Alternative Dosing And Prevention of Transfusions (ADAPT)

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

EARLY_PHASE1

Total Enrollment

100 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-06-16

Study Completion Date

2027-12-31

Brief Summary

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ADAPT is a prospective cohort study at Jinja Regional Referral Hospital (JRRH) primarily to assess the effect of hydroxyurea on blood transfusion utilization and secondarily to determine the feasibility of PK-guided hydroxyurea dosing.

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Detailed Description

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Hypothesis

* There will be a 50% reduction in the rate of blood transfusions received during the hydroxyurea treatment period compared with the pre-treatment period.
* A PK-guided starting dose will be generated for 80% of participants.
* Participants on PK-guided hydroxyurea treatment will require 25% fewer blood transfusions during their first year of hydroxyurea than those on dose escalation.

Conditions

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Sickle Cell Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

ADAPT is a prospective cohort study at Jinja Regional Referral Hospital (JRRH) primarily to assess the effect of hydroxyurea on blood transfusion utilization and secondarily to determine the feasibility of PK-guided hydroxyurea dosing.

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Treatment

All participants will receive an individualized PK hydroxyurea assessment. Participants for whom the PK-process successfully generates a dose in the predicted treatment range of 15-35 mg/kg/day, will start on that personalized dose. Participants for whom the process does not generate a starting hydroxyurea dose in the predicted treatment range, due to potential pitfalls in lab draws, serum storage, sample processing, or hydroxyurea analysis, will start at a default dose of 20.0 ± 2.5 mg/kg/day. For all participants, the hydroxyurea dose will be adjusted as needed based on blood counts to establish the optimal dose. Where necessary, a weekly dosing average will be determined, so that treatment can occur solely with locally available and affordable 500mg hydroxyurea capsules.

Group Type EXPERIMENTAL

Hydroxyurea

Intervention Type DRUG

All participants will receive an individualized PK hydroxyurea assessment. Participants for whom the PK-process successfully generates a dose in the predicted treatment range of 15-35 mg/kg/day, will start on that personalized dose. Participants for whom the process does not generate a starting hydroxyurea dose in the predicted treatment range, due to potential pitfalls in lab draws, serum storage, sample processing, or hydroxyurea analysis, will start at a default dose of 20.0 ± 2.5 mg/kg/day. For all participants, the hydroxyurea dose will be adjusted as needed based on blood counts to establish the optimal dose. Where necessary, a weekly dosing average will be determined, so that treatment can occur solely with locally available and affordable 500mg hydroxyurea capsules.

Interventions

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Hydroxyurea

All participants will receive an individualized PK hydroxyurea assessment. Participants for whom the PK-process successfully generates a dose in the predicted treatment range of 15-35 mg/kg/day, will start on that personalized dose. Participants for whom the process does not generate a starting hydroxyurea dose in the predicted treatment range, due to potential pitfalls in lab draws, serum storage, sample processing, or hydroxyurea analysis, will start at a default dose of 20.0 ± 2.5 mg/kg/day. For all participants, the hydroxyurea dose will be adjusted as needed based on blood counts to establish the optimal dose. Where necessary, a weekly dosing average will be determined, so that treatment can occur solely with locally available and affordable 500mg hydroxyurea capsules.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Patients with documented HbSS disease
* Age: ≥ 12 months and ≤ 10 years of age, at the time of enrollment
* Parent or guardian willing and able to provide informed consent
* Able to comply with all study related treatments, evaluations, and follow-up

Exclusion Criteria

* Current hydroxyurea treatment (or within the past 6 months)
* Regular blood transfusions (6 or more within the past 12 months)
* Transfusion within the last 30 days (temporary exclusion)
* Known malignancy or other known chronic illnesses including but not limited to active tuberculosis, renal disease
* Current participation in other therapeutic clinical trials, or within 6 months of prior disease-modifying treatments

Minimum Eligible Age

12 Months

Maximum Eligible Age

10 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No