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

NCT02090725 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: D04013
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Muscle Weakness

Keywords

fluctuating muscle weakness; hyporeflexia; autonomic Dysfunction

Outcome Measures

Primary Outcomes (1)

• 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.

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

Study Arms (1)

3-4 Diaminopyridine (DAP)

EXPERIMENTAL

Drug: 3-4 Diaminopyridine

Interventions

3-4 Diaminopyridine DRUG

Also known as: 3-4 DAP

3-4 Diaminopyridine (DAP)

Eligibility Criteria

• Age: 45 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• diagnosed with Lambert-Eaton Myasthenic Syndrome.
• subjects must be taking full dose of pyridostigmine

You may not qualify if:

• does subject have a history of prolonged QTc syndrome (which is a condition where there is prolongation between the start of the Q wave and the end of the T wave in the heart's electrical cycle).

Sponsors & Collaborators

• Jeffrey A. Cohen, MD: lead
• Jacobus Pharmaceutical: collaborator

Study Sites (1)

Dartmouth-Hitchcock Medical Center

Lebanon, New Hampshire, 03221, United States

Location

MeSH Terms

Conditions

Muscle Weakness; Reflex, Abnormal; Primary Dysautonomias

Condition Hierarchy (Ancestors)

Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Manifestations; Neurologic Manifestations; Nervous System Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Autonomic Nervous System Diseases

Results Point of Contact

• Title: Jeffrey A. Cohen, MD
• Organization: Dartmouth-Hitchcock

jeffrey.a.cohen@hitchcock.org

603-650-4211

Study Officials

• Jeffrey A. Cohen, MD

  Dartmouth-Hitchcock Medical Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Neurology Department Chair

Study Record Dates

• First Submitted: March 14, 2014
• First Posted: March 18, 2014
• Study Start: February 1, 2004
• Primary Completion: July 1, 2018
• Study Completion: November 28, 2018
• Last Updated: May 21, 2019
• Results First Posted: May 21, 2019

Record last verified: 2019-05

Locations

US (1)