drugset
drugset / Trial / NCT01372228

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

NCT01372228 ↗

Phase 1/2 Terminated 3 enrolled Talaris Therapeutics Inc. Duke University · collab
NaSingle-groupOpen-labelTreatment

Summary

The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.

Timeline

Start
2011-04
Primary completion
2016-04
Completion
2016-04

Outcome

Outcome not reported

Stopped: “company did not pursue this indication”

Drugs

EvaluationDrugModalityDoseRoute
Subject hematopoietic stem cell infusion Cell therapy — —

Indications

Hurler syndrome Hurler-Scheie syndrome Krabbe disease Niemann-Pick disease Pelizaeus-Merzbacher spectrum disorder Sandhoff disease Tay-Sachs disease adrenoleukodystrophy alpha-mannosidosis metachromatic leukodystrophy mucopolysaccharidosis type 2 mucopolysaccharidosis type 3