Amifampridine    (DrugBank: Amifampridine)

3 diseases
IDDisease name (Link within this page)Number of trials
3Spinal muscular atrophy2
11Myasthenia4
12Congenital myasthenic syndrome1

3. Spinal muscular atrophy    [ 179 clinical trials,   102 drugs,   (DrugBank: 26 drugs),   52 drug target genes,   78 drug target pathways]
Searched query = "Spinal muscular atrophy", "Myelopathic muscular atrophy", "SMA I", "Werdnig-Hoffman disease", "SMA II", "Dubowitz disease", "SMA III", "Kugelberg-Welander disease", "SMA IV"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 179 trials found
No.TrialIDDate_
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PhaseCountries
1NCT03819660
(ClinicalTrials.gov)
March 7, 201923/1/2019Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3Long Term Safety Study of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3Muscle AtrophyDrug: Amifampridine Phosphate 10 MG Oral TabletCatalyst Pharmaceuticals, Inc.NULLEnrolling by invitation6 Years50 YearsAll12Phase 2Italy
2NCT03781479
(ClinicalTrials.gov)
January 21, 201918/12/2018Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 PatientsA Randomized, Placebo-Controlled, Crossover Study to Evaluate the Safety and Efficacy of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3Muscular Atrophy, SpinalDrug: Amifampridine Phosphate;Drug: Placebo Oral TabletCatalyst Pharmaceuticals, Inc.NULLCompleted6 Years50 YearsAll12Phase 2Italy

11. Myasthenia    [ 226 clinical trials,   172 drugs,   (DrugBank: 45 drugs),   43 drug target genes,   118 drug target pathways]
Searched query = "Myasthenia", "MG"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
4 / 226 trials found
No.TrialIDDate_
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agemin
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1NCT03579966
(ClinicalTrials.gov)
July 1, 20185/6/2018Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis)Long Term Safety Study of Amifampridine Phosphate in Patients With MuSK Antibody Positive and AChR (Acetylcholine Receptor) Antibody Positive Myasthenia GravisMyasthenia Gravis, MuSKDrug: Amifampridine PhosphateCatalyst Pharmaceuticals, Inc.NULLActive, not recruiting18 YearsN/AAll70Phase 3United States
2NCT03304054
(ClinicalTrials.gov)
March 7, 201828/9/2017Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MGA Randomized, Placebo-control, Parallel Group Study to Evaluate the Effect of Amifampridine Phosphate in Patients With MuSK Antibody Positive Myasthenia Gravis, and a Sample of AChR Antibody Positive Myasthenia Gravis PatientsMyasthenia Gravis, GeneralizedDrug: Amifampridine Phosphate;Drug: Placebo Oral TabletCatalyst Pharmaceuticals, Inc.NULLCompleted18 YearsN/AAll70Phase 3United States
3EUCTR2015-003127-62-IT
(EUCTR)
02/10/201517/03/2017A Randomized, Placebo-Controlled Study to Evaluate the Effect of Amifampridine in Patients with MuSK Antibody Positive Myasthenia GravisA Randomized, Placebo-Controlled, Pilot Crossover Study to Evaluate the Effect of Amifampridine Phosphate(3,4-Diaminopyridine Phosphate) in Patients with MuSK Antibody Positive Myasthenia Gravis - MuSK-001 Patients with MuSK Antibody Positive Myasthenia Gravis
MedDRA version: 19.1;Level: LLT;Classification code 10062976;Term: Neuromuscular weakness;System Organ Class: 100000004859;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: FIRDAPSE - 10 MG - COMPRESSE - USO ORALE - BLISTER(ALU/PVC/PVDC) 100 X 1 COMPRESSE
Product Name: AMIFAMPRIDINA
INN or Proposed INN: AMIFAMPRIDINA
FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTANULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 2Italy
4EUCTR2010-021850-20-IT
(EUCTR)
23/11/201107/03/2012A Phase 3 placebo controlled study to evaluate safety and efficacy of Amifampridine phosphate.A Phase 3, Multicenter, Double-blind, Placebo-controlled Randomized Discontinuation Study Followed by an Open-label Extension Period to Evaluate the Efficacy and Safety of Amifampridine Phosphate (3,4-Diaminopyridine Phosphate) in Patients with Lambert-Eaton Myasthenic Syndrome (LEMS) Lambert-Eaton Myasthenic Syndrome (LEMS)
MedDRA version: 14.1;Level: LLT;Classification code 10028415;Term: Myasthenia;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders
MedDRA version: 14.1;Level: SOC;Classification code 10029205;Term: Nervous system disorders;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Body processes [G] - Digestive System and Oral Physiological Phenomena [G10]
Trade Name: FIRDAPSE 10mg Tablets
INN or Proposed INN: AMIFAMPRIDINE
Other descriptive name: 3,4-DAP
BIOMARIN PHARMACEUTICAL INC.NULLNot RecruitingFemale: yes
Male: yes
60Phase 3United States;Hungary;Spain;Bulgaria;Germany;Italy

12. Congenital myasthenic syndrome    [ 5 clinical trials,   7 drugs,   (DrugBank: 3 drugs),   5 drug target genes,   12 drug target pathways]
Searched query = "Congenital myasthenic syndrome", "End-plate acetylcholine receptor deficiency", "Sodium channel myasthenia", "End-plate acetylcholine esterase deficiency", "Dok-7 myasthenia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 5 trial found
No.TrialIDDate_
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Date_
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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1NCT02189720
(ClinicalTrials.gov)
July 20145/7/2014Expanded Access Study Amifampridine Phosphate in Congenital Myasthenic Syndrome (CMS)An Open-Label, Expanded Access Protocol for Amifampridine Phosphate Treatment in Patients With Congenital Myasthenic Syndrome (CMS)Congenital Myasthenic SyndromeDrug: Amifampridine PhosphateCatalyst Pharmaceuticals, Inc.NULLNo longer available2 YearsN/AAllUnited States