Hydroxyurea for Sickle Cell Anemia
(HOPS Trial)

Recruiting in Palo Alto (17 mi)

+12 other locations

Overseen byPatrick Niss, MD

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Children's Hospital Medical Center, Cincinnati

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Approved in 3 Jurisdictions

Trial Summary

What is the purpose of this trial?

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.

Research Team

Patrick Niss, MD

Principal Investigator

Lifespan

Eligibility Criteria

This trial is for children and young adults aged 6 months to 21 years with severe forms of sickle cell anemia who have decided, along with their healthcare providers, to start treatment with hydroxyurea. It's not open to those currently on chronic blood transfusions or erythrocytapheresis, pregnant or lactating individuals, patients treated with hydroxyurea in the past 3 months, or those taking other investigational drugs for sickle cell.

Inclusion Criteria

You have sickle cell anemia.

You are 6 months to 21 years old.

You and your healthcare providers have decided to start treatment with hydroxyurea.

Exclusion Criteria

Hemoglobin SC disease, HbS/β+-thalassemia

Current known pregnancy or lactation

You are currently receiving regular blood transfusions or erythrocytapheresis treatments.

See 2 more

Treatment Details

Interventions

Hydroxyurea (Anti-metabolites)

Trial OverviewThe HOPS trial is testing a new way of dosing hydroxyurea based on individual drug processing (pharmacokinetics) against the usual method based on body weight in kids with sickle cell anemia. The goal is to see if this new strategy leads to better levels of fetal hemoglobin compared to standard dosing.

Participant Groups

2Treatment groups

Experimental Treatment

Active Control

Group I: Alternative ArmExperimental Treatment1 Intervention

Participants randomized to the alternative arm will receive a pharmacokinetic guided starting dose of hydroxyurea based on PK labs drawn at a baseline visit to target an area under the curve (AUC) of 115 mg\*h/L in an attempt to approximate maximum tolerated dose (MTD). This dose will not exceed the maximum tolerated dose of 35 mg/kg/day.

Group II: Standard ArmActive Control1 Intervention

Participants randomized to the standard arm will receive a starting dose of hydroxyurea of 20 mg/kg/day.

Hydroxyurea is already approved in Canada for the following indications: