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Optimizing Hydroxyurea Therapy in Children With Sickle Cell Anemia In Malaria Endemic Areas: The NOHARM Maximum Tolerated Dose (MTD) Study

NCT07708714 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 250

Last updated 2026-07-16

No results posted yet for this study

Summary

NOHARM MTD is an extension of a previous study for children with Sickle Cell Anemia (SCA) who were enrolled in the NOHARM study. All children enrolled in NOHARM received hydroxyurea treatment at a fixed daily dose of 20 mg/kg/day. This dose was selected as a likely safe dose, but does not escalate hydroxyurea to maximum tolerated dose "MTD" as is commonly done in the US. Without this information, we cannot know whether hydroxyurea treatment at the MTD would be feasible (since it requires closer monitoring to avoid hematological toxicities), safe (since adverse events may be greater with MTD, risk of malaria may be altered by MTD, and risk of infections as a result of neutropenia could also be greater with MTD) or beneficial (MTD is associated with higher hemoglobin and fetal hemoglobin concentration).

Conditions

Interventions

DRUG

hydroxycarbamide

Hydroxyurea at optimized dose

Sponsors & Collaborators

  • Makerere University

    collaborator OTHER

  • Indiana University

    collaborator OTHER

  • Indian Institute of Science

    collaborator OTHER_GOV

  • Mulago Hospital, Uganda

    collaborator OTHER

  • Children's Hospital Medical Center, Cincinnati

    lead OTHER

Principal Investigators

  • Russell E. Ware, MD, PhD · Children's Hospital Medical Center, Cincinnati

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Model
SINGLE_GROUP

Eligibility

Min Age
11 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-05-13
Primary Completion
2030-11-30
Completion
2032-11-30

Countries

  • Uganda

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07708714 on ClinicalTrials.gov