Optimizing Hydroxyurea Dosage With Pharmakokinetic in Patients Suffering of Moderate to Severe Sickle Cell Anemia

Phase 2

Not yet recruiting

• Conditions: Sickle Cell Disease (SCD)

• Registration Number: NCT06761560
• Lead Sponsor: Yves Pastore

Brief 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. 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-12-12
• Status Verified: 2025-01
• Study First Submitted QC: 2025-01-05
• Last Update Submitted: 2025-01-05
• Study First Posted: 2025-01-07
• Last Update Posted: 2025-01-07
• Study Start: 2025-01-15
• Primary Completion: 2027-11-25
• Study Completion: 2027-11-25

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 29

Inclusion Criteria

Not provided

Exclusion Criteria

• 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
• 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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Primary Outcome Measures

Name	Time	Method
Evaluation of HU-PK at 6 months between group A and group B	At 6 months	Pharmakocinetic dosage of hydroxyurea will be determined at 6 months in group A and group B. We hypothesize that HU-PK in group B may be lower (suboptimal) compared to group A.

Secondary Outcome Measures

Name	Time	Method
Time to reach maximal tolerated dose (MTD)	3, 6, 9 and 12 months	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)
Fetal hemoglobin	at 3, 6 and 12 months	Comparing fetal hemoglobin between group A and B
Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) in group A and B	From enrollment to 12 months	Evaluation of the incidence adverse events (AE) and serious adverse events (SAE) in both groups
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	At 12 months	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.

Trial Locations

Locations (1)

CHU Sainte-Justine; 🇨🇦 Montreal, Quebec, Canada