Trial Outcomes & Findings for Controlled Trial of 3,4-Diaminopyridine (3-4DAP) in Lambert-Eaton Myasthenic Syndrome (LEMS) (NCT NCT02090725)

NCT ID: NCT02090725

Last Updated: 2019-05-21

Results Overview

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.

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 4 participants
• Primary outcome timeframe: Participants were followed until they withdrew or the study ended. Time frame ranged from 1 month to 3 years.
• Results posted on: 2019-05-21

Participant Flow

Participant milestones

Measure	3-4 Diaminopyridine (DAP) 3-4 Diaminopyridine Treatment will begin with 5mg three time a day. A common final dosage is 15mg four or five time a day, as clinically needed, and if tolerated. The upper limit is a total of 100mg/day.
Overall Study STARTED	4
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Measure	3-4 Diaminopyridine (DAP) 3-4 Diaminopyridine Treatment will begin with 5mg three time a day. A common final dosage is 15mg four or five time a day, as clinically needed, and if tolerated. The upper limit is a total of 100mg/day.
Overall Study Withdrawal by Subject	1
Overall Study participants moved from area	3

Baseline Characteristics

Controlled Trial of 3,4-Diaminopyridine (3-4DAP) in Lambert-Eaton Myasthenic Syndrome (LEMS)

Baseline characteristics by cohort

Measure	3-4 Diaminopyridine (DAP) n=4 Participants 3-4 Diaminopyridine
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	4 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	4 participants n=5 Participants

PRIMARY outcome

Timeframe: Participants were followed until they withdrew or the study ended. Time frame ranged from 1 month to 3 years.

Population: One participant was not on the study long enough to complete the one month assessment.

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.

Outcome measures

Measure	3-4 Diaminopyridine (DAP) n=3 Participants 3-4 Diaminopyridine
Number of Participants That Showed Improvement in Muscle Weakness During Their Last Study Related Visit	3 Participants

Adverse Events

3-4 Diaminopyridine (DAP)

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Jeffrey A. Cohen, MD

Dartmouth-Hitchcock

Phone: 603-650-4211

Email: jeffrey.a.cohen@hitchcock.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place