Ephedrine for the Treatment of Congenital Myasthenia

NCT ID: NCT00541216

Last Updated: 2007-10-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE1/PHASE2

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2007-10-31

Brief Summary

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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

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Conditions

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Myasthenic Syndromes, Congenital

Keywords

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congenital myasthenia ephedrine

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Interventions

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Ephedrine

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

12 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Hadassah Medical Organization

OTHER

Sponsor Role lead

Principal Investigators

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Simon Edvardson

Role: PRINCIPAL_INVESTIGATOR

Hadassah Medical Organization

Locations

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Hadassah Medical Organization, Jerusalem, Israel

Jerusalem, , Israel

Site Status

Countries

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Israel

Other Identifiers

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ephedrine-hmo-ctil

Identifier Type: -

Identifier Source: org_study_id