NCT04750707CompletedPhase 3
Hydroxyurea Therapy for Neurological and Cognitive Protection in Pediatric Sickle Cell Anemia in Uganda ( BRAINSAFE-II )
Hydroxyurea Therapy for Neurological and Cognitive Protection in Pediatric Sickle Cell Anemia in Uganda: A Single Arm Open Label Trial, "BRAIN SAFE II"
Source: ClinicalTrials.gov · Last updated 17 Apr 2025
Brief summary (registry, in English)
Worldwide, an estimated 200,000 babies are born with Sickle Cell Anemia (SCA) annually. Affected children suffer chronic ill health with some having frequent hospitalization. The patients are also at a high risk of brain injury arising from small and large cerebral blood vessel damage in SCA, also called sickle cell vasculopathy (SCV). SCV is associated with the high risk of stroke. Such injury may manifest with neurological and cognitive impairment. An abnormal blood flow to the brain, as measured by a Doppler Ultrasound scan is a known risk factor for stroke. The hypothesis is that hydroxyurea therapy will prevent, stabilize or improve SCV and its effects. The study is an open label, single arm clinical trial to test the impact of hydroxyurea treatment in 270 children with SCA starting at ages 3-9 years. Following baseline assessments, all participants will begin hydroxyurea therapy starting at about 20mg/kg/day. Changes in the frequency and severity of each test (neurological and cognitive tests and cerebral blood flow velocity) will be compared with their baseline tests (prior to hydroxyurea) by repeating these tests at 18 and 36 months. In a randomly selected subset of 90 participants, an evaluation of the impact of hydroxyurea on structural brain vascular injury using magnetic resonance brain imaging (MRI) and magnetic vessel imaging ,also called angiography (MRA) at baseline and at 36 months. Lastly, an assessment of changes to biomarkers of anemia, inflammation and malnutrition from before and during hydroxyurea therapy and determine their relationship to the outcomes. The proposed intervention with hydroxyurea is the first Africa-based trial to broadly prevent or ameliorate manifestations of SCV.
- Status (registry)
- Completed
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 270 (actual)
- First submitted
- 25 Jan 2021
- Start date
- 9 Mar 2021
- Primary completion date
- 10 Mar 2025
- Lead sponsor
- Global Health Uganda LTD
- Diseases
- Sickle cell disease
- NCT number
- NCT04750707
What you commit to
- Study type: Interventional · Phase 3
- Participants enrolled: 270
- From 9 Mar 2021 to the primary completion date, 10 Mar 2025
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Prior stroke as detected by standard Pediatric NIH Stroke Scale (PedNIHSS) examination
- Temporary exclusion criteria
- History of a sickle crisis within the prior 2 weeks, or blood transfusion within the past 90 days Participants with temporary exclusion can be enrolled later when stable or the excluding criteria has resolved
Other illness
- Found to be with acute illness at enrolment with fever in last 1 week, respiratory infection, etc.
Lab values
- Pre-existing hematological toxicity
- Hemoglobin \<4.0 gm/dL
- Hemoglobin \<6.0 gm/dL with Absolute Reticulocyte Count \<100 x 10 9/L
- Absolute Reticulocyte Count \<80 x 109/L with Hemoglobin \<7.0 gm/dL
- Platelets \<80 x 109/L
- Absolute Neutrophil Count \<1.0 x 109/L
Age
- History of neurological abnormality known before age 4 months (to avoid those with non SCA brain injury)
Other
- Child is currently enrolled in another clinical intervention trial
Who the study is looking for
- Documented laboratory diagnosis of HbSS or HbS-B 0 thalassemia (both types are treated equally in SCA studies)
- Ages 3 through 9 years (inclusive) at enrolment
- To ensure clinic follow-up, child has attended Mulago Hospital Sickle Cell Anaemia (SCA)clinic 2 times in the prior 4 years, or at least once in the past 2 years if younger than \< 4 years
- No history of hydroxyurea use for longer than 6 months
- Parent/legal guardian has provided a written consent (and if child is ≥8 years of age, has provided assent)
Age in the registry: 3 years to 9 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Global Health Uganda Coordinator of this site? Confirm its status | Kampala | Uganda | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.