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Ephedrine for the Treatment of Congenital Myasthenia

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified October 2007 by Hadassah Medical Organization.
Recruitment status was:  Enrolling by invitation
Sponsor:
ClinicalTrials.gov Identifier:
NCT00541216
First Posted: October 10, 2007
Last Update Posted: October 10, 2007
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by:
Hadassah Medical Organization
  Purpose

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 Intervention Phase
Myasthenic Syndromes, Congenital Drug: Ephedrine Phase 1 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by Hadassah Medical Organization:

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

Estimated Enrollment: 15
Study Start Date: October 2007
  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   12 Years to 75 Years   (Child, Adult, Senior)
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
Israel
Hadassah Medical Organization, Jerusalem, Israel
Jerusalem, Israel
Sponsors and Collaborators
Hadassah Medical Organization
Investigators
Principal Investigator: Simon Edvardson Hadassah Medical Organization
  More Information

ClinicalTrials.gov Identifier: NCT00541216     History of Changes
Other Study ID Numbers: ephedrine-hmo-ctil
First Submitted: October 7, 2007
First Posted: October 10, 2007
Last Update Posted: October 10, 2007
Last Verified: October 2007

Keywords provided by Hadassah Medical Organization:
congenital myasthenia
ephedrine

Additional relevant MeSH terms:
Lambert-Eaton Myasthenic Syndrome
Myasthenic Syndromes, Congenital
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
Pseudoephedrine
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
Bronchodilator Agents
Anti-Asthmatic Agents
Respiratory System Agents
Nasal Decongestants