Trial Outcomes & Findings for Hematopoietic Stem Cell Therapy for Patients With Refractory Myasthenia Gravis (NCT NCT00424489)

Results Overview

Survival

Recruitment status

TERMINATED

Study phase

PHASE1

Target enrollment

9 participants

Primary outcome timeframe

Up to 5 years

Results posted on

2018-08-31

Participant milestones
Measure	Hematopoietic Stem Cell Transplantation Autologous Hematopoietic Stem Cell Transplantation will be performed after conditioning Hematopoietic Stem Cell Transplantation: Autologous Hematopoietic Stem Cell Transplantation
Overall Study STARTED	9
Overall Study COMPLETED	6
Overall Study NOT COMPLETED	3

Reasons for withdrawal
Measure	Hematopoietic Stem Cell Transplantation Autologous Hematopoietic Stem Cell Transplantation will be performed after conditioning Hematopoietic Stem Cell Transplantation: Autologous Hematopoietic Stem Cell Transplantation
Overall Study Death	3

Hematopoietic Stem Cell Therapy for Patients With Refractory Myasthenia Gravis

Baseline characteristics by cohort
Measure	Hematopoietic Stem Cell Transplantation n=9 Participants Autologous Hematopoietic Stem Cell Transplantation will be performed after conditioning Hematopoietic Stem Cell Transplantation: Autologous Hematopoietic Stem Cell Transplantation
Age, Categorical <=18 years	1 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	8 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	45.22 years STANDARD_DEVIATION 16.49 • n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants
Region of Enrollment United States	9 Participants n=5 Participants

Timeframe: Up to 5 years

Population: Survival both treatment related and non-treatment related

Survival

Outcome measures
Measure	Hematopoietic Stem Cell Transplantation n=9 Participants Autologous Hematopoietic Stem Cell Transplantation will be performed after conditioning Hematopoietic Stem Cell Transplantation: Autologous Hematopoietic Stem Cell Transplantation
Survival	6 Participants

Serious events: 3 serious events

Other events: 3 other events

Deaths: 3 deaths

Serious adverse events
Measure	Hematopoietic Stem Cell Transplantation n=9 participants at risk Autologous Hematopoietic Stem Cell Transplantation will be performed after conditioning Hematopoietic Stem Cell Transplantation: Autologous Hematopoietic Stem Cell Transplantation
Infections and infestations Methicillin-Resistant Staphylococcus Aureus (MRSA)	11.1% 1/9 • Number of events 1 • Up to 5 years
Endocrine disorders Insulin induced hypoglycemia	11.1% 1/9 • Number of events 9 • Up to 5 years
Blood and lymphatic system disorders High Grade Lymphoma	11.1% 1/9 • Number of events 1 • Up to 5 years

Other adverse events
Measure	Hematopoietic Stem Cell Transplantation n=9 participants at risk Autologous Hematopoietic Stem Cell Transplantation will be performed after conditioning Hematopoietic Stem Cell Transplantation: Autologous Hematopoietic Stem Cell Transplantation
Infections and infestations GR+ bacteriemia	11.1% 1/9 • Number of events 1 • Up to 5 years
Infections and infestations pneumonia	11.1% 1/9 • Number of events 1 • Up to 5 years
Nervous system disorders Seizure	11.1% 1/9 • Number of events 1 • Up to 5 years
Gastrointestinal disorders bowl obstruction	11.1% 1/9 • Number of events 1 • Up to 5 years

Northwestern University

Phone: 312-695-4960