Sickle Cell Disease - Stroke Prevention in Nigeria Trial (SPIN)
Given large absolute numbers of individuals with sickle cell disease in Nigeria, hydroxyurea therapy for all individuals with sickle cell disease may not be initially feasible; however, a targeted strategy of hydroxyurea use for primary prevention of strokes is an alternative to the standard therapy (observation) for high-risk individuals. The investigators propose a feasibility study, Sickle Cell Disease - Stroke Prevention in Nigeria (SPIN) Trial, to determine whether hydroxyurea can be used for primary prevention of strokes in Nigerian children with sickle cell anemia.
Sickle Cell Anemia
Sickle Cell Disease
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Sickle Cell Disease - Stroke Prevention in Nigeria Trial|
- Hydroxyurea Therapy Acceptance and Adherence [ Time Frame: 3 years ] [ Designated as safety issue: No ]The primary outcome measure will be adherence to daily administration of hydroxyurea. If adherence rate is less than 55%, alternative strategies must be considered for the definitive Phase III Trial.
- Hydroxyurea Safety protocol for Children with Sickle Cell Anemia [ Time Frame: 12 Months ] [ Designated as safety issue: No ]We will evaluate the use of a standard safety protocol, non-dose escalating, for hydroxyurea in children with sickle cell anemia using a protocol similar to the recently completed National Heart Lung and Blood Institute (NHLBI) Baby HUG study, published in Lancet.(1) We expect the proportion of serious adverse reactions, as well as hydroxyurea-related morbidity and mortality, to be very small compared to the benefits. We will compare the frequency of severe adverse events and hydroxyurea toxicity related events that are associated with hospitalization in those receiving hydroxyurea (n= 40) to those who had normal transcranial Doppler measurements (n= 210) over the course of one year.
- Feasibility of a Definitive Phase III Trial for Hydroxyurea Therapy to Prevent Strokes in Sickle Cell Disease [ Time Frame: 24 Months ] [ Designated as safety issue: No ]During the course of the current study, we will prepare a manual of operations and case report forms for the proposed trial. We will also solidify working relationships with our colleagues and collaborators at Aminu Kano Teaching Hospital in Kano, Nigeria; and develop and organize all committees, collaborators and study procedures necessary for initiation of a successful, definitive, Phase III Trial.
|Study Start Date:||February 2013|
|Estimated Study Completion Date:||September 2015|
|Estimated Primary Completion Date:||September 2015 (Final data collection date for primary outcome measure)|
We propose to enroll 40 children with SCA and an elevated TCD measurement between 5 and 12 years of age in this one arm feasibility study of hydroxyurea therapy, with follow-up of at least 12 months per subject. The study intervention will include HU to begin at ~ 20 mg/kg/day(range 17.5 - 26 mg/kg/day). No dose escalation will occur. Given the success of the first year of enrollment and the favorable response of TCD measurement after 3 months on HU therapy, the study investigators have participants as an internal pilot. The definitive phase III trial will now compare low dose HU therapy to the result of no treatment arm from the STOP Trial.
Hydroxyurea will be prescribed as an investigational therapy by the treating physician. Recommended guidelines for titration of hydroxyurea to maximal tolerated dose are below. The study intervention will include hydroxyurea to begin at ~20 mg/kg/day (range 17.5 - 26 mg/kg/day). No dose escalation will occur as this dose was shown to have some efficacy in infants with SCA and was associated with rare myelosuppression.(1)
Please refer to this study by its ClinicalTrials.gov identifier: NCT01801423
|Contact: Michael R. DeBaun, MD, MPHfirstname.lastname@example.org|
|Aminu Kano Teaching Hospital||Recruiting|
|Kano, Nigeria, P.MB. 3452|
|Contact: Najibah Galadanchi, MBBS, FMCPath 234-803-700-5452 email@example.com|
|Principal Investigator: Najibah Galadanchi, MBBS,FMCPath|
|Principal Investigator:||Michael R. DeBaun, MD, MPH||Vanderbilt University|
|Principal Investigator:||Muktar Aliyu, MBBS, MPH, DrPH||Vanderbilt University|
|Principal Investigator:||Lori Jordan, MD, PhD||Vanderbilt University|