Evaluating the Safety and Effectiveness of Stem Cell Transplants From Unrelated Donors in Children With Sickle Cell Disease (BMT CTN #0601) (The SCURT Study)

Status: Recruiting
Diagnosis: Pediatric Hematology/Blood Related, Pediatric Hematopoetic Stem Cell Transplant (HSCT)
NCT ID: NCT00745420 (View complete trial on ClinicalTrials.gov)
DFCI Protocol ID: 09-141


Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause organ damage, stroke, and intense pain episodes. A blood stem cell transplant is a treatment option for someone with a severe form of the disease. Prior to undergoing a transplant, people typically receive a conditioning regimen of high doses of chemotherapy and other medications to prepare the body to accept the transplant. A conditioning regimen that uses lower doses of chemotherapy and medications may be safer for transplant recipients. This study will evaluate the safety and effectiveness of blood stem cell transplants, using either bone marrow or umbilical cord blood from unrelated donors, in children with severe SCD who receive a reduced intensity conditioning regimen prior to the transplant.


Conducting Institutions:
Dana-Farber Cancer Institute, Children's Hospital Boston

Overall PI:
Leslie Lehmann, MD, Dana-Farber Cancer Institute

Site-responsible Investigators:

Dana-Farber Cancer Institute: Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP, ctip@partners.org

Eligibility Criteria

Inclusion Criteria: - SCD (genotype hemoglobin SS disease [Hb SS], genotype hemoglobin SC disease [HbSC],sickle ß°[Sß°] thalassemia, or sickle ß^+[Sß^+]thalassemia) with one or more of the following: 1. Clinically significant neurologic event (stroke) or any neurologic defect lasting more than 24 hours and accompanied by an infarct on cerebral MRI 2. Minimum of two episodes of acute chest syndrome in the 2 years before study entry, defined as new pulmonary alveolar consolidation involving at least one complete lung segment (associated with acute symptoms including fever, chest pain, tachypnea, wheezing, rales, or cough that is not attributed to asthma or bronchiolitis) despite adequate supportive care measures 3. History of three or more severe pain events per year in the 2 years before study entry - Lansky performance score greater than or equal to 40 - Hemoglobin S (Hb S) level less than or equal to 45% - An 8 of 8 human leucocyte antigen (HLA)-A, -B, -C, and -DRB1 allele-matched unrelated bone marrow donor OR an unrelated umbilical cord blood (UCB) donor HLA-matched at 5 of 6 HLA-A, -B (low/intermediate resolution) and -DRB1 (high resolution molecular typing) loci - The UCB unit must contain a pre-cryopreserved total nucleated cell dose (TNC) of at least 3.0 x 10^7 per kilogram. Exclusion Criteria: - Cirrhosis of the liver, with uncontrolled bacterial, viral, or fungal infection in the 1 month before study entry - Seropositivity for HIV - Patients with HLA-matched family donors - Has undergone a prior hematopoietic stem cell transplant - Pregnant or breastfeeding
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