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MNGIE Allogeneic Hematopoietic Stem Cell Transplant Safety Study

NCT02427178 · Status: WITHDRAWN · Phase: PHASE1 · Type: INTERVENTIONAL

Last updated 2022-08-01

No results posted yet for this study

Summary

The purpose of this study is to find out if a stem cell transplant is safe for patients with a very rare disease. The stem cell transplant is called AHSCT (for "allogeneic hematopoetic stem cell transplantation"). The rare disease is called MNGIE (for "Mitochondrial NeuroGastroIntestinal Encephalomyopathy"). Patients with MNGIE will be transplanted with stem cells from an individual who is human leukocyte antigen (HLA) 10/10 matched. The purpose of the transplant is the production of thymidine phosphorylase.

Conditions

  • Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE)

Interventions

BIOLOGICAL

Hematopoietic Allogeneic Stem Cells

HLA 10/10 matched allogeneic bone marrow cells will be infused into recipient (patient).

Sponsors & Collaborators

  • Cornell University

    collaborator OTHER

  • National Institute of Neurological Disorders and Stroke (NINDS)

    collaborator NIH

  • Michio Hirano, MD

    lead OTHER

Principal Investigators

  • Michio Hirano, MD · Columbia University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
5 Years
Max Age
55 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-03-31
Primary Completion
2022-06-30
Completion
2023-06-30

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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 NCT02427178 on ClinicalTrials.gov