Ephedrine for the Treatment of Congenital Myasthenia

This study is enrolling participants by invitation only.
Sponsor:
Information provided by:
Hadassah Medical Organization
ClinicalTrials.gov Identifier:
NCT00541216
First received: October 7, 2007
Last updated: October 9, 2007
Last verified: October 2007

October 7, 2007
October 9, 2007
October 2007
Not Provided
strength and fatiguability: walking, straight arm raising, spirometry. [ Time Frame: 5 weeks ]
Same as current
Complete list of historical versions of study NCT00541216 on ClinicalTrials.gov Archive Site
Not Provided
Not Provided
Not Provided
Not Provided
 
Ephedrine for the Treatment of Congenital Myasthenia
Not Provided

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).

Not Provided
Interventional
Phase 1
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Myasthenic Syndromes, Congenital
Drug: Ephedrine
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Enrolling by invitation
15
Not Provided
Not Provided

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.
Both
12 Years to 75 Years
Not Provided
Contact information is only displayed when the study is recruiting subjects
Israel
 
NCT00541216
ephedrine-hmo-ctil
No
Not Provided
Hadassah Medical Organization
Not Provided
Principal Investigator: Simon Edvardson Hadassah Medical Organization
Hadassah Medical Organization
October 2007

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP