NCT01441557

Pilot Study on the Usefulness of 3,4-diaminopyridine in the Treatment of Botulism

Completed Phase 2/Phase 3 Last updated 16 September 2025

What this trial tests

Phase 2/Phase 3 trial testing 3,4-diaminopyridine in Botulism in 3 participants. Completed in 1 March 2012.

Timeline

1 September 2011

Primary endpoint
1 February 2012

1 March 2012

Quick facts

Lead sponsor	Centre Hospitalier Universitaire, Amiens
Phase	Phase 2/Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	single group
Masking	none
Primary purpose	other
Enrollment	3
Start date	1 September 2011
Primary completion	1 February 2012
Estimated completion	1 March 2012
Sites	1 location across France

Drugs / interventions tested

• 3,4-diaminopyridine — full drug profile →

Conditions studied

• Botulism — all drugs for Botulism →

Sponsor

Centre Hospitalier Universitaire, Amiens

Who can join

18 and older, any sex, with Botulism. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• electrophysiological
  Time frame: 90 min
  Primary endpoint: Clinical, electrophysiological and respiratory before and after administration of 3,4-diaminopyridine.

Sponsor's own 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.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. 3,4-Diaminopyridine may improve neuromuscular block during botulism.
   Friggeri A, Marçon F, Marciniak S, Lemaire-Hurtel AS, et al · · 2013 · cited 11× · PMID 24007658 · DOI 10.1186/cc12880