Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
Phase 1
Terminated
- Conditions
- Metachromatic Leukodystrophy (MLD)Pelizaeus Merzbacher (PMD)Alpha-mannosidosisHunter Syndrome (MPS II)Sanfilippo Syndrome (MPS III)Krabbe Disease (Globoid Leukodystrophy)Sandhoff DiseaseTay Sachs DiseaseAdrenoleukodystrophy (ALD and AMN)Niemann-Pick Disease
- Interventions
- Biological: hematopoietic stem cell infusion
- Registration Number
- NCT01372228
- Lead Sponsor
- Talaris Therapeutics Inc.
- Brief Summary
The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.
- Detailed Description
The objective for the study is to establish chimerism following reduced intensity conditioning with no grade III/IV GVHD. The primary endpoint we will follow is production of the missing enzyme at ≥ 10% of the normal level at day 180 post-transplant in \> 90% of patients.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 3
Inclusion Criteria
Not provided
Exclusion Criteria
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Inherited Metabolic Disorder Patients hematopoietic stem cell infusion Recipients are treated with hematopoietic stem cell infusion from living donors
- Primary Outcome Measures
Name Time Method Production of missing enzyme at levels greater than or equal to 10% of normal Day 180 post transplant to three years
- Secondary Outcome Measures
Name Time Method Enriched Hematopoetic Stem Cell Engraftment One month to three years
Trial Locations
- Locations (1)
Duke University Medical Center
🇺🇸Durham, North Carolina, United States