Myasthenic Syndromes, Congenital Clinical Trial
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).
n/a
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
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Phase 3 | |
| No longer available |
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