Status:
COMPLETED
Stem Cell Transplant for Hemoglobinopathy
Lead Sponsor:
Masonic Cancer Center, University of Minnesota
Collaborating Sponsors:
National Marrow Donor Program
Conditions:
Sickle Cell Disease
Thalassemia
Eligibility:
All Genders
Up to 50 years
Phase:
PHASE2
PHASE3
Brief Summary
This study tests the clinical outcomes of one of two preparative regimens (determined by available donor source) in patients with non-malignant hemoglobinopathies. The researchers hypothesize that the...
Detailed Description
Prior to transplantation, subjects will receive either: Cyclophosphamide, Fludarabine, Campath, Total body irradiation (TBI) Or Busulfan, Cyclophosphamide, antithymocyte globulin (ATG), granulocyte...
Eligibility Criteria
Inclusion
- Patients with Sickle Cell Disease/Thalassemia (SCD/THAL) 0-50 years of age with an acceptable stem cell donor and disease characteristic defined by the following:
- Stroke, central nervous system (CNS) hemorrhage or a neurologic event lasting longer than 24 hours, or abnormal cerebral magnetic resonance imaging (MRI) or cerebral arteriogram or MRI angiographic study and impaired neuropsychological testing
- Acute chest syndrome with a history of recurrent hospitalizations or exchange transfusions
- Recurrent vaso-occlusive pain 3 or more episodes per year for 3 years or more years or recurrent priapism,
- Impaired neuropsychological function and abnormal cerebral MRI scan
- Stage I or II sickle lung disease,
- Sickle nephropathy (moderate or severe proteinuria or a glomerular filtration rate \[GFR\] 30-50% of the predicted normal value)
- Bilateral proliferative retinopathy and major visual impairment in at least one eye
- Osteonecrosis of multiple joints with documented destructive changes
- Requirement for chronic transfusions but with red blood cell (RBC) alloimmunization \>2 antibodies during long term transfusion therapy
- Patients with transfusion dependent alpha- or beta-thalassemia 0-35 years of age with an acceptable stem cell donor as defined in the criteria in section above.
- Patients with other non-malignant hematologic disorders that are transfusion-dependent or involve other potentially life-threatening cytopenias (including but not limited to Severe Congenital Neutropenia, Diamond-Blackfan Anemia and Shwachman-Diamond Syndrome) who are 0-35 years of age with an acceptable stem cell donor
- Second Transplants
- Patients with sickle cell disease or thalassemia who have failed to engraft or have autologous recovery after a myeloablative SCT regimen or non-myeloablative regimen are eligible for this protocol.
- Regimen A2 will be utilized for patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or for any patient who has pre-existing organ dysfunction making them ineligible for a myeloablative preparative regimen.
- Regimen B will be utilized for patients with sickle cell disease or thalassemia who have an HLA-identical sibling donor.
- Patients must meet above criteria.
- If the patient has received prior radiation therapy, eligibility to receive additional radiation therapy must be determined by Dr. Dusenbery
- If first transplant was a non-myeloablative regimen, the second transplant can occur at any time
- If the first transplant was a myeloablative regimen, then the second transplant must be \> 6 months from the first transplant
Exclusion
- Patients with one or more of the following:
- Karnofsky or Lansky performance score \<70
- Acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy
- Stage III-IV lung disease
- GFR\<30% predicted
- Pregnant or lactating females
- Active serious infection whereby patient has been on intravenous antibiotics for one week prior to study entry. Any patient with AIDS or ARC or HIV seropositivity
- Psychologically incapable of undergoing bone marrow transplant (BMT) with associated strict isolation or documented history of medical non-compliance
- Patients not able to receive total lymphocytic irradiation (TLI) due to prior radiation therapy
Key Trial Info
Start Date :
June 1 2002
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2020
Estimated Enrollment :
22 Patients enrolled
Trial Details
Trial ID
NCT00176852
Start Date
June 1 2002
End Date
January 1 2020
Last Update
February 27 2020
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States, 55455