Stem Cell Transplant in Sickle Cell Disease and Thalassemia

Description

Sickle cell disease is a genetic disorder in which a mutation in the beta chain of human hemoglobin results in abnormal blood hemoglobin, causing red blood cells to sickle under stress with resulting symptoms including severe pains and strokes. Beta thalassemia is another genetic disorder in which there are abnormal beta hemoglobin chains, causing anemia. In both disorders, frequent red blood cell transfusions may be required to sustain life, but these often result in complications including multiple hospitalizations, iron overload, or bacterial or viral infections such as hepatitis. Standard drugs and therapies used in the treatment of sickle cell disease and/or beta thalassemia provide only supportive care, and may result in long-term side effects, and inadequate control of the disease process. Bone marrow transplant has been increasingly used for the long-term treatment and cure of sickle cell disease and beta thalassemia. Although, not without acute and potential long term side effects, this alternative offers long term control and potential cure of the disease. Most of the side effects seen with bone marrow transplant are directly related to the high intensity of chemotherapy used (ablative). The primary purpose of this study is to see if giving lower doses of chemotherapy (moderately ablative) will result in successful bone marrow replacement without as severe side-effects but with permanent control of the disease. Patients will receive a chemotherapy regimen with busulfan, fludarabine, and alemtuzumab followed by an infusion of stem cells, either from a family-related or cord-blood matched donor.

Study Start Date

September 2004

Estimated Completion Date

November 2017

Interventions

  • Drug: Fludarabine
  • Drug: Alemtuzumab
  • Procedure: Allogeneic stem cell transplant
  • Drug: Busulfan

Specialties

  • Oncology: Anemia/Polycythemia,BMT/SCT
  • Pharmacy: Chemotherapy/Oncology
  • Physician Assistant: Clinical Pharmacology,Hematology/Oncology

MeSH Terms

  • Alemtuzumab
  • Anemia
  • Anemia, Sickle Cell
  • Beta-Thalassemia
  • Busulfan
  • Fludarabine

Study ID

Columbia University -- AAAA7701

Status

Unknown

Trial ID

NCT00408447

Study Type

Interventional

Trial Phase

Phase 2

Enrollment Quota

70

Sponsor

Columbia University

Inclusion Criteria Sickle Cell Disease:
  • Diagnosis of Homozygous Hemoglobin S Disease or Heterozygous Hemoglobin SC or SB/+ thalassemia, or Sickle/variant resulting in Chronic Hemolytic Anemia with HgB < 10 or equal to 10 mg/dL
  • Age < or equal to 21
  • Matched sibling donor and asymptomatic Thalassemia: Homozygous B-thalassemia Clinical syndrome of B-thalassemia intermedia (including double heterozygotes, e.g. Hgb E-Beta thalassemia) who either:
  • Require regular transfusions, or
  • Have evidence of severe ineffective erythropoiesis
  • marked marrow hyperplasia and the resultant cosmetic abnormalities and/or retardation of growth and development as a result of severe anemia. and:
  • Lucarelli Stage 1 or 2
  • Age < or equal to 21 Additionally, Patient must have adequate organ function as below:
  • Adequate renal function defined as serum creatinine < or equal to 1.5 x normal, or creatinine clearance or radioisotope GFR =40 ml/min/m2 or >60 ml/min/1.73 m2 or an equivalent GFR as determined by the institutional normal range.
  • Adequate liver function defined as SGOT (AST) or SGPT (ALT) < 5.0 x normal
  • Adequate cardiac function defined as shortening fraction of >=28% by echocardiogram, or ejection fraction of >=48% by radionuclide angiogram or echocardiogram
  • Adequate pulmonary function defined as uncorrected DLCO>=35% by pulmonary function test
  • for children who are uncooperative, no evidence of dyspnea at rest Exclusion Criteria
  • Karnofsky/Lansky Performance Score <60%
  • Demonstrated lack of compliance with medical care
  • Pregnant or nursing
  • Sickle Cell Disease: Grade III-IV* residual non-motor neurologic
  • Grade IV* Hematuria
  • Thalassemia: Lucarelli Stage 3
  • Gender

    Both

    Ages

    1 Month to 21 Years

    Accepts Healthy Volunteers

    No

    Study Locations and Contact Information (2)

    Study Location Distance Name Phone Email
    Morgan Stanley Childrens Hospital New YorkPresbyterian Columbia University - New York, New York 44.8 miles None None None
    Morgan Stanley Childrens Hospital New YorkPresbyterian Columbia University - New York, New York 44.8 miles Monica Bhatia MD 212-305-9138 mb2476@columbia.edu

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