Sickle Cell DiseasePhase 1NCT05036512
A Study to Evaluate GBT021601 in Single and Multiple Doses in Healthy Participants
A Phase 1 study testing GBT021601 in people with Sickle Cell Disease, run by Pfizer at 4 sites in 2 countries.
Trial summary
This first in human study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and food effect of GBT021601, a hemoglobin S (HbS) polymerization inhibitor, in healthy participants. 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?
- Yes, some participants may receive a placebo
- How many people
- 129 participants
- Where
- 4 sitesin 2 countries
- Study dates
- 9 Dec 2020to 7 Feb 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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
- Healthy males and females ≥ 18 to ≤ 55 years of age
- Body mass index ≥ 18.0 to ≤ 30.0 kg/m2
- Body weight ≥ 50 kg at screening and Day -1
Who cannot join
- Positive pregnancy test or currently breastfeeding.
Trial locations and contacts
4 sites in 2 countries.
4 sites in 2 countries
Australia3 sites
Harry Perkins Institute of Medical Research
Nedlands, Western Australia, 6009
Linear Clinical Research
Nedlands, Western Australia, 6009
Oxford Compounding
North Perth, Western Australia, 6006
United States1 site
ICON Early Phase Services, LLC
San Antonio, Texas, 78209
About this study
Official title: A Double-Blind, Randomized, Placebo-Controlled, Single and Multiple Ascending Dose (SAD/MAD) Study to Evaluate the Safety, Tolerability, PK, and Food Effect of GBT021601, a Hemoglobin S Polymerization Inhibitor, in Healthy Participants.
This first in human study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and food effect of GBT021601, a hemoglobin S (HbS) polymerization inhibitor, in healthy participants.
Read the detailed description
This is a randomized, double-blind, placebo controlled, single and multiple ascending dose study 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
Also called Placebo
Administered orally with water as a single dose in the morning.
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Placebo as a tablet or capsule with dose based off of preceding cohort's data. Receives: Drug: GBT021601 |
| GBT021601 | Experimental | GBT021601 as a tablet or capsule with dose based off of preceding cohort's data. Receives: Drug: GBT021601 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety, as assessed by frequency and severity of adverse events (AEs)
AEs will be coded to system organ class and preferred term using the Medical Dictionary for Regulatory Activities (MedDRA) and summarized.
Measured: 119 days from screening Part A, 134 days from screening Part B
Safety, as assessed by changes in Heart Rate.
Number of participants with changes in heart rate (bpm) as compared to baseline.
Measured: 119 days from screening Part A, 134 days from screening Part B
Safety, as assessed by changes in eGFR
Number of participants with changes in eGFR from baseline
Measured: 119 days from screening Part A, 134 days from screening Part B
Safety, as assessed by changes in alanine aminotransferase (ALT)
Number of participants with changes in alanine aminotransferase (ALT)
Measured: 119 days from screening Part A, 134 days from screening Part B
Safety, as assessed by changes in Blood pressure
Number of participants with changes in systolic (mmHg) and diastolic (mmHg) blood
Measured: 119 days from screening Part A, 134 days from screening Part B
Plasma concentration
Time of Cmax
Measured: 119 days from screening Part A
Plasma concentration
Cmax on D1-D15
Measured: 134 days from screening Part B
Secondary outcomes (3)
Determine whole blood concentration of GBT021601
Hemoximetry will be used to assess oxygen saturation in whole blood by generating oxygen equilibrium curves (OECs) which relate the extent of Hb-O2 saturation to the partial pressure of O2 (pO2) and measure the binding affinity of O2 to Hb.
Measured: 119 days from screening Part A
Determine plasma concentration of GBT021601.
With dosing data from each cohort determine the steady-state maximum plasma/whole blood concentration (Cmax).
Measured: 134 days from screening Part B
Safety, as assessed by changes in QTcF
Number of participants with changes in the QTcF interval from baseline
Measured: 119 days from screening Part A, 134 days from screening Part B
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Sep 2021
- Study start9 Dec 2020
- Primary completion2 Nov 2022
- Study completion7 Feb 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 129 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05036512
- Sponsor's protocol ID
- GBT021601-011
- Other IDs
- C5351001
- Status last verified
- Jul 2023
- First submitted
- 13 Aug 2021
- Last updated
- 25 Jul 2023
- Results posted
- No
- 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
Lisbon EA, Brown RC, Redfern A, Duchin K, Barth A, Roberts ES, Gaputan P, Dufu K, Lo A, Pochron MP, Archer DR, Chin W, Kong K, Washington CB, Adenola A, Zimmerman EI. Safety, Pharmacokinetics, and Pharmacodynamics of Osivelotor for Sickle Cell Disease: First-in-Human Studies in Healthy Participants and Patients. Clin Pharmacol Ther. 2026 Jan;119(1):120-130. doi: 10.1002/cpt.70028. Epub 2025 Sep 2.
PubMed 40891510
Study at a glance
From the registry record, last updated by the sponsor on 25 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-Blind, Randomized, Placebo-Controlled, Single and Multiple Ascending Dose (SAD/MAD) Study to Evaluate the Safety, Tolerability, PK, and Food Effect of GBT021601, a Hemoglobin S Polymerization Inhibitor, in Healthy Participants.
- ClinicalTrials.gov ID
- NCT05036512
- Acronym / sponsor ID
- GBT021601-011
- Conditions
- Sickle Cell Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 129 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- 9 Dec 2020 (actual)
- Primary completion
- 2 Nov 2022 (actual)
- Study completion
- 7 Feb 2023 (actual)
- First posted
- 5 Sep 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This first in human study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and food effect of GBT021601, a hemoglobin S (HbS) polymerization inhibitor, in healthy participants.
Detailed description
This is a randomized, double-blind, placebo controlled, single and multiple ascending dose study in healthy participants.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: GBT021601Placebo as a tablet or capsule with dose based off of preceding cohort's data. |
| GBT021601 | Experimental | Drug: GBT021601GBT021601 as a tablet or capsule with dose based off of preceding cohort's data. |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- Healthy males and females ≥ 18 to ≤ 55 years of age
- Body mass index ≥ 18.0 to ≤ 30.0 kg/m2
- Body weight ≥ 50 kg at screening and Day -1
Key exclusion criteria (1 listed)
- Positive pregnancy test or currently breastfeeding.
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 |
|---|---|---|
| GBT021601Also: PlaceboAdministered orally with water as a single dose in the morning. | Drug | GBT021601, Placebo |
Primary outcome
Safety, as assessed by frequency and severity of adverse events (AEs)
Time frame: 119 days from screening Part A, 134 days from screening Part B
AEs will be coded to system organ class and preferred term using the Medical Dictionary for Regulatory Activities (MedDRA) and summarized.
Safety, as assessed by changes in Heart Rate.
Time frame: 119 days from screening Part A, 134 days from screening Part B
Number of participants with changes in heart rate (bpm) as compared to baseline.
Safety, as assessed by changes in eGFR
Time frame: 119 days from screening Part A, 134 days from screening Part B
Number of participants with changes in eGFR from baseline
Safety, as assessed by changes in alanine aminotransferase (ALT)
Time frame: 119 days from screening Part A, 134 days from screening Part B
Number of participants with changes in alanine aminotransferase (ALT)
Safety, as assessed by changes in Blood pressure
Time frame: 119 days from screening Part A, 134 days from screening Part B
Number of participants with changes in systolic (mmHg) and diastolic (mmHg) blood
Plasma concentration
Time frame: 119 days from screening Part A
Time of Cmax
Plasma concentration
Time frame: 134 days from screening Part B
Cmax on D1-D15
3 secondary outcomes
Determine whole blood concentration of GBT021601
Time frame: 119 days from screening Part A
Determine plasma concentration of GBT021601.
Time frame: 134 days from screening Part B
Safety, as assessed by changes in QTcF
Time frame: 119 days from screening Part A, 134 days from screening Part B
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jul 2023.
- Derived from this study: PubMed 40891510Lisbon EA, Brown RC, Redfern A, Duchin K, Barth A, Roberts ES, Gaputan P, Dufu K, Lo A, Pochron MP, Archer DR, Chin W, Kong K, Washington CB, Adenola A, Zimmerman EI. Safety, Pharmacokinetics, and Pharmacodynamics of Osivelotor for Sickle Cell Disease: First-in-Human Studies in Healthy Participants and Patients. Clin Pharmacol Ther. 2026 Jan;119(1):120-130. doi: 10.1002/cpt.70028. Epub 2025 Sep 2.
- 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.
Related trials for Sickle Cell Disease
RecruitingObservational study
Blood Sampling for Research Related to Sickle Cell Disease
Sickle Cell Trait, Sickle Cell Disease
Active, not recruitingPhase 2/3
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
Sickle Cell Disease
Enrolling by invitationObservational study
Blood Collection for Research Related to Certain Diseases Involving Blood Vessels
Sickle Cell Disease
RecruitingPhase 1/2
Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels
Myeloid Chimerism
Not yet recruitingObservational study
MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT)
Antiphospholipid (aPL)-Positive, Rheumatic Arthritis
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 CT.gov Call Center study director
Pfizer
4 sites in 2 countries: 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 4 sites in 2 countries.
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 3 inclusion and 1 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 25 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Australia, United States. 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. Some participants in this study may receive a placebo. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Safety, as assessed by frequency and severity of adverse events (AEs) (time frame: 119 days from screening Part A, 134 days from screening Part B). The study lists 7 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 NCT05036512, 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 25 Jul 2023.