Ephedrine for the Treatment of Congenital Myasthenia
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00541216|
Recruitment Status : Unknown
Verified October 2007 by Hadassah Medical Organization.
Recruitment status was: Enrolling by invitation
First Posted : October 10, 2007
Last Update Posted : October 10, 2007
|First Submitted Date ICMJE||October 7, 2007|
|First Posted Date ICMJE||October 10, 2007|
|Last Update Posted Date||October 10, 2007|
|Study Start Date ICMJE||October 2007|
|Primary Completion Date||Not Provided|
|Current Primary Outcome Measures ICMJE
||strength and fatiguability: walking, straight arm raising, spirometry. [ Time Frame: 5 weeks ]|
|Original Primary Outcome Measures ICMJE||Same as current|
|Change History||No Changes Posted|
|Current Secondary Outcome Measures ICMJE||Not Provided|
|Original Secondary Outcome Measures ICMJE||Not Provided|
|Current Other Pre-specified Outcome Measures||Not Provided|
|Original Other Pre-specified Outcome Measures||Not Provided|
|Brief Title ICMJE||Ephedrine for the Treatment of Congenital Myasthenia|
|Official Title ICMJE||Not Provided|
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 Description||Not Provided|
|Study Type ICMJE||Interventional|
|Study Phase ICMJE||Phase 1
|Study Design ICMJE||Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Condition ICMJE||Myasthenic Syndromes, Congenital|
|Intervention ICMJE||Drug: Ephedrine|
|Study Arms ICMJE||Not Provided|
|Publications *||Not Provided|
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
|Recruitment Status ICMJE||Unknown status|
|Estimated Enrollment ICMJE
|Original Estimated Enrollment ICMJE||Same as current|
|Study Completion Date ICMJE||Not Provided|
|Primary Completion Date||Not Provided|
|Eligibility Criteria ICMJE||
|Ages ICMJE||12 Years to 75 Years (Child, Adult, Older Adult)|
|Accepts Healthy Volunteers ICMJE||Not Provided|
|Contacts ICMJE||Contact information is only displayed when the study is recruiting subjects|
|Listed Location Countries ICMJE||Israel|
|Removed Location Countries|
|NCT Number ICMJE||NCT00541216|
|Other Study ID Numbers ICMJE||ephedrine-hmo-ctil|
|Has Data Monitoring Committee||No|
|U.S. FDA-regulated Product||Not Provided|
|IPD Sharing Statement ICMJE||Not Provided|
|Current Responsible Party||Not Provided|
|Original Responsible Party||Same as current|
|Current Study Sponsor ICMJE||Hadassah Medical Organization|
|Original Study Sponsor ICMJE||Same as current|
|Collaborators ICMJE||Not Provided|
|PRS Account||Hadassah Medical Organization|
|Verification Date||October 2007|
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP