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	EUCTR2017-001223-49-BG (EUCTR)	16/01/2018	11/10/2017	A clinical trial to evaluate the safety and efficacy of Ataluren versus placebo in people who have specific genetic disease Duchenne Muscular Dystrophy	A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension	Nonsense Mutation Duchenne Muscular Dystrophy MedDRA version: 20.0;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]	Trade Name: Translarna 125 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Trade Name: Translarna 250 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Trade Name: Translarna 1000 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN	PTC Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	340	Phase 3	United States;Taiwan;Hong Kong;Thailand;Turkey;Russian Federation;India;Mexico;Canada;Jordan;Argentina;Brazil;Malaysia;Poland;Australia;Bulgaria;China;Japan;Korea, Republic of
2	EUCTR2013-005489-20-FR (EUCTR)	18/10/2016	18/06/2015	Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy	A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 18.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]		PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
3	NCT02819557 (ClinicalTrials.gov)	June 9, 2016	16/6/2016	Study of Ataluren in =2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy	A Phase 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patients Aged =2 to <5 Years Old With Nonsense Mutation Dystrophinopathy	Duchenne Muscular Dystrophy	Drug: Ataluren	PTC Therapeutics	NULL	Completed	2 Years	5 Years	Male	14	Phase 2	United States
4	EUCTR2013-005489-20-BG (EUCTR)	25/08/2015	26/06/2015	Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy	A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 18.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.0;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: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
5	EUCTR2013-005489-20-CZ (EUCTR)	08/01/2015	24/10/2014	Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy	A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
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
6	EUCTR2013-005489-20-DE (EUCTR)	29/09/2014	28/07/2014	Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy	A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]		PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
7	EUCTR2013-005489-20-IT (EUCTR)	10/09/2014	30/07/2014	Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy	A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
8	EUCTR2013-005489-20-ES (EUCTR)	01/09/2014	06/08/2014	Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy	A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
9	EUCTR2013-005489-20-BE (EUCTR)	28/08/2014	11/07/2014	Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy	A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
10	EUCTR2013-005489-20-GB (EUCTR)	22/08/2014	11/07/2014	Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy	A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]		PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
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
11	EUCTR2013-005489-20-SE (EUCTR)	22/08/2014	11/07/2014	Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy	A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]		PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Sweden;Korea, Republic of
12	NCT02090959 (ClinicalTrials.gov)	March 20, 2014	17/3/2014	An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy	A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy	Muscular Dystrophy, Duchenne;Muscular Dystrophies;Muscular Disorders, Atrophic;Muscular Diseases;Musculoskeletal Diseases;Neuromuscular Diseases;Nervous System Diseases;Genetic Diseases, X-Linked;Genetic Diseases, Inborn	Drug: Ataluren	PTC Therapeutics	NULL	Terminated	7 Years	15 Years	Male	219	Phase 3	United States;Australia;Belgium;Brazil;Bulgaria;Canada;Chile;Czechia;France;Germany;Israel;Italy;Korea, Republic of;Poland;Spain;Sweden;Switzerland;Turkey;United Kingdom;Czech Republic
13	EUCTR2012-004527-20-PL (EUCTR)	12/02/2014	27/11/2013	Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy	A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A	Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren	PTC Therapeutics, Inc	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden
14	EUCTR2012-004527-20-SE (EUCTR)	30/08/2013	24/06/2013	Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy	A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A	Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren	PTC Therapeutics, Inc	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Sweden;Korea, Republic of
15	EUCTR2012-004527-20-CZ (EUCTR)	30/08/2013	24/06/2013	Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy	A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A	Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren	PTC Therapeutics, Inc	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden
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
16	EUCTR2012-004527-20-DE (EUCTR)	20/08/2013	24/06/2013	Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy	A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren	PTC Therapeutics, Inc	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden
17	EUCTR2012-004527-20-GB (EUCTR)	08/08/2013	24/06/2013	Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy	A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A	Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]		PTC Therapeutics, Inc	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;France;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden
18	EUCTR2012-004527-20-BE (EUCTR)	30/07/2013	24/06/2013	Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy	A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A	Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren	PTC Therapeutics, Inc	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Poland;Belgium;Brazil;Australia;Germany;Korea, Republic of;Sweden
19	EUCTR2012-004527-20-ES (EUCTR)	17/07/2013	12/07/2013	Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy	A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A	Nonsense mutation dystrophinopathy MedDRA version: 16.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 16.0;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: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren	PTC Therapeutics, Inc	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden
20	EUCTR2012-004527-20-IT (EUCTR)	01/07/2013	27/06/2013	Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy	A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A	Nonsense mutation dystrophinopathy MedDRA version: 16.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 16.0;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: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren	PTC Therapeutics, Inc	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;United Kingdom;Italy;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden
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
21	EUCTR2011-004853-18-IT (EUCTR)	18/09/2012	27/09/2012	An Open-Label Study for Previously Treated Ataluren (PTC124) Patients with Nonsense Mutation Dystrophinopathy	An Open-Label Study for Previously Treated Ataluren (PTC124) Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 15.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 15.0;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: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: na Product Name: ataluren Product Code: PTC124 INN or Proposed INN: na	PTC THERAPEUTICS, INC.	NULL	Not Recruiting			Female: no Male: yes	96	Phase 3	Canada;Spain;Belgium;Australia;Israel;Germany;United Kingdom;Italy;Sweden
22	EUCTR2011-004853-18-GB (EUCTR)	21/08/2012	29/03/2012	Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy	An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]		PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	96	Phase 3	France;Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
23	EUCTR2011-004853-18-ES (EUCTR)	13/06/2012	17/04/2012	Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy	An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy - N/A	Nonsense mutation dystrophinopathy 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;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren	PTC Therapeutics, Inc	NULL	Not Recruiting			Female: no Male: yes	96	Phase 3	Canada;Belgium;Spain;Australia;Israel;Germany;Italy;United Kingdom;Sweden
24	NCT01557400 (ClinicalTrials.gov)	May 20, 2012	15/3/2012	Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada	An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients With Nonsense Mutation Dystrophinopathy	Duchenne Muscular Dystrophy;Becker Muscular Dystrophy;Dystrophinopathy	Drug: Ataluren	PTC Therapeutics	NULL	Completed	N/A	N/A	Male	94	Phase 3	Australia;Belgium;Canada;France;Germany;Israel;Italy;Spain;Sweden;United Kingdom
25	EUCTR2011-004853-18-FR (EUCTR)	02/05/2012	13/08/2012	Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy	An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 14.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]		PTC Therapeutics, Inc	NULL	Not Recruiting			Female: no Male: yes	96	Phase 3	France;Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
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
26	EUCTR2011-004853-18-DE (EUCTR)	02/05/2012	12/03/2012	Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy	An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;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: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	96	Phase 3	Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
27	EUCTR2011-004853-18-BE (EUCTR)	27/04/2012	03/01/2012	Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy	An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy	Nonsense mutation dystrophinopathy MedDRA version: 16.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 16.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren	PTC Therapeutics, Inc	NULL	Not Recruiting			Female: no Male: yes	96	Phase 3	Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
28	EUCTR2011-004853-18-SE (EUCTR)	26/04/2012	27/02/2012	Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy	An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy - N/A	Nonsense mutation dystrophinopathy MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren	PTC Therapeutics, Inc	NULL	Not Recruiting			Female: no Male: yes	96	Phase 3	Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
29	NCT01247207 (ClinicalTrials.gov)	November 30, 2010	22/11/2010	Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)	An Open-Label, Safety Study for Previously Treated Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy	Duchenne Muscular Dystrophy	Drug: Ataluren	PTC Therapeutics	NULL	Enrolling by invitation	N/A	N/A	Male	160	Phase 3	United States;Canada
30	EUCTR2009-013169-24-GB (EUCTR)	19/01/2010	17/03/2010	A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients with Nonsense–Mutation-Mediated Duchenne/Becker Muscular Dystrophy - Study of Ataluren in Nonambulatory Patients with DMD/BMD	A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients with Nonsense–Mutation-Mediated Duchenne/Becker Muscular Dystrophy - Study of Ataluren in Nonambulatory Patients with DMD/BMD	Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy MedDRA version: 12.0;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 12.0;Classification code 10059117;Term: Becker's muscular dystrophy	Product Name: Ataluren 125 mg Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: Ataluren 250 mg Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: Ataluren 1000 mg Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844	PTC Therapeutics Inc	NULL	Not Recruiting			Female: no Male: yes	30	Phase 2a	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
31	NCT01009294 (ClinicalTrials.gov)	January 13, 2010	5/11/2009	Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)	A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy	Duchenne Muscular Dystrophy;Becker Muscular Dystrophy	Drug: Ataluren;Drug: Chronic Corticosteroid Therapy	PTC Therapeutics	Genzyme, a Sanofi Company	Terminated	7 Years	N/A	Male	6	Phase 2	United States;United Kingdom
32	EUCTR2008-007648-32-ES (EUCTR)	09/07/2009	04/05/2009	Un estudio de extensión en Fase 2b de PTC124 en sujetos con distrofia muscular de Duchenne y Becker mediada por mutación terminadora.	Un estudio de extensión en Fase 2b de PTC124 en sujetos con distrofia muscular de Duchenne y Becker mediada por mutación terminadora.	Distrofia muscular de Duchenne, Distrofia muscular de Becker MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy	Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844	PTC Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	174		United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
33	EUCTR2008-007648-32-GB (EUCTR)	22/06/2009	29/04/2009	A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy	A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy	Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy	Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	174	Phase 2b	Germany;United Kingdom;Belgium;France;Spain;Italy;Sweden
34	EUCTR2008-007648-32-DE (EUCTR)	22/06/2009	08/04/2009	A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy	A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy	Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy	Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	174	Phase 2b	France;Spain;Belgium;Germany;Italy;United Kingdom;Sweden
35	EUCTR2008-007648-32-SE (EUCTR)	02/06/2009	17/04/2009	A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy	A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy	Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy	Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	174	Phase 2b	United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
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
36	EUCTR2008-007648-32-FR (EUCTR)	28/05/2009	03/04/2009	A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy	A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy	Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy	Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844	PTC Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	174	Phase 2b	United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
37	EUCTR2008-007648-32-IT (EUCTR)	27/05/2009	22/05/2009	A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy - ND	A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy - ND	Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801 MedDRA version: 9.1;Classification code 10059117	Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN	PTC THERAPEUTICS, INC.	NULL	Not Recruiting			Female: no Male: yes	174	Phase 2b	United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
38	EUCTR2007-005478-29-GB (EUCTR)	27/04/2009	22/02/2008	A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy	A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy	Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy		PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	174	Phase 2	France;Spain;Belgium;Germany;Italy;United Kingdom;Sweden
39	EUCTR2008-007648-32-BE (EUCTR)	14/04/2009	25/02/2009	A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy	A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy	Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy	Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	174	Phase 2b	United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
40	NCT00847379 (ClinicalTrials.gov)	January 31, 2009	16/2/2009	Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)	A Phase 2B Extension Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy	Duchenne Muscular Dystrophy;Becker Muscular Dystrophy	Drug: Ataluren	PTC Therapeutics	Genzyme, a Sanofi Company	Terminated	5 Years	N/A	Male	173	Phase 2	United States;Australia;Belgium;Canada;France;Germany;Israel;Italy;Spain;Sweden;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
41	NCT00759876 (ClinicalTrials.gov)	August 13, 2008	23/9/2008	Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)	A Phase 2a Extension Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne Muscular Dystrophy	Duchenne Muscular Dystrophy	Drug: Ataluren	PTC Therapeutics	Genzyme, a Sanofi Company	Terminated	N/A	N/A	Male	36	Phase 2	United States
42	EUCTR2007-005478-29-IT (EUCTR)	03/06/2008	14/07/2008	A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy - ND	A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy - ND	Duchenne Muscular Dystrophy, Becker Muscular Dystrophy. MedDRA version: 9.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy	Product Code: PTC124	PTC THERAPEUTICS, INC.	NULL	Not Recruiting			Female: no Male: yes	165	Phase 2b	United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
43	EUCTR2007-005478-29-ES (EUCTR)	20/05/2008	10/03/2008	Estudio de fase 2b sobre la eficacia y la seguridad de PTC124 en pacientes con distrofia muscular de Duchenne y Becker mediada por mutación terminadora	Estudio de fase 2b sobre la eficacia y la seguridad de PTC124 en pacientes con distrofia muscular de Duchenne y Becker mediada por mutación terminadora	Distrofia muscular de Duchenne, Distrofia muscular de Becker MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy	Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: PTC124 Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844	PTC Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	165		United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
44	EUCTR2007-005478-29-SE (EUCTR)	16/04/2008	22/02/2008	A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy	A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy	Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy	Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	174	Phase 2b	United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
45	EUCTR2007-005478-29-DE (EUCTR)	14/04/2008	28/01/2008	A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy	A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy	Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy	Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	174	Phase 2b	United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
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
46	EUCTR2007-005478-29-FR (EUCTR)	17/03/2008	24/01/2008	A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy	A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy	Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy	Product Name: PTC124 Product Code: PTC124 Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844	PTC Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	165	Phase 2b	United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
47	EUCTR2007-005478-29-BE (EUCTR)	11/03/2008	27/02/2008	A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy	A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy	Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy	Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844	PTC Therapeutics, Inc.	NULL	Not Recruiting			Female: no Male: yes	174	Phase 2b	United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
48	NCT00592553 (ClinicalTrials.gov)	February 29, 2008	1/1/2008	Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)	A Phase 2B Efficacy and Safety Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy	Duchenne Muscular Dystrophy;Becker Muscular Dystrophy	Drug: Ataluren;Drug: Placebo	PTC Therapeutics	NULL	Completed	5 Years	N/A	Male	174	Phase 2	United States;Australia;Belgium;Canada;France;Germany;Israel;Italy;Spain;Sweden;United Kingdom
49	NCT00264888 (ClinicalTrials.gov)	December 2005	9/12/2005	Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy	A Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Duchenne Muscular Dystrophy	Duchenne Muscular Dystrophy	Drug: PTC124	PTC Therapeutics	Muscular Dystrophy Association	Completed	5 Years	N/A	Male	38	Phase 2	United States
50	EUCTR2012-004527-20-FR (EUCTR)		25/09/2015	Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy	A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A	Nonsense mutation dystrophinopathy MedDRA version: 18.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]		PTC Therapeutics, Inc	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;France;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden
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
51	EUCTR2017-001223-49-PL (EUCTR)		05/06/2018	A clinical trial to evaluate the safety and efficacy of Ataluren versus placebo in people who have specific genetic disease Duchenne Muscular Dystrophy	A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension	Nonsense Mutation Duchenne Muscular Dystrophy MedDRA version: 20.0;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]	Trade Name: Translarna 125 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Trade Name: Translarna 250 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Trade Name: Translarna 1000 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN	PTC Therapeutics, Inc.	NULL	NA			Female: no Male: yes	340	Phase 3	United States;Taiwan;Hong Kong;Thailand;Turkey;Russian Federation;India;Mexico;Canada;Jordan;Argentina;Poland;Brazil;Malaysia;Australia;Bulgaria;China;Japan;Korea, Republic of

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

EUCTR2017-001223-49-BG
(EUCTR)

16/01/2018

11/10/2017

A clinical trial to evaluate the safety and efficacy of Ataluren versus placebo in people who have specific genetic disease Duchenne Muscular Dystrophy

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension

Nonsense Mutation Duchenne Muscular Dystrophy
MedDRA version: 20.0;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]

Trade Name: Translarna 125 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Trade Name: Translarna 250 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Trade Name: Translarna 1000 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN

PTC Therapeutics, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

340

Phase 3

United States;Taiwan;Hong Kong;Thailand;Turkey;Russian Federation;India;Mexico;Canada;Jordan;Argentina;Brazil;Malaysia;Poland;Australia;Bulgaria;China;Japan;Korea, Republic of

EUCTR2013-005489-20-FR
(EUCTR)

18/10/2016

18/06/2015

Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy

A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy

Nonsense mutation dystrophinopathy
MedDRA version: 18.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 18.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

PTC Therapeutics, Inc.

NULL

Not Recruiting

Female: no
Male: yes

220

Phase 3

United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden

NCT02819557
(ClinicalTrials.gov)

June 9, 2016

16/6/2016

Study of Ataluren in =2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy

A Phase 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patients Aged =2 to <5 Years Old With Nonsense Mutation Dystrophinopathy

Duchenne Muscular Dystrophy

Drug: Ataluren

PTC Therapeutics

NULL

Completed

2 Years

5 Years

Male

Phase 2

United States

EUCTR2013-005489-20-BG
(EUCTR)

25/08/2015

26/06/2015

Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy

A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy

Nonsense mutation dystrophinopathy
MedDRA version: 18.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 18.0;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: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren

PTC Therapeutics, Inc.

NULL

Not Recruiting

Female: no
Male: yes

220

Phase 3

United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden

EUCTR2013-005489-20-CZ
(EUCTR)

08/01/2015

24/10/2014

Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy

A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy

Nonsense mutation dystrophinopathy
MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

PTC Therapeutics, Inc.

NULL

Not Recruiting

Female: no
Male: yes

220

Phase 3

United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden

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

EUCTR2013-005489-20-DE
(EUCTR)

29/09/2014

28/07/2014

Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy

A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy

Nonsense mutation dystrophinopathy
MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

PTC Therapeutics, Inc.

NULL

Not Recruiting

Female: no
Male: yes

220

Phase 3

United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden

EUCTR2013-005489-20-IT
(EUCTR)

10/09/2014

30/07/2014

Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy

A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy

Nonsense mutation dystrophinopathy
MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

PTC Therapeutics, Inc.

NULL

Not Recruiting

Female: no
Male: yes

220

Phase 3

United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden

EUCTR2013-005489-20-ES
(EUCTR)

01/09/2014

06/08/2014

Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy

A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy

PTC Therapeutics, Inc.

NULL

Not Recruiting

Female: no
Male: yes

220

Phase 3

United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden

EUCTR2013-005489-20-BE
(EUCTR)

28/08/2014

11/07/2014

Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy

A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy

PTC Therapeutics, Inc.

NULL

Not Recruiting

Female: no
Male: yes

220

Phase 3

United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Bulgaria;Germany;Korea, Republic of;Sweden

EUCTR2013-005489-20-GB
(EUCTR)

22/08/2014

11/07/2014

Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy

A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy

PTC Therapeutics, Inc.

NULL

Not Recruiting

Female: no
Male: yes

220

Phase 3

United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden

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

EUCTR2013-005489-20-SE
(EUCTR)

22/08/2014

11/07/2014

Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy

A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy

PTC Therapeutics, Inc.

NULL

Not Recruiting

Female: no
Male: yes

220

Phase 3

United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Sweden;Korea, Republic of

NCT02090959
(ClinicalTrials.gov)

March 20, 2014

17/3/2014

An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy

A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy

Muscular Dystrophy, Duchenne;Muscular Dystrophies;Muscular Disorders, Atrophic;Muscular Diseases;Musculoskeletal Diseases;Neuromuscular Diseases;Nervous System Diseases;Genetic Diseases, X-Linked;Genetic Diseases, Inborn

Drug: Ataluren

PTC Therapeutics

NULL

Terminated

7 Years

15 Years

Male

219

Phase 3

United States;Australia;Belgium;Brazil;Bulgaria;Canada;Chile;Czechia;France;Germany;Israel;Italy;Korea, Republic of;Poland;Spain;Sweden;Switzerland;Turkey;United Kingdom;Czech Republic

EUCTR2012-004527-20-PL
(EUCTR)

12/02/2014

27/11/2013

Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy

A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A

PTC Therapeutics, Inc

NULL

Not Recruiting

Female: no
Male: yes

220

Phase 3

United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden

EUCTR2012-004527-20-SE
(EUCTR)

30/08/2013

24/06/2013

Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy

A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A

PTC Therapeutics, Inc

NULL

Not Recruiting

Female: no
Male: yes

220

Phase 3

United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Sweden;Korea, Republic of

EUCTR2012-004527-20-CZ
(EUCTR)

30/08/2013

24/06/2013

Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy

A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A

PTC Therapeutics, Inc

NULL

Not Recruiting

Female: no
Male: yes

220

Phase 3

United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden

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

EUCTR2012-004527-20-DE
(EUCTR)

20/08/2013

24/06/2013

Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy

A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy

PTC Therapeutics, Inc

NULL

Not Recruiting

Female: no
Male: yes

220

Phase 3

United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden

EUCTR2012-004527-20-GB
(EUCTR)

08/08/2013

24/06/2013

Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy

A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A

Nonsense mutation dystrophinopathy
MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

PTC Therapeutics, Inc

NULL

Not Recruiting

Female: no
Male: yes

220

Phase 3

United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;France;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden

EUCTR2012-004527-20-BE
(EUCTR)

30/07/2013

24/06/2013

Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy

A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A

PTC Therapeutics, Inc

NULL

Not Recruiting

Female: no
Male: yes

220

Phase 3

United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Poland;Belgium;Brazil;Australia;Germany;Korea, Republic of;Sweden

EUCTR2012-004527-20-ES
(EUCTR)

17/07/2013

12/07/2013

Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy

A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A

Nonsense mutation dystrophinopathy
MedDRA version: 16.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 16.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

PTC Therapeutics, Inc

NULL

Not Recruiting

Female: no
Male: yes

220

Phase 3

United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden

EUCTR2012-004527-20-IT
(EUCTR)

01/07/2013

27/06/2013

Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy

A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A

PTC Therapeutics, Inc

NULL

Not Recruiting

Female: no
Male: yes

220

Phase 3

United States;Spain;Turkey;Chile;Israel;United Kingdom;Italy;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden

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

EUCTR2011-004853-18-IT
(EUCTR)

18/09/2012

27/09/2012

An Open-Label Study for Previously Treated Ataluren (PTC124) Patients with Nonsense Mutation Dystrophinopathy

Nonsense mutation dystrophinopathy
MedDRA version: 15.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 15.0;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: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: na
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: na

PTC THERAPEUTICS, INC.

NULL

Not Recruiting

Female: no
Male: yes

Phase 3

Canada;Spain;Belgium;Australia;Israel;Germany;United Kingdom;Italy;Sweden

EUCTR2011-004853-18-GB
(EUCTR)

21/08/2012

29/03/2012

Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy

An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy

PTC Therapeutics, Inc.

NULL

Not Recruiting

Female: no
Male: yes

Phase 3

France;Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden

EUCTR2011-004853-18-ES
(EUCTR)

13/06/2012

17/04/2012

Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy

An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy - N/A

Nonsense mutation dystrophinopathy
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;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

PTC Therapeutics, Inc

NULL

Not Recruiting

Female: no
Male: yes

Phase 3

Canada;Belgium;Spain;Australia;Israel;Germany;Italy;United Kingdom;Sweden

NCT01557400
(ClinicalTrials.gov)

May 20, 2012

15/3/2012

Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada

An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients With Nonsense Mutation Dystrophinopathy

Duchenne Muscular Dystrophy;Becker Muscular Dystrophy;Dystrophinopathy

Drug: Ataluren

PTC Therapeutics

NULL

Completed

N/A

Male

Phase 3

Australia;Belgium;Canada;France;Germany;Israel;Italy;Spain;Sweden;United Kingdom

EUCTR2011-004853-18-FR
(EUCTR)

02/05/2012

13/08/2012

Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy

An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy

Nonsense mutation dystrophinopathy
MedDRA version: 14.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

PTC Therapeutics, Inc

NULL

Not Recruiting

Female: no
Male: yes

Phase 3

France;Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden

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

EUCTR2011-004853-18-DE
(EUCTR)

02/05/2012

12/03/2012

Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy

An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy

Nonsense mutation dystrophinopathy
MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

PTC Therapeutics, Inc.

NULL

Not Recruiting

Female: no
Male: yes

Phase 3

Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden

EUCTR2011-004853-18-BE
(EUCTR)

27/04/2012

03/01/2012

Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy

An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy

Nonsense mutation dystrophinopathy
MedDRA version: 16.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 16.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

PTC Therapeutics, Inc

NULL

Not Recruiting

Female: no
Male: yes

Phase 3

Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden

EUCTR2011-004853-18-SE
(EUCTR)

26/04/2012

27/02/2012

Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy

An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy - N/A

PTC Therapeutics, Inc

NULL

Not Recruiting

Female: no
Male: yes

Phase 3

Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden

NCT01247207
(ClinicalTrials.gov)

November 30, 2010

22/11/2010

Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

An Open-Label, Safety Study for Previously Treated Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy

Duchenne Muscular Dystrophy

Drug: Ataluren

PTC Therapeutics

NULL

Enrolling by invitation

N/A

Male

160

Phase 3

United States;Canada

EUCTR2009-013169-24-GB
(EUCTR)

19/01/2010

17/03/2010

A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients with Nonsense–Mutation-Mediated Duchenne/Becker Muscular Dystrophy - Study of Ataluren in Nonambulatory Patients with DMD/BMD

Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy
MedDRA version: 12.0;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
MedDRA version: 12.0;Classification code 10059117;Term: Becker's muscular dystrophy

Product Name: Ataluren 125 mg
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: Ataluren 250 mg
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: Ataluren 1000 mg
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844

PTC Therapeutics Inc

NULL

Not Recruiting

Female: no
Male: yes

Phase 2a

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

NCT01009294
(ClinicalTrials.gov)

January 13, 2010

5/11/2009

Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)

A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy

Duchenne Muscular Dystrophy;Becker Muscular Dystrophy

Drug: Ataluren;Drug: Chronic Corticosteroid Therapy

PTC Therapeutics

Genzyme, a Sanofi Company

Terminated

7 Years

N/A

Male

Phase 2

United States;United Kingdom

EUCTR2008-007648-32-ES
(EUCTR)

09/07/2009

04/05/2009

Un estudio de extensión en Fase 2b de PTC124 en sujetos con distrofia muscular de Duchenne y Becker mediada por mutación terminadora.

Distrofia muscular de Duchenne, Distrofia muscular de Becker
MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy

Product Name: ATALUREN
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: ATALUREN
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: ATALUREN
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844

PTC Therapeutics, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

174

United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden

EUCTR2008-007648-32-GB
(EUCTR)

22/06/2009

29/04/2009

A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy

Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy

Product Name: Ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: ATALUREN
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: ATALUREN
Product Code: PTC124
INN or Proposed INN: ATALUREN
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844

PTC Therapeutics, Inc.

NULL

Not Recruiting

Female: no
Male: yes

174

Phase 2b

Germany;United Kingdom;Belgium;France;Spain;Italy;Sweden

EUCTR2008-007648-32-DE
(EUCTR)

22/06/2009

08/04/2009

A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy

PTC Therapeutics, Inc.

NULL

Not Recruiting

Female: no
Male: yes

174

Phase 2b

France;Spain;Belgium;Germany;Italy;United Kingdom;Sweden

EUCTR2008-007648-32-SE
(EUCTR)

02/06/2009

17/04/2009

A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy

PTC Therapeutics, Inc.

NULL

Not Recruiting

Female: no
Male: yes

174

Phase 2b

United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden

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

EUCTR2008-007648-32-FR
(EUCTR)

28/05/2009

03/04/2009

A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy

PTC Therapeutics, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

174

Phase 2b

United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden

EUCTR2008-007648-32-IT
(EUCTR)

27/05/2009

22/05/2009

A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy - ND

Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
MedDRA version: 9.1;Level: LLT;Classification code 10013801
MedDRA version: 9.1;Classification code 10059117

Product Name: Ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Product Name: Ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Product Name: Ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN

PTC THERAPEUTICS, INC.

NULL

Not Recruiting

Female: no
Male: yes

174

Phase 2b

United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden

EUCTR2007-005478-29-GB
(EUCTR)

27/04/2009

22/02/2008

A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy

PTC Therapeutics, Inc.

NULL

Not Recruiting

Female: no
Male: yes

174

Phase 2

France;Spain;Belgium;Germany;Italy;United Kingdom;Sweden

EUCTR2008-007648-32-BE
(EUCTR)

14/04/2009

25/02/2009

A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy

PTC Therapeutics, Inc.

NULL

Not Recruiting

Female: no
Male: yes

174

Phase 2b

United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden

NCT00847379
(ClinicalTrials.gov)

January 31, 2009

16/2/2009

Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

A Phase 2B Extension Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy

Duchenne Muscular Dystrophy;Becker Muscular Dystrophy

Drug: Ataluren

PTC Therapeutics

Genzyme, a Sanofi Company

Terminated

5 Years

N/A

Male

173

Phase 2

United States;Australia;Belgium;Canada;France;Germany;Israel;Italy;Spain;Sweden;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

NCT00759876
(ClinicalTrials.gov)

August 13, 2008

23/9/2008

Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)

A Phase 2a Extension Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Drug: Ataluren

PTC Therapeutics

Genzyme, a Sanofi Company

Terminated

N/A

Male

Phase 2

United States

EUCTR2007-005478-29-IT
(EUCTR)

03/06/2008

14/07/2008

A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy - ND

Duchenne Muscular Dystrophy, Becker Muscular Dystrophy.
MedDRA version: 9.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy

Product Code: PTC124

PTC THERAPEUTICS, INC.

NULL

Not Recruiting

Female: no
Male: yes

165

Phase 2b

United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden

EUCTR2007-005478-29-ES
(EUCTR)

20/05/2008

10/03/2008

Estudio de fase 2b sobre la eficacia y la seguridad de PTC124 en pacientes con distrofia muscular de Duchenne y Becker mediada por mutación terminadora

Product Name: PTC124
Product Code: PTC124
INN or Proposed INN: PTC124
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844

PTC Therapeutics, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

165

United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden

EUCTR2007-005478-29-SE
(EUCTR)

16/04/2008

22/02/2008

A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy

Product Name: PTC124
Product Code: PTC124
INN or Proposed INN: ataluren
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844

PTC Therapeutics, Inc.

NULL

Not Recruiting

Female: no
Male: yes

174

Phase 2b

United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden

EUCTR2007-005478-29-DE
(EUCTR)

14/04/2008

28/01/2008

A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy

Product Name: PTC124
Product Code: PTC124
INN or Proposed INN: ataluren
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: PTC124
Product Code: PTC124
INN or Proposed INN: ataluren
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Product Name: PTC124
Product Code: PTC124
INN or Proposed INN: ataluren
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844

PTC Therapeutics, Inc.

NULL

Not Recruiting

Female: no
Male: yes

174

Phase 2b

United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden

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

EUCTR2007-005478-29-FR
(EUCTR)

17/03/2008

24/01/2008

A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy

Product Name: PTC124
Product Code: PTC124
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844

PTC Therapeutics, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

165

Phase 2b

United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden

EUCTR2007-005478-29-BE
(EUCTR)

11/03/2008

27/02/2008

A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy

Product Name: PTC124
Product Code: PTC124
INN or Proposed INN: ataluren
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844

PTC Therapeutics, Inc.

NULL

Not Recruiting

Female: no
Male: yes

174

Phase 2b

United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden

NCT00592553
(ClinicalTrials.gov)

February 29, 2008

1/1/2008

Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

A Phase 2B Efficacy and Safety Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy

Duchenne Muscular Dystrophy;Becker Muscular Dystrophy

Drug: Ataluren;Drug: Placebo

PTC Therapeutics

NULL

Completed

5 Years

N/A

Male

174

Phase 2

United States;Australia;Belgium;Canada;France;Germany;Israel;Italy;Spain;Sweden;United Kingdom

NCT00264888
(ClinicalTrials.gov)

December 2005

9/12/2005

Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy

A Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Drug: PTC124

PTC Therapeutics

Muscular Dystrophy Association

Completed

5 Years

N/A

Male

Phase 2

United States

EUCTR2012-004527-20-FR
(EUCTR)

25/09/2015

Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy

A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A

PTC Therapeutics, Inc

NULL

Not Recruiting

Female: no
Male: yes

220

Phase 3

United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;France;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden

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

EUCTR2017-001223-49-PL
(EUCTR)

05/06/2018

A clinical trial to evaluate the safety and efficacy of Ataluren versus placebo in people who have specific genetic disease Duchenne Muscular Dystrophy

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension

PTC Therapeutics, Inc.

NULL

Female: no
Male: yes

340

Phase 3

United States;Taiwan;Hong Kong;Thailand;Turkey;Russian Federation;India;Mexico;Canada;Jordan;Argentina;Poland;Brazil;Malaysia;Australia;Bulgaria;China;Japan;Korea, Republic of