Trials / Terminated
TerminatedNCT01372228
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
- Status
- Terminated
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 3 (actual)
- Sponsor
- Talaris Therapeutics Inc. · Industry
- Sex
- All
- Age
- —
- Healthy volunteers
- Not accepted
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.
Conditions
- Hurler Syndrome (MPS I)
- Hurler-Scheie Syndrome
- Hunter Syndrome (MPS II)
- Sanfilippo Syndrome (MPS III)
- Krabbe Disease (Globoid Leukodystrophy)
- Metachromatic Leukodystrophy (MLD)
- Adrenoleukodystrophy (ALD and AMN)
- Sandhoff Disease
- Tay Sachs Disease
- Pelizaeus Merzbacher (PMD)
- Niemann-Pick Disease
- Alpha-mannosidosis
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | hematopoietic stem cell infusion | Enriched hematopoetic stem cell infusion |
Timeline
- Start date
- 2011-04-01
- Primary completion
- 2016-04-01
- Completion
- 2016-04-01
- First posted
- 2011-06-13
- Last updated
- 2023-04-12
Locations
1 site across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT01372228. Inclusion in this directory is not an endorsement.