Status:
TERMINATED
Hematopoietic Stem Cell Therapy for Patients With Refractory Myasthenia Gravis
Lead Sponsor:
Northwestern University
Conditions:
Myasthenia Gravis
Eligibility:
All Genders
15-65 years
Phase:
PHASE1
Brief 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 ...
Detailed Description
There will be no randomization in this study. All subjects who are determined to be eligible for the study treatment will receive high dose cyclophosphamide and ATG followed by infusion of autologous ...
Eligibility Criteria
Inclusion
- Inclusion Criteria (Patients must fulfill all of the following)
- Established diagnosis of myasthenia gravis defined as clinical evidence of muscle weakness and fatigue ability and supported, an abnormal EMG-NCV repetitive nerve stimulation (or single-fiber EMG) or Lambert-Eaton Myasthenic Syndrome without evidence of malignancy.
- Ages 15-65 years.
- Positive antibody preferred (anti-AchR, MuSK, voltage gated calcium channel, anti-striational).
- Failure of thymectomy (except for Lambert-Eaton Myasthenic Syndrome).
- Failure anticholinesterase therapy, corticosteroids, and at least two of the following: azathioprine, cyclosporin, CellCept, cyclophosphamide, plasma exchange, or IVIG. Failure is defined as at least 6 months of the above drug therapy and an Osserman score of IIB, III, or IV and not clinically improving.
- And at least one of the following:
- History of myasthenia crises (requiring mechanical ventilation) despite thymectomy and immunosuppressive therapy.
- Hospitalized or on ventilator support for myasthenia gravis within the last 18 months despite thymectomy and immunosuppressive therapy.
- Inability to maintain nutrition due to muscle weakness.
- A Karnofsky performance status of 70% or less (may or may not be able to care for self, but unable to carry on normal activity or unable to do active work).
- Exclusion Criteria
- Significant end organ damage such as:
- LVEF \<40% or deterioration of LVEF during exercise test on MUGA or echocardiogram.
- Untreated life-threatening arrhythmia, active ischemic heart disease or heart failure.
- DLCO \< 40% of predicted value.
- Serum creatinine \> 2.5 mg/dl.
- Liver cirrhosis, transaminases \>3x of normal limits, or bilirubin \>2.0 unless due to Gilberts disease.
- HIV positive.
- Uncontrolled diabetes mellitus, or any other illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive treatment.
- Prior history of malignancy except localized basal cell or squamous skin cancer. Other malignancies for which the patient is judged to be cured by local surgical therapy, such as (but not limited to) head and neck cancer, or stage I or II breast cancer, will be considered on an individual basis.
- Positive pregnancy test, inability or unable to pursue effective means of birth control, failure to willingly accept or comprehend irreversible sterility as a side effect of therapy.
- Psychiatric illness or mental deficiency making compliance with treatment or informed consent impossible.
- Inability to give informed consent
- Congenital myasthenia gravis
- Neonatal myasthenia gravis
- Osserman grade 1 or 2
- Pure red cell aplasia
- Any patient on insulin
Exclusion
Key Trial Info
Start Date :
February 1 2002
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 1 2016
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT00424489
Start Date
February 1 2002
End Date
June 1 2016
Last Update
August 31 2018
Active Locations (1)
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1
Northwestern University, Feinberg School of Medicine
Chicago, Illinois, United States, 60611