Lambert Eaton Myasthenic SyndromeNCT00704925
Treatment of Lambert-Eaton Syndrome With 3,4 DAP
A study testing 3, 4 DAP in people with Lambert Eaton Myasthenic Syndrome, run by David P. Richman, MD at 1 site in United States.
Trial summary
A new drug called 3,4-Diaminopyridine (3,4-DAP) is currently under investigation for treatment of the symptoms of Lambert-Eaton Myasthenic Syndrome (LEMS). This is an expanded access trial, which means that although data from this study will be collected and reported to the US Food and Drug Administration (FDA)and the… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Lambert Eaton Myasthenic Syndrome
- What is being tested?
- 3, 4 DAP
- Type of treatment
- Pill or oral medicine
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Be 18 years or older, diagnosed with LEMS
- If female, have negative pregnancy test and if premenopausal, be willing to practice an effective form of birth control during the study.
Who cannot join
- Is known to have a sensitivity to 3, 4 DAP
- Has a history of past or current seizures
- Has a history of past or current severe asthma
- Is believed by the investigator to be unable to comply with the protocol
- Unable to give informed consent
Trial locations and contacts
1 site in 1 country.
United States1 site
University of California, Davis
Sacramento, California, 95817
About this study
Official title: Treatment of Lambert-Eaton Syndrome With 3, 4-Diaminopyridine
A new drug called 3,4-Diaminopyridine (3,4-DAP) is currently under investigation for treatment of the symptoms of Lambert-Eaton Myasthenic Syndrome (LEMS). This is an expanded access trial, which means that although data from this study will be collected and reported to the US Food and Drug Administration (FDA)and the drug manufacturer, this is not a formal study of drug in LEMS.
If you decide volunteer, you will be evaluated by a neurologist to determine your eligibility to receive 3, 4-DAP by a review of your medical history, medication regimen (the medications you are taking) and a neurological examination. If you are a female of child-bearing potential, a serum pregnancy test will be done to ensure that you are not pregnant. Once it is determined that this treatment is appropriate for your care, you will begin taking 3, 4 DAP by mouth in slowly increasing doses. Treatment will begin with 5mg three times a day, as clinically needed, and if tolerated. You will be monitored for strength and side effects by routine clinic visits at initial intervals of once a month, increasing to intervals of every 12 months as permitted. Blood will be drawn (approximately 1 tablespoon) at every clinic visit or as often as the investigator deems necessary to assess your liver/kidney function and blood counts. You will have an EKG (a test to see how your heart is functioning) at your first study visit, after 6 months of taking 3,4 DAP and again every 2 years. Treatment will be continued indefinitely if a good clinical response is achieved. This study is planned to last indefinitely.
The dosage of 3, 4DAP is individually adjusted. The usual range is 10-15 mg 3-4 times per day for the full effect and will increase by 50% every two weeks to 10-15 mg three to six times a day, as needed and if tolerated. Dosages above the full effect level will not provide an additional benefit and should not be used. 3, 4 DAP is a convulsant (causes seizures). A total of 100 mg/day is the maximum dosage allowed.
Read the detailed description
Patients with clinically-confirmed LEMS will receive 3, 4-DAP by mouth in slowly increasing doses. Treatment will begin with 5 mg, three times a day and will increase by 50% every two weeks to 10-15 mg, three to six times a day, as clinically needed, and if tolerated. Patients will be monitored for strength and side effects via routine out-patient clinic visits at initial intervals of 1 month, increasing to intervals of 12 months as permitted. Results of treatment and adverse events will be reported to the FDA. Treatment will be continued indefinitely if a good clinical response is achieved and side effects are tolerable.
- Conditions
- Lambert Eaton Myasthenic Syndrome
- Being tested
- 3, 4 DAP
- Keywords
- Lambert Eaton Myasthenic Syndrome, 3,4 DAP, 3,4 Diaminopyridine
- Medical subject headings
- Lambert-Eaton Myasthenic Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
3, 4 DAPDrug
Also called 3,4 diaminopyridine
Patients with clinically-confirmed LEMS will receive 3, 4 DAP by mouth in slowly increasing doses. Treatment will begin with 5 mg three times a day and will increase by 50% every two weeks to 10-15 mg three to six times a day, as clinically needed, and if tolerated.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jun 2008 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lambert Eaton Myasthenic SyndromeStudies 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
- David P. Richman, MD
- Sponsor type
- University, hospital or other
- Collaborators
- Jacobus Pharmaceutical
- Responsible party
- Sponsor-investigator, David P. Richman, MD, University of California, Davis
- Investigators
- David Richman, MD, Principal investigator, University of California, Davis
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00704925
- Sponsor's protocol ID
- 200311036
- Status last verified
- Oct 2019
- First submitted
- 23 Jun 2008
- Last updated
- 30 Oct 2019
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of Lambert-Eaton Syndrome With 3, 4-Diaminopyridine
- ClinicalTrials.gov ID
- NCT00704925
- Acronym / sponsor ID
- 200311036
- Conditions
- Lambert Eaton Myasthenic Syndrome
- Keywords
- Lambert Eaton Myasthenic Syndrome, 3,4 DAP, 3,4 Diaminopyridine
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- David P. Richman, MDUniversity, hospital or other
- Collaborators
- Jacobus Pharmaceutical
- First posted
- 25 Jun 2008 (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.
A new drug called 3,4-Diaminopyridine (3,4-DAP) is currently under investigation for treatment of the symptoms of Lambert-Eaton Myasthenic Syndrome (LEMS). This is an expanded access trial, which means that although data from this study will be collected and reported to the US Food and Drug Administration (FDA)and the drug manufacturer, this is not a formal study of drug in LEMS.
If you decide volunteer, you will be evaluated by a neurologist to determine your eligibility to receive 3, 4-DAP by a review of your medical history, medication regimen (the medications you are taking) and a neurological examination. If you are a female of child-bearing potential, a serum pregnancy test will be done to ensure that you are not pregnant. Once it is determined that this treatment is appropriate for your care, you will begin taking 3, 4 DAP by mouth in slowly increasing doses. Treatment will begin with 5mg three times a day, as clinically needed, and if tolerated. You will be monitored for strength and side effects by routine clinic visits at initial intervals of once a month, increasing to intervals of every 12 months as permitted. Blood will be drawn (approximately 1 tablespoon) at every clinic visit or as often as the investigator deems necessary to assess your liver/kidney function and blood counts. You will have an EKG (a test to see how your heart is functioning) at your first study visit, after 6 months of taking 3,4 DAP and again every 2 years. Treatment will be continued indefinitely if a good clinical response is achieved. This study is planned to last indefinitely.
The dosage of 3, 4DAP is individually adjusted. The usual range is 10-15 mg 3-4 times per day for the full effect and will increase by 50% every two weeks to 10-15 mg three to six times a day, as needed and if tolerated. Dosages above the full effect level will not provide an additional benefit and should not be used. 3, 4 DAP is a convulsant (causes seizures). A total of 100 mg/day is the maximum dosage allowed.
Detailed description
Patients with clinically-confirmed LEMS will receive 3, 4-DAP by mouth in slowly increasing doses. Treatment will begin with 5 mg, three times a day and will increase by 50% every two weeks to 10-15 mg, three to six times a day, as clinically needed, and if tolerated. Patients will be monitored for strength and side effects via routine out-patient clinic visits at initial intervals of 1 month, increasing to intervals of 12 months as permitted. Results of treatment and adverse events will be reported to the FDA. Treatment will be continued indefinitely if a good clinical response is achieved and side effects are tolerable.
Study design
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Be 18 years or older, diagnosed with LEMS
- If female, have negative pregnancy test and if premenopausal, be willing to practice an effective form of birth control during the study.
Key exclusion criteria (5 listed)
- Is known to have a sensitivity to 3, 4 DAP
- Has a history of past or current seizures
- Has a history of past or current severe asthma
- Is believed by the investigator to be unable to comply with the protocol
- Unable to give informed consent
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 DAPAlso: 3,4 diaminopyridinePatients with clinically-confirmed LEMS will receive 3, 4 DAP by mouth in slowly increasing doses. Treatment will begin with 5 mg three times a day and will increase by 50% every two weeks to 10-15 mg three to six times a day, as clinically needed, and if tolerated. | 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 30 Oct 2019.
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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.
David Richman, MD principal investigator
University of California, Davis
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 DAP in people with Lambert Eaton Myasthenic Syndrome, run by David P. Richman, 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?
The sponsor lists 2 inclusion and 5 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 30 Oct 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.
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?
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 30 Oct 2019.