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Active, Not Recruiting

NCT Number: NCT05662098

Alternative Dosing And Prevention of Transfusions (ADAPT)

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies

Age range

12 month–10 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Early Phase 1

Primary location

Jinja Regional Referral Hospital (JRRH), Department of Paediatrics, Sickle Cell Clinic

Jinja, Uganda

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Hydroxyurea

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.

Primary outcomes

  1. To compare the rates of blood transfusions overall and by specific indications in children with sickle cell anaemia (SCA), prior to and during hydroxyurea treatment

    Time frame: One year (Enrollment - Month 15)

    The incidence rate ratio of transfusions overall and by specific indication during the screening phase as compared to the treatment phase

Secondary outcomes

  1. To determine clinical and laboratory factors associated with reduction in blood transfusions for children with SCA on hydroxyurea treatment

    Time frame: One year (Enrollment - Month 15)

    The relative risk of transfusion due to the most common clinical diagnoses and laboratory factors for children with SCA on hydroxyurea treatment.

Other outcomes

  1. To assess the feasibility and safety of a pharmacokinetic (PK)-based hydroxyurea dose within the predicted treatment range for Uganda

    Time frame: One year (Enrollment - Month 15)

    The percentage of successful PK-dosing assessments, defined as assessments completed in entirety resulting in the generation of a PK-guided starting dose.

    The incidence rate ratio of clinical and laboratory adverse events among those started on the PK-guided hydroxyurea dose during the screening phase compared with the treatment phase.

  2. To quantify rates of SCA-related complications (including stroke, sepsis, and pain) in participants receiving PK-guided hydroxyurea dosing and within the overall cohort on hydroxyurea treatment

    Time frame: One year (Enrollment - Month 15)

    The number of participants with sickle cell-related complications (including stroke, sepsis and pain) in participants receiving PK-guided hydroxyurea dosing compared to the rate of events in the default dosing group.

Sponsors and collaborators

Lead sponsor

Children's Hospital Medical Center, Cincinnati

Other

Collaborators

  • Jinja Regional Referral Hospital (JRRH), Sickle Cell Clinic, Jinja, Uganda

Registry information

Official study title

Alternative Dosing And Prevention of Transfusions (ADAPT): A Prospective Study to Reduce Transfusion Requirements for Children With Sickle Cell Anemia Using Pharmacokinetics-based Hydroxyurea Dosing

Important dates

Study start
2022
Primary completion
2026
Study completion
2027
First posted
Dec 22, 2022
Registry last updated
Apr 9, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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