DDrare: Database of Drug Development for Rare Diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
11	重症筋無力症	2

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2015-003127-62-IT (EUCTR)	02/10/2015	17/03/2017	A Randomized, Placebo-Controlled Study to Evaluate the Effect of Amifampridine in Patients with MuSK Antibody Positive Myasthenia Gravis	A 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 BESTA	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	20	Phase 2	Italy
2	EUCTR2010-021850-20-IT (EUCTR)	23/11/2011	07/03/2012	A 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.	NULL	Not Recruiting			Female: yes Male: yes	60	Phase 3	United States;Hungary;Spain;Bulgaria;Germany;Italy

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2015-003127-62-IT
(EUCTR)

02/10/2015

17/03/2017

A Randomized, Placebo-Controlled Study to Evaluate the Effect of Amifampridine in Patients with MuSK Antibody Positive Myasthenia Gravis

A 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 BESTA

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

Italy

EUCTR2010-021850-20-IT
(EUCTR)

23/11/2011

07/03/2012

A 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.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

United States;Hungary;Spain;Bulgaria;Germany;Italy