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Hematopoietic Stem Cell Therapy for Patients With Refractory Myasthenia Gravis

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ClinicalTrials.gov Identifier: NCT00424489
Recruitment Status : Terminated (No participants enrolled for more than two years. No plan to continue study.)
First Posted : January 19, 2007
Results First Posted : August 6, 2018
Last Update Posted : August 31, 2018
Sponsor:
Information provided by (Responsible Party):
Richard Burt, MD, Northwestern University

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Myasthenia Gravis
Interventions Biological: Hematopoietic Stem Cell Transplantation
Drug: Cyclophosphamide
Drug: ATG (rabbit)
Drug: Mesna
Drug: Methylprednisolone
Drug: G-CSF
Enrollment 9
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description

Autologous Hematopoietic Stem Cell Transplantation will be performed after conditioning

Hematopoietic Stem Cell Transplantation: Autologous Hematopoietic Stem Cell Transplantation

Period Title: Overall Study
Started 9
Completed 6
Not Completed 3
Reason Not Completed
Death             3
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description

Autologous Hematopoietic Stem Cell Transplantation will be performed after conditioning

Hematopoietic Stem Cell Transplantation: Autologous Hematopoietic Stem Cell Transplantation

Overall Number of Baseline Participants 9
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
<=18 years
1
  11.1%
Between 18 and 65 years
8
  88.9%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 9 participants
45.22  (16.49)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants
Female
4
  44.4%
Male
5
  55.6%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 9 participants
9
 100.0%
1.Primary Outcome
Title Survival
Hide Description Survival
Time Frame Up to 5 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Survival both treatment related and non-treatment related
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description:

Autologous Hematopoietic Stem Cell Transplantation will be performed after conditioning

Hematopoietic Stem Cell Transplantation: Autologous Hematopoietic Stem Cell Transplantation

Overall Number of Participants Analyzed 9
Measure Type: Count of Participants
Unit of Measure: Participants
6
  66.7%
Time Frame Up to 5 years
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Hematopoietic Stem Cell Transplantation
Hide Arm/Group Description

Autologous Hematopoietic Stem Cell Transplantation will be performed after conditioning

Hematopoietic Stem Cell Transplantation: Autologous Hematopoietic Stem Cell Transplantation

All-Cause Mortality
Hematopoietic Stem Cell Transplantation
Affected / at Risk (%)
Total   3/9 (33.33%)    
Show Serious Adverse Events Hide Serious Adverse Events
Hematopoietic Stem Cell Transplantation
Affected / at Risk (%) # Events
Total   3/9 (33.33%)    
Blood and lymphatic system disorders   
High Grade Lymphoma  [1]  1/9 (11.11%)  1
Endocrine disorders   
Insulin induced hypoglycemia  [2]  1/9 (11.11%)  9
Infections and infestations   
Methicillin-Resistant Staphylococcus Aureus (MRSA)  [3]  1/9 (11.11%)  1
Indicates events were collected by systematic assessment
[1]
One participant died of late lymphoma.
[2]
One known diabetic on tight insulin control died of insulin overdose.
[3]
One patient with prior splenectomy and thymectomy died of sepsis bacteremia.
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Hematopoietic Stem Cell Transplantation
Affected / at Risk (%) # Events
Total   3/9 (33.33%)    
Gastrointestinal disorders   
bowl obstruction  [1]  1/9 (11.11%)  1
Infections and infestations   
GR+ bacteriemia  [2]  1/9 (11.11%)  1
pneumonia  [3]  1/9 (11.11%)  1
Nervous system disorders   
Seizure  [4]  1/9 (11.11%)  1
Indicates events were collected by systematic assessment
[1]
One patient hospitalized for bowl obstruction that resolved without surgical intervention.
[2]
One patient developed pneumonia, resolved with antibiotics.
[3]
One patient with post transplant pneumonia resolved with antibiotics.
[4]
One participant was hospitalized after having seizure. The cause was idiopathic and workup was negative except for pituitary cyst, treated with Keppra with no further seizures.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Dr. Richard Burt
Organization: Northwestern University
Phone: 312-695-4960
Responsible Party: Richard Burt, MD, Northwestern University
ClinicalTrials.gov Identifier: NCT00424489     History of Changes
Other Study ID Numbers: Myasthenia Gravis Auto 2002
First Submitted: January 18, 2007
First Posted: January 19, 2007
Results First Submitted: June 20, 2017
Results First Posted: August 6, 2018
Last Update Posted: August 31, 2018