Sickle Cell Disease - Stroke Prevention in Nigeria Trial (SPIN)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01801423|
Recruitment Status : Completed
First Posted : February 28, 2013
Last Update Posted : August 3, 2020
|Condition or disease||Intervention/treatment||Phase|
|Sickle Cell Anemia Sickle Cell Disease Stroke||Drug: Hydroxyurea||Not Applicable|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||29 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|
|Actual Study Start Date :||April 24, 2013|
|Actual Primary Completion Date :||January 31, 2019|
|Actual Study Completion Date :||January 31, 2019|
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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
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|