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Hematopoietic Stem Cell Therapy for Patients With Refractory Myasthenia Gravis

NCT00424489 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2018-08-31

Study results available
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Summary

MG may be neonatal, congenital, or autoimmune. Neonatal MG arises from transplacental transfer of ACh receptor antibodies from a mother with autoimmune MG to the fetus. Neonatal MG resolves with post delivery clearance of maternal antibodies. Congenital MG results from a genetic defect in the ACh receptor. Patients with congenital MG do not have ACh receptor antibodies. Both neonatal and congenital MG are excluded from this study. Autoimmune MG, which is the most common form of MG, affects approximately 25,000 Americans. Like most autoimmune diseases, it is associated with particular HLA genotypes, has a female predominance, and environmental factors involved in breaking tolerance to the ACh receptor are unknown. Patients with refractory and severe autoimmune MG will be considered candidates for this study.

The purpose of this study is to assess the toxicity/feasibility (phase I) of autologous hematopoietic stem cell transplantation for refractory myasthenia gravis.

Conditions

Interventions

BIOLOGICAL

Hematopoietic Stem Cell Transplantation

Autologous Hematopoietic Stem Cell Transplantation

DRUG

Cyclophosphamide

DRUG

ATG (rabbit)

DRUG

Mesna

DRUG

Methylprednisolone

DRUG

G-CSF

Sponsors & Collaborators

Principal Investigators

  • Richard Burt, MD · Northwestern University and Northwestern Memorial Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
15 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2002-02-28
Primary Completion
2016-06-30
Completion
2016-06-30

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs
Diseases
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 NCT00424489 on ClinicalTrials.gov