Sickle Cell Disease - Stroke Prevention in Nigeria Trial (SPIN)
|ClinicalTrials.gov Identifier: NCT01801423|
Recruitment Status : Active, not recruiting
First Posted : February 28, 2013
Last Update Posted : March 28, 2017
|Condition or disease||Intervention/treatment|
|Sickle Cell Anemia Sickle Cell Disease Stroke||Drug: Hydroxyurea|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||60 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Primary Prevention of Strokes in Nigerian Children With Sickle Cell Disease Affiliated Titles: Sickle Cell Disease - Stroke Prevention in Nigeria (SPIN) Trial|
|Study Start Date :||February 2013|
|Estimated Primary Completion Date :||September 2021|
|Estimated Study Completion Date :||September 2021|
U.S. FDA Resources
Study investigators propose to enroll 60 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)
- Hydroxyurea Therapy Acceptance and Adherence [ Time Frame: 5 years ]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 ]Study investigators 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) Study investigators expect the proportion of serious adverse reactions, as well as hydroxyurea-related morbidity and mortality, to be very small compared to the benefits. Study investigators will compare the frequency of severe adverse events and hydroxyurea toxicity related events that are associated with hospitalization in those receiving hydroxyurea (n= 60) 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 ]During the course of the current study, study investigators will prepare a manual of operations and case report forms for the proposed trial. Investigators 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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01801423
|Aminu Kano Teaching Hospital|
|Kano, Nigeria, P.MB. 3452|
|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|