MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Public ClinicalTrials.gov record NCT02171104. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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).
Study identification
- NCT ID
- NCT02171104
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 149 participants
Conditions and interventions
Conditions
- Mucopolysaccharidosis Disorders
- Hurler Syndrome
- Hunter Syndrome
- Maroteaux Lamy Syndrome
- Sly Syndrome
- Alpha-Mannosidosis
- Fucosidosis
- Aspartylglucosaminuria
- Glycoprotein Metabolic Disorders
- Sphingolipidoses
- Recessive Leukodystrophies
- Globoid Cell Leukodystrophy
- Metachromatic Leukodystrophy
- Niemann-Pick B
- Niemann-Pick C Subtype 2
- Sphingomyelin Deficiency
- Peroxisomal Disorders
- Adrenoleukodystrophy With Cerebral Involvement
- Zellweger Syndrome
- Neonatal Adrenoleukodystrophy
- Infantile Refsum Disease
- Acyl-CoA Oxidase Deficiency
- D-Bifunctional Enzyme Deficiency
- Multifunctional Enzyme Deficiency
- Alpha-methylacyl-CoA Racmase Deficiency
- Mitochondrial Neurogastrointestingal Encephalopathy
- Severe Osteopetrosis
- Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation)
- Inherited Metabolic Disorders
Interventions
- 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
- cALD SR-B (Standard-Risk, Regimen B) Drug
- cALD HR-D (High-Risk, Regimen C) Drug
- cALD HR-D (High-Risk, Regimen D) Drug
Biological · Drug
Eligibility (public fields only)
- Age range
- Up to 55 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jul 10, 2014 (Actual)
- Primary completion
- Jan 5, 2026 (Actual)
- Completion
- Jul 14, 2029 (Estimated)
- Last update posted
- Jan 7, 2026
2014 – 2029
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Masonic Cancer Center, University of Minnesota | Minneapolis | Minnesota | 55455 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02171104, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jan 7, 2026 · Synced Oct 18, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT02171104 live on ClinicalTrials.gov.