Ephedrine for the Treatment of Congenital Myasthenia

• ClinicalTrials.gov Identifier: NCT00541216
• Recruitment Status: Unknown
• First Posted: October 10, 2007
• Last Update Posted: October 10, 2007
• Sponsor: Hadassah Medical Organization
• Information provided by: Hadassah Medical Organization

Tracking Information

• First Submitted Date: October 7, 2007
• First Posted Date: October 10, 2007
• Last Update Posted Date: October 10, 2007
• Study Start Date: October 2007
• Primary Completion Date: Not Provided
• Current Primary Outcome Measures (submitted: October 9, 2007): strength and fatiguability: walking, straight arm raising, spirometry. [ Time Frame: 5 weeks ]
• Original Primary Outcome Measures: Same as current
• Change History: No Changes Posted
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Ephedrine for the Treatment of Congenital Myasthenia
• Official Title: Not Provided

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 Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1; Phase 2
• Study Design: Allocation: Randomized; Intervention Model: Crossover Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Myasthenic Syndromes, Congenital
• Intervention: Drug: Ephedrine
• Study Arms: Not Provided
• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Unknown status
• Estimated Enrollment (submitted: October 9, 2007): 15
• Original Estimated Enrollment: Same as current
• Study Completion Date: Not Provided
• Primary Completion Date: Not Provided

Eligibility Criteria

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 12 Years to 75 Years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: Not Provided
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Israel

Administrative Information

• NCT Number: NCT00541216
• Other Study ID Numbers: ephedrine-hmo-ctil
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Not Provided
• Original Responsible Party: Same as current
• Current Study Sponsor: Hadassah Medical Organization
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: Simon Edvardson; Hadassah Medical Organization

• PRS Account: Hadassah Medical Organization
• Verification Date: October 2007