NCT03128515: NOHARM-MTD

Optimizing Hydroxyurea Therapy in Children With SCA In Malaria Endemic Areas

Completed Phase 3 Last updated 20 February 2020

What this trial tests

Phase 3 trial testing Hydroxyurea in Sickle Cell Anemia in 187 participants. Completed in 28 January 2020.

Timeline

26 July 2017

Primary endpoint
7 April 2019

28 January 2020

Quick facts

Lead sponsor	Indiana University
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	187
Start date	26 July 2017
Primary completion	7 April 2019
Estimated completion	28 January 2020
Sites	1 location across Uganda

Drugs / interventions tested

• Hydroxyurea — full drug profile →

Conditions studied

• Sickle Cell Anemia — all drugs for Sickle Cell Anemia →
• Sickle Cell Disease — all drugs for Sickle Cell Disease →
• Malaria — all drugs for Malaria →

Sponsor

Indiana University

Who can join

Adults 24 Months to 72 Months, any sex, with Sickle Cell Anemia or Sickle Cell Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The Novel use Of Hydroxyurea in an African Region with Malaria (NOHARM) study is the first placebo-controlled randomized clinical trial of hydroxyurea treatment in a malaria endemic region. NOHARM has now achieved full enrollment; all children have completed the blinded portion of the protocol and are in the open-label study treatment portion.

This extension study of maximum tolerated dose (MTD), addresses the next critical set of questions about the optimal dosing and monitoring of hydroxyurea treatment for children with SCA in low-resource settings. By providing guidance about optimal hydroxyurea treatment, the NOHARM MTD Study will directly inform policies that can transform the health of African children living with SCA.

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

1. Hydroxyurea Dose Escalation for Sickle Cell Anemia in Sub-Saharan Africa.
   John CC, Opoka RO, Latham TS, Hume HA, et al · · 2020 · cited 119× · PMID 32579813 · DOI 10.1056/nejmoa2000146
2. Effective use of hydroxyurea for sickle cell anemia in low-resource countries.
   Power-Hays A, Ware RE. · · 2020 · cited 45× · PMID 32205588 · DOI 10.1097/moh.0000000000000582
3. Hydroxyurea dose optimisation for children with sickle cell anaemia in sub-Saharan Africa (REACH): extended follow-up of a multicentre, open-label, phase 1/2 trial.
   Aygun B, Lane A, Smart LR, Santos B, et al · · 2024 · cited 21× · PMID 38701812 · DOI 10.1016/s2352-3026(24)00078-4
4. Changing the Clinical Paradigm of Hydroxyurea Treatment for Sickle Cell Anemia Through Precision Medicine.
   Dong M, McGann PT. · · 2021 · cited 18× · PMID 32869281 · DOI 10.1002/cpt.2028
5. Rationale, Development, and Validation of HdxSim, a Clinical Decision Support Tool for Model-Informed Precision Dosing of Hydroxyurea for Children with Sickle Cell Anemia.
   Power-Hays A, Dong M, Punt N, Mizuno T, et al · · 2024 · cited 11× · PMID 38018175 · DOI 10.1002/cpt.3119
6. Interventions for preventing silent cerebral infarcts in people with sickle cell disease.
   Estcourt LJ, Kimber C, Hopewell S, Trivella M, et al · · 2020 · cited 10× · PMID 32250453 · DOI 10.1002/14651858.cd012389.pub3
7. "Maximum tolerated dose" vs "fixed low-dose" hydroxyurea for treatment of adults with sickle cell anemia.
   Akingbola TS, Tayo BO, Ezekekwu CA, Sonubi O, et al · · 2019 · cited 8× · PMID 30663794 · DOI 10.1002/ajh.25412