Muscle WeaknessPhase 23-4DAPNCT02090725
Controlled Trial of 3,4-Diaminopyridine (3-4DAP) in Lambert-Eaton Myasthenic Syndrome (LEMS)
A Phase 2 study testing 3-4 Diaminopyridine in people with Muscle Weakness, run by Jeffrey A. Cohen, MD at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 45 to 65 years
- Conditions the study is for
- Muscle Weakness
- What is being tested?
- 3-4 Diaminopyridine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2004to 28 Nov 2018
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 45 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Inclusion Criteria: -Male or female majority between 45 and 60 years of age
- diagnosed with Lambert-Eaton Myasthenic Syndrome.
- subjects must be taking full dose of pyridostigmine
Exclusion Criteria: - does subject have a history of liver problems?
- 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).
Trial locations and contacts
1 site in 1 country.
United States1 site
Dartmouth-Hitchcock Medical Center
Lebanon, New Hampshire, 03221
About this study
Official title: Controlled Trial of 3,4-Diaminopyridine in LEMS
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.
Read the detailed description
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.
- Conditions
- Muscle Weakness
- Being tested
- 3-4 Diaminopyridine
- Keywords
- fluctuating muscle weakness, hyporeflexia, autonomic Dysfunction
- Medical subject headings
- Muscle Weakness, Reflex, Abnormal, Primary Dysautonomias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
3-4 DiaminopyridineDrug
Also called 3-4 DAP
| Study group | Role | What they receive |
|---|---|---|
| 3-4 Diaminopyridine (DAP) | Experimental | Not described Receives: Drug: 3-4 Diaminopyridine |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Participants were followed until they withdrew or the study ended. Time frame ranged from 1 month to 3 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2014 (estimated)
- Study startFeb 2004
- Primary completionJul 2018
- Study completion28 Nov 2018
Why it stopped: Drug receive FDA approval
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Muscle WeaknessStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Jeffrey A. Cohen, MD
- Sponsor type
- University, hospital or other
- Collaborators
- Jacobus Pharmaceutical
- Responsible party
- Sponsor-investigator, Jeffrey A. Cohen, MD, Dartmouth-Hitchcock Medical Center
- Investigators
- Jeffrey A. Cohen, MD, Principal investigator, Dartmouth-Hitchcock Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02090725
- Sponsor's protocol ID
- D04013
- Acronym
- 3-4DAP
- Status last verified
- May 2019
- First submitted
- 14 Mar 2014
- Last updated
- 21 May 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 May 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Controlled Trial of 3,4-Diaminopyridine in LEMS
- ClinicalTrials.gov ID
- NCT02090725
- Acronym / sponsor ID
- 3-4DAP / D04013
- Conditions
- Muscle Weakness
- Keywords
- fluctuating muscle weakness, hyporeflexia, autonomic Dysfunction
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Drug receive FDA approval
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Jeffrey A. Cohen, MDUniversity, hospital or other
- Collaborators
- Jacobus Pharmaceutical
- Study start
- Feb 2004
- Primary completion
- Jul 2018 (actual)
- Study completion
- 28 Nov 2018 (actual)
- First posted
- 18 Mar 2014 (estimated)
- Results
- Posted 21 May 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 3-4 Diaminopyridine (DAP) | Experimental | Drug: 3-4 Diaminopyridine |
Who may qualify
- Ages
- 45 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
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 |
|---|---|---|
| 3-4 DiaminopyridineAlso: 3-4 DAP | Drug | 3-4 Diaminopyridine (DAP) |
Primary outcome
Number of Participants That Showed Improvement in Muscle Weakness During Their Last Study Related Visit
Time frame: Participants were followed until they withdrew or the study ended. Time frame ranged from 1 month to 3 years.
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 May 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Jeffrey A. Cohen, MD principal investigator
Dartmouth-Hitchcock Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing 3-4 Diaminopyridine in people with Muscle Weakness, run by Jeffrey A. Cohen, MD at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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: stopped early, as of the sponsor's last update on 21 May 2019. 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 United States. 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 phase mean?
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?
Yes. Results are posted in the registry (first posted 21 May 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants That Showed Improvement in Muscle Weakness During Their Last Study Related Visit (time frame: Participants were followed until they withdrew or the study ended. Time frame ranged from 1 month to 3 years.). 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 21 May 2019.