NCT06526117

Brief Summary

The primary goal of this study is to complete a multicenter single-arm, type I hybrid trial to assess the effectiveness of hydroxyurea therapy for primary stroke prevention in high-risk children with sickle cell anemia (SCA) living in Nigeria in routine care settings.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
220

participants targeted

Target at P75+ for phase_4

Timeline
51mo left

Started Aug 2024

Longer than P75 for phase_4

Geographic Reach
1 country

2 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress30%
Aug 2024Jun 2030

First Submitted

Initial submission to the registry

July 23, 2024

Completed
6 days until next milestone

First Posted

Study publicly available on registry

July 29, 2024

Completed
7 days until next milestone

Study Start

First participant enrolled

August 5, 2024

Completed
5.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2029

Expected
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

June 30, 2030

Last Updated

March 5, 2026

Status Verified

March 1, 2026

Enrollment Period

5.4 years

First QC Date

July 23, 2024

Last Update Submit

March 2, 2026

Conditions

Keywords

primary stroke preventionhydroxyurealow income countrysub-Saharan Africa

Outcome Measures

Primary Outcomes (1)

  • Incidence Rate of Clinical Stroke or TIA

    The incidence rate of clinical stroke or TIA in participants treated with low- or moderate-dose hydroxyurea.

    5 years

Secondary Outcomes (1)

  • Incidence of All-Cause Hospitalizations

    5 years

Study Arms (1)

Low or Moderate Dose Hydroxyurea

EXPERIMENTAL

Initial treatment with Hydroxyurea at 10 mg/kg/day (range 7 to 15 mg/kg/day) for primary stroke prevention. Subsequent treatment with moderate-dose hydroxyurea (20 mg/kg/day (range 17.5 - 26 mg/kg/day)) based after at least two severe pain events requiring physician contact during the trial.

Drug: Hydroxyurea

Interventions

The study intervention will include initial treatment with low-dose hydroxyurea therapy at 10 mg/kg/day (range 7 - 15 mg/kg/day), with subsequent increase to moderate dose hydroxyurea therapy at 20 mg/kg/day (range 17.5 - 26 mg/kg/day) after at least two severe events requiring physician contact.

Also known as: Hydrea
Low or Moderate Dose Hydroxyurea

Eligibility Criteria

Age5 Years - 12 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Diagnosis of HbSS or HbSB0 confirmed by high-performance liquid chromatography (HPLC);
  • Informed consent from the parent/legal guardian and assent from the patient at least 7 years of age;
  • Two TCD flow velocity readings of \>or equal to180 cm/sec and \< 220 cm/sec or one TCD velocity reading \> or equal to 220 cm/sec; typically the repeat TCD is performed on the same day so treatment can start immediately;
  • Age between 5 and 12 years (assessment can take place up until the 13th birthday), which includes the peak age of onset of strokes in SCA, \~ 6 yo; and
  • Ability to swallow the hydroxyurea capsule.

You may not qualify if:

  • Prior stroke or TIA by history, or concern for moderate or severe neurological deficit based on a positive validated "10 questions" screening;
  • Other significant organ system dysfunction or other contraindication to hydroxyurea;
  • Children who are already on therapy with either blood transfusion or hydroxyurea therapy;
  • Significant cytopenias (absolute neutrophil count (ANC) \<1500, platelets \<150,000/ul, reticulocytes \<80,000/ul, unless Hb is \> 9 g/dl\], renal insufficiency (creatinine \> 0.8 mg/dl); and
  • History of seizures or diagnosis of epilepsy, and 6) metal in the body that would make MRI unsafe. The rationale for excluding children under 5 years old: Despite being a vulnerable age group for strokes, children younger than 5 years were excluded because a significant proportion of this population is unable to swallow a capsule, the only stable form of hydroxyurea available in Nigeria.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Aminu Kano Teaching Hospital

Kano, Nigeria

RECRUITING

Murtala Muhammad Specialist Hospital

Kano, Nigeria

RECRUITING

Related Publications (2)

  • Adams RJ, McKie VC, Hsu L, Files B, Vichinsky E, Pegelow C, Abboud M, Gallagher D, Kutlar A, Nichols FT, Bonds DR, Brambilla D. Prevention of a first stroke by transfusions in children with sickle cell anemia and abnormal results on transcranial Doppler ultrasonography. N Engl J Med. 1998 Jul 2;339(1):5-11. doi: 10.1056/NEJM199807023390102.

    PMID: 9647873BACKGROUND
  • Abdullahi SU, Jibir BW, Bello-Manga H, Gambo S, Inuwa H, Tijjani AG, Idris N, Galadanci A, Hikima MS, Galadanci N, Borodo A, Tabari AM, Haliru L, Suleiman A, Ibrahim J, Greene BC, Ghafuri DL, Rodeghier M, Slaughter JC, Kirkham FJ, Neville K, Kassim A, Trevathan E, Jordan LC, Aliyu MH, DeBaun MR. Hydroxyurea for primary stroke prevention in children with sickle cell anaemia in Nigeria (SPRING): a double-blind, multicentre, randomised, phase 3 trial. Lancet Haematol. 2022 Jan;9(1):e26-e37. doi: 10.1016/S2352-3026(21)00368-9.

    PMID: 34971579BACKGROUND

MeSH Terms

Conditions

Anemia, Sickle CellStroke

Interventions

Hydroxyurea

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCerebrovascular DisordersBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesVascular DiseasesCardiovascular Diseases

Intervention Hierarchy (Ancestors)

UreaAmidesOrganic Chemicals

Study Officials

  • Michael R DeBaun, MD, MPH

    Vanderbilt University Medical Center

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Michael R DeBaun, MD, MPH

CONTACT

Leshana Saint Jean, PHD

CONTACT

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NA
Masking
NONE
Purpose
PREVENTION
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor, Vice Chair, Chair, Clinical Research, Director, Vanderbilt/Meharry Center of SCD

Study Record Dates

First Submitted

July 23, 2024

First Posted

July 29, 2024

Study Start

August 5, 2024

Primary Completion (Estimated)

December 31, 2029

Study Completion (Estimated)

June 30, 2030

Last Updated

March 5, 2026

Record last verified: 2026-03

Data Sharing

IPD Sharing
Will not share

Locations