Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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: Completed Phase 2 study, sponsored by Masonic Cancer Center, University of Minnesota.
NCT00383448 is a Phase 2 study that has completed, run by Masonic Cancer Center, University of Minnesota. The registered enrollment target is 38 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (71% lower). According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
Donor Cell Engraftment is defined as the process of transplanted stem cells reproducing new cells.
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 |
What the finished NCT00383448 record still lists
NCT00383448 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 38 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (71% lower).
The record links to 0 conditions, and to 5 interventions - of which Melphalan is the first listed.
NCT00383448 does not publish any study locations in the registry export this page uses.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00383448's enrollment target sits among peer trials
38 1467th of 2000 higher than 517 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
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT01684904 · 38 participants · NA
Proton Therapy for Esophageal Cancer
- NCT03310879 · 38 participants · Phase 2
Study of the CDK4/6 Inhibitor Abemaciclib in Solid Tumors Harboring Genetic Alterations in Genes Encoding D-Type Cyclins or Amplification of CDK4 or CDK6
- NCT03896269 · 38 participants · Phase 1
CPX-351 in Treating Patients With Relapsed or Refractory High Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia
- NCT04085315 · 38 participants · Phase 1
Alisertib in Combination With Osimertinib in Metastatic EGFR-mutant Lung Cancer
Similar registry start date
- NCT05988463 · started 2027-01-01 · Phase 1
Dose-Escalation Study of Artesunate Patients With IPF
- NCT07219706 · started 2026-12-10 · NA
Dietary Fiber and Time Restricted Eating
- NCT02831335 · started 2026-12-01
Technical Development of Multi-Parametric Renocerebral MRI
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia