Trials / Completed
CompletedNCT02845596
Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia
- Status
- Completed
- Phase
- N/A
- Study type
- Interventional
- Enrollment
- 40 (estimated)
- Sponsor
- Michael Pulsipher · Academic / Other
- Sex
- All
- Age
- 25 Years
- Healthy volunteers
- Not accepted
Summary
The purpose of this study is to determine the feasibility of comparing outcomes of patients treated de novo with immunosuppressive therapy (IST) versus matched unrelated donor (MUD) hematopoietic stem cell transplant (HSCT) for pediatric acquired severe aplastic anemia.
Detailed description
A major challenge in treating pediatric Severe Aplastic Anemia (SAA) is the determination of best primary therapy for patients who lack a fully matched related donor for HSCT. Good survival outcomes have been seen with IST, but initial and late failures, CSA dependence, persistent cytopenias and secondary Myelodysplastic Syndrome (MDS) / Acute Myeloid Leukemia (AML) in a portion of patients leave considerable room for improvement. MUD HSCT survival in SAA has markedly improved, but a direct comparison of this approach with IST is necessary to determine whether this approach is feasible and will lead to better Event Free Survival. This trial will address the feasibility of randomization, test whether patients can be evaluated in a timely fashion and safely begin therapy with MUD HSCT or IST, and give a preliminary assessment of the safety of up-front MUD HSCT. If successful, this trial will lead to a future prospective trial comparing directly IST to MUD HSCT in this disease.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | cyclosporine | cyclosporine |
| PROCEDURE | Matched Unrelated Donor Hematopoietic Stem Cell Transplant | Matched Unrelated Donor (MUD) Hematopoietic Stem Cell Transplantation (HSCT) |
| DRUG | horse anti-thymocyte globulin (ATG) | horse anti-thymocyte globulin (ATG) |
| DRUG | rabbit anti-thymocyte globulin (ATG) | rabbit anti-thymocyte globulin (ATG) |
| DRUG | methotrexate | methotrexate |
| DRUG | fludarabine | fludarabine |
| DRUG | cyclophosphamide | cyclophosphamide |
| RADIATION | low-dose total body irradiation (TBI) | low-dose total body irradiation (TBI) |
| PROCEDURE | Immunosuppressive Therapy (IST) | Immunosuppressive Therapy (IST) |
Timeline
- Start date
- 2016-08-01
- Primary completion
- 2020-11-03
- Completion
- 2023-07-19
- First posted
- 2016-07-27
- Last updated
- 2025-05-11
Locations
13 sites across 1 country: United States
Source: ClinicalTrials.gov record NCT02845596. Inclusion in this directory is not an endorsement.