A Phase 2/3 interventional study of 3,4-diaminopyridine in Botulism, sponsored by Centre Hospitalier Universitaire, Amiens. Completed at 1 site in France. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-09-19.
Sponsored by Centre Hospitalier Universitaire, Amiens · Phase 2/3, Interventional, and Other
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.
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.
20 studies on the registry are indexed under Botulism; 3 are open to participants now.
This study's enrollment of 3 is below the median of 29 across 14 interventional studies indexed under Botulism.
Browse Botulism studies →Centre Hospitalier Universitaire, Amiens is the lead sponsor of 586 studies on the registry; 185 are open to participants now.
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Exclusion Criteria:
test dose 10 mg then 20 mg, then depending on tolerance and efficacy full-dose administration(from 10mg /8h to 20 mg /8h)
electrophysiological
Primary endpoint: Clinical, electrophysiological and respiratory before and after administration of 3,4-diaminopyridine.
Time frame: 90 min
This study is completed, as verified in Sep 2025. You cannot join it, but the record below documents what was studied.
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