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
Study identification
- NCT ID
- NCT02171104
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 2
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Enrollment
- 149 participants
Conditions and interventions
Conditions
- Acyl-CoA Oxidase Deficiency
- Adrenoleukodystrophy With Cerebral Involvement
- Alpha-Mannosidosis
- Alpha-methylacyl-CoA Racmase Deficiency
- Aspartylglucosaminuria
- D-Bifunctional Enzyme Deficiency
- Fucosidosis
- Globoid Cell Leukodystrophy
- Glycoprotein Metabolic Disorders
- Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation)
- Hunter Syndrome
- Hurler Syndrome
- Infantile Refsum Disease
- Inherited Metabolic Disorders
- Maroteaux Lamy Syndrome
- Metachromatic Leukodystrophy
- Mitochondrial Neurogastrointestingal Encephalopathy
- Mucopolysaccharidosis Disorders
- Multifunctional Enzyme Deficiency
- Neonatal Adrenoleukodystrophy
- Niemann-Pick B
- Niemann-Pick C Subtype 2
- Peroxisomal Disorders
- Recessive Leukodystrophies
- Severe Osteopetrosis
- Sly Syndrome
- Sphingolipidoses
- Sphingomyelin Deficiency
- Zellweger Syndrome
Interventions
- IMD Preparative Regimen Drug
- Osteopetrosis Haploidentical Only Preparative Regimen Drug
- Osteopetrosis Only Preparative Regimen Drug
- Stem Cell Transplantation Biological
- cALD HR-D (High-Risk, Regimen C) Drug
- cALD HR-D (High-Risk, Regimen D) Drug
- cALD SR-A (Standard-Risk, Regimen A) Drug
- cALD SR-B (Standard-Risk, Regimen B) Drug
Drug · Biological
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 9, 2014
- Primary completion
- Jan 4, 2026
- Completion
- Jul 13, 2029
- Last update posted
- Jan 6, 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 6, 2026 · Synced May 8, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT02171104 live on ClinicalTrials.gov.