DDrare: Database of Drug Development for Rare Diseases

11. 重症筋無力症
［臨床試験数：226，薬物数：172（DrugBank：45），標的遺伝子数：43，標的パスウェイ数：118］

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	EUCTR2018-002247-28-IT (EUCTR)	04/02/2019	04/11/2020	To evaluate the efficacy, safety and tolerability of M281 injection for the treatment of patients with Myasthenia gravis, a neuromuscular disease	A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of M281 Administered to Adults with Generalized Myasthenia Gravis - VIVACITY	Treatment of MG, with an initial focus on patients with gMG treated with M281 injection and evaluation of the expected reduction of circulating levels of antibodies by blocking IgG recycling, including the pathogenic autoantibodies that cause MG, and to ameliorate manifestations of the disease. MedDRA version: 21.1;Level: PT;Classification code 10028417;Term: Myasthenia gravis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: M281 Injection Product Code: [M281] INN or Proposed INN: M281	MOMENTA PHARMACEUTICALS, Inc	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	60	Phase 2	United States;Canada;Spain;Poland;Belgium;Germany;United Kingdom;Italy

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agemin

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EUCTR2018-002247-28-IT
(EUCTR)

04/02/2019

04/11/2020

To evaluate the efficacy, safety and tolerability of M281 injection for the treatment of patients with Myasthenia gravis, a neuromuscular disease

A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of M281 Administered to Adults with Generalized Myasthenia Gravis - VIVACITY

Treatment of MG, with an initial focus on patients with gMG treated with M281 injection and evaluation of the expected reduction of circulating levels of antibodies by blocking IgG recycling, including the pathogenic autoantibodies that cause MG, and to ameliorate manifestations of the disease.
MedDRA version: 21.1;Level: PT;Classification code 10028417;Term: Myasthenia gravis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Product Name: M281 Injection
Product Code: [M281]
INN or Proposed INN: M281

MOMENTA PHARMACEUTICALS, Inc

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

United States;Canada;Spain;Poland;Belgium;Germany;United Kingdom;Italy