Comparing Pharmacokinetic based dosage change with Pharmacokinetic dosing for sickle cell disease (SCD)
Official title: Optimizing Hydroxyurea Dosage With Pharmakokinetic in Patients Suffering of Moderate to Severe Sickle Cell Anemia
- Phase 2
- 3 groups
- One site, in Montreal
- Recruiting
Interventions
- Other intervention
Pharmacokinetic based dosage change
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
- Other intervention
Pharmacokinetic dosing
Patient with sickle cell disease will undergo one pharmacokinetic test after taking 12 months of hydroxyurea to evaluate HU-AUC at that timepoint
Canadian Sites (1)
1 of 1 recruiting
- Recruiting
CHU Sainte-Justine
Montreal, Quebec
Eligibility Criteria
See who this study is looking for20 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +(Group A and B) :
- +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.
- +Inclusion CriteriaInclusion criteriaThe things you must have or be for a study to consider you.Read more → (Group C) :
- +Have had confirmed diagnosis of SCD at CHU Sainte-Justine biochemistry lab with hemoglobin electrophoresis.
- +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 MTDMaximum tolerated doseThe highest amount that can be given before side effects become unacceptable.Read more → for at least 6 months.
- +Be between age of 6months old and 18 years old.
- +Have consented for participation in the study.
Exclusion
- −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 enrollmentEnrolmentThe number of participants a study plans to include, or has included.Read more →.
- −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 controlControl groupThe group a new treatment is measured against.Read more →
- −Conditions which in the opinion of the investigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more →, would compromise participation in the study will be excluded.
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study does not fall into the standard research categories.
Phase 2Phase 2A middle-stage study looking at what a treatment does and watching for side effects.Read more →/3 — a combined study that runs the middle stage and the large comparison stage together.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
There are 3 groups in this study.
Group A receives PharmacokineticPharmacokineticsThe study of how the body absorbs, distributes, and clears a treatment.Read more → based dosage change.
Registry label: A: Group A
Group B receives no study treatment and is followed for comparison.
Registry label: B: Group B
Group C receives Pharmacokinetic dosing.
Registry label: C: Group C
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
One group receives no study treatment at all, and is followed for comparison.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study lists an age range of 6 months to 18 years.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 29 people.
The study is currently expected to finish around November 2028.
The main measurement is taken over: At 6 months.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Evaluation of HU-PKPharmacokineticsThe study of how the body absorbs, distributes, and clears a treatment.Read more → at 6 months between group A and group B — measured over At 6 months.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT06761560 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About 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
From the trial registry — its own words, unedited.
What a diagnostic test is here: A scan, blood test, or other test being studied for how well it detects or measures something.
Read the full explanation → · in clinical review
About 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
From the trial registry — its own words, unedited.
What a diagnostic test is here: A scan, blood test, or other test being studied for how well it detects or measures something.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: an age range of 6 months to 18 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
One group receives no study treatment and is followed for comparison.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists an age range of 6 months to 18 years.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: At 6 months.
The study as a whole is currently expected to finish around 2028-11-25.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 29 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists one location: Montreal, Quebec, Canada.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About This Trial
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.
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See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.