BotulismPhase 2/3NCT01441557
Pilot Study on the Usefulness of 3,4-diaminopyridine in the Treatment of Botulism
A Phase 2/3 study testing 3,4-diaminopyridine in people with Botulism, run by Centre Hospitalier Universitaire, Amiens at 1 site in France.
Trial summary
Main objectives: Evaluate the effectiveness of an administration of 3,4-diaminopyridine (FIRDAPSE ®) in severe botulinic poisoning in measuring the effect on electrophysiological and respiratory parameters Secondary Objective: Study the natural history of electrophysiological and respiratory parameters during the… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Botulism
- What is being tested?
- 3,4-diaminopyridine
- How many people
- 3 participants
- Where
- 1 siteFrance
- Study dates
- Sep 2011to Mar 2012
- Phase
- Phase 2/3
- 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
- botulism
- ventilation
Who cannot join
- renal failure
- cardiac failure
- pregnancy
- under age 18
- hepatic failure
Trial locations and contacts
1 site in 1 country.
About this study
Main objectives:
Evaluate the effectiveness of an administration of 3,4-diaminopyridine (FIRDAPSE ®) in severe botulinic poisoning in measuring the effect on electrophysiological and respiratory parameters
Read more
Secondary Objective:
Study the natural history of electrophysiological and respiratory parameters during the botulinic intoxication
Primary endpoint:
Clinical, electrophysiological and respiratory before and after administration of 3,4-diaminopyridine.
Study Design:
Pilot study, prospective, interventional.
Study population:
Case series (n = 8 patients) suffering from botulinic type A, respiratory failure, but with no other organ failure
Experimental treatment :
3,4-diaminopyridine, FIRDAPSE ® (BioMarin) The dosage will be gradually increased according to a predetermined scheme and will not exceed 60 mg / day and 20 mg / dose.
Statistics:
Intra-individual comparison of physiological parameters measured before and after administration of 3,4-diaminopyridine. Electromyographic and respiratory parameters will be measured for each patient. Then a dose of 10 mg of 3,4-diaminopyridine will be administrated. If this dose is well tolerated and provides a relative improvement of 10% for at least one of the parameters studied, the dose will be maintained at 10 mg for 48 hours 3 times a day then increased to 20 mg.
The primary endpoint is the change in the amplitude of muscle response evaluated by the subtraction of amplitude at T1.5 and T0.
Read the detailed description
Main objectives:
Evaluate the effectiveness of an administration of 3,4-diaminopyridine (FIRDAPSE ®) in severe botulinic poisoning in measuring the effect on electrophysiological and respiratory parameters
Secondary Objective:
Study the natural history of electrophysiological and respiratory parameters during the botulinic intoxication
Primary endpoint:
Clinical, electrophysiological and respiratory before and after administration of 3,4-diaminopyridine.
Study Design:
Pilot study, prospective, interventional.
Study population:
Case series (n = 8 patients) suffering from botulinic type A, respiratory failure, but with no other organ failure
Experimental treatment :
3,4-diaminopyridine, FIRDAPSE ® (BioMarin) The dosage will be gradually increased according to a predetermined scheme and will not exceed 60 mg / day and 20 mg / dose.
Statistics:
Intra-individual comparison of physiological parameters measured before and after administration of 3,4-diaminopyridine. Electromyographic and respiratory parameters will be measured for each patient. Then a dose of 10 mg of 3,4-diaminopyridine will be administrated. If this dose is well tolerated and provides a relative improvement of 10% for at least one of the parameters studied, the dose will be maintained at 10 mg for 48 hours 3 times a day then increased to 20 mg.
The primary endpoint is the change in the amplitude of muscle response evaluated by the subtraction of amplitude at T1.5 and T0.
Quantitative variables are expressed as mean ± standard deviation or median (minimum - maximum) and qualitative variables as percentages.
The null hypothesis (the evolution is identical between the two doses) will be rejected in favour of the alternative hypothesis (the evolution is different) using the mixed model analysis of variance on the threshold of 5% for the first kind with adjusting the amplitude at T0, age, the time between patient admission and the first administration of 3,4-diaminopyridine. The evolution of latency and respiratory parameters will be analyzed with the same mixed ANOVA without risk adjustment alpha.
For the secondary objective, the evolution curves of the amplitude of muscle response on the one hand and respiratory function on the other hand, will be built for each patient.
The mean (or median) of amplitude and respiratory function at weaning will be calculated with a confidence level of 95% (or range). The average time between the first 3,4-diaminopyridine administration and withdrawal of mechanical ventilation will also be calculated.
For each dose, the average evolution of the amplitude will be calculated with a confidence level of 95%.
Statistical analysis will be carried out using SAS software version 9.2.
- Conditions
- Botulism
- Being tested
- 3,4-diaminopyridine
- Keywords
- mechanical ventilation
- Medical subject headings
- Botulism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
3,4-diaminopyridineDrug
test dose 10 mg then 20 mg, then depending on tolerance and efficacy full-dose administration(from 10mg /8h to 20 mg /8h)
What is being measured
The primary outcome is the main question the study is designed to answer.
electrophysiological
Primary endpoint:
Clinical, electrophysiological and respiratory before and after administration of 3,4-diaminopyridine.
Measured: 90 min
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Sep 2011 (estimated)
- Study startSep 2011
- Primary completionFeb 2012
- Study completionMar 2012
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 BotulismStudies of the same condition, open and under way.
- More trials run by Centre Hospitalier Universitaire, AmiensThe sponsor's other registered studies.
- 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 2/3
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Universitaire, Amiens
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Herve DUPONT, Md, PhD, Principal investigator, CHU Amiens
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01441557
- Sponsor's protocol ID
- PI11-PR-DUPONT
- Status last verified
- Sep 2025
- First submitted
- 26 Sep 2011
- Last updated
- 19 Sep 2025
- Results posted
- No
Publications
Friggeri A, Marcon F, Marciniak S, Lemaire-Hurtel AS, Seydi A, Ammenouche N, Levrard M, Mahjoub Y, Airapetian N, Tinturier F, Petitjean M, Dupont H. 3,4-Diaminopyridine may improve neuromuscular block during botulism. Crit Care. 2013 Sep 5;17(5):449. doi: 10.1186/cc12880. No abstract available.
PubMed 24007658
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study on the Usefulness of 3,4-diaminopyridine in the Treatment of Botulism
- ClinicalTrials.gov ID
- NCT01441557
- Acronym / sponsor ID
- PI11-PR-DUPONT
- Conditions
- Botulism
- Keywords
- mechanical ventilation
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Centre Hospitalier Universitaire, AmiensUniversity, hospital or other
- Study start
- Sep 2011
- Primary completion
- Feb 2012 (actual)
- Study completion
- Mar 2012 (actual)
- First posted
- 27 Sep 2011 (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.
Main objectives:
Evaluate the effectiveness of an administration of 3,4-diaminopyridine (FIRDAPSE ®) in severe botulinic poisoning in measuring the effect on electrophysiological and respiratory parameters
Secondary Objective:
Study the natural history of electrophysiological and respiratory parameters during the botulinic intoxication
Primary endpoint:
Clinical, electrophysiological and respiratory before and after administration of 3,4-diaminopyridine.
Study Design:
Pilot study, prospective, interventional.
Study population:
Case series (n = 8 patients) suffering from botulinic type A, respiratory failure, but with no other organ failure
Experimental treatment :
3,4-diaminopyridine, FIRDAPSE ® (BioMarin) The dosage will be gradually increased according to a predetermined scheme and will not exceed 60 mg / day and 20 mg / dose.
Statistics:
Intra-individual comparison of physiological parameters measured before and after administration of 3,4-diaminopyridine. Electromyographic and respiratory parameters will be measured for each patient. Then a dose of 10 mg of 3,4-diaminopyridine will be administrated. If this dose is well tolerated and provides a relative improvement of 10% for at least one of the parameters studied, the dose will be maintained at 10 mg for 48 hours 3 times a day then increased to 20 mg.
The primary endpoint is the change in the amplitude of muscle response evaluated by the subtraction of amplitude at T1.5 and T0.
Detailed description
Main objectives:
Evaluate the effectiveness of an administration of 3,4-diaminopyridine (FIRDAPSE ®) in severe botulinic poisoning in measuring the effect on electrophysiological and respiratory parameters
Secondary Objective:
Study the natural history of electrophysiological and respiratory parameters during the botulinic intoxication
Primary endpoint:
Clinical, electrophysiological and respiratory before and after administration of 3,4-diaminopyridine.
Study Design:
Pilot study, prospective, interventional.
Study population:
Case series (n = 8 patients) suffering from botulinic type A, respiratory failure, but with no other organ failure
Experimental treatment :
3,4-diaminopyridine, FIRDAPSE ® (BioMarin) The dosage will be gradually increased according to a predetermined scheme and will not exceed 60 mg / day and 20 mg / dose.
Statistics:
Intra-individual comparison of physiological parameters measured before and after administration of 3,4-diaminopyridine. Electromyographic and respiratory parameters will be measured for each patient. Then a dose of 10 mg of 3,4-diaminopyridine will be administrated. If this dose is well tolerated and provides a relative improvement of 10% for at least one of the parameters studied, the dose will be maintained at 10 mg for 48 hours 3 times a day then increased to 20 mg.
The primary endpoint is the change in the amplitude of muscle response evaluated by the subtraction of amplitude at T1.5 and T0.
Quantitative variables are expressed as mean ± standard deviation or median (minimum - maximum) and qualitative variables as percentages.
The null hypothesis (the evolution is identical between the two doses) will be rejected in favour of the alternative hypothesis (the evolution is different) using the mixed model analysis of variance on the threshold of 5% for the first kind with adjusting the amplitude at T0, age, the time between patient admission and the first administration of 3,4-diaminopyridine. The evolution of latency and respiratory parameters will be analyzed with the same mixed ANOVA without risk adjustment alpha.
For the secondary objective, the evolution curves of the amplitude of muscle response on the one hand and respiratory function on the other hand, will be built for each patient.
The mean (or median) of amplitude and respiratory function at weaning will be calculated with a confidence level of 95% (or range). The average time between the first 3,4-diaminopyridine administration and withdrawal of mechanical ventilation will also be calculated.
For each dose, the average evolution of the amplitude will be calculated with a confidence level of 95%.
Statistical analysis will be carried out using SAS software version 9.2.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- botulism
- ventilation
Key exclusion criteria (5 listed)
- renal failure
- cardiac failure
- pregnancy
- under age 18
- hepatic failure
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-diaminopyridinetest dose 10 mg then 20 mg, then depending on tolerance and efficacy full-dose administration(from 10mg /8h to 20 mg /8h) | Drug | – |
Primary outcome
electrophysiological
Time frame: 90 min
Primary endpoint: Clinical, electrophysiological and respiratory before and after administration of 3,4-diaminopyridine.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2025.
- Results publication: PubMed 24007658Friggeri A, Marcon F, Marciniak S, Lemaire-Hurtel AS, Seydi A, Ammenouche N, Levrard M, Mahjoub Y, Airapetian N, Tinturier F, Petitjean M, Dupont H. 3,4-Diaminopyridine may improve neuromuscular block during botulism. Crit Care. 2013 Sep 5;17(5):449. doi: 10.1186/cc12880. No abstract available.
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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.
Herve DUPONT, Md, PhD principal investigator
CHU Amiens
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing 3,4-diaminopyridine in people with Botulism, run by Centre Hospitalier Universitaire, Amiens at 1 site in France.
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: finished, as of the sponsor's last update on 19 Sep 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 France. 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 combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
electrophysiological (time frame: 90 min). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01441557, 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 19 Sep 2025.