Sickle Cell Disease (SCD)Phase 2/3OPTIMANCT06761560
Optimizing Hydroxyurea Dosage With Pharmakokinetic in Patients Suffering of Moderate to Severe Sickle Cell Anemia
A Phase 2/3 study testing Pharmacokinetic based dosage change and Pharmacokinetic dosing in people with Sickle Cell Disease (SCD), run by Yves Pastore at 1 site in Canada.
Trial summary
The goal of this study is to evaluate if patients with sickle cell disease can achieve a maximum tolerate dose of hydroxuyrea (HU) over a period of 12 months faster with pharmacokinetic testing than the standard of care bloodwork follow-up. Read more about this study
- Who is it for?
- Ages 6 months to 18 years
- Conditions the study is for
- Sickle Cell Disease (SCD)
- What is being tested?
- Pharmacokinetic based dosage change and Pharmacokinetic dosing
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 29 participants
- Where
- 1 siteCanada
- Study dates
- 3 Feb 2026to 25 Nov 2028
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
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 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Have had confirmed diagnosis of SCD at CHU Sainte-Justine biochemistry lab with hemoglobin electrophoresis.
- Patients with SS, SBThal0.
- Agree to take hydroxyurea for a period of 12 months
- Be between age of 6months old and 18 years old.
- Have consented for participation in the study.
- Have had confirmed diagnosis of SCD at CHU Sainte-Justine biochemistry lab with hemoglobin electrophoresis.
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- Patients with SS, SBThal0.
- Have taken hydroxyurea for a period of at least 12 months, and have received HU at a stable dose and at MTD for at least 6 months.
- Be between age of 6months old and 18 years old.
- Have consented for participation in the study.
Who cannot join
- Patients with sickle cell genotype other than SS or SBThal0 (SC, SBThal+, SE or SD)
- Patients on chronic transfusion program
- Patients have received a blood transfusion in the last 4 weeks of study enrollment.
- Have received a hematopoietic stem-cell transplantation
- Creatinine >2x normal for age
- ALT>2x normal for age
Show all 9Show fewer
- Sexually active females unwilling to comply with reliable method of birth control
- Pregnancy
- Conditions which in the opinion of the investigator, would compromise participation in the study will be excluded.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Bianka Courcelle, Research nurse, RN contact
Canada1 site, 1 recruiting
- Recruiting
CHU Sainte-Justine
Montreal, Quebec, H3T 1C5
Yves Pastore, MD, 514-345-4931 ext. 5027, yves.pastore.med@ssss.gouv.qc.ca
Karyne Daigle, RN, 514-345-4931 ext. 3455, karyne.daigle.hsj@ssss.gouv.qc.ca
About this study
The goal of this study is to evaluate if patients with sickle cell disease can achieve a maximum tolerate dose of hydroxuyrea (HU) over a period of 12 months faster with pharmacokinetic testing than the standard of care bloodwork follow-up. Pharmacokinetic test is used to evaluate the process by which drugs are absorbed, distributed in the body, localized in the tissues, and is excreted.
Patient will be a randomized (coin toss method) into 2 groups. Group A will have an increase of their HU dosage with pharmacokinetic results and Group B will have an increase of their HU dosage following the standard of care bloodwork follow-up.
Group C will include patient with sickle cell disease that has been taking HU for at least 12 months and will undergo a pharmacokinetic dosage to check the level of HU only one time.
- Conditions
- Sickle Cell Disease (SCD)
- Being tested
- Pharmacokinetic based dosage change, Pharmacokinetic dosing
- Keywords
- hydroxyurea, pharmacokinetic, sickle cell disease
- 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.
Pharmacokinetic based dosage changeDiagnostic test
This study will compare 2 groups of sickle cell patients that are receiving hydroxyurea. Group A will have an increase in their dosage based on the pharmacokinetic result over a period of 12 months and Group B will have an increase in their dosage based on the standard of care follow-up over a period of 12 months. The aim is to evaluate if the group A can reach MTD faster than than the Group B
Pharmacokinetic dosingDiagnostic test
Patient with sickle cell disease will undergo one pharmacokinetic test after taking 12 months of hydroxyurea to evaluate HU-AUC at that timepoint
| Study group | Role | What they receive |
|---|---|---|
| Group A | Experimental | Patient starting hydroxyurea that will have PK sample procurements at different timpoints over a period of 12 months until HU-AUC shows MTD Receives: Diagnostic Test: Pharmacokinetic based dosage change |
| Group B | No intervention | Patient starting hydroxuyrea that will be followed as per standard of care over a period of 12 months until MTD has been reached |
| Group C | Experimental | Patient will undergo one PK sample procurement to evaluate level of HU-AUC after 12 months of taking hydroxyurea. Receives: Diagnostic Test: Pharmacokinetic dosing |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluation of HU-PK at 6 months between group A and group B
Pharmakocinetic dosage of hydroxyurea will be determined at 6 months in group A and group B. The investigator hypothesize that HU-PK in group B may be lower (suboptimal) compared to group A.
Measured: At 6 months
Secondary outcomes (4)
Time to reach maximal tolerated dose (MTD)
Time (weeks) to achieve MTD in groups A and B will be determined by evaluating the % of patients reaching MTD (at 3 , 6, 9 and 12 months) in each group.
MTD is defined by hematological parameters: Absolute neutrophile count 0.8-1.5x10*9/L or platelet 80-120x10*9/L or absolute reticulocyte count 50-80x10*9/L)
Measured: 3, 6, 9 and 12 months
Fetal hemoglobin
Comparing fetal hemoglobin between group A and B
Measured: at 3, 6 and 12 months
Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) in group A and B
Evaluation of the incidence adverse events (AE) and serious adverse events (SAE) in both groups
Measured: From enrollment to 12 months
Evaluation of % of patients reaching AUC of 115 +/- 15mg*h/L at 12 months compared to the percentage of patients in group C reaching the same AUC
Percentage of patients in the HU-AUC (group A) with an AUC of 115 mg*h/L at 12 months will be compared to the percentage of patients in group C with an AUC of 115 +/-15 mg*h/L.
Measured: At 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jan 2025
- Study start3 Feb 2026
- Primary completion25 Nov 2028 (estimated)
- Study completion25 Nov 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (14)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell Disease (SCD)Studies 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 2/3
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Group A will receive dosing of hydroxyurea depending on the HU-AUC result at different timepoint until MTD Group B will receive dosing of hydroxyurea following the standard of care bloodwork follow-up until MTD Group C will undergo one pharmacokinetic testing after taking hydroxyurea for at least 12 months
- Masking
- None (open label)
- Enrolment
- 29 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Yves Pastore
- Sponsor type
- University, hospital or other
- Collaborators
- St. Justine's Hospital
- Responsible party
- Sponsor-investigator, Yves Pastore, St. Justine's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06761560
- Sponsor's protocol ID
- 2023-4674
- Acronym
- OPTIMA
- Status last verified
- Aug 2026
- First submitted
- 12 Dec 2024
- Last updated
- 21 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: This is a pilot study with a small group of participant.
Publications
Verduzco LA, Nathan DG. Sickle cell disease and stroke. Blood. 2009 Dec 10;114(25):5117-25. doi: 10.1182/blood-2009-05-220921.
PubMed 19797523Meier ER. Treatment Options for Sickle Cell Disease. Pediatr Clin North Am. 2018 Jun;65(3):427-443. doi: 10.1016/j.pcl.2018.01.005.
PubMed 29803275McGann PT, Niss O, Dong M, Marahatta A, Howard TA, Mizuno T, Lane A, Kalfa TA, Malik P, Quinn CT, Ware RE, Vinks AA. Robust clinical and laboratory response to hydroxyurea using pharmacokinetically guided dosing for young children with sickle cell anemia. Am J Hematol. 2019 Aug;94(8):871-879. doi: 10.1002/ajh.25510. Epub 2019 Jun 12.
PubMed 31106898Dong M, McGann PT. Changing the Clinical Paradigm of Hydroxyurea Treatment for Sickle Cell Anemia Through Precision Medicine. Clin Pharmacol Ther. 2021 Jan;109(1):73-81. doi: 10.1002/cpt.2028. Epub 2020 Oct 8.
PubMed 32869281Marahatta A, Ware RE. Hydroxyurea: Analytical techniques and quantitative analysis. Blood Cells Mol Dis. 2017 Sep;67:135-142. doi: 10.1016/j.bcmd.2017.08.009. Epub 2017 Aug 8.
PubMed 28847416Hai X, Guo M, Gao C, Zhou J. Quantification of hydroxyurea in human plasma by HPLC-MS/MS and its application to pharmacokinetics in patients with chronic myeloid leukaemia. J Pharm Biomed Anal. 2017 Apr 15;137:213-219. doi: 10.1016/j.jpba.2017.01.008. Epub 2017 Jan 10.
PubMed 28131939Wang WC, Ware RE, Miller ST, Iyer RV, Casella JF, Minniti CP, Rana S, Thornburg CD, Rogers ZR, Kalpatthi RV, Barredo JC, Brown RC, Sarnaik SA, Howard TH, Wynn LW, Kutlar A, Armstrong FD, Files BA, Goldsmith JC, Waclawiw MA, Huang X, Thompson BW; BABY HUG investigators. Hydroxycarbamide in very young children with sickle-cell anaemia: a multicentre, randomised, controlled trial (BABY HUG). Lancet. 2011 May 14;377(9778):1663-72. doi: 10.1016/S0140-6736(11)60355-3.
PubMed 21571150Thornburg CD, Files BA, Luo Z, Miller ST, Kalpatthi R, Iyer R, Seaman P, Lebensburger J, Alvarez O, Thompson B, Ware RE, Wang WC; BABY HUG Investigators. Impact of hydroxyurea on clinical events in the BABY HUG trial. Blood. 2012 Nov 22;120(22):4304-10; quiz 4448. doi: 10.1182/blood-2012-03-419879. Epub 2012 Aug 22.
PubMed 22915643Yahouedehou SCMA, Adorno EV, da Guarda CC, Ndidi US, Carvalho SP, Santiago RP, Aleluia MM, de Oliveira RM, Goncalves MS. Hydroxyurea in the management of sickle cell disease: pharmacogenomics and enzymatic metabolism. Pharmacogenomics J. 2018 Dec;18(6):730-739. doi: 10.1038/s41397-018-0045-1. Epub 2018 Sep 12.
PubMed 30206297Switzer JA, Hess DC, Nichols FT, Adams RJ. Pathophysiology and treatment of stroke in sickle-cell disease: present and future. Lancet Neurol. 2006 Jun;5(6):501-12. doi: 10.1016/S1474-4422(06)70469-0.
PubMed 16713922Quinn CT. Sickle cell disease in childhood: from newborn screening through transition to adult medical care. Pediatr Clin North Am. 2013 Dec;60(6):1363-81. doi: 10.1016/j.pcl.2013.09.006.
PubMed 24237976Hebbel RP. Ischemia-reperfusion injury in sickle cell anemia: relationship to acute chest syndrome, endothelial dysfunction, arterial vasculopathy, and inflammatory pain. Hematol Oncol Clin North Am. 2014 Apr;28(2):181-98. doi: 10.1016/j.hoc.2013.11.005.
PubMed 24589261Kuypers FA. Hemoglobin s polymerization and red cell membrane changes. Hematol Oncol Clin North Am. 2014 Apr;28(2):155-79. doi: 10.1016/j.hoc.2013.12.002. Epub 2014 Jan 22.
PubMed 24589260Pleasants S. Epidemiology: a moving target. Nature. 2014 Nov 13;515(7526):S2-3. doi: 10.1038/515S2a. No abstract available.
PubMed 25390139
Study at a glance
From the registry record, last updated by the sponsor on 21 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Optimizing Hydroxyurea Dosage With Pharmakokinetic in Patients Suffering of Moderate to Severe Sickle Cell Anemia
- ClinicalTrials.gov ID
- NCT06761560
- Acronym / sponsor ID
- OPTIMA / 2023-4674
- Conditions
- Sickle Cell Disease (SCD)
- Keywords
- hydroxyurea, pharmacokinetic, sickle cell disease
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 29 participants (estimated)
- Lead sponsor
- Yves PastoreUniversity, hospital or other
- Collaborators
- St. Justine's Hospital
- Study start
- 3 Feb 2026 (actual)
- Primary completion
- 25 Nov 2028 (estimated)
- Study completion
- 25 Nov 2028 (estimated)
- First posted
- 7 Jan 2025 (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.
The goal of this study is to evaluate if patients with sickle cell disease can achieve a maximum tolerate dose of hydroxuyrea (HU) over a period of 12 months faster with pharmacokinetic testing than the standard of care bloodwork follow-up. Pharmacokinetic test is used to evaluate the process by which drugs are absorbed, distributed in the body, localized in the tissues, and is excreted.
Patient will be a randomized (coin toss method) into 2 groups. Group A will have an increase of their HU dosage with pharmacokinetic results and Group B will have an increase of their HU dosage following the standard of care bloodwork follow-up.
Group C will include patient with sickle cell disease that has been taking HU for at least 12 months and will undergo a pharmacokinetic dosage to check the level of HU only one time.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- Group A will receive dosing of hydroxyurea depending on the HU-AUC result at different timepoint until MTD Group B will receive dosing of hydroxyurea following the standard of care bloodwork follow-up until MTD Group C will undergo one pharmacokinetic testing after taking hydroxyurea for at least 12 months
| Arm | Type | Receives |
|---|---|---|
| Group A | Experimental | Diagnostic Test: Pharmacokinetic based dosage changePatient starting hydroxyurea that will have PK sample procurements at different timpoints over a period of 12 months until HU-AUC shows MTD |
| Group B | No intervention | –Patient starting hydroxuyrea that will be followed as per standard of care over a period of 12 months until MTD has been reached |
| Group C | Experimental | Diagnostic Test: Pharmacokinetic dosingPatient will undergo one PK sample procurement to evaluate level of HU-AUC after 12 months of taking hydroxyurea. |
Who may qualify
- Ages
- 6 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- Have had confirmed diagnosis of SCD at CHU Sainte-Justine biochemistry lab with hemoglobin electrophoresis.
- Patients with SS, SBThal0.
- Agree to take hydroxyurea for a period of 12 months
- Be between age of 6months old and 18 years old.
- Have consented for participation in the study.
Key exclusion criteria (9 listed)
- Patients with sickle cell genotype other than SS or SBThal0 (SC, SBThal+, SE or SD)
- Patients on chronic transfusion program
- Patients have received a blood transfusion in the last 4 weeks of study enrollment.
- Have received a hematopoietic stem-cell transplantation
- Creatinine >2x normal for age
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 |
|---|---|---|
| Pharmacokinetic based dosage changeThis study will compare 2 groups of sickle cell patients that are receiving hydroxyurea. Group A will have an increase in their dosage based on the pharmacokinetic result over a period of 12 months and Group B will have an increase in their dosage based on the standard of care follow-up over a period of 12 months. The aim is to evaluate if the group A can reach MTD faster than than the Group B | Diagnostic test | Group A |
| Pharmacokinetic dosingPatient with sickle cell disease will undergo one pharmacokinetic test after taking 12 months of hydroxyurea to evaluate HU-AUC at that timepoint | Diagnostic test | Group C |
Primary outcome
Evaluation of HU-PK at 6 months between group A and group B
Time frame: At 6 months
Pharmakocinetic dosage of hydroxyurea will be determined at 6 months in group A and group B. The investigator hypothesize that HU-PK in group B may be lower (suboptimal) compared to group A.
4 secondary outcomes
Time to reach maximal tolerated dose (MTD)
Time frame: 3, 6, 9 and 12 months
Fetal hemoglobin
Time frame: at 3, 6 and 12 months
Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) in group A and B
Time frame: From enrollment to 12 months
Evaluation of % of patients reaching AUC of 115 +/- 15mg*h/L at 12 months compared to the percentage of patients in group C reaching the same AUC
Time frame: At 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Aug 2026.
- Background: PubMed 19797523Verduzco LA, Nathan DG. Sickle cell disease and stroke. Blood. 2009 Dec 10;114(25):5117-25. doi: 10.1182/blood-2009-05-220921.
- Background: PubMed 29803275Meier ER. Treatment Options for Sickle Cell Disease. Pediatr Clin North Am. 2018 Jun;65(3):427-443. doi: 10.1016/j.pcl.2018.01.005.
- Background: PubMed 31106898McGann PT, Niss O, Dong M, Marahatta A, Howard TA, Mizuno T, Lane A, Kalfa TA, Malik P, Quinn CT, Ware RE, Vinks AA. Robust clinical and laboratory response to hydroxyurea using pharmacokinetically guided dosing for young children with sickle cell anemia. Am J Hematol. 2019 Aug;94(8):871-879. doi: 10.1002/ajh.25510. Epub 2019 Jun 12.
- Background: PubMed 32869281Dong M, McGann PT. Changing the Clinical Paradigm of Hydroxyurea Treatment for Sickle Cell Anemia Through Precision Medicine. Clin Pharmacol Ther. 2021 Jan;109(1):73-81. doi: 10.1002/cpt.2028. Epub 2020 Oct 8.
- Background: PubMed 28847416Marahatta A, Ware RE. Hydroxyurea: Analytical techniques and quantitative analysis. Blood Cells Mol Dis. 2017 Sep;67:135-142. doi: 10.1016/j.bcmd.2017.08.009. Epub 2017 Aug 8.
- Background: PubMed 28131939Hai X, Guo M, Gao C, Zhou J. Quantification of hydroxyurea in human plasma by HPLC-MS/MS and its application to pharmacokinetics in patients with chronic myeloid leukaemia. J Pharm Biomed Anal. 2017 Apr 15;137:213-219. doi: 10.1016/j.jpba.2017.01.008. Epub 2017 Jan 10.
- Background: PubMed 21571150Wang WC, Ware RE, Miller ST, Iyer RV, Casella JF, Minniti CP, Rana S, Thornburg CD, Rogers ZR, Kalpatthi RV, Barredo JC, Brown RC, Sarnaik SA, Howard TH, Wynn LW, Kutlar A, Armstrong FD, Files BA, Goldsmith JC, Waclawiw MA, Huang X, Thompson BW; BABY HUG investigators. Hydroxycarbamide in very young children with sickle-cell anaemia: a multicentre, randomised, controlled trial (BABY HUG). Lancet. 2011 May 14;377(9778):1663-72. doi: 10.1016/S0140-6736(11)60355-3.
- Background: PubMed 22915643Thornburg CD, Files BA, Luo Z, Miller ST, Kalpatthi R, Iyer R, Seaman P, Lebensburger J, Alvarez O, Thompson B, Ware RE, Wang WC; BABY HUG Investigators. Impact of hydroxyurea on clinical events in the BABY HUG trial. Blood. 2012 Nov 22;120(22):4304-10; quiz 4448. doi: 10.1182/blood-2012-03-419879. Epub 2012 Aug 22.
- Background: PubMed 30206297Yahouedehou SCMA, Adorno EV, da Guarda CC, Ndidi US, Carvalho SP, Santiago RP, Aleluia MM, de Oliveira RM, Goncalves MS. Hydroxyurea in the management of sickle cell disease: pharmacogenomics and enzymatic metabolism. Pharmacogenomics J. 2018 Dec;18(6):730-739. doi: 10.1038/s41397-018-0045-1. Epub 2018 Sep 12.
- Background: PubMed 16713922Switzer JA, Hess DC, Nichols FT, Adams RJ. Pathophysiology and treatment of stroke in sickle-cell disease: present and future. Lancet Neurol. 2006 Jun;5(6):501-12. doi: 10.1016/S1474-4422(06)70469-0.
- Background: PubMed 24237976Quinn CT. Sickle cell disease in childhood: from newborn screening through transition to adult medical care. Pediatr Clin North Am. 2013 Dec;60(6):1363-81. doi: 10.1016/j.pcl.2013.09.006.
- Background: PubMed 24589261Hebbel RP. Ischemia-reperfusion injury in sickle cell anemia: relationship to acute chest syndrome, endothelial dysfunction, arterial vasculopathy, and inflammatory pain. Hematol Oncol Clin North Am. 2014 Apr;28(2):181-98. doi: 10.1016/j.hoc.2013.11.005.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: No. This is a pilot study with a small group of participant.
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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.
Bianka Courcelle, Research nurse, RN contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Pharmacokinetic based dosage change and Pharmacokinetic dosing in people with Sickle Cell Disease (SCD), run by Yves Pastore at 1 site in Canada.
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 10 inclusion and 9 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: looking for participants now, as of the sponsor's last update on 21 Aug 2026. 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 Canada. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yves Pastore, MD (514-345-4931 ext. 5027, yves.pastore.med@ssss.gouv.qc.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluation of HU-PK at 6 months between group A and group B (time frame: At 6 months). 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 NCT06761560, which also lists 14 linked publications.
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 21 Aug 2026.