Stem Cell Transplant for Hemoglobinopathy (NCT00176852) | Clinical Trial Compass
CompletedPhase 2/3
Stem Cell Transplant for Hemoglobinopathy
United States22 participantsStarted 2002-06
Plain-language 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 these regimens will have a positive effect on post transplant engraftment and the incidence of graft-versus-host-disease.
Regimen A2 has replaced Regimen A in this study. Two patients were treated on Regimen A but did not have evidence of initial engraftment thus triggering the stopping rule for that arm of this study.
Who can participate
Age range50 Years
SexALL
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Inclusion Criteria:
* 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 (incl…
What they're measuring
1
Number of Patients Who Experienced Grade 3-5 Treatment Related Toxicity
Timeframe: 1 year
Trial details
NCT IDNCT00176852
SponsorMasonic Cancer Center, University of Minnesota