Sickle Cell DiseasePhase 1/2HUPKNCT03763656

Pharmacokinetics of Oral Hydroxyurea Solution

A Phase 1/2 study testing Oral Hydroxyurea (100 mg/mL) Solution in people with Sickle Cell Disease and Sickle-Cell; Hemoglobin Disease, Thalassemia, run by Nova Laboratories Limited at 6 sites in 2 countries.

Completed

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

Official record

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Trial summary

Who is it for?: Ages 6 months to 17 years
Conditions the study is for: Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia, Sickle Cell-beta-thalassemia
What is being tested?: Oral Hydroxyurea (100 mg/mL) Solution
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 33 participants
Where: 6 sitesin 2 countries
Study dates: 3 Jan 2019to 29 Dec 2021
Phase: Phase 1/2
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 6 months to 17 years
All sexes
No healthy volunteers
Child

Who may be able to join

Male or female aged from 6 months to 17.99 years of age (i.e. to the day before 18th birthday).
Diagnosis of sickle cell anemia (HbSS and HbSβº).
Parent(s)/legal guardian able and willing to provide written informed consent for the child to take part in the study.
Where applicable, the child should assent to undergo blood sampling for pharmacokinetic and biochemistry purposes and to allow physiological measurements to be made.

Who cannot join

Any clinically significant medical condition or abnormality, which, in the opinion of the Investigator, might have compromised the safety of the patient or which might have interfered with the study.
Hydroxyurea use within 6 months before enrolment.
Renal insufficiency (known creatinine more than twice the upper limit of normal (ULN) for age and >1.0 mg/dL [88.4 μmol/L]).
Clinical evidence of hepatic compromise with alanine aminotransferase (ALT) >3 times the ULN (a temporary swing in ALT did not result in exclusion).
Other significant organ system dysfunction based on the site Investigators discretion.
Severe active infections: fungal, viral or bacterial (as confirmed by culture), examples included tuberculosis, malaria, active hepatitis, osteomyelitis or any other illness that would have precluded the use of HU in normal clinical practice.

Show all 13Show fewer

Active chronic leg ulcers.
Known allergy to oral HU solution or any of the excipients.
Positive pregnancy test for females of child-bearing potential (in post-menarcheal females) before initiation of treatment, unless participant was sexually abstinent. Note: True abstinence was considered as being in line with the preferred and usual lifestyle of the participant. Periodic abstinence (such as calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.
Inadequate contraception measures in sexually active females (post-menarcheal females) and males of child-bearing age (see Section 9.5.1.10.4).
Breastfeeding at study initiation.
Participation in another clinical trial of an IMP.
Known infection with HIV.

Trial locations and contacts

6 sites in 2 countries.

Country

City, postcode or hospital

6 sites in 2 countries

United Kingdom5 sites

Birmingham Women's and Children's NHS Foundation Trust
Birmingham
Alder Hey Children's NHS Foundation Trust
Liverpool
Evelina London Children's Hospital
London
King's College Hospital NHS Foundation Trust
London
The Royal London Children's Hospital, Barts Health NHS Trust
London

Other trials in United Kingdom

Jamaica1 site

Dr Angela E Rankine- Mullings
Kingston

Other trials in Jamaica

About this study

Official title: A Prospective Open Label, Pharmacokinetic Study of an Oral Hydroxyurea Solution in Children With Sickle Cell Anemia.

An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months to 17.99 years (i.e. to the day before 18th birthday), with a 12 to 15 month treatment period for each participant. The study treatment duration will be for 6 months at the maximum tolerated dose [MTD], which is usually reached by 6 months after initiation of treatment. For patients in whom time to MTD is longer than 6 months or not achieved at all, the maximum duration of study treatment will be 15 months.

Conditions: Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia, Sickle Cell-beta-thalassemia, Sickle Cell Hemoglobin C
Being tested: Oral Hydroxyurea (100 mg/mL) Solution
Keywords: Hydroxyurea, Hydroxycarbamide, Xromi
Medical subject headings: Anemia, Sickle Cell, Hemoglobinopathies, Thalassemia, Hemoglobin SC Disease

What participants receive

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

Oral Hydroxyurea (100 mg/mL) SolutionDrug
Participants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.

Study groups in NCT03763656
Study group	Role	What they receive
Open Label	Experimental	Novel oral solution formulation of hydroxyurea Receives: Drug: Oral Hydroxyurea (100 mg/mL) Solution

What is being measured

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

Clearance (CL/F)
Model estimated Pharmacokinetic Parameter (PK population: n=32, having received at least one dose of study drug).
Measured: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)
Volume of Distribution (V/F)
Model estimated Pharmacokinetic Parameter (PK population: n=32, having received at least one dose of study drug).
Measured: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)
Time to Maximum Concentration (Tmax)
Mean Tmax (h) pharmacokinetic parameter derived using the final population PK model.
Measured: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)
Maximum Plasma Concentration Cmax (ug/mL)
Mean Cmax (ug/mL) pharmacokinetic parameter derived using the final population PK model.
Measured: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)
Area Under Plasma Concentration Time Curve (AUC 0-Inf)
Mean AUC 0-Infinity (hr*ug/mL) pharmacokinetic parameters derived using the final population PK model.
Measured: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)
Terminal Half-life (Hours)
Mean Terminal Half-life (hours) pharmacokinetic parameter derived using the final population PK model.
Measured: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)

Secondary outcomes (25)

Adverse Events
Incidence of Adverse events during the course of the trial from screening to final follow up phone call at Week 64.
Relatedness refers to 'at least possibly related to the IMP', with severity/toxicity assessed using the CTCAE Toxicity Grade and seriousness based on the standard definition for SAE in accordance with GCP. With the exception of SCA related events, requiring >7day hospitalisation, or extension of hospitalisation before being reported as an SAE due to their frequency within the disease population. All other non-SCA related SAEs were reportable.
Measured: Screening Up to Week 64
Absolute Neutrophil Count (ANC)
Safety review for haematological toxicity (mild myelosuppression target: 1-3x10^9/L)
Measured: Baseline and Week 60 (or final visit); max 15 months on treatment
White Blood Cell Count (Leukocytes)
Impact of Hydroxyurea on Safety, Hematology and Biochemistry
Measured: Baseline to Week 60 or Final Visit; max 15 months on treatment
Platelets
Impact of Hydroxyurea on Safety, Hematology and Biochemistry
Measured: Baseline to Week 60 (or Final Visit), max 15 months on treatment
Mean Corpuscular Hemoglobin (MCH)
Impact of Hydroxyurea on Safety, Hematology and Biochemistry
Measured: Baseline to Week 60 (or Final Visit), max 15 months on treatment
Hematocrit
Impact of Hydroxyurea on Safety, Hematology and Biochemistry
Measured: Up to Week 60
Bilirubin
Impact of Hydroxyurea on Safety, Hematology and Biochemistry
Measured: Up to Week 60
Elevation in Liver Function Tests (LFTs)
Impact of Hydroxyurea on Safety, Hematology and Biochemistry, including Liver Function Tests (LFTs): Alkaline Phosphatase (ALP), Gamma Glutamyl Transferase (GGT), Aspartate Amino Transferase (AST), Alanine Aminotransferase (ALT)
Measured: Up to Week 60
Hemoglobin
Safety and efficacy of hydroxyurea therapy
Measured: Baseline to Week 60 (or Final Visit); max 15 months on treatment
Bacterial Infections
Impact of Hydroxyurea on Safety, Hematology and Biochemistry
Measured: Up to Week 60
Viral Infections
Impact of Hydroxyurea on Safety, Hematology and Biochemistry
Measured: Up to Week 60
Fungal Infections
Impact of Hydroxyurea on Safety, Hematology and Biochemistry
Measured: Up to Week 60
Leg Ulcers
Impact of Hydroxyurea on Safety, Hematology and Biochemistry
Measured: Up to Week 60
Fetal Hemoglobin
Biomarker endpoints of Hydroxyurea efficacy
Measured: Baseline to Week 60 (or Final Visit); max 15 months on treatment
Mean Corpuscular Volume (MCV)
Biomarker endpoints of Hydroxyurea efficacy
Measured: Baseline to Week 60 (or Final Visit); max 15 months on treatment
Cystatin C
Biomarker Endpoints
Measured: PK1 (day 1), week 20-32 (6 months) and Week 60 (Final Visit)
Incidence of Acute Vaso-Occlusive Pain Crises (VOC)
Hospitalization Incidence for 12 Months Prior to and After First Dose of IMP (Safety Population) for vaso-occlusive crisis (Mean [SD])
Measured: 12 months prior to treatment and time-averaged 12 months post-treatment; maximum 15 months on IMP
Number and Frequency of Blood Transfusions
Clinical status endpoints, related to blood transfusions for treatment of SCA, this may be hospitalization, A\&E, or in-clinic treatment from safety population (n=32)
Measured: Up to Week 60
Acute Chest Syndrome (ACS)
Hospitalization Incidence for 12 Months Prior to and After First Dose of IMP (Safety Population) for Acute Chest Syndrome (Mean [SD])
Measured: 12 months prior to treatment and time averaged 12 months post-treatment; maximum 15 months on IMP
Hospitalizations
Clinical Status endpoints, all hospitalisations (not ER/Accident without hospitalization) (Mean [SD])
Measured: 12 months prior to treatment and time-averaged 12 months post-treatment; maximum 15 months on IMP
Dose Escalation i.e. Maximum Tolerated Dose (MTD)
Summary of maximum tolerated dose achieved in mg/kg (Mean [SD])
Measured: Screening Up to Final Visit (Week 60 or Withdrawal), maximum 15 months on IMP
Other SCA-related Hospitalizations
Clinical parameters (symptoms), secondary clinical status endpoints (not including ACS, VOC, Other Non-SCA related) (mean [SD])
Measured: 12 months prior to treatment and time averaged 12 months post-treatment; maximum 15 months on IMP
Parent/Caregiver Palatability and Acceptability Questionnaire
Clinical status endpoints. Visual Analogue scale used to determine, taste, smell, aftertaste, acceptability and ease of dosing (1-100mm scale) with 1 being worst possible and 100 being best possible.
Assessed for <6 years of age by parents/guardians. Assessed for >6 years of age, combination of parent/guardian and participant responses.
Measured: Taken once at any point after 8 weeks on study medication (or at early Withdrawal)
Vitamin D
Biochemistry
Measured: From Screening Up to Final Visit (Week 60 or WD)
Other Non-SCA-related Hospitalizations
Clinical parameters (symptoms), secondary clinical status endpoints (not including ACS, VOC, Other SCA-related)
Measured: 12 months prior to treatment and 12 months post-treatment; maximum 15 months on IMP

Timeline

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

3 Jan 201929 Dec 2021

First posted4 Dec 2018
Study start3 Jan 2019
Primary completion19 May 2021
Study completion29 Dec 2021

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.
Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
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 1/2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Model description: Open label, observational, pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months-17.99 years over a 12-15-month period
Masking: None (open label)
Enrolment: 33 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03763656
Sponsor's protocol ID: INV543
Other IDs: 2017-004568-37, 2017
Acronym: HUPK
Status last verified: Aug 2024
First submitted: 3 Dec 2018
Last updated: 28 Oct 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No

Want to know more?

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

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

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?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Sickle Cell Disease trials

Study at a glance

From the registry record, last updated by the sponsor on 28 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Prospective Open Label, Pharmacokinetic Study of an Oral Hydroxyurea Solution in Children With Sickle Cell Anemia.
ClinicalTrials.gov ID: NCT03763656
Acronym / sponsor ID: HUPK / INV543
Conditions: Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia, Sickle Cell-beta-thalassemia, Sickle Cell Hemoglobin C
Keywords: Hydroxyurea, Hydroxycarbamide, Xromi
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 33 participants (actual)
Lead sponsor: Nova Laboratories LimitedIndustry (pharma, biotech, devices)
Study start: 3 Jan 2019 (actual)
Primary completion: 19 May 2021 (actual)
Study completion: 29 Dec 2021 (actual)
First posted: 4 Dec 2018 (actual)
Results: Posted 28 Oct 2024

Clinical summary

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

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1
Model description: Open label, observational, pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months-17.99 years over a 12-15-month period

Arm	Type	Receives
Open Label	Experimental	Drug: Oral Hydroxyurea (100 mg/mL) SolutionNovel oral solution formulation of hydroxyurea

Who may qualify

Ages: 6 months to 17 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (4 listed)

Male or female aged from 6 months to 17.99 years of age (i.e. to the day before 18th birthday).
Diagnosis of sickle cell anemia (HbSS and HbSβº).
Parent(s)/legal guardian able and willing to provide written informed consent for the child to take part in the study.
Where applicable, the child should assent to undergo blood sampling for pharmacokinetic and biochemistry purposes and to allow physiological measurements to be made.

Key exclusion criteria (13 listed)

Any clinically significant medical condition or abnormality, which, in the opinion of the Investigator, might have compromised the safety of the patient or which might have interfered with the study.
Hydroxyurea use within 6 months before enrolment.
Renal insufficiency (known creatinine more than twice the upper limit of normal (ULN) for age and >1.0 mg/dL [88.4 μmol/L]).
Clinical evidence of hepatic compromise with alanine aminotransferase (ALT) >3 times the ULN (a temporary swing in ALT did not result in exclusion).
Other significant organ system dysfunction based on the site Investigators discretion.

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
Oral Hydroxyurea (100 mg/mL) SolutionParticipants received Oral Hydroxyurea 15 mg/kg once daily. Escalated by 5 mg/kg/day every 8-12 weeks until maximum tolerated dose achieved, up to a maximum 35 mg/kg/day.	Drug	Open Label

Primary outcome

Clearance (CL/F)
Time frame: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)
Model estimated Pharmacokinetic Parameter (PK population: n=32, having received at least one dose of study drug).
Volume of Distribution (V/F)
Time frame: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)
Model estimated Pharmacokinetic Parameter (PK population: n=32, having received at least one dose of study drug).
Time to Maximum Concentration (Tmax)
Time frame: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)
Mean Tmax (h) pharmacokinetic parameter derived using the final population PK model.
Maximum Plasma Concentration Cmax (ug/mL)
Time frame: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)
Mean Cmax (ug/mL) pharmacokinetic parameter derived using the final population PK model.
Area Under Plasma Concentration Time Curve (AUC 0-Inf)
Time frame: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)
Mean AUC 0-Infinity (hr*ug/mL) pharmacokinetic parameters derived using the final population PK model.
Terminal Half-life (Hours)
Time frame: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)
Mean Terminal Half-life (hours) pharmacokinetic parameter derived using the final population PK model.

25 secondary outcomes

Adverse Events
Time frame: Screening Up to Week 64
Absolute Neutrophil Count (ANC)
Time frame: Baseline and Week 60 (or final visit); max 15 months on treatment
White Blood Cell Count (Leukocytes)
Time frame: Baseline to Week 60 or Final Visit; max 15 months on treatment
Platelets
Time frame: Baseline to Week 60 (or Final Visit), max 15 months on treatment
Mean Corpuscular Hemoglobin (MCH)
Time frame: Baseline to Week 60 (or Final Visit), max 15 months on treatment
Hematocrit
Time frame: Up to Week 60
Bilirubin
Time frame: Up to Week 60
Elevation in Liver Function Tests (LFTs)
Time frame: Up to Week 60
Hemoglobin
Time frame: Baseline to Week 60 (or Final Visit); max 15 months on treatment
Bacterial Infections
Time frame: Up to Week 60
Viral Infections
Time frame: Up to Week 60
Fungal Infections
Time frame: Up to Week 60
Leg Ulcers
Time frame: Up to Week 60
Fetal Hemoglobin
Time frame: Baseline to Week 60 (or Final Visit); max 15 months on treatment
Mean Corpuscular Volume (MCV)
Time frame: Baseline to Week 60 (or Final Visit); max 15 months on treatment
Cystatin C
Time frame: PK1 (day 1), week 20-32 (6 months) and Week 60 (Final Visit)
Incidence of Acute Vaso-Occlusive Pain Crises (VOC)
Time frame: 12 months prior to treatment and time-averaged 12 months post-treatment; maximum 15 months on IMP
Number and Frequency of Blood Transfusions
Time frame: Up to Week 60
Acute Chest Syndrome (ACS)
Time frame: 12 months prior to treatment and time averaged 12 months post-treatment; maximum 15 months on IMP
Hospitalizations
Time frame: 12 months prior to treatment and time-averaged 12 months post-treatment; maximum 15 months on IMP
Dose Escalation i.e. Maximum Tolerated Dose (MTD)
Time frame: Screening Up to Final Visit (Week 60 or Withdrawal), maximum 15 months on IMP
Other SCA-related Hospitalizations
Time frame: 12 months prior to treatment and time averaged 12 months post-treatment; maximum 15 months on IMP
Parent/Caregiver Palatability and Acceptability Questionnaire
Time frame: Taken once at any point after 8 weeks on study medication (or at early Withdrawal)
Vitamin D
Time frame: From Screening Up to Final Visit (Week 60 or WD)
Other Non-SCA-related Hospitalizations
Time frame: 12 months prior to treatment and 12 months post-treatment; maximum 15 months on IMP

Results and safety

Results posted to the registry on 28 Oct 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Oral Hydroxyurea	33	24

Primary outcome: Clearance (CL/F)

Time frame: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36). Mean (95% Confidence Interval). Unit: L/hr

Measure	Oral Hydroxyurea n = 32
Value	3.61 (3.29 to 3.92)

Population analysed: Clearance value for patient of 15.05 kg

Primary outcome: Volume of Distribution (V/F)

Time frame: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36). Mean (95% Confidence Interval). Unit: L

Measure	Oral Hydroxyurea n = 32
Value	11.4 (9.52 to 13.2)

Population analysed: Volume of distribution for central and peripheral compartments for patient of 15.05 kg

Primary outcome: Time to Maximum Concentration (Tmax)

Time frame: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36). Mean (Standard Deviation). Unit: hours

Measure	Oral Hydroxyurea n = 32
Tmax (Overall)	1.253 (0.412)
Tmax (Age Group 6 months - 1.99 years)	1.265 (0.322)
Tmax (Age Group 2 - 5.99 years)	1.238 (0.455)
Tmax (Age Group 6 - 17.99 years)	1.269 (0.423)

Population analysed: PK population = 32 participants, who have received at least one dose at 15 mg/kg and have at least one sample of PK plasma above the lower limit of quantification

Primary outcome: Maximum Plasma Concentration Cmax (ug/mL)

Time frame: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36). Mean (Standard Deviation). Unit: ug/mL

Measure	Oral Hydroxyurea n = 32
Cmax (Overall)	13.124 (1.742)
Cmax (Age Group 6 months - 1.99 years)	12.807 (1.399)
Cmax (Age Group 2 - 5.99 years)	13.068 (1.939)
Cmax (Age Group 6 - 17.99 years)	13.404 (1.714)

Population analysed: PK population: n=32 safety population, having received at least one dose of study drug at 15mg/kg and one sample of PK plasma above the lower limit of quantification.

25 secondary outcomes are reported in the full results.

Adverse events

Time frame: Screening to safety follow up call at 64 weeks (maximum 15 months on treatment).. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Oral Hydroxyurea	6 of 32 (18.8%)	31 of 32 (96.9%)	0 of 32

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 28 Oct 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

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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.

Angela E Rankine- Mullings, MD principal investigator

University of the West Indies, Mona, Kingston, Jamaica

6 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 13 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 28 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in United Kingdom, Jamaica. 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 combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 28 Oct 2024). 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?

Clearance (CL/F) (time frame: 0, 0.25, 0.5, 1, 1.5, 2, 3, 4, 5, 6, and 12 hours post-dose on Day 1 and Day 2 (Week 20-36)). The study lists 6 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 NCT03763656.

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 28 Oct 2024.