Myasthenic Syndromes, CongenitalPhase 1/2NCT00541216
Ephedrine for the Treatment of Congenital Myasthenia
A Phase 1/2 study testing Ephedrine in people with Myasthenic Syndromes, Congenital, run by Hadassah Medical Organization at 1 site in Israel.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 to 75 years
- Conditions the study is for
- Myasthenic Syndromes, Congenital
- What is being tested?
- Ephedrine
- How many people
- 15 participants
- Where
- 1 siteIsrael
- Study dates
- Oct 2007to not given
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 75 years
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Male or female patients , with congenital myasthenia, belonging to a previously reported kindred diagnosed with COLQ deficiency.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read more
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).
- Conditions
- Myasthenic Syndromes, Congenital
- Being tested
- Ephedrine
- Keywords
- congenital myasthenia, ephedrine
- Medical subject headings
- Myasthenic Syndromes, Congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EphedrineDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
strength and fatiguability: walking, straight arm raising, spirometry.
Measured: 5 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Oct 2007 (estimated)
- Study startOct 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myasthenic Syndromes, CongenitalStudies of the same condition, open and under way.
- More trials run by Hadassah Medical OrganizationThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hadassah Medical Organization
- Sponsor type
- University, hospital or other
- Investigators
- Simon Edvardson, Principal investigator, Hadassah Medical Organization
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00541216
- Sponsor's protocol ID
- ephedrine-hmo-ctil
- Status last verified
- Oct 2007
- First submitted
- 7 Oct 2007
- Last updated
- 10 Oct 2007
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Oct 2007. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ephedrine for the Treatment of Congenital Myasthenia
- ClinicalTrials.gov ID
- NCT00541216
- Acronym / sponsor ID
- ephedrine-hmo-ctil
- Conditions
- Myasthenic Syndromes, Congenital
- Keywords
- congenital myasthenia, ephedrine
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Hadassah Medical OrganizationUniversity, hospital or other
- Study start
- Oct 2007
- First posted
- 10 Oct 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
Who may qualify
- Ages
- 12 to 75 years
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Male or female patients , with congenital myasthenia, belonging to a previously reported kindred diagnosed with COLQ deficiency.
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ephedrine | Drug | – |
Primary outcome
strength and fatiguability: walking, straight arm raising, spirometry.
Time frame: 5 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Oct 2007.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Simon Edvardson principal investigator
Hadassah Medical Organization
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Ephedrine in people with Myasthenic Syndromes, Congenital, run by Hadassah Medical Organization at 1 site in Israel.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 10 Oct 2007. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
strength and fatiguability: walking, straight arm raising, spirometry. (time frame: 5 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Oct 2007.