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Phase 1/Phase 2
12 Years
75 Years
Open (Enrolling by invite only)
Both
Myasthenic Syndromes, Congenital

Thank you

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

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