Ephedrine for the Treatment of Congenital Myasthenia

October 9, 2007 updated by: Hadassah Medical Organization

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).

Study Overview

Status

Unknown

Conditions

Myasthenic Syndromes, Congenital

Intervention / Treatment

Drug: Ephedrine

Study Type

Interventional

Enrollment (Anticipated)

Phase

Phase 2
Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Israel
- - Jerusalem, Israel
    - Hadassah Medical Organization, Jerusalem, Israel

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

8 years to 71 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Primary Purpose: Treatment
Allocation: Randomized
Interventional Model: Crossover Assignment
Masking: Quadruple

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
strength and fatiguability: walking, straight arm raising, spirometry. Time Frame: 5 weeks	5 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Hadassah Medical Organization

Investigators

Principal Investigator: Simon Edvardson, Hadassah Medical Organization

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

October 1, 2007

Study Registration Dates

First Submitted

October 7, 2007

First Submitted That Met QC Criteria

October 9, 2007

First Posted (Estimate)

October 10, 2007

Study Record Updates

Last Update Posted (Estimate)