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Stem Cell Transplantation

Hematopoietic Allogeneic Stem Cells for Mitochondrial Neurogastrointestinal Encephalomyopathy (MASS Trial)

Phase 1
Waitlist Available
Research Sponsored by Michio Hirano, MD
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
5 to 55 years of age
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 100 days
Awards & highlights

MASS Trial Summary

This trial is to see if it's safe to give stem cells to people with a very rare disease called MNGIE, which affects the nervous system, muscles, and gastrointestinal system. The stem cells come from a donor who is a perfect match for the patient.

MASS Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

MASS Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~100 days
This trial's timeline: 3 weeks for screening, Varies for treatment, and 100 days for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
neutrophil count (cells/L)
Secondary outcome measures
chimerism percentage
micromole Thd
micromole/l dUrd
+1 more

MASS Trial Design

1Treatment groups
Experimental Treatment
Group I: Open labelExperimental Treatment1 Intervention
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.

Find a Location

Who is running the clinical trial?

Cornell UniversityOTHER
165 Previous Clinical Trials
14,089,460 Total Patients Enrolled
National Institute of Neurological Disorders and Stroke (NINDS)NIH
1,337 Previous Clinical Trials
648,844 Total Patients Enrolled
Michio Hirano, MDLead Sponsor
1 Previous Clinical Trials
18 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Who else is applying?

How old are they?
18 - 65
What site did they apply to?
Columbia University
What portion of applicants met pre-screening criteria?
Met criteria
~0 spots leftby Mar 2025