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NCT02171104 · ClinicalTrials.gov registry record · Phase 2
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
A Phase 2 study of Hunter Syndrome and Hurler Syndrome, sponsored by Masonic Cancer Center, University of Minnesota.
- Active
- Registry status
- Phase 2
- Development phase
- 149
- Enrollment target
- 1
- Study location
NCT02171104: Active Phase 2 study of Hunter Syndrome and Hurler Syndrome, sponsored by Masonic Cancer Center, University of Minnesota.
NCT02171104 is a Phase 2 study of Hunter Syndrome and Hurler Syndrome that is active but no longer recruiting, run by Masonic Cancer Center, University of Minnesota. The registered enrollment target is 149 participants, below the 326-participant average among 5 other Hunter Syndrome trials with a reported enrollment target (54% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02171104, a Phase 2 study of Hunter Syndrome and Hurler Syndrome, is active but no longer recruiting, sponsored by Masonic Cancer Center, University of Minnesota.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 149 participants
- Enrollment target
- 1
- Study location
Study Summary
This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe osteopetrosis (OP).
Primary Outcome
Defined as neutrophil recovery by Day +42 post-transplant and ≥ 80% donor cells on the myeloid fraction of peripheral blood at Day +100 post-transplant
Conditions Studied
Interventions
- BIOLOGICAL Stem Cell Transplantation
- DRUG IMD Preparative Regimen
- DRUG Osteopetrosis Only Preparative Regimen
- DRUG Osteopetrosis Haploidentical Only Preparative Regimen
- DRUG cALD SR-A (Standard-Risk, Regimen A)
Study Locations (1)
Minnesota
- Masonic Cancer Center, University of Minnesota - Minneapolis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 149 participants |
| Start Date | 2014-07-10 |
| Est. Completion | 2029-07-14 |
| Phase | Phase 2 |
What the registry record for NCT02171104 still lists
NCT02171104 is an interventional study that assigns participants to a tested intervention. The registered 149 participants enrollment target is mid-sized for trials with a published cap, below the 326-participant average among 5 other Hunter Syndrome trials with a reported enrollment target (54% lower).
The record links to 10 conditions, with Hunter Syndrome appearing as the primary indexed condition, and to 5 interventions - of which Stem Cell Transplantation is the first listed.
NCT02171104 reports a single indexed study location in Minnesota.
Frequently Asked Questions
What is clinical trial NCT02171104 about?
NCT02171104 is a clinical study titled "MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis". This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patient...
What is the current status of trial NCT02171104?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 149 participants. The study started on 2014-07-10. Estimated completion is 2029-07-14.
What conditions does trial NCT02171104 study?
This clinical trial studies the following conditions: Hunter Syndrome, Hurler Syndrome, Maroteaux Lamy Syndrome, Fucosidosis, Alpha-Mannosidosis.
What interventions are being tested in trial NCT02171104?
The interventions under investigation include: Stem Cell Transplantation (BIOLOGICAL), IMD Preparative Regimen (DRUG), Osteopetrosis Only Preparative Regimen (DRUG), Osteopetrosis Haploidentical Only Preparative Regimen (DRUG), cALD SR-A (Standard-Risk, Regimen A) (DRUG).
Who is sponsoring clinical trial NCT02171104?
This trial is sponsored by Masonic Cancer Center, University of Minnesota, which has 249 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT02171104 being conducted?
This trial has 1 study location across Minnesota. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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