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 range
50 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This trial tracked Grade 3–5 treatment-related toxicity as its main outcome — what does that severity range actually mean in practice, and how did the results from this completed study compare to what's expected with standard care for my specific condition?
2Since this trial covered several different conditions including sickle cell disease and thalassemia, can you tell me whether the safety and toxicity data from this study is relevant to my particular diagnosis, or were outcomes meaningfully different across those patient groups?
3This was a Phase 2/3 stem cell transplant trial, which means it was evaluating both safety and effectiveness — based on the completed results, what did doctors learn about whether the risks of transplant were justified by the outcomes for patients like me?
4Stem cell transplantation is a major procedure with significant recovery demands — if this trial's completed data supports transplant as an option for my condition, how would the logistics and long-term follow-up care fit into my current situation?
5Are there standard treatments for my condition that I should try before considering a transplant approach like the one studied in this trial, or does the evidence from this completed study suggest transplant should be considered earlier in the treatment process?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
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