Trial Information
Inclusion Criteria:
- Male or female patients , with congenital myasthenia, belonging to a previously
reported kindred diagnosed with COLQ deficiency.
Exclusion Criteria:
- History of allergy to Ephedrine or any inactive component.
- Significant abnormalities in screening Cardiovascular parameters (blood pressure,
pulse).
- Surgery within 6 weeks of screening.
- Concurrent use of any other medication except steroids.
- Pregnancy.
- Thyrotoxicosis.
- Co-morbid conditions or other neurological disorders that would confound assessment
of clinical parameters.
- Participation in another clinical trial within 30 days of study start.
- Patients who are non-cooperative or parents/ legal guardians who are unwilling to
sign consent form.
Type of Study:
Interventional
Study Design:
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
Outcome Measure:
strength and fatiguability: walking, straight arm raising, spirometry.
Outcome Time Frame:
5 weeks
Principal Investigator
Simon Edvardson
Investigator Role:
Principal Investigator
Investigator Affiliation:
Hadassah Medical Organization
Authority:
Israel: Israeli Health Ministry Pharmaceutical Administration
Study ID:
ephedrine-hmo-ctil
NCT ID:
NCT00541216
Start Date:
October 2007
Completion Date:
Related Keywords:
- Myasthenic Syndromes, Congenital
- congenital myasthenia
- ephedrine
- Lambert-Eaton Myasthenic Syndrome
- Myasthenic Syndromes, Congenital