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	EUCTR2017-000397-10-GB (EUCTR)	20/01/2020	15/03/2019	Study in which all the patients take the same investigational drug with theaim of evaluate the long-term safety, tolerability, and efficacy ofGIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophywho have been already treated in one of the GIVINOSTAT studies in thepast	Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies. - Givinostat DMD long term study	Duchenne Muscular Dystrophy (DMD) 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]	Product Name: GIVINOSTAT (hyrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT	ITALFARMACO S.p.A.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	185	Phase 3	France;United States;Canada;Belgium;Spain;Netherlands;Germany;Italy;United Kingdom
2	EUCTR2017-000397-10-NL (EUCTR)	01/10/2019	06/11/2019	Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the past	Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies - Givinostat DMD long term study	Duchenne Muscular Dystrophy (DMD) 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]	Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT	ITALFARMACO S.p.A.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	185	Phase 3	United States;France;Canada;Spain;Belgium;Germany;Netherlands;United Kingdom;Italy
3	EUCTR2017-000397-10-BE (EUCTR)	12/06/2019	12/02/2019	Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the past	Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies - Givinostat DMD long term study	Duchenne Muscular Dystrophy (DMD) 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]	Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT	ITALFARMACO S.p.A.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	185	Phase 3	United States;France;Canada;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy
4	EUCTR2017-000397-10-ES (EUCTR)	16/05/2019	11/04/2019	Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the past	Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies - Givinostat DMD study long term	Duchenne Muscular Dystrophy (DMD) 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]		ITALFARMACO S.p.A.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	185	Phase 3	France;United States;Canada;Belgium;Spain;Netherlands;Germany;Italy;United Kingdom
5	EUCTR2017-001629-41-NL (EUCTR)	15/11/2018	31/05/2018	Study to evaluate the micro-macroscopic effects on muscles, the safety and tolerability, and the efficacy of givinostat in patients with Becker Muscular Dystrophy	A randomised, double-blind, placebo-controlled study to evaluate the micro-macroscopic effects on muscles, the safety and tolerability, and the efficacy ofgivinostat in patients with Becker Muscular Dystrophy - NA	Distrofia Muscolare di Becker (DMB) MedDRA version: 20.0;Level: PT;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: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT	ITALFARMACO S.P.A.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	48	Phase 2	Netherlands
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	NCT03238235 (ClinicalTrials.gov)	December 12, 2017	12/7/2017	Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy	A Randomised, Double Blind, Placebo Controlled Study to Evaluate the Micro-macroscopic Effects on Muscles, the Safety and Tolerability, and the Efficacy of Givinostat in Patients With Becker Muscular Dystrophy (BMD)	Becker Muscular Dystrophy	Drug: givinostat;Drug: placebo	Italfarmaco	NULL	Active, not recruiting	18 Years	65 Years	Male	51	Phase 2	Italy;Netherlands
7	NCT03373968 (ClinicalTrials.gov)	October 24, 2017	4/12/2017	Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study	Open Label, Long-term Safety, Tolerability, and Efficacy Study of GIVINOSTAT in All DMD Patients Who Have Been Previously Treated in One of the GIVINOSTAT Studies	Duchenne Muscular Dystrophy	Drug: Givinostat	Italfarmaco	Cromsource	Enrolling by invitation	7 Years	N/A	Male	100	Phase 2;Phase 3	United States;Belgium;Canada;France;Germany;Italy;Netherlands;Spain;United Kingdom
8	EUCTR2017-000397-10-IT (EUCTR)	31/07/2017	30/01/2018	Studio nel quale tutti i pazienti prendono lo stesso farmaco sperimentale con lo scopo di valutare a lungo termine la sicurezza, la tollerabilità e l'efficacia del GIVINOSTAT in pazienti affetti da distrofia muscolare di Duchenne che sono stati precedentemente trattati in uno studio con GIVINOSTAT.	Studio in aperto a lungo termine volto a valutare la sicurezza, la tollerabilità e l'efficacia di GIVINOSTAT in pazienti affetti da distrofia muscolare di Duchenne che sono stati precedentemente trattati in studio con GIVINOSTAT. - Studio con Givinostat in DMD a lungo termine	Distrofia Muscolare di Duchenne (DMD) 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]		ITALFARMACO S.p.A.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	100	Phase 3	France;United States;Canada;Belgium;Spain;Netherlands;Germany;United Kingdom;Italy
9	EUCTR2016-000401-36-NL (EUCTR)	28/06/2017	14/12/2016	Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.	Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. - EPIDYS	Duchenne Muscular Dystrophy (DMD) 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]		ITALFARMACO S.p.A.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	242	Phase 3	Serbia;France;United States;Canada;Belgium;Spain;Israel;Germany;Netherlands;Italy;United Kingdom
10	NCT02851797 (ClinicalTrials.gov)	June 1, 2017	27/7/2016	Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy	Randomised, Double Blind, Placebo Controlled, Multicentre Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy	Duchenne Muscular Dystrophy	Drug: givinostat;Drug: placebo	Italfarmaco	Syneos Health	Active, not recruiting	6 Years	17 Years	Male	169	Phase 3	United States;Belgium;Canada;France;Germany;Israel;Italy;Netherlands;Serbia;Spain;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
11	EUCTR2016-000401-36-IT (EUCTR)	24/05/2017	08/02/2017	Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.	Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.	Duchenne Muscular Dystrophy (DMD) MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT	ITALFARMACO S.p.A.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	192	Phase 3	France;United States;Canada;Spain;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy
12	EUCTR2016-000401-36-DE (EUCTR)	15/05/2017	28/10/2016	Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.	Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. - EPIDYS	Duchenne Muscular Dystrophy (DMD) 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]		ITALFARMACO S.p.A.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	242	Phase 3	France;United States;Canada;Spain;Belgium;Israel;Netherlands;Germany;Italy;United Kingdom
13	EUCTR2016-000401-36-GB (EUCTR)	07/03/2017	26/10/2016	Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.	Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. - EPIDYS	Duchenne Muscular Dystrophy (DMD) 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]	Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT	ITALFARMACO S.p.A.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	169	Phase 3	United States;Serbia;France;Canada;Spain;Belgium;Israel;Netherlands;Germany;Italy;United Kingdom
14	EUCTR2016-000401-36-ES (EUCTR)	10/02/2017	10/02/2017	Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.	Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.	Duchenne Muscular Dystrophy (DMD) MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT	ITALFARMACO S.p.A.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	192	Phase 3	France;United States;Canada;Belgium;Spain;Israel;Netherlands;Germany;Italy;United Kingdom
15	EUCTR2016-000401-36-BE (EUCTR)	27/01/2017	27/10/2016	Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.	Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. - EPIDYS	Duchenne Muscular Dystrophy (DMD) 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]		ITALFARMACO S.p.A.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	242	Phase 3	Serbia;France;United States;Canada;Spain;Belgium;Israel;Netherlands;Germany;Italy;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
16	NCT01761292 (ClinicalTrials.gov)	April 2013	20/12/2012	A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD	A Two-Part Study to Assess the Safety and Tolerability, Pharmacokinetics, and Effects on Histology and Different Clinical Parameters of Givinostat in Ambulant Children With Duchenne Muscular Dystrophy	Duchenne Muscular Dystrophy (DMD)	Drug: Givinostat	Italfarmaco	NULL	Completed	7 Years	11 Years	Male	20	Phase 1;Phase 2	Italy
17	EUCTR2012-002566-12-IT (EUCTR)	04/02/2013	17/10/2012	A Two-Part Study to Assess the Safety and Tolerability, Pharmacokinetics, and Effects on Histology and Different Clinical Parameters of Givinostat in Ambulant Children with Duchenne Muscular Dystrophy	A Two-Part Study to Assess the Safety and Tolerability, Pharmacokinetics, and Effects on Histology and Different Clinical Parameters of Givinostat in Ambulant Children with Duchenne Muscular Dystrophy - DSC	Duchenne Muscular Dystrophy MedDRA version: 14.1;Level: SOC;Classification code 10028395;Term: Musculoskeletal and connective tissue disorders;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: Givinostat Product Code: ITF2357 INN or Proposed INN: Givinostat Other descriptive name: givinostat Product Name: Givinostat Product Code: ITF2357 INN or Proposed INN: Givinostat Other descriptive name: Givinostat	ITALFARMACO	NULL	Not Recruiting			Female: no Male: yes	20	Phase 2	Italy
18	EUCTR2017-000397-10-DE (EUCTR)		22/01/2019	Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the past	Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies - Givinostat DMD long term study	Duchenne Muscular Dystrophy (DMD) 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]	Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT	ITALFARMACO S.p.A.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	185	Phase 3	United States;Serbia;France;Canada;Spain;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy
19	EUCTR2016-000401-36-FR (EUCTR)		16/05/2017	Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.	Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.	Duchenne Muscular Dystrophy (DMD) 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]	Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT	ITALFARMACO S.p.A.	NULL	NA			Female: no Male: yes	192	Phase 3	United States;France;Canada;Spain;Belgium;Israel;Netherlands;Germany;Italy;United Kingdom
20	EUCTR2017-000397-10-FR (EUCTR)		09/09/2019	Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the past	Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies - Givinostat DMD long term study	Duchenne Muscular Dystrophy (DMD) 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]	Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT	ITALFARMACO S.p.A.	NULL	NA			Female: no Male: yes	185	Phase 3	United States;France;Canada;Belgium;Spain;Netherlands;Germany;Italy;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

EUCTR2017-000397-10-GB
(EUCTR)

20/01/2020

15/03/2019

Study in which all the patients take the same investigational drug with theaim of evaluate the long-term safety, tolerability, and efficacy ofGIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophywho have been already treated in one of the GIVINOSTAT studies in thepast

Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies. - Givinostat DMD long term study

Duchenne Muscular Dystrophy (DMD)
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]

Product Name: GIVINOSTAT (hyrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT

ITALFARMACO S.p.A.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

185

Phase 3

France;United States;Canada;Belgium;Spain;Netherlands;Germany;Italy;United Kingdom

EUCTR2017-000397-10-NL
(EUCTR)

01/10/2019

06/11/2019

Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the past

Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies - Givinostat DMD long term study

Product Name: Givinostat (hydrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT

ITALFARMACO S.p.A.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

185

Phase 3

United States;France;Canada;Spain;Belgium;Germany;Netherlands;United Kingdom;Italy

EUCTR2017-000397-10-BE
(EUCTR)

12/06/2019

12/02/2019

Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the past

Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies - Givinostat DMD long term study

Product Name: Givinostat (hydrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT

ITALFARMACO S.p.A.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

185

Phase 3

United States;France;Canada;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy

EUCTR2017-000397-10-ES
(EUCTR)

16/05/2019

11/04/2019

Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the past

Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies - Givinostat DMD study long term

Duchenne Muscular Dystrophy (DMD)
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]

ITALFARMACO S.p.A.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

185

Phase 3

France;United States;Canada;Belgium;Spain;Netherlands;Germany;Italy;United Kingdom

EUCTR2017-001629-41-NL
(EUCTR)

15/11/2018

31/05/2018

Study to evaluate the micro-macroscopic effects on muscles, the safety and tolerability, and the efficacy of givinostat in patients with Becker Muscular Dystrophy

A randomised, double-blind, placebo-controlled study to evaluate the micro-macroscopic effects on muscles, the safety and tolerability, and the efficacy ofgivinostat in patients with Becker Muscular Dystrophy - NA

Distrofia Muscolare di Becker (DMB)
MedDRA version: 20.0;Level: PT;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: Givinostat (hydrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT

ITALFARMACO S.P.A.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

Phase 2

Netherlands

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

NCT03238235
(ClinicalTrials.gov)

December 12, 2017

12/7/2017

Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy

A Randomised, Double Blind, Placebo Controlled Study to Evaluate the Micro-macroscopic Effects on Muscles, the Safety and Tolerability, and the Efficacy of Givinostat in Patients With Becker Muscular Dystrophy (BMD)

Becker Muscular Dystrophy

Drug: givinostat;Drug: placebo

Italfarmaco

NULL

Active, not recruiting

18 Years

65 Years

Male

Phase 2

Italy;Netherlands

NCT03373968
(ClinicalTrials.gov)

October 24, 2017

4/12/2017

Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study

Open Label, Long-term Safety, Tolerability, and Efficacy Study of GIVINOSTAT in All DMD Patients Who Have Been Previously Treated in One of the GIVINOSTAT Studies

Duchenne Muscular Dystrophy

Drug: Givinostat

Italfarmaco

Cromsource

Enrolling by invitation

7 Years

N/A

Male

100

Phase 2;Phase 3

United States;Belgium;Canada;France;Germany;Italy;Netherlands;Spain;United Kingdom

EUCTR2017-000397-10-IT
(EUCTR)

31/07/2017

30/01/2018

Studio nel quale tutti i pazienti prendono lo stesso farmaco sperimentale con lo scopo di valutare a lungo termine la sicurezza, la tollerabilità e l'efficacia del GIVINOSTAT in pazienti affetti da distrofia muscolare di Duchenne che sono stati precedentemente trattati in uno studio con GIVINOSTAT.

Studio in aperto a lungo termine volto a valutare la sicurezza, la tollerabilità e l'efficacia di GIVINOSTAT in pazienti affetti da distrofia muscolare di Duchenne che sono stati precedentemente trattati in studio con GIVINOSTAT. - Studio con Givinostat in DMD a lungo termine

Distrofia Muscolare di Duchenne (DMD)
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]

ITALFARMACO S.p.A.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

100

Phase 3

France;United States;Canada;Belgium;Spain;Netherlands;Germany;United Kingdom;Italy

EUCTR2016-000401-36-NL
(EUCTR)

28/06/2017

14/12/2016

Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.

Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. - EPIDYS

Duchenne Muscular Dystrophy (DMD)
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]

ITALFARMACO S.p.A.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

242

Phase 3

Serbia;France;United States;Canada;Belgium;Spain;Israel;Germany;Netherlands;Italy;United Kingdom

NCT02851797
(ClinicalTrials.gov)

June 1, 2017

27/7/2016

Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy

Randomised, Double Blind, Placebo Controlled, Multicentre Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Drug: givinostat;Drug: placebo

Italfarmaco

Syneos Health

Active, not recruiting

6 Years

17 Years

Male

169

Phase 3

United States;Belgium;Canada;France;Germany;Israel;Italy;Netherlands;Serbia;Spain;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

EUCTR2016-000401-36-IT
(EUCTR)

24/05/2017

08/02/2017

Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.

Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.

Duchenne Muscular Dystrophy (DMD)
MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Product Name: Givinostat (hydrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT

ITALFARMACO S.p.A.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

192

Phase 3

France;United States;Canada;Spain;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy

EUCTR2016-000401-36-DE
(EUCTR)

15/05/2017

28/10/2016

Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.

Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. - EPIDYS

Duchenne Muscular Dystrophy (DMD)
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]

ITALFARMACO S.p.A.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

242

Phase 3

France;United States;Canada;Spain;Belgium;Israel;Netherlands;Germany;Italy;United Kingdom

EUCTR2016-000401-36-GB
(EUCTR)

07/03/2017

26/10/2016

Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.

Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. - EPIDYS

Product Name: Givinostat (hydrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT

ITALFARMACO S.p.A.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

169

Phase 3

United States;Serbia;France;Canada;Spain;Belgium;Israel;Netherlands;Germany;Italy;United Kingdom

EUCTR2016-000401-36-ES
(EUCTR)

10/02/2017

Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.

Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.

Product Name: Givinostat (hydrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT

ITALFARMACO S.p.A.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

192

Phase 3

France;United States;Canada;Belgium;Spain;Israel;Netherlands;Germany;Italy;United Kingdom

EUCTR2016-000401-36-BE
(EUCTR)

27/01/2017

27/10/2016

Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.

Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. - EPIDYS

Duchenne Muscular Dystrophy (DMD)
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]

ITALFARMACO S.p.A.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

242

Phase 3

Serbia;France;United States;Canada;Spain;Belgium;Israel;Netherlands;Germany;Italy;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

NCT01761292
(ClinicalTrials.gov)

April 2013

20/12/2012

A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD

A Two-Part Study to Assess the Safety and Tolerability, Pharmacokinetics, and Effects on Histology and Different Clinical Parameters of Givinostat in Ambulant Children With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy (DMD)

Drug: Givinostat

Italfarmaco

NULL

Completed

7 Years

11 Years

Male

Phase 1;Phase 2

Italy

EUCTR2012-002566-12-IT
(EUCTR)

04/02/2013

17/10/2012

A Two-Part Study to Assess the Safety and Tolerability, Pharmacokinetics, and Effects on Histology and Different Clinical Parameters of Givinostat in Ambulant Children with Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy
MedDRA version: 14.1;Level: SOC;Classification code 10028395;Term: Musculoskeletal and connective tissue disorders;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Product Name: Givinostat
Product Code: ITF2357
INN or Proposed INN: Givinostat
Other descriptive name: givinostat
Product Name: Givinostat
Product Code: ITF2357
INN or Proposed INN: Givinostat
Other descriptive name: Givinostat

ITALFARMACO

NULL

Not Recruiting

Female: no
Male: yes

Phase 2

Italy

EUCTR2017-000397-10-DE
(EUCTR)

22/01/2019

Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the past

Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies - Givinostat DMD long term study

Product Name: Givinostat (hydrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT

ITALFARMACO S.p.A.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

185

Phase 3

United States;Serbia;France;Canada;Spain;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy

EUCTR2016-000401-36-FR
(EUCTR)

16/05/2017

Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.

Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.

Product Name: Givinostat (hydrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT

ITALFARMACO S.p.A.

NULL

Female: no
Male: yes

192

Phase 3

United States;France;Canada;Spain;Belgium;Israel;Netherlands;Germany;Italy;United Kingdom

EUCTR2017-000397-10-FR
(EUCTR)

09/09/2019

Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the past

Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies - Givinostat DMD long term study

Product Name: Givinostat (hydrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT

ITALFARMACO S.p.A.

NULL

Female: no
Male: yes

185

Phase 3

United States;France;Canada;Belgium;Spain;Netherlands;Germany;Italy;United Kingdom