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NCT00383448 · ClinicalTrials.gov registry record · Phase 2
HSCT for High Risk Inherited Inborn Errors
A Phase 2 study, sponsored by Masonic Cancer Center, University of Minnesota.
- Completed
- Registry status
- Phase 2
- Development phase
- 38
- Enrollment target
NCT00383448 is a Phase 2 study that has completed, run by Masonic Cancer Center, University of Minnesota. The registered enrollment target is 38 participants.
The verdict
NCT00383448, a Phase 2 study, has completed, sponsored by Masonic Cancer Center, University of Minnesota.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 38 participants
- Enrollment target
Study Summary
Hematopoietic stem cell transplantation has proven effective therapy for individuals with adrenoleukodystrophy (ALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD, or Krabbe disease). This protocol also considers other inherited metabolic diseases such as, but not limited to, GM1 gangliosidosis, Tay Sachs disease, Sanfilippo syndrome or Sandhoff disease, I-cell disease (mucolipidosis II). For patients with advanced or rapidly progressive disease, the morbidity and mortality with transplantation is unacceptably high. Unfortunately, there are no viable alternative therapeutic options for these patients; if transplantation is not performed the patients are sent home to die. Our group at Minnesota has developed a new protocol incorporating transplantation using a reduced intensity conditioning regimen designed to decrease toxicity associated with the transplant procedure. This regimen will make use of the drug clofarabine, which has lympholytic and immune suppressive properties without the neurologic toxicity observed in the related compound, fludarabine, commonly used for transplantation. In addition, several agents providing anti-oxidant and anti-inflammatory properties will be used to assist in the stabilization of the disease processes. This revised transplant protocol will test the following: 1) the ability to achieve engraftment with the reduced intensity protocol, 2) the mortality associated with transplant by day 100, 3) patient outcomes, based on differential neurologic, neuropsychologic, imaging and biologic evaluations prior to transplantation and at designated points after transplantation (day 100, 6 months, 1, 2 and 5 years). Additional biologic studies will include pharmacokinetics of clofarabine and mycophenolate mofetil (MMF). In addition, for patients undergoing lumbar puncture studies, cerebrospinal fluid (CSF) will be requested for determinations of biologic parameters.
Interventions
- DRUG Melphalan
- DRUG Alemtuzumab
- DRUG Clofarabine
- PROCEDURE Total body Irradiation
- BIOLOGICAL Hematopoietic Stem Cell Transplantation
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 38 participants |
| Start Date | 2006-09 |
| Est. Completion | 2014-09 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00383448
The ClinicalTrials.gov registry entry for NCT00383448 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 38 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Masonic Cancer Center, University of Minnesota, which has 249 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 5 interventions - of which Melphalan is the first listed.
NCT00383448 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00383448 about?
NCT00383448 is a clinical study titled "HSCT for High Risk Inherited Inborn Errors". Hematopoietic stem cell transplantation has proven effective therapy for individuals with adrenoleukodystrophy (ALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD, or Krabbe disease). This protocol also considers other inherited metabolic diseases such as, but not limited t...
What is the current status of trial NCT00383448?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 38 participants. The study started on 2006-09. Estimated completion is 2014-09.
What interventions are being tested in trial NCT00383448?
The interventions under investigation include: Melphalan (DRUG), Alemtuzumab (DRUG), Clofarabine (DRUG), Total body Irradiation (PROCEDURE), Hematopoietic Stem Cell Transplantation (BIOLOGICAL).
Who is sponsoring clinical trial NCT00383448?
This trial is sponsored by Masonic Cancer Center, University of Minnesota, which has 249 total clinical trials registered on ClinicalTrials.gov.
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