Optimizing Hydroxyurea Therapy in Children With SCA In Malaria Endemic Areas

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03128515
• Date of registration: 22/03/2017
• Prospective Registration: Yes
• Primary sponsor: Indiana University
• Public title: Optimizing Hydroxyurea Therapy in Children With SCA In Malaria Endemic Areas NOHARM-MTD
• Scientific title: Optimizing Hydroxyurea Therapy in Children With Sickle Cell Anemia In Malaria Endemic Areas: The NOHARM Maximum Tolerated Dose (MTD) Study
• Date of first enrolment: July 26, 2017
• Target sample size: 187
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03128515
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Uganda

Contacts

• Name: Chandy C John, M.D.
• Affiliation: Indiana University

Key inclusion & exclusion criteria

Inclusion Criteria:

- Children with confirmed SCA who participated in the NOHARM study of hydroxyurea at the
Mulago Hospital Sickle Cell Clinic (MHSCC), will be eligible for the MTD study after
completing both 12-months of blinded study treatment and then an additional 12-months
of open-label hydroxyurea for the second year of the study.

- The age range for enrollment into NOHARM, which began in 2014, was 1-4 years.
Therefore, the children who will be enrolled in the follow up MTD study will be 3-6
years of age.

Exclusion Criteria:

- Not willing to come for all scheduled clinical visits or accept randomization



Age minimum: 24 Months
Age maximum: 72 Months
Gender: All

Health Condition(s) or Problem(s) studied

Sickle Cell Disease

Malaria

Sickle Cell Anemia

Intervention(s)

Drug: Hydroxyurea

Primary Outcome(s)

Proportion of children with average hemoglobin =9.0 g/dL or average HbF =20% [Time Frame: Over 24 month period on study drug]

Secondary Outcome(s)

Clinical malaria incidence [Time Frame: Over 24 month period on study drug]

Change in weight-for-height z-score [Time Frame: Over 24 month period on study drug]

Incidence of hematologic toxicities [Time Frame: Over 24 month period on study drug]

Change in creatinine levels [Time Frame: Over 24 month period on study drug]

Cerebrovascular function [Time Frame: At study treatment initiation then at 12 months and 24 months after study initiation]

Change in weight-for-age z-score [Time Frame: Over 24 month period on study drug]

Vaso-occlusive crises [Time Frame: Over 24 month period on study drug]

Change in cystatin C [Time Frame: Over 24 month period on study drug]

Change in height-for-age z-score [Time Frame: Over 24 month period on study drug]

Change in splenic function [Time Frame: Over 24 month period on study drug]

Incidence of severe adverse events (SAE) [Time Frame: Over 24 month period on study drug]

Secondary ID(s)

00456728

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Mulago Hospital, Uganda

Doris Duke Charitable Foundation

Makerere University

Children's Hospital Medical Center, Cincinnati