Trials / Completed
CompletedNCT01019876
Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases
Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Selected Non-Malignant Diseases
- Status
- Completed
- Phase
- Phase 2 / Phase 3
- Study type
- Interventional
- Enrollment
- 38 (actual)
- Sponsor
- Columbia University · Academic / Other
- Sex
- All
- Age
- 30 Years
- Healthy volunteers
- Not accepted
Summary
This study proposes the use of a reduced intensity chemotherapy/radiation therapy regimen followed by stem cell transplantation, as compared to standard ablative chemotherapy regimens associated with stem cell transplantation, in a population of patients with non-malignant diseases (non-cancer). Eligible patients will have a non-malignant disease in one of the following four strata: bone marrow failure syndromes, immunodeficiencies, inborn errors of metabolism, or histiocytoses. Patients will be assigned to therapy according to diagnosis. Patients will be stratified by disease into one of four strata and treatment regimens will be based on specific disease criteria and conditions. Although these diseases are non-malignant in name, they are often malignant by nature of the disease progression, treatment and associated complications.
Detailed description
This treatment program proposes the use of a reduced intensity chemotherapy regimen, which has been shown to be effective for inducing remission and cure in these diseases. Studies have shown that the use of non-myeloablative chemotherapy regimens have resulted in 75-100% engraftment (replacement of the recipient bone marrow with the donor bone marrow), and significantly reduced transplant related complications as compared to the standard myeloablative chemotherapy regimens.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Fludarabine | |
| DRUG | Cyclophosphamide | |
| DRUG | Busulfan | |
| DRUG | Alemtuzumab | |
| DRUG | Rabbit Anti-thymocyte Globulin | |
| DRUG | Horse Anti-thymocyte Globulin |
Timeline
- Start date
- 2002-09-01
- Primary completion
- 2021-09-07
- Completion
- 2021-09-07
- First posted
- 2009-11-25
- Last updated
- 2024-10-31
Locations
1 site across 1 country: United States
Source: ClinicalTrials.gov record NCT01019876. Inclusion in this directory is not an endorsement.