on Day 1
GBT021601 Concentration in Whole Blood and Plasma at 0.50 Hours
Time frame: 0.50 hours post dosing on Day 1
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 0.50 Hours
Time frame: 0.50 hours post dosing on Day 1
GBT021601 Concentration in Whole Blood and Plasma at 1 Hours
Time frame: 1 hours post dosing on Day 1
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 1 Hours
Time frame: 1 hours post dosing on Day 1
GBT021601 Concentration in Whole Blood and Plasma at 2 Hours
Time frame: 2 hours post dosing on Day 1
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 2 Hours
Time frame: 2 hours post dosing on Day 1
GBT021601 Concentration in Whole Blood and Plasma at 3 Hours
Time frame: 3 hours post dosing on Day 1
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 3 Hours
Time frame: 3 hours post dosing on Day 1
GBT021601 Concentration in Whole Blood and Plasma at 4 Hours
Time frame: 4 hours post dosing on Day 1
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 4 Hours
Time frame: 4 hours post dosing on Day 1
GBT021601 Concentration in Whole Blood and Plasma at 6 Hours
Time frame: 6 hours post dosing on Day 1
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 6 Hours
Time frame: 6 hours post dosing on Day 1
GBT021601 Concentration in Whole Blood and Plasma at 8 Hours
Time frame: 8 hours post dosing on Day 1
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 8 Hours
Time frame: 8 hours post dosing on Day 1
GBT021601 Concentration in Whole Blood and Plasma at 12 Hours
Time frame: 12 hours post dosing on Day 1
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 12 Hours
Time frame: 12 hours post dosing on Day 1
GBT021601 Concentration in Whole Blood and Plasma at 24 Hours
Time frame: 24 hours post dosing on Day 1
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 24 Hours
Time frame: 24 hours post dosing on Day 1
GBT021601 Concentration in Whole Blood and Plasma at 48 Hours
Time frame: 48 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 48 Hours
Time frame: 48 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 72 Hours
Time frame: 72 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 72 Hours
Time frame: 72 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 96 Hours
Time frame: 96 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 96 Hours
Time frame: 96 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 144 Hours
Time frame: 144 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 144 Hours
Time frame: 144 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 168 Hours
Time frame: 168 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 168 Hours
Time frame: 168 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 312 Hours
Time frame: 312 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 312 Hours
Time frame: 312 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 480 Hours
Time frame: 480 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 480 Hours
Time frame: 480 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 648 Hours
Time frame: 648 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 648 Hours
Time frame: 648 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 816 Hours
Time frame: 816 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 816 Hours
Time frame: 816 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 984 Hours
Time frame: 984 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 984 Hours
Time frame: 984 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 1320 Hours
Time frame: 1320 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 1320 Hours
Time frame: 1320 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 1656 Hours
Time frame: 1656 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 1656 Hours
Time frame: 1656 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 1992 Hours
Time frame: 1992 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 1992 Hours
Time frame: 1992 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 2328 Hours
Time frame: 2328 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 2328 Hours
Time frame: 2328 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 2664 Hours
Time frame: 2664 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 2664 Hours
Time frame: 2664 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 3240 Hours
Time frame: 3240 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 3240 Hours
Time frame: 3240 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 3576 Hours
Time frame: 3576 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 3576 Hours
Time frame: 3576 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 4248 Hours
Time frame: 4248 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 4248 Hours
Time frame: 4248 hours post dosing
GBT021601 Concentration in Whole Blood and Plasma at 4920 Hours
Time frame: 4920 hours post dosing
14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at 4920 Hours
Time frame: 4920 hours post dosing
Cumulative Amount of [14C]-GBT021601 Total Radioactivity Excreted in Urine and Feces
Time frame: Day 1 (prior to dosing), after dosing from 0-6 hours, 6-12 hrs, 12-24 hrs postdose for urine, 24-hr intervals up to Day 29; 816 hrs , 984 hrs, 1320 hrs, 1656 hrs, 1992 hrs, 2328 hrs, 2664 hrs, 3240 hrs, 3576 hrs, 4248 hrs, and 4920 hrs post-dose
Data are reported as aggregate of all scheduled time points.
Cumulative Fraction of [14C]-GBT021601 Total Radioactivity Excreted in Urine and Feces
Time frame: Day 1 (prior to dosing), after dosing from 0-6 hours, 6-12 hrs, 12-24 hrs postdose for urine, 24-hr intervals up to Day 29; 816 hrs , 984 hrs, 1320 hrs, 1656 hrs, 1992 hrs, 2328 hrs, 2664 hrs, 3240 hrs, 3576 hrs, 4248 hrs, and 4920 hrs post-dose
Data are reported as aggregate of all scheduled time points.
Area Under the Plasma Concentration-Time Curve From Time Zero (0) to 24 Hours (AUC0-24) of GBT021601 in Whole Blood and Plasma
Time frame: Day 1 (prior to dosing) at 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24 hours
Area under the plasma concentration-time curve up to 24 hours post dose. Data reported in Hour*microgram/millimeter (h*mcg/mL).
Area Under the Plasma Concentration-Time Curve From Time Zero (0) to 24 Hours (AUC0-24) of 14C-GBT021601 in Whole Blood and Plasma
Time frame: Day 1 (prior to dosing) at 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24 hours
Area under the plasma concentration-time curve up to 24 hours postdose. Data reported in hour*microgram equivalent/milliliter (h*mcgEq/mL).
Area Under the Concentration-Time From Time Zero to the Last Measurable Concentration (AUC0-last) of GBT021601 in Whole Blood and Plasma
Time frame: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Area Under the Concentration-Time From Time Zero to the Last Measurable Concentration (AUC0-last) of 14C-GBT021601 in Whole Blood and Plasma
Time frame: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Area Under the Plasma Concentration-Time Curve From Time Zero to Infinity (AUC0-inf) of GBT021601 in Whole Blood and Plasma
Time frame: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Area under the plasma concentration-time curve from time 0 to infinity calculated as: AUC0-inf=AUC0-t+Clast/kel, where Clast is the last measurable plasma concentration.
Area Under the Plasma Concentration-Time Curve From Time Zero to Infinity (AUC0-inf) of 14C-GBT021601 in Whole Blood and Plasma
Time frame: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Area under the plasma concentration-time curve from time 0 to infinity calculated as: AUC0-inf=AUC0-t+Clast/kel, where Clast is the last measurable plasma concentration and Kel is apparent terminal elimination rate constant.
Maximum Observed Concentration (Cmax) of GBT021601 in Whole Blood and Plasma
Time frame: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Maximum Observed Concentration (Cmax) of 14C-GBT021601 in Whole Blood and Plasma
Time frame: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Time to Attain Maximum Observed Concentration (Tmax) of GBT021601 in Whole Blood and Plasma
Time frame: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Time to Attain Maximum Observed Concentration (Tmax) of 14C-GBT021601 in Whole Blood and Plasma
Time frame: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Apparent Terminal Elimination Half-Life (t1/2) of GBT021601 in Whole Blood and Plasma
Time frame: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Apparent terminal elimination half-life, calculated as 0.693/kel where kel is Apparent terminal elimination rate constant.
Apparent Terminal Elimination Half-Life (t1/2) of 14C-GBT021601 in Whole Blood and Plasma
Time frame: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Apparent terminal elimination half-life, calculated as 0.693/kel where kel is Apparent terminal elimination rate constant.
Renal Clearance (CLR) of GBT021601 and 14C-GBT021601 in Urine
Time frame: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Renal clearance was calculated as CLR = Ae0-inf/AUC0-inf. Where Ae indicates Cumulative amount of drug excreted and AUC0-inf indicates Area under the plasma concentration-time curve from time 0 to infinity.
Apparent Volume of Distribution at Terminal Phase (Vz/F) of GBT021601 in Whole Blood and Plasma
Time frame: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Apparent volume of distribution at terminal phase, calculated as: (CL/F) Apparent total clearance, calculated as dose/AUC0-inf/Kel.
Apparent Volume of Distribution at Terminal Phase (Vz/F) of 14C-GBT021601 in Whole Blood and Plasma
Time frame: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Apparent volume of distribution at terminal phase, calculated as: (CL/F) Apparent total clearance, calculated as dose/AUC0-inf/Kel.
Apparent Total Clearance (CL/F) of GBT021601 in Whole Blood and Plasma
Time frame: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Apparent total clearance, calculated as dose/AUC0-inf.
Apparent Total Clearance (CL/F) of 14C-GBT021601 in Whole Blood and Plasma
Time frame: Day 1 (prior to dosing), 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144,168, 312, 480, 648, 816, 984,1320,1656,1992, 2328, 2664, 3240, 3576, 4248 and 4920 hours post-dose
Apparent total clearance, calculated as dose/AUC0-inf.
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 0-6 Hours
Time frame: 0-6 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 6-12 Hours
Time frame: 6-12 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 12-24 Hours
Time frame: 12-24 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 0-24 Hours
Time frame: 0-24 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 24 Hours
Time frame: 24 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 48 Hours
Time frame: 48 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 72 Hours
Time frame: 72 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 96 Hours
Time frame: 96 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 120 Hours
Time frame: 120 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 144 Hours
Time frame: 144 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 168 Hours
Time frame: 168 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 192 Hours
Time frame: 192 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 216 Hours
Time frame: 216 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 240 Hours
Time frame: 240 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 264 Hours
Time frame: 264 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 288 Hours
Time frame: 288 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 312 Hours
Time frame: 312 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 336 Hours
Time frame: 336 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 360 Hours
Time frame: 360 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 384 Hours
Time frame: 384 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 408 Hours
Time frame: 408 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 432 Hours
Time frame: 432 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 456 Hours
Time frame: 456 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 480 Hours
Time frame: 480 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 504 Hours
Time frame: 504 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 528 Hours
Time frame: 528 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 552 Hours
Time frame: 552 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 576 Hours
Time frame: 576 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 600 Hours
Time frame: 600 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 624 Hours
Time frame: 624 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 648 Hours
Time frame: 648 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 816 Hours
Time frame: 816 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 984 Hours
Time frame: 984 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 1320 Hours
Time frame: 1320 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 1656 Hours
Time frame: 1656 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 1992 Hours
Time frame: 1992 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 2328 Hours
Time frame: 2328 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 2664 Hours
Time frame: 2664 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 3240 Hours
Time frame: 3240 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 3576 Hours
Time frame: 3576 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 4248 Hours
Time frame: 4248 hours post dose
Distribution of 14C- GBT021601 Total Radioactivity in Urine and Feces at 4920 Hours
Time frame: 4920 hours post dose

5 secondary outcomes

Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Time frame: From Day 1 up to maximum of Day 57
Number of Participants With Clinically Significant Laboratory Values
Time frame: Day 1 of dosing to Day 207
Number of Participants With Clinically Significant Vital Signs Values
Time frame: Day 1 of dosing to Day 207
Number of Participants With Clinically Significant ECG Values
Time frame: Day 1
Number of Participants With Clinically Significant Physical Examination Values
Time frame: Screening (-28 Day to -2 Day, day with respect to dosing)

Results and safety

Results posted to the registry on 8 Jan 2025, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
GBT021601+[14C]-GBT021601	9	6

Primary outcome: GBT021601 Concentration in Whole Blood and Plasma at Predose

Time frame: Predose (within 60 minutes prior to GBT021601 dosing). Mean (Standard Deviation). Unit: Microgram/ milliliter (mcg/mL)

Measure	GBT021601+[14C]-GBT021601 n = 9
Whole blood	NA (NA)
Plasma	NA (NA)

Population analysed: Pharmacokinetic (PK) analysis set included all participants who had received 1 dose of GBT021601 and provided at least 1 measurable post dose PK concentration of GBT021601 or total radioactivity in whole blood or plasma.

Primary outcome: 14C-GBT021601 Total Radioactivity Concentration in Whole Blood and Plasma at Predose

Time frame: Predose (within 60 minutes prior to 14C-GBT021601 dosing). Mean (Standard Deviation). Unit: mcgEq/mL

Measure	GBT021601+[14C]-GBT021601 n = 9
Whole blood	0 (0)
Plasma	0 (0)

Population analysed: PK analysis set included all participants who had received 1 dose of GBT021601 and provided at least 1 measurable post dose PK concentration of GBT021601 or total radioactivity in whole blood or plasma.

Primary outcome: GBT021601 Concentration in Whole Blood and Plasma at 0.25 Hours

Time frame: 0.25 hours post dosing on Day 1. Mean (Standard Deviation). Unit: mcg/mL

Measure	GBT021601+[14C]-GBT021601 n = 9
Whole blood	6.368 (5.4178)
Plasma	0.46267 (0.373675)

Primary outcome: 14C-GBT021601 Total Radioactivity Concentrations in Whole Blood and Plasma at 0.25 Hours

Time frame: 0.25 hours post dosing on Day 1. Mean (Standard Deviation). Unit: mcgEq/mL

Measure	GBT021601+[14C]-GBT021601 n = 9
Whole Blood	5.8206 (4.64396)
Plasma	0.84819 (0.595349)

5 secondary outcomes are reported in the full results.

Adverse events

Time frame: From Day 1 up to maximum of Day 207. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
GBT021601+[14C]-GBT021601	0 of 9 (0%)	4 of 9 (44.4%)	0 of 9

Limitations and caveats (from the sponsor): Data for metabolite profiling was not reported for \[14C\]-GBT021601 in whole blood and plasma because it was planned to be collected as part of separate, pooled analysis.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 8 Jan 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 42183775Zimmerman EI, Callegari E, Sharma R, Adenola A, Volpe J, Seetharaman K, Zhang C, Duchin K, Walker GS. A Phase 1 Microtracer Study Evaluating the Mass Balance, Excretion, and Pharmacokinetics of Osivelotor in Healthy Participants. Clin Transl Sci. 2026 Jun;19(6):e70601. doi: 10.1111/cts.70601.
To obtain contact information for a study center near you, click here.Link provided by the sponsor.
Individual participant dataSharing planned: No. Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Pfizer Pfizer CT.gov Call Center study director

Pfizer

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing GBT021601 in people with Sickle Cell Disease, run by Pfizer at 1 site in Netherlands.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 8 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 8 Jan 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

GBT021601 Concentration in Whole Blood and Plasma at Predose (time frame: Predose (within 60 minutes prior to GBT021601 dosing)). The study lists 121 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05718687, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jan 2025.