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

Phase 1

Terminated

• Conditions: Metachromatic Leukodystrophy (MLD); Pelizaeus Merzbacher (PMD); Alpha-mannosidosis; Hunter Syndrome (MPS II); Sanfilippo Syndrome (MPS III); Krabbe Disease (Globoid Leukodystrophy); Sandhoff Disease; Tay Sachs Disease; Adrenoleukodystrophy (ALD and AMN); Niemann-Pick Disease

• 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.

Study Timeline

• Study Start: 2011-04
• Study First Submitted: 2011-06-10
• Study First Submitted QC: 2011-06-10
• Study First Posted: 2011-06-13
• Primary Completion: 2016-04
• Study Completion: 2016-04
• Status Verified: 2023-04
• Last Update Submitted: 2023-04-10
• Last Update Posted: 2023-04-12

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

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