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MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
NCT02171104 · View on ClinicalTrials.gov ↗
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).
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02171104
The ClinicalTrials.gov registry entry for NCT02171104 describes a study currently listed as active not recruiting. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 149 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 154 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT02171104 reports 1 study location spanning 1 distinct geographic area — top geographies include Minnesota. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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, Fucosidosis, Alpha-Mannosidosis, Sly Syndrome. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
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). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT02171104?
This trial is sponsored by Masonic Cancer Center, University of Minnesota, which has 154 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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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