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

NCT01441557 | Completed Phase 2

• 1 other identifier: PI11-PR-DUPONT
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief 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 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.

Conditions

Botulism

Keywords

mechanical ventilation

Outcome Measures

Primary Outcomes (1)

• electrophysiological

  Primary endpoint: Clinical, electrophysiological and respiratory before and after administration of 3,4-diaminopyridine.

  90 min

Interventions

3,4-diaminopyridine DRUG

test dose 10 mg then 20 mg, then depending on tolerance and efficacy full-dose administration(from 10mg /8h to 20 mg /8h)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• botulism
• ventilation

You may not qualify if:

• renal failure
• cardiac failure
• pregnancy
• under age 18
• hepatic failure

Sponsors & Collaborators

• Centre Hospitalier Universitaire, Amiens: lead

Study Sites (1)

CHU

Amiens, 80000, France

Location

Related Publications (1)

• 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.

  PMID: 24007658 RESULT

MeSH Terms

Conditions

Botulism

Interventions

Amifampridine

Condition Hierarchy (Ancestors)

Clostridium Infections; Gram-Positive Bacterial Infections; Bacterial Infections; Bacterial Infections and Mycoses; Infections; Neuromuscular Junction Diseases; Neuromuscular Diseases; Nervous System Diseases; Neurotoxicity Syndromes; Foodborne Diseases; Poisoning; Chemically-Induced Disorders

Intervention Hierarchy (Ancestors)

4-Aminopyridine; Aminopyridines; Amines; Organic Chemicals; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Herve DUPONT, Md, PhD

  CHU Amiens

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 26, 2011
• First Posted: September 27, 2011
• Study Start: September 1, 2011
• Primary Completion: February 1, 2012
• Study Completion: March 1, 2012
• Last Updated: September 19, 2025

Record last verified: 2025-09

Locations

FR (1)