Cooperative Assessment of Late Effects for SCD Curative Therapies
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age4-65
SponsorVanderbilt University Medical Center
About this trial
Sickle Cell Disease is one of the most common genetic diseases in the United States, occurring in approximately 1 in 400 births. Approximately 100,000 individuals are diagnosed with SCD in the United States. Mortality for children with SCD has decreased substantially over the past 4 decades, with \>99% of those born in high resource settings, including the United States, France, and England, now surviving to 18 years of age. However, the life expectancy of adults with SCD is severely shortened. Dysfunction of the heart, lung, and kidney is directly associated with decreased life expectancy. With the variety of curative therapies that are now available for SCD, long-term health outcomes studies are time-sensitive. As of now, efforts to determine long-term health outcomes following curative therapies for SCD have been limited. Though curative therapies initially should provide a cure for symptoms of SCD, there is the risk of late health outcomes to consider. Defining health outcomes following curative therapy is essential to improve personalized decision-making when considering curative versus disease-modifying therapeutic options. The primary goal of this study is to determine whether curative therapies for individuals with SCD will result in improved or worsening heart, lung, and kidney damage when compared to individuals with SCD receiving standard therapy. The investigators will also explore whether certain genes are associated with a good or bad outcome after curative therapy for SCD.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Confirmed laboratory diagnosis of SCD
Ability to give informed consent
Ability to provide pre- and post-curative therapy data
Treated with either one HSCT or with standard disease-modifying therapy
Disqualifiers
None
Trial population
Participants will be patient volunteers from the sickle cell disease clinics and transplant centers of participating study sites.
Trial design
Design model
Cohort
Time perspective
Other
Treatments tested in this trial
Not listed
Trial groups
750 Participants
are grouped into 4 trial groupsGroup A: Pediatric Myeloablative allo-HSCT
Group B: Pediatric Standard Disease-Modifying Therapy
Group C: Adult Non-Myeloablative allo-HSCT
Group D: Adult Standard Disease-Modifying Therapy
Trial outcomes
Primary outcomes
1
Measurement of longitudinal change in FEV1
Measurements of forced expiratory volume in 1 second (FEV1) based on the global lung index will be acquired from children with SCD receiving myeloablative curative therapies and adults with SCD receiving nonmyeloablative allo-HSCT, as well as from the respective control children and adult cohorts with SCD who received standard therapy. FEV1 will be reported in liters. (References: Eur Respir J. Volume 40 Issue 6: pages 1324-1343, 2012 June 27; Am J Hematol. Volume 93 Issue 3: pages 408-415, 2018 March).
Time frame
Through study completion, an average of four years2
Percent predicted value of longitudinal change in FEV1
Percent predicted of forced expiratory volume in 1 second (FEV1) based on the global lung index will be acquired from children with SCD receiving myeloablative curative therapies and adults with SCD receiving nonmyeloablative allo-HSCT, as well as from the respective control children and adult cohorts with SCD who received standard therapy. FEV1 will be reported in percentage. (References: Eur Respir J. Volume 40 Issue 6: pages 1324-1343, 2012 June 27; Am J Hematol. Volume 93 Issue 3: pages 408-415, 2018 March).
Time frame
Through study completion, an average of four years3
Measurement of longitudinal change in FVC
Measurements of forced volume capacity (FVC) based on the global lung index will be acquired from children with SCD receiving myeloablative curative therapies and adults with SCD receiving nonmyeloablative allo-HSCT, as well as from the respective control children and adult cohorts with SCD who received standard therapy. FVC will be reported in liters. (References: Eur Respir J. Volume 40 Issue 6: pages 1324-1343, 2012 June 27; Am J Hematol. Volume 93 Issue 3: pages 408-415, 2018 March).
Time frame
Through study completion, an average of four years4
Percent predicted value of longitudinal change in FVC
Percent predicted of forced volume capacity (FVC) on the global lung index will be acquired from children with SCD receiving myeloablative curative therapies and adults with SCD receiving nonmyeloablative allo-HSCT, as well as from the respective control children and adult cohorts with SCD who received standard therapy. FVC will be reported in percentage. (References: Eur Respir J. Volume 40 Issue 6: pages 1324-1343, 2012 June 27; Am J Hematol. Volume 93 Issue 3: pages 408-415, 2018 March).
Time frame
Through study completion, an average of four yearsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Vanderbilt University Medical Center
Lead sponsor
Johns Hopkins University
Collaborator
Children's National Research Institute
Collaborator
Emory University
Collaborator
University of Illinois at Chicago
Collaborator
Children's Healthcare of Atlanta
Collaborator
Source ClinicalTrials.gov