BrUOG 419 - Promoting Utilization and Safety of Hydroxyurea Using Precision in Africa (PUSHUP)

Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY

Sickle cell anemia (SCA) is among the world's most common and devastating blood disorders, affecting more than 300,000 newborns per year. Most infants with SCA are born in the low-resource settings of sub- Saharan Africa, where an estimated 50-90% will die before 5 years of age due to lack of early diagnosis and appropriate care. Hydroxyurea is a safe and effective once-daily oral medication that has become the standard of care for the treatment of children with SCA in high-resource settings. There is now a growing body of evidence to support the safety and clinical benefits of hydroxyurea for the treatment of SCA in sub-Saharan Africa. The requirement for frequent laboratory monitoring, uncertainties about appropriate, most effective dosing, and the concern for hematologic laboratory toxicities, however, will continue to limit widespread hydroxyurea utilization and real-world effectiveness. The investigators have recently developed and prospectively evaluated an individualized, pharmacokinetics-guided hydroxyurea dosing strategy for children with SCA that has demonstrated optimal clinical and laboratory benefits with minimal toxicity. In this research study, the investigators aim to extend this precision medicine approach to Africa.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 6 months
Maximum Age: 12
Healthy Volunteers: f
View:

• Diagnosis of sickle cell anemia (HbSS or HbS/B0-thalassemia)

• Age 6 months- 12 years of age at enrollment

• Parent or guardian willing and able to provide written or informed consent

• Weight ≥ 7.5 kg (temporary exclusion)

Locations
Other Locations
Angola
Hospital Geral dos Cajueiros
RECRUITING
Luanda
Contact Information
Primary
BrUOG
BrUOG@brown.edu
401-863-3000
Time Frame
Start Date: 2023-11-15
Estimated Completion Date: 2027-09-01
Participants
Target number of participants: 400
Treatments
Active_comparator: Weight Based Starting Dose
25 mg/kg starting dose Hydroxyurea
Experimental: PK-guided starting dose
Individualized, PK-guided starting dose Hydroxyurea
Sponsors
Leads: Brown University
Collaborators: National Heart, Lung, and Blood Institute (NHLBI), Novartis

This content was sourced from clinicaltrials.gov