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
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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).
|Condition or disease||Intervention/treatment||Phase|
|Myasthenic Syndromes, Congenital||Drug: Ephedrine||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||15 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Study Start Date :||October 2007|
- strength and fatiguability: walking, straight arm raising, spirometry. [ Time Frame: 5 weeks ]
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|Ages Eligible for Study:||12 Years to 75 Years (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
- Male or female patients , with congenital myasthenia, belonging to a previously reported kindred diagnosed with COLQ deficiency.
- 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.
- 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.
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
|Hadassah Medical Organization, Jerusalem, Israel|
|Principal Investigator:||Simon Edvardson||Hadassah Medical Organization|
|Other Study ID Numbers:||
|First Posted:||October 10, 2007 Key Record Dates|
|Last Update Posted:||October 10, 2007|
|Last Verified:||October 2007|
Lambert-Eaton Myasthenic Syndrome
Myasthenic Syndromes, Congenital
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
Neoplasms by Site
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Neuromuscular Junction Diseases
Immune System Diseases
Genetic Diseases, Inborn
Central Nervous System Stimulants
Physiological Effects of Drugs
Peripheral Nervous System Agents
Molecular Mechanisms of Pharmacological Action