DDrare: Database of Drug Development for Rare Diseases

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.

Drug = 250 mg CK-2017357; 3,4-DAP; 3,4-Diaminopyridine; 3,4-diaminopyridine; 500 mg CK-2017357; ALXN1210; AMIFAMPRIDINA; AMIFAMPRIDINE; ARGX-113; ATG (rabbit); AZA; Abatacept; Abatacept Injection; Acetaminophen; Amifampridine; Amifampridine Phosphate; Anti-C5 antibody; Anti-Thymocyte Globulin; Azathioprine; B-peptide-CRM197 conjugate; BBR 2778; BELIMUMAB; BENLYSTA® (belimumab); BORTEZOMIB; Belimumab; Benlysta (belimumab); Bortezomib; CD19-directed humanised afucosylated monoclonal antibody; CELLCEPT; CFZ533; CV-MG01; Carmustine; CellCept; CellCept 500 mg; CellCept 500 mg film-coated tablets; CellCept 500 mg tablets; CellCept®500 mg tablets; Cellcept; Cellcept 500 mg tablets; Chloride; Cyclophosphamide; Cyclosporine; Cytarabine; DAP; DAS-001; DIFENIDRAMINA CLORIDRATO; Descartes-08; Difenidramina; Diphenhydramine; Diphenhydramine hydrochloride; ECULIZUMAB; EFALIZUMAB; EN101; EPHEDRINE HYDROCHLORIDE; Eculizumab; Efalizumab; Efedrina; Efgartigimod; Ephedrine; Etoposide; FIRDAPSE - 10 MG - COMPRESSE - USO ORALE - BLISTER(ALU/PVC/PVDC) 100 X 1 COMPRESSE; FIRDAPSE 10mg Tablets; Fc- and CDR-modified humanised monoclonal antibody against C5; Firdapse; G-CSF; GAMUNEX 10%; GB-0998 (Intravenous immunoglobulin); GSK1550188; Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF); HBM9161 Injection (340 mg); HBM9161 Injection (680mg and 340 mg); HIZENTRA ®; HUMAN NORMAL IMMUNOGLOBULIN (IV); Hematopoietic Stem Cell Transplantation; Hidrocloruro de efedrina; Human normal immunoglobulin; Human normal immunoglobulin G (IgG); IGIV-C; IMMUNOGLOBULIN G; INEBILIZUMAB; IVIG; IVIg; Immune Globulin (Human), 10% Caprylate/Chromatography Purified; Immune Globulin (Human), 10% Caprylate/Chromatography Purified (IGIV-C); Immune globulin; Inebilizumab; Influenza vaccine; Intravenous ImmuneGlobulin; Leflunomide; M281; M281 Injection; METILPREDNISOLONE; MONARSEN; MONITOR; MabThera; Mabthera; Magnesium; Magnesium sulfate; Melphalan; Mesna; Mestinon; Methotrexate; Methylprednisolone; Metilprdnisolone; Mutagrip; Mycophenolate; Mycophenolate Mofetil; Mycophenolate mofetil; Mycophenolate mofetil (CellCept); Mycophenolate mofetil [CellCept]; Mycophenolic acid; Other: Laboratory Biomarker Analysis; PARACETAMOLO; PIXANTRONE; PRIVIGEN; PYRIDOSTIGMINE; Paracetamolo; Phosphate; Pixantrone; Prednisolone; Prednisone; Prednisone - Azathioprine; Prednisone alone; Procedure: Autologous Hematopoietic Stem Cell Transplantation; Procedure: PLEX; Procedure: Peripheral Blood Stem Cell Transplantation; Procedure: Plasmapheresis; Procedure: Syngeneic Bone Marrow Transplantation; Procedure: thymectomy plus prednisone; Pyridostigmine; RC18 160mg; RC18 240 mg; RITUXIMAB; ROZANOLIXIZUMAB; RTX; RVT-1401; Rabbit; Raptiva; Raptiva 100 mg/ml powder and solvent for solution for injection; Ravulizumab; Rituximab; Rituximab (Rituxan); Rozanolixizumab; SALBUTAMOL SULFATE PH. EUR.; SOLIRIS; Salbutamol; Salbutamol 4Mg Tablet; Salbutamol WZF 4 mg; Sodium Chloride solution; Sodium chloride; Soliris; Subcutaneous immunoglobulins; Sulfate; Symptomatic Treatment, Steroids, Immunosuppressive Agents( azathioprine, tacrolimus, Mycophenolate Mofetil); Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg); T-peptide-CRM197 conjugate; TAK-079; Tacrolimus; Tacrolimus capsule; Tetanus vaccin; Tofacitinib; Tofacitinib 5 MG; UCB7665; Ultomiris; Velcade; Velcade®; Zilucoplan; Zilucoplan (RA101495); [M281]; [NA]; [TAK-079, TSF-021, TSF79]; [acetominofene]

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