Phase 2
N=21
Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia
Severe Thalassemia
Bottom Line
View on ClinicalTrials.gov: NCT01005576 ↗Enrolled (actual)
21
Serious AEs
47.6%
Results posted
Oct 2017
Primary outcome: Primary: Primary Objective: Event-free Survival at 1 Year. — 17 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan (Drug)
- Age
- Pediatric · 1+ yrs
- Sex
- All
- Sponsor
- Washington University School of Medicine
- Primary completion
- Apr 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Primary Objective: Event-free Survival at 1 Year. |
17 | — |
| SECONDARY Development of Graft Versus Host Disease (GVHD) |
12; 9 | — |
| SECONDARY Median Time to ANC Engraftment |
14 | — |
| SECONDARY Median Time to Platelet Engraftment |
27.5 | — |
| SECONDARY Incidence of Disease Recurrence |
1 | — |
Summary
This study is being done to determine if blood cell transplants, with either bone marrow or cord blood from unrelated donors, are effective in children with severe thalassemia and if this treatment approach has acceptable risks and side effects.
This study includes a preparative regimen with Hydroxyurea, Alemtuzumab, Fludarabine, Thiotepa and Melphalan that provides intense host immunosuppression without myeloablation. The primary hypothesis is that this regimen will promote stable engraftment of unrelated donor hematopoietic cells, support normal erythropoiesis, and result in an event free survival of > 75% of children with thalassemia major.
Eligibility Criteria
Inclusion Criteria
- 1-16.00 years old
- Have transfusion dependent thalassemia major
- Shall not have an HLA-matched family donor
- Must have a suitably matched unrelated marrow donor or UCB product
- Lansky score >/= 70
- Adequate pulmonary, renal, liver, and other organ function as defined in protocol
- Negative pregnancy test
- Adequate total nucleated cell or CD34+ dose of product as defined in protocol
- Iron chelation must be discontinued >/= 48 hours prior to conditioning regimen
Exclusion Criteria
- Pregnant or breastfeeding
- HIV positive
- Prior allogeneic marrow or stem cell transplantation
Data sourced from ClinicalTrials.gov (NCT01005576). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.