Myasthenic Syndromes, CongenitalNCT01765140
Treatment Use of 3,4-Diaminopyridine
A study testing 3,4-diaminopyridine in people with Myasthenic Syndromes, Congenital, run by Vern C. Juel, M.D. at 1 site in United States.
Trial summary
This protocol provided 3,4 diaminopyridine (DAP) under a treatment-use IND to patients with congenital myasthenic syndrome (CMS). It is now closed. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Myasthenic Syndromes, Congenital
- What is being tested?
- 3,4-diaminopyridine
- How many people
- Not given
- Where
- 1 siteUnited States
- Study dates
- Start not givento not given
- Phase
- Expanded access
- 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 Any age
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of congenital myasthenic syndrome (CMS)
- Women of childbearing potential must have negative pregnancy test and agree to practice adequate contraception while taking DAP
- Must be competent to give consent
Who cannot join
- Known seizure disorder
- Pregnancy
- Known cardiac arrhythmia or evidence of significant arrhythmia on screening ECG
- Known hepatic, renal or hematologic disease
Trial locations and contacts
1 site in 1 country.
United States1 site
Duke University Hospital
Durham, North Carolina, 27710
About this study
Official title: Treatment Use of 3,4-Diaminopyridine in Congenital Myasthenic Syndrome
This protocol provided 3,4 diaminopyridine (DAP) under a treatment-use IND to patients with congenital myasthenic syndrome (CMS). It is now closed.
Read the detailed description
CMS diagnoses were made based on clinical, electrophysiologic and molecular genetic findings. All patients were referred to the PI for DAP treatment. This study enrolled minors and adults.
CMS patients under age 18 years were included if their parent or guardian gave written permission. Minors who turned 18 while in the program were re-consented as adults.
The dose of DAP was determined individually for each patient. Adults were started with a dose of 10 mg 3-4 times daily, increased over several weeks to the dose that produced the maximum symptomatic response, not to exceed 100 mg daily. Pyridostigmine bromide (PB) was often added at low doses and increased to the dose that produced the best response, not to exceed 360 mg daily. In children, equivalent doses of these medications were calculated on a surface area basis. The doses of DAP and PB were periodically adjusted to assure that the smallest effective doses are used.
- Conditions
- Myasthenic Syndromes, Congenital
- Being tested
- 3,4-diaminopyridine
- Keywords
- 3,4 diaminopyridine (DAP)
- 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.
3,4-diaminopyridineDrug
Also called DAP
Treatment use of 3,4-DAP for patients with congenital myasthenic syndrome (CMS)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jan 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Myasthenic Syndromes, CongenitalStudies 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- Vern C. Juel, M.D.
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Vern C. Juel, M.D., Duke University
- Investigators
- Vern C. Juel, M.D., Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01765140
- Sponsor's protocol ID
- Pro00007811
- Status last verified
- Jan 2025
- First submitted
- 6 Jan 2013
- Last updated
- 20 Jan 2025
- Results posted
- No
Publications
Sanders DB, Juel VC, Harati Y, Smith AG, Peltier AC, Marburger T, Lou JS, Pascuzzi RM, Richman DP, Xie T, Demmel V, Jacobus LR, Ales KL, Jacobus DP; Dapper Study Team. 3,4-diaminopyridine base effectively treats the weakness of Lambert-Eaton myasthenia. Muscle Nerve. 2018 Apr;57(4):561-568. doi: 10.1002/mus.26052. Epub 2018 Feb 2.
PubMed 29280483
Study at a glance
From the registry record, last updated by the sponsor on 20 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment Use of 3,4-Diaminopyridine in Congenital Myasthenic Syndrome
- ClinicalTrials.gov ID
- NCT01765140
- Acronym / sponsor ID
- Pro00007811
- Conditions
- Myasthenic Syndromes, Congenital
- Keywords
- 3,4 diaminopyridine (DAP)
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- Vern C. Juel, M.D.University, hospital or other
- First posted
- 10 Jan 2013 (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.
This protocol provided 3,4 diaminopyridine (DAP) under a treatment-use IND to patients with congenital myasthenic syndrome (CMS). It is now closed.
Detailed description
CMS diagnoses were made based on clinical, electrophysiologic and molecular genetic findings. All patients were referred to the PI for DAP treatment. This study enrolled minors and adults.
CMS patients under age 18 years were included if their parent or guardian gave written permission. Minors who turned 18 while in the program were re-consented as adults.
The dose of DAP was determined individually for each patient. Adults were started with a dose of 10 mg 3-4 times daily, increased over several weeks to the dose that produced the maximum symptomatic response, not to exceed 100 mg daily. Pyridostigmine bromide (PB) was often added at low doses and increased to the dose that produced the best response, not to exceed 360 mg daily. In children, equivalent doses of these medications were calculated on a surface area basis. The doses of DAP and PB were periodically adjusted to assure that the smallest effective doses are used.
Study design
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Diagnosis of congenital myasthenic syndrome (CMS)
- Women of childbearing potential must have negative pregnancy test and agree to practice adequate contraception while taking DAP
- Must be competent to give consent
Key exclusion criteria (4 listed)
- Known seizure disorder
- Pregnancy
- Known cardiac arrhythmia or evidence of significant arrhythmia on screening ECG
- Known hepatic, renal or hematologic disease
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: DAPTreatment use of 3,4-DAP for patients with congenital myasthenic syndrome (CMS) | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jan 2025.
- Results publication: PubMed 29280483Sanders DB, Juel VC, Harati Y, Smith AG, Peltier AC, Marburger T, Lou JS, Pascuzzi RM, Richman DP, Xie T, Demmel V, Jacobus LR, Ales KL, Jacobus DP; Dapper Study Team. 3,4-diaminopyridine base effectively treats the weakness of Lambert-Eaton myasthenia. Muscle Nerve. 2018 Apr;57(4):561-568. doi: 10.1002/mus.26052. Epub 2018 Feb 2.
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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.
Vern C. Juel, M.D. principal investigator
Duke University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing 3,4-diaminopyridine in people with Myasthenic Syndromes, Congenital, run by Vern C. Juel, M.D. 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?
The sponsor lists 3 inclusion and 4 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 20 Jan 2025. 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.
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.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01765140, which also lists 1 linked publication.
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 20 Jan 2025.