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	EUCTR2011-004667-76-SE (EUCTR)	19/12/2011	21/10/2011	An open single center study investigating the effects and side-effects of CRD007 in children with Duchenne Muscular Dystrophy (DMD -an inherited disorder which results in muscle degeneration) or Becker Muscular Dystrophy (BMD- an inherited disorder characterized by slowly progressive muscle weakness of the legs and pelvis) or children being symptomatic carriers for DMD or BMD.	An open-label, un-controlled, single-centre trial investigating the efficacy and safety of CRD007 tablets administered twice daily for 12 weeks in children with Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD) or children being symptomatic carriers for DMD or BMD. - CRD007 for the treatment of DMD, BMD and symptomatic carriers	Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD) and symptomatic carriers for DMD or BMD MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Classification code 10059117;Term: Becker's muscular dystrophy;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: pemirolast Product Code: CRD007 INN or Proposed INN: PEMIROLAST	Cardoz AB	NULL	Not Recruiting			Female: yes Male: yes			Sweden

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EUCTR2011-004667-76-SE
(EUCTR)

19/12/2011

21/10/2011

An open single center study investigating the effects and side-effects of CRD007 in children with Duchenne Muscular Dystrophy (DMD -an inherited disorder which results in muscle degeneration) or Becker Muscular Dystrophy (BMD- an inherited disorder characterized by slowly progressive muscle weakness of the legs and pelvis) or children being symptomatic carriers for DMD or BMD.

An open-label, un-controlled, single-centre trial investigating the efficacy and safety of CRD007 tablets administered twice daily for 12 weeks in children with Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD) or children being symptomatic carriers for DMD or BMD. - CRD007 for the treatment of DMD, BMD and symptomatic carriers

Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD) and symptomatic carriers for DMD or BMD
MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1;Classification code 10059117;Term: Becker's muscular dystrophy;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Product Name: pemirolast
Product Code: CRD007
INN or Proposed INN: PEMIROLAST

Cardoz AB

NULL

Not Recruiting

Female: yes
Male: yes

Sweden