A Phase 2 interventional study of 3-4 Diaminopyridine in Muscle Weakness, sponsored by Jeffrey A. Cohen, MD. Terminated at 1 site in United States. Open to participants aged 45 Years to 65 Years. Per ClinicalTrials.gov, last updated 2019-05-21.
Sponsored by Jeffrey A. Cohen, MD · Phase 2, Interventional, and Treatment
The main purpose for this study is to provide access to 3,4 DAP, a drug which has demonstrated to be effective in treating weakness associated with Lambert-Eaton Myasthenic Syndrome. LEMS is a rare autoimmune cause of a defect in neuromuscular transmission. The disorder is clinically characterized by fluctuating muscle weakness, hyporeflexia and autonomic dysfunction.
More than half of LEMS cases are associated with malignancy, usually small cell lung cancer. These paraneoplastic cases progress more quickly than primary autoimmune LEMS. An overlap syndrome with other autoimmune diseases is often detected in LEMS patients.
3,4 DAP is effective in LEMS because it increases calcium influx into the nerve terminal by blocking potassium efflux and thereby prolonging the presynaptic action potential. 3,4 DAP is less likely to provoke epileptic seizures than its precursor, 4-aminopyridine, because it is less able to cross the blood-brain barrier. 3,4 DAP is effective in increasing strength and improving autonomic symptoms in LEMS patients of both the primary autoimmune and paraneoplastic etiologies.
601 studies on the registry are indexed under Muscle Weakness; 99 are open to participants now.
This study's enrollment of 4 is below the median of 46 across 444 interventional studies indexed under Muscle Weakness.
Browse Muscle Weakness studies →This is the only study on the registry with Jeffrey A. Cohen, MD as lead sponsor.
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Inclusion Criteria: -Male or female majority between 45 and 60 years of age
Exclusion Criteria: - does subject have a history of liver problems?
Drug: 3-4 Diaminopyridine
Also known as: 3-4 DAP
Number of Participants That Showed Improvement in Muscle Weakness During Their Last Study Related Visit
Muscle weakness will be assessed monthly for the first 3 months based on clinical assessment during office visits. Muscle weakness will then be assessed every 6 months once the patient is stabilized based on clinical assessments during office visits. The assessment of whether there was an improvement in muscle weakness, based on the PI's clinical judgment, was noted during the last study visit completed by the participant.
Time frame: Participants were followed until they withdrew or the study ended. Time frame ranged from 1 month to 3 years.
| Milestone | 3-4 Diaminopyridine (DAP) |
|---|---|
| Started | 4 |
| Completed | 0 |
| Not completed | 4 |
| Withdrew: Withdrawal by subject | 1 |
| Withdrew: Participants moved from area | 3 |
Muscle weakness will be assessed monthly for the first 3 months based on clinical assessment during office visits. Muscle weakness will then be assessed every 6 months once the patient is stabilized based on clinical assessments during office visits. The assessment of whether there was an improvement in muscle weakness, based on the PI's clinical judgment, was noted during the last study visit completed by the participant.
| Participants | 3-4 Diaminopyridine (DAP) |
|---|---|
| Number of Participants That Showed Improvement in Muscle Weakness During Their Last Study Related Visit | 3 |
Collected over Participants were followed until they withdrew from the trial. Adverse events were evaluated at Month 1, Month 2, Month 3, then every 6 months once patient is stable. The total time period over which adverse events were evaluated ranged from 2 weeks to 3 years.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| 3-4 Diaminopyridine (DAP) | 0/4 (0%) | 0/4 (0%) | 0/4 (0%) |
| Age, Categorical(Participants) | 3-4 Diaminopyridine (DAP) |
|---|---|
| <=18 years | 0 |
| Between 18 and 65 years | 4 |
| >=65 years | 0 |
| Sex: Female, Male(Participants) | 3-4 Diaminopyridine (DAP) |
|---|---|
| Female | 1 |
| Male | 3 |
| Race (NIH/OMB)(Participants) | 3-4 Diaminopyridine (DAP) |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 0 |
| White | 4 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | 3-4 Diaminopyridine (DAP) |
|---|---|
| United States | 4 |
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