Trials / Active Not Recruiting
Active Not RecruitingNCT02171104
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG
- Status
- Active Not Recruiting
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 149 (actual)
- Sponsor
- Masonic Cancer Center, University of Minnesota · Academic / Other
- Sex
- All
- Age
- 55 Years
- Healthy volunteers
- Not accepted
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
- 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
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | Stem Cell Transplantation | Infusion given on Day 0 |
| DRUG | IMD Preparative Regimen | * Anti-thymocyte Globulin (ATG) * Fludarabine * Busulfan |
| DRUG | Osteopetrosis Only Preparative Regimen | * Anti-thymocyte Globulin (ATG) * Fludarabine * Busulfan * Thiotepa |
| DRUG | Osteopetrosis Haploidentical Only Preparative Regimen | * Rituximab * Alemtuzumab * Busulfan * Fludarabine |
| DRUG | cALD SR-A (Standard-Risk, Regimen A) | N-acetylcysteine start day +1 through day +28 |
| DRUG | cALD SR-B (Standard-Risk, Regimen B) | N-acetylcysteine start day +1through day +56 |
| DRUG | cALD HR-D (High-Risk, Regimen C) | N-acetylcysteine and celecoxib start day of admission (prior to conditioning regimen) and continue through day +100 |
| DRUG | cALD HR-D (High-Risk, Regimen D) | N-acetylcysteine, celecoxib, vitamin E and alpha lipoic acid start day of admission (prior to conditioning regimen) and continue through day +100 |
Timeline
- Start date
- 2014-07-10
- Primary completion
- 2026-01-05
- Completion
- 2029-07-14
- First posted
- 2014-06-23
- Last updated
- 2026-01-07
Locations
1 site across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT02171104. Inclusion in this directory is not an endorsement.