logo Hematology/Oncology Research Studies: Open for Enrollment Date: 05/27/20


Bone Marrow Transplantation Compared to Standard Care in Adolescents and Young Adults with Severe Sickle Cell Disease

NCT#/Phase: NCT02766465
Phase 2
IRB#: 2016-046
Description: This is a prospective multi-center trial of hematopoietic stem cell transplantation or standard of care based on availability of HLA-matched related or unrelated donor after confirmation of clinical eligibility. In order to minimize bias assignment to either treatment arm, clinical eligibility to both treatment arms are similar and donor availability is not known at referral. This study will compare the overall survival at 2 years between those patients assigned to the donor arm and the no donor arm. Patients will be assigned to each treatment arm depending on the availability of a matched sibling or unrelated donor. Those patients with a donor are expected to proceed with a bone marrow transplant. Those patients who do not have a donor will receive standard therapy.
Inclusion: Individuals with severe sickle cell disease who are between 15 - 40 years of age and have not had HLA typing and donor search. Individuals must have had one or more of the following: Clinically significant neurologic event (stroke) or any neurological deficit lasting > 24 hours; History of two or more episodes of acute chest syndrome (ACS) in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. asthma therapy); Three or more pain crises per year in the 2-year period preceding referral; Administration of regular RBC transfusion therapy, defined as receiving 8 or more transfusions per year for = 1 year to prevent vaso-occlusive clinical complications; An echocardiographic finding of tricuspid valve regurgitant jet (TRJ) velocity = 2.7 m/sec.
Enrollment Status: Open for enrollment
Sponsor: BMT-CTN, Medical College of Wisconsin
Principal Investigator(s): Mark Walters, MD
Contact(s): Marci Moriarty, RN (510-428-3385 x5396)
Julie Decaris (510-428-3385 x2739)
Study Link: https://clinicaltrials.gov/ct2/show/NCT02766465

UCSF Benioff Children's Hospital Oakland • 747 52nd Street, Oakland, California 94609 &bull Phone: (510) 428-3000