Hydroxyurea Optimization Through Precision Study

  • STATUS
    Recruiting
  • End date
    Dec 31, 2022
  • participants needed
    116
  • sponsor
    Children's Hospital Medical Center, Cincinnati
Updated on 7 April 2021
anemia
hydroxyurea
thalassemia
sickle hemoglobin
hemoglobin f

Summary

Hydroxyurea Optimization through Precision Study (HOPS) is a prospective, multi-center, randomized trial that will directly compare a novel, individualized dosing strategy of hydroxyurea to standard weight-based dosing for children with SCA. The primary objective of the study is to evaluate whether a pharmacokinetics-based starting hydroxyurea dose thieves superior fetal hemoglobin response to to standard weight-based initial dosing. Patients will be recruited from the pediatric sickle cell clinic at Cincinnati Children's Hospital Medical Center and from additional pediatric sickle cell centers within the United States.

Description

The trial will recruit patients who have decided to initiate hydroxyurea therapy. All participants will have pharmacokinetics studies performed at baseline, following a 20 mg/kg oral dose of hydroxyurea. Pharmacokinetic sampling will use a sparse sampling approach, requiring collection of blood at 3 time points (15 minutes, 60 minutes, 180 minutes) following the hydroxyurea dose. Enrolled participants will be randomized to receive either hydroxyurea using a starting dose of 20 mg/kg/day (Standard Arm) or a personalized PK-guided dose (Alternative Arm) to target an area under the curve (AUC) of 115 mg*h/L based to approximate hydroxyurea exposure seen when patients are escalated to maximum tolerated dose (MTD).

Following randomization and selection of the initial dose, participants in both arms will follow the same procedures of laboratory medication holds for hematological toxicity. The primary endpoint is fetal hemoglobin (HbF) six months following the initiation of hydroxyurea therapy with the hypothesis that participants starting with a PK-guided dose will achieve HbF at least 5% greater than those starting with a 20 mg/kg dose. Based upon the estimated number of new hydroxyurea starts at each site, it is anticipated that it will take 24 months to enroll the 116 participants required to achieve sufficient power to assess the primary endpoint. The study will conclude for each participant 12 months following hydroxyurea initiation.

Details
Condition SICKLE CELL ANEMIA, Sickle Cell Disease
Treatment hydroxyurea
Clinical Study IdentifierNCT03789591
SponsorChildren's Hospital Medical Center, Cincinnati
Last Modified on7 April 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Is your age between 1 yrs and 21 yrs?
Gender: Male or Female
Do you have any of these conditions: Sickle Cell Disease or SICKLE CELL ANEMIA?
Do you have any of these conditions: SICKLE CELL ANEMIA or Sickle Cell Disease?
Do you have any of these conditions: Sickle Cell Disease or SICKLE CELL ANEMIA?
Do you have any of these conditions: Sickle Cell Disease or SICKLE CELL ANEMIA?
Do you have any of these conditions: Sickle Cell Disease or SICKLE CELL ANEMIA?
Do you have any of these conditions: Sickle Cell Disease or SICKLE CELL ANEMIA?
Diagnosis of sickle cell anemia (HbSS, HbSD, HbS/0-thalassemia, or similarly severe SCA genotype)
Age 6 months to 21 years at the time of enrollment
Clinical decision by patient, family, and healthcare providers to initiate hydroxyurea therapy

Exclusion Criteria

Current treatment with chronic, monthly blood transfusions or erythrocytapheresis
Treatment with hydroxyurea within the past 3 months
Hemoglobin SC disease, HbS/+-thalassemia
Current treatment with other investigational sickle cell medications
Current known pregnancy or lactation
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