Clinical Trial: Ephedrine for the Treatment of Congenital Myasthenia

Study Status: Recruiting
Recruit Status: Unknown status
Study Type: Interventional

Official Title:

Brief Summary:

Previous research has demonstrated possible efficacy of Ephedrine in the treatment of congenital myasthenia caused by end-plate acetylcholinesterase deficiency.

The aim of the current study is to test the hypothesis that Ephedrine may be beneficial to these patients.

To test this hypothesis we will perform a double blind, placebo-controlled, crossover study clinical efficacy and safety study.

Drug naïve patients who agree to participate will be randomized to two groups. Each group will be treated in a blinded manner for 5 weeks with either placebo or Ephedrine HCl in an escalating dose up to 100 mg per day divided in two doses. After five weeks the groups will cross over and continue treatment or placebo for a further five weeks.

Evaluations of strength and fatiguability will be done at baseline, at the end of each five week period and after a further two weeks.

Safety will be assessed weekly by the investigators using interview and physical examination.

Outcome measures will include Barthel index, Quality of life questionnaire, Timed up and go, spirometry, timed elevation of limbs, and force measurements.

All patients will report to the clinic as per study schedule (See Appendix A). Specifically, the 12 clinic visits will include: baseline (1), safety and efficacy assessments(10) and closeout (1).

Detailed Summary:
Sponsor: Hadassah Medical Organization

Current Primary Outcome: strength and fatiguability: walking, straight arm raising, spirometry. [ Time Frame: 5 weeks ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Hadassah Medical Organization

Dates:
Date Received: October 7, 2007
Date Started: October 2007
Date Completion:
Last Updated: October 9, 2007
Last Verified: October 2007