Pilot Study on the Usefulness of 3,4-diaminopyridine in the Treatment of Botulism
Completed
Centre Hospitalier Universitaire, Amiens
Phase 2/Phase 3
2011-09-01
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.
Botox injections into the thyroarytenoid muscle are a predictable and effective treatment for
SD, but typically result in transient symptoms of voice weakness and breathiness during the
first 2-3 weeks after injection. We hypothesize that voice weakness and breathiness after
Botox treatment can be alleviated using amifampridine.
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