MNGIE Allogeneic Hematopoietic Stem Cell Transplant Safety Study (MASS)

This study is currently recruiting participants.

See

Contacts and Locations

Verified January 2017 by Michio Hirano, Columbia University

Sponsor:

Collaborators:

Cornell University

National Institute of Neurological Disorders and Stroke (NINDS)

Information provided by (Responsible Party):

Michio Hirano, Columbia University

ClinicalTrials.gov Identifier:

NCT02427178

First received: February 10, 2015

Last updated: January 25, 2017

Last verified: January 2017

History of Changes

Tracking Information

First Received Date ^ICMJE

February 10, 2015

Last Updated Date

January 25, 2017

Start Date ^ICMJE

March 2015

Estimated Primary Completion Date

June 2022 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

neutrophil count (cells/L) [ Time Frame: 42 days ]

engraftment success

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

Complete list of historical versions of study NCT02427178 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

number of patient survival days [ Time Frame: 100 days ]
is the patient al
chimerism percentage [ Time Frame: 100 days ]
percent of donor cell chimerism at 100 days
micromole/l dUrd [ Time Frame: 100 days ]
level of deoxyuridine
micromole Thd [ Time Frame: 100 days ]
level of thymidine

Original Secondary Outcome Measures ^ICMJE

Same as current

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

MNGIE Allogeneic Hematopoietic Stem Cell Transplant Safety Study

Official Title ^ICMJE

MNGIE (Mitochondrial Neurogastrointestinal Encephalomyopathy) AHSCT (Allogeneic Hematopoietic Stem Cell Transplant) Safety Study

Brief 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.

Detailed Description

Patients who have been identified as having MNGIE by genetic testing and/or reduced thymidine phosphorylase levels will be considered for this study. The study team physician will evaluate the condition of the patient and determine if they are eligible. An HLA matched donor is necessary for transplantation. If a suitable donor is found the transplant process can proceed. The patient receives immunosuppressive therapy ( 1 week in the hospital) with subsequent IV transfer of stem cells from the donor. The patient remains in the hospital for approximately 1 month to monitor the transplant. The patient is required to attend research visits at days 0, 100, 6m, 18m and 24 m.

Study Type ^ICMJE

Interventional

Study Phase

Phase 1

Study Design ^ICMJE

Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment

Condition ^ICMJE

Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE)

Intervention ^ICMJE

Biological: Hematopoietic Allogeneic Stem Cells

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

Study Arms

Experimental: Open label

Hematopoietic allogeneic stem cells will be transplanted:

HLA testing will be performed on potential stem cell donors. HLA 10/10 matched donors are eligible, however there are additional criteria that will be applied to determine an acceptable donor. Patients will receive 2 X10 6 CD34 cells/kg weight.

Intervention: Biological: Hematopoietic Allogeneic Stem Cells

Publications *

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Recruiting

Estimated Enrollment ^ICMJE

Estimated Completion Date

June 2023

Estimated Primary Completion Date

June 2022 (Final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

Inclusion Criteria:

Homozygous or compound heterozygous mutations in the TYMP gene
Plasma thymidine level >3micromole/L
Plasma deoxyuridine >7.5 micromole/L
5 to 55 years of age
Appropriate stem cell donor (HLA 10/10 matched)
Karnofsky performance of at least 55

Exclusion Criteria:

Severe cognitive impairment
Severe psychiatric illness
Moderate to severe lung disease
Prior episode of peritonitis due to perforated diverticula
Prior episode of intestinal pseudo-obstruction
Moderate to severe hepatopathy
Moderate to severe diabetes Mellitus
Moderate to severe cardiomyopathy
Moderate to severe nephropathy
Pregnancy or planning to become pregnant during study
Hypersensitivity to E.coli derived products
HIV disease
Positive to anti-donor HLA DP

Sex/Gender

Sexes Eligible for Study:

All

Ages

5 Years to 55 Years (Child, Adult)

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact: Kris Engelstad, MS CGC

1-212-305-6834

ke4@cumc.columbia.edu

Listed Location Countries ^ICMJE

United States

Removed Location Countries

Administrative Information

NCT Number ^ICMJE

NCT02427178

Other Study ID Numbers ^ICMJE

AAAI1718
U54NS078059 ( U.S. NIH Grant/Contract )

Has Data Monitoring Committee

Yes

U.S. FDA-regulated Product

Not Provided

IPD Sharing Statement

Plan to Share IPD:	Yes
Plan Description:	When applicable, we will submit a manuscript describing the results

Responsible Party

Michio Hirano, Columbia University

Study Sponsor ^ICMJE

Michio Hirano

Collaborators ^ICMJE

Cornell University
National Institute of Neurological Disorders and Stroke (NINDS)

Investigators ^ICMJE

Principal Investigator:

Michio Hirano, MD

Columbia University

PRS Account

Columbia University

Verification Date

January 2017

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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