DDrare: Database of Drug Development for Rare Diseases

113. 筋ジストロフィー
［臨床試験数：567，薬物数：442（DrugBank：93），標的遺伝子数：55，標的パスウェイ数：151］

Searched query = "Muscular dystrophy", "Dystrophinopathies", "Myotilinopathy", "Laminopathy", "Caveolinopathy", "LGMD1C", "Desminopathy", "Sarcoglycanopathy", "α-dystroglycanopathy", "FCMD", "Walker-Warburg syndrome", "Muscle-eye-brain disease", "Myotonic dystrophy", "Integrin α7 deficient CMD", "Rigid spine syndrome"
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 = (+)- Epicatechin; (+)-Epicatechin; (-)-Epicatechin; (-)-epicatechin; 12 mg/kg GSK2402968; 2011-2012 seasonal flu vaccine Intramuscular; 2011-2012 seasonal flu vaccine Subcutaneous; 29180.00.00, 34681.00.00, 34681.01.00, 34681.02.00; 3 mg/kg GSK2402968; 4-Benzyl-2-naphthalen-1-yl-1,2,4- thiadiazolidine-3,5-dione; 4-benzyl-2-naphthalen-1-yl-1,2,4-thiadiazolidine-3,5-dione; 5-(ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole; 6 mg/kg GSK2402968; 750 mg metformin and 7.5 g L-citrulline daily p.o.; 9 mg/kg GSK2402968; AAV1-gamma-sarcoglycan vector injection; ACE-031 (Extension of cohort 1 from core study, A031-03); ACE-031 (Extension of cohort 2 from core study, A031-03); ACE-031 (Extension of cohort 3 from core study, A031-03); ACE-031 0.5 mg/kg q4wk; ACE-031 1.0 mg/kg q2wk; ACE-083; ASP0367; ATALUREN; ATYR1940; AVI-4658; AVI-4658 (Eteplirsen); AVI-4658 (PMO); AVI-4658 for Injection; Acetate; Actonel Once a week 5mg film coated tablets; Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene; Albuterol; Allogeneic Cardiosphere-Derived Cells (CAP-1002); Anti-Myostatin Adnectin; Anti-myostati; Anti-myostatin Adnectin; Arginine; Aspirin; Ataluren; Ataluren 1000 mg; Ataluren 125 mg; Ataluren 250 mg; Autologous bone marrow mononuclear cell transplantation; BIO101; BLS-M22; BMN 044; BMN 044 IV 6 mg/kg; BMN 044 IV 9 mg/kg; BMN 044 SC 6 mg/kg; BMN 045; BMN 053; BMN053; BMS-986089; BMS-986089-01 (RO7239361) , anti-myostatin; BMS-986089-01 (RO7239361) Injections, 15 mg/Syringe (21.4 mg/mL); BMS-986089-01 (RO7239361) Injections, 35 mg/Syringe (50 mg/mL); BMS-986089-01 (RO7239361) Injections, 50 mg/Syringe (71.4 mg/mL); BMS-986089-01 (RO7239361) Injections, 7.5 mg/Syringe (10.7 mg/mL); BMS-986089-01 (RO7239361), anti-myostatin; BMS-986089-01 Injections, 15 mg/Syringe (21.4 mg/mL); BMS-986089-01 Injections, 35 mg/Syringe (50 mg/mL); BMS-986089-01 Injections, 50 mg/Syringe (71.4 mg/mL); BMS-986089-01 Injections, 7.5 mg/Syringe (10.7 mg/mL); BMS-986089-01, Anti-myostatin; BMS-986089-01, anti-myostatin; BOTULINUM TOXIN TYPE A; Balance; Behavioral: In-home Exercise Training; Behavioral: Regular exercise; Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell; Bisoprolol; Bisoprolol Fumarate; Bisphosphonate treatment; Blood test; Botulinum toxin type A; CALCIFEDIOL; CAP-1002; CARVEDILOL; CASIMERSEN; CAT-1004; CRD007; Cabaletta; Calcichew D3 Forte; Calcifediol; Canakinumab; Canakinumab Injection [Ilaris]; Cardicor; Cardicor (Merck brand); Cardiovascular profile; Carvedilol; Casimersen; Chronic Corticosteroid Therapy; Cialis 10 mg film-coated tablets; Cialis 2.5 mg film-coated tablets; Cialis 20 mg film-coated tablets; Cialis 5 mg film-coated tablets; Citrulline; Coenzyme Q10; Coenzyme Q10 and Lisinopril; Cosyntropin; Cosyntropin acetate; Cr13C; Creatine; Creatine Monohydrate; Creatine monohydrate; Creatine-13C; Cyclosporin; Cyclosporin A; DEFLAN*10CPR 6MG; DEFLAZACORT; DELTACORTENE*20CPR 5MG; DHA; DILATREND; DRISAPERSEN SODIUM; DS-5141b; Decortin; Deflazacort; Deflazacort 6 mg Tablets; Dehydroepiandrosterone; Dehydroepiandrosterone 100 and 400 mg; Device: Activity Monitor; Dexmedetomidine; Diagnostic Test: 30 seconds sit to stand test; Diagnostic Test: Beck depression inventory; Diagnostic Test: Berg balance scale; Diagnostic Test: Brief Pain Inventory Short-Form; Diagnostic Test: DM1-ActivC; Diagnostic Test: Fatigue and Daytime Sleepiness Scale; Diagnostic Test: Functional Index-2; Diagnostic Test: GSGC; Diagnostic Test: Hand opening time; Diagnostic Test: Manual muscle testing; Diagnostic Test: McGill pain questionnaire; Diagnostic Test: Myotonia (from Individualised Neuromuscular Quality of Life Questionnaire); Diagnostic Test: Myotonia Behaviour scale; Diagnostic Test: Pressure pain threshold; Diagnostic Test: Quantitative muscle testing; Diagnostic Test: Quick motor function test; Diagnostic Test: R-PAct; Diagnostic Test: Scale for Assessment and Rating of Ataxia; Diagnostic Test: Six minute walking test; Dosing Extension; Drisapersen; EDASALONEXENT; EDG-5506; EGCG; EMD 87580; EMEA/H/C/000638; EMFLAZA® (DEFLAZACORT); EMFLAZA® (deflazacort); ENALAPRIL; EPA; EPA and DHA; ERX-963; ETEPLIRSEN; EV substance code: SUB188638; EXONDYS 51™; Edasalonexent; EnaHexal; EnaHexal®, 10mg; EnalHexal®, 5 mg; Enalapril; Epicatechin; Epigallocatechin; Epigallocatechin-Gallate; Eplerenone; Eteplirsen; Eteplirsen Injection; Eucardic; Exondys 51; Ezutromid; FG-3019; FTX-1821; Filgrastim; Flavocoxid; GIVINOSTAT; GIVINOSTAT (hyrochloride monohydrate); GOLODIRSEN; GSK2402968; GSK2402968 3mg/kg/week; GSK2402968 6 mg/kg/week; GSK2402968 6mg/kg/week; Genetic: RNA extraction; Gentamicin; Gentamicin infusions twice a week for six months; Givinostat; Givinostat (hydrochloride monohydrate); Givinostat (idrocloruro monoidrato); Glutamine; Gold; Golodirsen; Granulocyte colony-stimulating factor (Filgrastim); Guanidine; H44AON188; H51AON23; HIDROFEROL; HLA-identical allogeneic mesoangioblasts; HMABs; HT-100; Haemostasis tests; Hidroferol; Hidroferol®; Hormonal profile; Human Umbilical Cord Mesenchymal Stem Cells; Human umbilical cord mesenchymal stem cells; IBUPROFEN; IBUPROFEN 200MG; IDEBENONE; IGF-1; IONIS-DMPKRx; ISOSORBIDE DINITRATE; ITF2357; Ibuprofen; Idebenone; Idebenone 150 mg film-coated tablets; Ifetroban; Inflammatory profile; Isosorbide; Isosorbide Dinitrate; Isosorbide Dinitrate 20 mg; Isosorbide Dinitrate 40 mg; Isosorbide Dinitrate 60 mg; Isosorbide Dinitrate 80 mg; Isosorbide dinitrate; Ketamine; L-Arginine; L-Citrulline and Metformin & L-Citrulline; L-Glutamine; L-arginine; LOSMAPIMOD; Lentiviral Vector Trans-Skip gene modified Autologous Mesoangioblasts; Lisinopril; Lonafarnib; Losartan; Losmapimod; METFORMIN; METFORMINA - 500 30 COMPRESSE 500 MG; METOPROLOL SUCCINATE; MNK-1411; MONITOR; MYO-029; MYODM; Metabolic profile; Metformin; Metformin 500mg; Metformin and Metformin & L-Citrulline; Metformina; Methylphenidate; MetoHEXAL® Succ ® 23,75 mg Retardtabletten; MetoHEXAL® Succ ® 47,5 mg Retardtabletten; MetoHEXAL® Succ ® 95 mg Retardtabletten; Metoprolol; Mexiletine; Monocytes and megacaryocytes culture and RNA extraction; Myoblast autologous graft; Myoblast transplantation; Myoblastes autologues; N(4,5Bis methanesulfonyl2methylbenzoyl) guanidine hydrochloride monohydrate; N-(4,5-Bis methanesulfonyl-2-methyl-benzoyl)guanidine hydrochloride monohydrate; NPC-14; NS-065/NCNP-01; NS-089/NCNP-02; Nanosuspension); Nebivolol; Needle biopsy of the vastus lateralis muscle; Nitrate; Nébivolol; Oligomero morfolino fosforodiamidato per skipping dell’esone 45; Oligomero morfolino fosforodiamidato per skipping dell’esone 53; Omigapil; Other: Dystrophin levels; Other: Echocardiogram; Other: Ibuprofen and Isosorbide Dinitrate combination; Other: Magnetic Resonance Imaging (MRI); Other: Metabolic Exercise Testing using stationary bicycle; Other: Pulmonary Function Testing (PFT); Other: Standard steroid treatment; Other: beetroot juice extract; Oxatomide; Oxatomide (tinset); P-188 NF; PAMREVLUMAB; PB1046 Injection; PEMIROLAST; PF-06252616; PF-06939926; PFT; PRD2175434; PREDNISONE; PRO044; PRO044 IV; PRO044 IV 6 mg/kg; PRO044 IV 9 mg/kg; PRO044 SC; PRO044 SC 6 mg/kg; PRO045; PRO045, 0.15 mg/kg/week; PRO045, 1.0 mg/kg/week; PRO045, 3.0 mg/kg/week; PRO045, 6.0 mg/kg/week; PRO045, 9.0 mg/kg/week; PRO045, selected dose; PRO051; PRO053; PS188; PS188 (company code); PS220; PS524; PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844; PTC124; Pamrevlumab; Pamrevlumab (FG-3019); Pemirolast; Pending; Pentoxifylline; Peptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping; Perindopril; Perindopril 2mg Tablets; Phosphorodiamidate Morpholino Oligomer; Phosphorodiamidate morpholino oligomer for exon 45 skipping; Phosphorodiamidate morpholino oligomer for exon 53 skipping; Powder for Oral Suspension); Prednisolone; Prednison; Prednisone; Prednisone 5 mg tablets; Procedure: Increased exercise intensity; Procedure: Saline injection; Procedure: Taking of blood; Protein-carbohydrate supplementation; RAAV1.CMV.huFollistatin344; RAAV1.CMV.huFollistin344; RAAV2.5-CMV-minidystrophin (d3990); RAAV9-CK8-h-µD5; RAAVrh74.MCK.GALGT2; RAAVrh74.MCK.micro-Dystrophin; RAXONE - 150 MG- COMPRESSE RIVESTITE CON FILM- USO ORALE- FLACONE (HDPE)- 180 COMPRESSE; RO7239361; RO7239361 (BMS-986089); RO7239361 Injections, 15 mg/Syringe (21.4 mg/mL); RO7239361 Injections, 35 mg/Syringe (50 mg/mL); RO7239361 Injections, 50 mg/Syringe (71.4 mg/mL); RO7239361 Injections, 7.5 mg/Syringe (10.7 mg/mL); Ramipril; Ranolazine; Raxone; Recombinant human anti-GDF-8 antibody; Regimen Selection Phase Group 1 (COMPLETED); Regimen Selection Phase Group 2; Regimen Selection Phase Group 3; Resveratrol; Revatio; RhIGF-I/rhIGFBP-3; RhIGFBP-3; Rimeporide; Risedronate; Risedronate sodium; SGT-001; SMT C1100; SMT C1100 (F5; SMT C1100 (F5); SMT C1100 (F6; SMT C1100 (F6); SNT-MC17; SRP-4045; SRP-4053; SRP-5051; Sandimmun Optoral; ScAAV9.U7.ACCA; ScAAVrh74.tMCK.hSGCA; Sildenafil; Sleep parameters; Sodium Nitrate; Sodium Nitrate - double dose; Sodium nitrate; SomatoKine/IPLEX; Somatropin; Spironolactone; Stem Cell; Stem Cells; Sunphenon; Sunphenon EGCG; Sustanon; Sustanon (testosterone); Sustanon 250; Suvodirsen; TADALAFIL; TAMOX; TAMOXIFEN; TAMOXIFEN CITRATE; TAS-205; TETRACOSACTIDE HEXAACETATE; TRIATEC; Tacrolimus; Tadalafil; Tadalafil 20 MG; Tadalafil and Sildenafil; Tamoxifen; Tamoxifen 20mg Hexal® Filmtabletten; Tehalose 30gr; Temerit; Testosterone; Testosterone Enanthate; Testosterone decanoate; Testosterone enanthate; Testosterone isocaproate; Testosterone phenylpropionate; Testosterone propionate; Tetracosactide; Tideglusib; Tinset; Tranilast; Translarna; Translarna 1000 mg granules for oral suspension; Translarna 125 mg granules for oral suspension; Translarna 250 mg granules for oral suspension; Treatment Phase Group 4; Trehalose; Triatec; Umbilical Cord Mesenchymal Stem Cell; Umbilical Cord Mesenchymal Stem Cells; VAMOROLONE; VBP15; VILTOLARSEN; Vamarolone; Vamorolone; Vamorolone 0.25 mg/day/day; Vamorolone 0.25 mg/kg/day; Vamorolone 0.75 mg/day/day; Vamorolone 0.75 mg/kg/day; Vamorolone 2.0 mg/day/day; Vamorolone 2.0 mg/kg/day; Vamorolone 6.0 mg/day/day; Vamorolone 6.0 mg/kg/day; Viltolarsen; Vit C Vit E Zn Se; WVE-210201; WVE-210201 (suvodirsen); Xeomin; Zn; [WVE-210201]

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	EUCTR2011-005042-35-BE (EUCTR)	26/06/2013	21/03/2013	A study to test if PRO053 is safe and effective in people who suffer from Duchenne muscular dystrophy	A Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of PRO053 in subjects with Duchenne muscular dystrophy	Duchenne muscular dystrophy resulting from a mutation correctable by PRO053-induced DMD exon 53 skipping MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: BMN053 Product Code: BMN053 INN or Proposed INN: PS524	BioMarin Nederland B.V.	NULL	Not Recruiting			Female: no Male: yes	42	Phase 1;Phase 2	United States;Turkey;Chile;Israel;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan
2	NCT01957059 (ClinicalTrials.gov)	June 2013	2/7/2013	A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)	A Phase I/II, Open-label, Dose Escalating With 48 Week Treatment Study to Assess the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of BMN053 (Previously Known as PRO053) in Subjects With Duchenne Muscular Dystrophy.	Duchenne Muscular Dystrophy	Drug: Regimen Selection Phase Group 2;Drug: Regimen Selection Phase Group 3;Drug: Treatment Phase Group 4;Drug: Regimen Selection Phase Group 1 (COMPLETED);Drug: Dosing Extension	BioMarin Pharmaceutical	NULL	Terminated	5 Years	18 Years	Male	9	Phase 1;Phase 2	Belgium;France;Italy;Netherlands;United Kingdom

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

EUCTR2011-005042-35-BE
(EUCTR)

26/06/2013

21/03/2013

A study to test if PRO053 is safe and effective in people who suffer from Duchenne muscular dystrophy

A Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of PRO053 in subjects with Duchenne muscular dystrophy

Duchenne muscular dystrophy resulting from a mutation correctable by PRO053-induced DMD exon 53 skipping
MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Product Name: BMN053
Product Code: BMN053
INN or Proposed INN: PS524

BioMarin Nederland B.V.

NULL

Not Recruiting

Female: no
Male: yes

Phase 1;Phase 2

United States;Turkey;Chile;Israel;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan

NCT01957059
(ClinicalTrials.gov)

June 2013

2/7/2013

A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)

A Phase I/II, Open-label, Dose Escalating With 48 Week Treatment Study to Assess the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of BMN053 (Previously Known as PRO053) in Subjects With Duchenne Muscular Dystrophy.

Duchenne Muscular Dystrophy

Drug: Regimen Selection Phase Group 2;Drug: Regimen Selection Phase Group 3;Drug: Treatment Phase Group 4;Drug: Regimen Selection Phase Group 1 (COMPLETED);Drug: Dosing Extension

BioMarin Pharmaceutical

NULL

Terminated

5 Years

18 Years

Male

Phase 1;Phase 2

Belgium;France;Italy;Netherlands;United Kingdom