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NCT01043640 · ClinicalTrials.gov registry record · Phase 2
Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
A Phase 2 study, sponsored by Masonic Cancer Center, University of Minnesota.
- Completed
- Registry status
- Phase 2
- Development phase
- 46
- Enrollment target
NCT01043640: Completed Phase 2 study, sponsored by Masonic Cancer Center, University of Minnesota.
NCT01043640 is a Phase 2 study that has completed, run by Masonic Cancer Center, University of Minnesota. The registered enrollment target is 46 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (65% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01043640, a Phase 2 study, has completed, sponsored by Masonic Cancer Center, University of Minnesota.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 46 participants
- Enrollment target
Study Summary
Rationale: Chemotherapy administration before a donor stem cell transplant is necessary to stop the patient's immune system from rejecting the donor's stem cells. When healthy stem cells from a donor are infused into the patient, the donor white blood cells can provide the missing enzyme that causes the metabolic disease. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving a monoclonal antibody, alemtuzumab, before transplant and cyclosporine and mycophenolate mofetil before and after transplant may stop this from happening. This may be an effective treatment for inherited metabolic disorders. Purpose: The design of this study is to achieve donor cell engraftment in patients with standard-risk inherited metabolic diseases with limited peri-transplant morbidity and mortality. This will be achieved through the administration of the chemotherapy regimen described. The intention is to follow transplanted patient for years after transplant monitoring them for complications of their disease and assisting families with a multifaceted interdisciplinary approach.
Primary Outcome
Donor derived engraftment is defined as 80 percent or greater donor cells in the recipient's bone marrow and blood cells.
Interventions
- DRUG Cyclophosphamide
- DRUG Busulfan
- DRUG Cyclosporine A
- PROCEDURE Allogeneic stem cell transplantation
- DRUG Campath-1H
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 46 participants |
| Start Date | 2009-12 |
| Est. Completion | 2017-06 |
| Phase | Phase 2 |
What the finished NCT01043640 record still lists
NCT01043640 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 46 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (65% lower).
The record links to 0 conditions, and to 5 interventions - of which Cyclophosphamide is the first listed.
NCT01043640 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01043640 about?
NCT01043640 is a clinical study titled "Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders". Rationale: Chemotherapy administration before a donor stem cell transplant is necessary to stop the patient's immune system from rejecting the donor's stem cells. When healthy stem cells from a donor are infused into the patient, the donor white blood cells can provide the missing enzyme that causes...
What is the current status of trial NCT01043640?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 46 participants. The study started on 2009-12. Estimated completion is 2017-06.
What interventions are being tested in trial NCT01043640?
The interventions under investigation include: Cyclophosphamide (DRUG), Busulfan (DRUG), Cyclosporine A (DRUG), Allogeneic stem cell transplantation (PROCEDURE), Campath-1H (DRUG).
Who is sponsoring clinical trial NCT01043640?
This trial is sponsored by Masonic Cancer Center, University of Minnesota, which has 249 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01043640's enrollment target sits among peer trials
46 1286th of 2000 higher than 705 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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