Ephedrine for the Treatment of Congenital Myasthenia

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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

View this study on Beta.ClinicalTrials.gov

Sponsor:

Information provided by:

Hadassah Medical Organization

Study Description

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).

Condition or disease	Intervention/treatment	Phase
Myasthenic Syndromes, Congenital	Drug: Ephedrine	Phase 1 Phase 2

Study Design

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Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	15 participants
Allocation:	Randomized
Intervention Model:	Crossover Assignment
Masking:	Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose:	Treatment
Study Start Date :	October 2007

Resource links provided by the National Library of Medicine

MedlinePlus Genetics related topics: Congenital myasthenic syndrome

Drug Information available for: Ephedrine Hydrochloride Pseudoephedrine Ephedrine sulfate Ephedrine Pseudoephedrine hydrochloride Pseudoephedrine sulfate

Genetic and Rare Diseases Information Center resources: Lambert Eaton Myasthenic Syndrome Congenital Myasthenic Syndromes Myasthenia Gravis

U.S. FDA Resources

Arms and Interventions

Outcome Measures

Primary Outcome Measures :

strength and fatiguability: walking, straight arm raising, spirometry. [ Time Frame: 5 weeks ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:	12 Years to 75 Years (Child, Adult, Older Adult)
Sexes Eligible for Study:	All

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.

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00541216

Locations

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Israel
Hadassah Medical Organization, Jerusalem, Israel
Jerusalem, Israel

Sponsors and Collaborators

Hadassah Medical Organization

Investigators

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Principal Investigator:	Simon Edvardson	Hadassah Medical Organization

More Information

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ClinicalTrials.gov Identifier:	NCT00541216
Other Study ID Numbers:	ephedrine-hmo-ctil
First Posted:	October 10, 2007 Key Record Dates
Last Update Posted:	October 10, 2007
Last Verified:	October 2007

Keywords provided by Hadassah Medical Organization:


congenital myasthenia ephedrine

Additional relevant MeSH terms:

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Lambert-Eaton Myasthenic Syndrome Myasthenic Syndromes, Congenital Myasthenia Gravis Paraneoplastic Syndromes, Nervous System Nervous System Neoplasms Neoplasms by Site Neoplasms Paraneoplastic Syndromes Autoimmune Diseases of the Nervous System Nervous System Diseases Neurodegenerative Diseases Neuromuscular Junction Diseases Neuromuscular Diseases	Autoimmune Diseases Immune System Diseases Genetic Diseases, Inborn Ephedrine Central Nervous System Stimulants Physiological Effects of Drugs Sympathomimetics Autonomic Agents Peripheral Nervous System Agents Vasoconstrictor Agents Adrenergic Agents Neurotransmitter Agents Molecular Mechanisms of Pharmacological Action

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