Evaluating the Safety and Effectiveness of Bone Marrow Transplants in Children With Sickle Cell Disease (BMT CTN 0601)

Completed · Phase 2

Conditions studied: Sickle Cell Disease

In brief

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 bone marrow from unrelated donors, in children with severe SCD who receive a reduced intensity conditioning regimen prior to the transplant.

Key facts

Study ID

NCT00745420

Run by

Medical College of Wisconsin

People needed

30

Starts

2008-08-01

Expected to finish

2016-09-01

Last updated by the study team

2022-12-09

Who can join

Age: 3 and older, up to 19. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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:
• Patients must have symptomatic SCD (genotype Hb SS, Hb SC, Sß° thalassemia or Sß+ thalassemia), AND have 1 or more of the following clinical complications:(i) Clinically significant neurologic event (stroke) or any neurologic deficit lasting more than 24 hours that is accompanied by an infarct on cerebral MRI; OR (ii) patients who have a Transcranial Doppler (TCD) velocity that exceeds 200 cm/sec by the non-imaging technique (or TCD measurement greater than 185 cm/sec by the imaging technique) measured at a minimum of 2 separate occasions one month or more apart; OR,
• 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
• History of 3 or more severe pain events per year in the 2 years before study entry
• Lansky/Karnofsky performance score greater than or equal to 40
• Patients must have an unrelated adult bone marrow donor who is Human Leukocyte Antigen (HLA)-matched at 8 of 8 HLA-A, -B, -C and -DRB1 at high resolution using DNA-based typing.
• Patients with adequate physical function: a)Cardiac: Left ventricular ejection fraction (LVEF) greater than 40%, or LV shortening fraction greater than 26%; b) Pulmonary: Pulse oxymetry with a baseline O2 saturation of greater than or equal to 85% is required for all patients, Carbon Monoxide Diffusing Capacity (DLCO) greater than 40% (corrected for hemoglobin) for patients in whom pulmonary function testing can be performed; c) Renal: Serum creatinine less than or equal to 1.5 x upper limit of normal for age and glomerular filtration rate (GFR) greater than 100 mL/min/1.73 m. For patients older than or equal to 16 years of age, GFR should be greater than 70 mL/min/1.73 m\^2; d) Hepatic: Serum conjugated (direct) bilirubin less than 2x upper limit of normal for age as per local laboratory; alanine transaminase (ALT) and aspartate transaminase (AST) less than 5 times upper limit of normal as per local laboratory.
• If the patient has been receiving chronic transfusion therapy for more than or equal to 1 year AND has clinical evidence of iron overload (serum ferritin level of greater than 1000 ng/ml), a liver biopsy shall be obtained within 90 days of starting conditioning therapy (alemtuzumab). Histologic exam of the liver must document absence of bridging fibrosis or cirrhosis of the liver. In other cases, a liver biopsy is optional.
• Hemoglobin S (Hb S) level less than or equal to 45%, seven days prior to initiation of alemtuzumab

You may not qualify if…

• Evidence of uncontrolled bacterial, viral or fungal infections (currently taking medication and progression of clinical symptoms) within 1 month prior to starting the conditioning regimen. Patients with fever or suspected minor infection should await resolution of symptoms before starting the conditioning regimen
• Pregnant or breastfeeding
• Patients with 8/8 HLA-matched family donors able to donate
• Seropositivity for HIV
• Prior allogeneic marrow or stem cell transplant
• Iron chelation must be discontinued more than or equal to 48 hours before initiating the conditioning regimen
• Hydroxyurea (if receiving this therapy) must be discontinued more than or equal to 48 hours before initiating the conditioning regimen

Where it is running

• University of Alabama at Birmingham — Birmingham, Alabama, United States
• Children's National Medical Center — Washington D.C., District of Columbia, United States
• University of Miami — Miami, Florida, United States
• Children's Healthcare of Atlanta — Atlanta, Georgia, United States
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• Children's Hospital of New Orleans/LSUMC CCOP — New Orleans, Louisiana, United States
• University of Michigan Medical Center — Ann Arbor, Michigan, United States
• University of Mississippi Medical Center — Jackson, Mississippi, United States
• Children's Mercy Hospital and Clinics — Kansas City, Missouri, United States
• Washington University, St. Louis Children's Hospital — St Louis, Missouri, United States
• Cohen Children's Hospital — New Hyde Park, New York, United States
• Columbia University Medical Center — New York, New York, United States
• University of North Carolina Hospital at Chapel Hill — Chapel Hill, North Carolina, United States
• University Hospitals of Cleveland — Cleveland, Ohio, United States
• Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
• Medical University of South Carolina — Charleston, South Carolina, United States
• Baylor College of Medicine/The Methodist Hospital — Houston, Texas, United States
• Virginia Commonwealth University — Richmond, Virginia, United States
• Medical College of Wisconsin — Milwaukee, Wisconsin, United States

Full record on ClinicalTrials.gov

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