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

NCT01372228 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2023-04-12

No results posted yet for this study

Summary

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

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

BIOLOGICAL

hematopoietic stem cell infusion

Enriched hematopoetic stem cell infusion

Sponsors & Collaborators

  • Duke University

    collaborator OTHER

  • Talaris Therapeutics Inc.

    lead INDUSTRY

Principal Investigators

  • Suzanne T Ildstad, MD · Talaris Therapeutics Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-04-30
Primary Completion
2016-04-30
Completion
2016-04-30
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01372228 on ClinicalTrials.gov