Ignite Creation Date:
2025-12-26 @ 11:48 AM
Ignite Modification Date:
2025-12-26 @ 11:48 AM
Study NCT ID:
NCT00541216
Status:
UNKNOWN
Last Update Posted:
2007-10-10
First Post:
2007-10-07
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Ephedrine for the Treatment of Congenital Myasthenia
Sponsor:
Hadassah Medical Organization
Organization:
Study Overview
Official Title:
None
Status:
UNKNOWN
Status Verified Date:
2007-10
Last Known Status:
ENROLLING_BY_INVITATION
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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).
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:
None
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: