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	NCT02958202 (ClinicalTrials.gov)	April 2016	27/10/2016	Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)	A Multi Center, Multi National, Open Label, Extension Study to Evaluate the Long-term Efficacy and Safety of BMN 044 (PRO044) in Subjects With Duchenne Muscular Dystrophy	Duchenne Muscular Dystrophy	Drug: BMN 044 IV 6 mg/kg;Drug: BMN 044 IV 9 mg/kg;Drug: BMN 044 SC 6 mg/kg	BioMarin Pharmaceutical	NULL	Terminated	5 Years	N/A	Male	7	Phase 2	Belgium;Italy;Netherlands;Sweden
2	EUCTR2015-003681-87-BE (EUCTR)	12/01/2016	16/11/2015	A study to assess the effect of BMN 044 in subjects with Duchenne muscular dystrophy (Extension study)	A multi center, multi national, open label, extension study to evaluate the long-term efficacy and safety of BMN 044 (PRO044) in subjects with Duchenne muscular dystrophy	Duchenne muscular dystrophy MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: BMN 044 Product Code: BMN 044 INN or Proposed INN: PS188 (company code) Other descriptive name: PS188 Product Name: BMN 044 Product Code: BMN 044 INN or Proposed INN: PS188 (company code) Other descriptive name: PS188	BioMarin Pharmaceutical Inc.	NULL	Not Recruiting			Female: no Male: yes	50	Phase 2	United States;Belgium;Netherlands;Italy;Sweden
3	EUCTR2013-003605-26-NL (EUCTR)	13/04/2015	19/11/2014	A study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01)	A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy	Duchenne muscular dystrophy MedDRA version: 17.1;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: PRO044 Product Code: PRO044 INN or Proposed INN: PS188 (company code) Other descriptive name: PS188	Prosensa Therapeutics B.V.	NULL	Not Recruiting			Female: no Male: yes	18	Phase 2	Belgium;Netherlands;Sweden
4	EUCTR2013-003605-26-BE (EUCTR)	23/01/2015	04/12/2014	A study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01)	A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy	Duchenne muscular dystrophy MedDRA version: 18.0;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: PRO044 Product Code: PRO044 INN or Proposed INN: PS188 (company code) Other descriptive name: PS188	Prosensa Therapeutics B.V.	NULL	Not Recruiting			Female: no Male: yes	18	Phase 2	Belgium;Netherlands;Sweden
5	EUCTR2013-003605-26-IT (EUCTR)	02/12/2014	11/09/2014	A study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01)	A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy	Duchenne muscular dystrophy MedDRA version: 17.0;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: PRO044 Product Code: PRO044 INN or Proposed INN: PS188 (company code) Other descriptive name: PS188	Prosensa Therapeutics B.V.	NULL	Not Recruiting			Female: no Male: yes	18	Phase 2	Belgium;Netherlands;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
6	NCT02329769 (ClinicalTrials.gov)	December 2014	22/12/2014	Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)	A Phase II, Open Label, Extension Study to Assess the Effect of PRO044 in Patients With Duchenne Muscular Dystrophy	Duchenne Muscular Dystrophy	Drug: PRO044 SC 6 mg/kg;Drug: PRO044 IV 6 mg/kg;Drug: PRO044 IV 9 mg/kg	BioMarin Pharmaceutical	NULL	Terminated	9 Years	20 Years	Male	15	Phase 2	Belgium;Italy;Netherlands;Sweden
7	EUCTR2013-003605-26-SE (EUCTR)	26/11/2014	26/09/2014	A study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01)	A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy	Duchenne muscular dystrophy MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]	Product Name: PRO044 Product Code: PRO044 INN or Proposed INN: PS188 (company code) Other descriptive name: PS188	BioMarin Nederland B.V.	NULL	Not Recruiting			Female: no Male: yes	18	Phase 2	Belgium;Netherlands;Sweden
8	EUCTR2009-013762-63-NL (EUCTR)	16/04/2010	25/08/2009	A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy	A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy	Duchenne Muscular Dystrophy	Product Name: PRO044 Product Code: PRO044 INN or Proposed INN: h44AON188	Prosensa Therapeutics B.V	NULL	Not Recruiting			Female: no Male: yes	18	Phase 1;Phase 2a	Belgium;Netherlands;Italy;Sweden
9	EUCTR2009-013762-63-IT (EUCTR)	25/02/2010	27/04/2010	A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy - ND	A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy - ND	Duchenne Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801	Product Name: PRO044 Product Code: PRO044	PROSENSA THERAPEUTICS BV	NULL	Not Recruiting			Female: no Male: yes	18	Phase 1;Phase 2a	Belgium;Netherlands;Italy;Sweden
10	EUCTR2009-013762-63-SE (EUCTR)	19/02/2010	03/11/2009	A study to assess the effect and safety of multiple subcutaneous and intravenous doses of PRO044 in patients with Duchenne Disease	A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous and intravenous doses of PRO044 in patients with Duchenne muscular dystrophy	Duchenne Muscular Dystrophy MedDRA version: 14.1;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: PRO044 Product Code: PRO044 INN or Proposed INN: h44AON188	Prosensa Therapeutics B.V	NULL	Not Recruiting			Female: no Male: yes	24	Phase 1;Phase 2a	Belgium;Netherlands;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
11	EUCTR2009-013762-63-BE (EUCTR)	09/12/2009	03/11/2009	A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy	A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy	Duchenne Muscular Dystrophy	Product Name: PRO044 Product Code: PRO044 INN or Proposed INN: h44AON188	Prosensa Therapeutics B.V	NULL	Not Recruiting			Female: no Male: yes	24	Phase 1;Phase 2	Belgium;Netherlands;Italy;Sweden
12	NCT01037309 (ClinicalTrials.gov)	December 2009	21/12/2009	Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)	A Phase I/IIa, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous and Intravenous Doses of PRO044 in Patients With Duchenne Muscular Dystrophy	Duchenne Muscular Dystrophy	Drug: PRO044 SC;Drug: PRO044 IV	BioMarin Pharmaceutical	NULL	Completed	5 Years	16 Years	Male	18	Phase 1;Phase 2	Belgium;Italy;Netherlands;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

NCT02958202
(ClinicalTrials.gov)

April 2016

27/10/2016

Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)

A Multi Center, Multi National, Open Label, Extension Study to Evaluate the Long-term Efficacy and Safety of BMN 044 (PRO044) in Subjects With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Drug: BMN 044 IV 6 mg/kg;Drug: BMN 044 IV 9 mg/kg;Drug: BMN 044 SC 6 mg/kg

BioMarin Pharmaceutical

NULL

Terminated

5 Years

N/A

Male

Phase 2

Belgium;Italy;Netherlands;Sweden

EUCTR2015-003681-87-BE
(EUCTR)

12/01/2016

16/11/2015

A study to assess the effect of BMN 044 in subjects with Duchenne muscular dystrophy (Extension study)

A multi center, multi national, open label, extension study to evaluate the long-term efficacy and safety of BMN 044 (PRO044) in subjects with Duchenne muscular dystrophy

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

Product Name: BMN 044
Product Code: BMN 044
INN or Proposed INN: PS188 (company code)
Other descriptive name: PS188
Product Name: BMN 044
Product Code: BMN 044
INN or Proposed INN: PS188 (company code)
Other descriptive name: PS188

BioMarin Pharmaceutical Inc.

NULL

Not Recruiting

Female: no
Male: yes

Phase 2

United States;Belgium;Netherlands;Italy;Sweden

EUCTR2013-003605-26-NL
(EUCTR)

13/04/2015

19/11/2014

A study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01)

A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy

Duchenne muscular dystrophy
MedDRA version: 17.1;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: PRO044
Product Code: PRO044
INN or Proposed INN: PS188 (company code)
Other descriptive name: PS188

Prosensa Therapeutics B.V.

NULL

Not Recruiting

Female: no
Male: yes

Phase 2

Belgium;Netherlands;Sweden

EUCTR2013-003605-26-BE
(EUCTR)

23/01/2015

04/12/2014

A study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01)

A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy

Duchenne muscular dystrophy
MedDRA version: 18.0;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: PRO044
Product Code: PRO044
INN or Proposed INN: PS188 (company code)
Other descriptive name: PS188

Prosensa Therapeutics B.V.

NULL

Not Recruiting

Female: no
Male: yes

Phase 2

Belgium;Netherlands;Sweden

EUCTR2013-003605-26-IT
(EUCTR)

02/12/2014

11/09/2014

A study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01)

A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy

Duchenne muscular dystrophy
MedDRA version: 17.0;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: PRO044
Product Code: PRO044
INN or Proposed INN: PS188 (company code)
Other descriptive name: PS188

Prosensa Therapeutics B.V.

NULL

Not Recruiting

Female: no
Male: yes

Phase 2

Belgium;Netherlands;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

NCT02329769
(ClinicalTrials.gov)

December 2014

22/12/2014

Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)

A Phase II, Open Label, Extension Study to Assess the Effect of PRO044 in Patients With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Drug: PRO044 SC 6 mg/kg;Drug: PRO044 IV 6 mg/kg;Drug: PRO044 IV 9 mg/kg

BioMarin Pharmaceutical

NULL

Terminated

9 Years

20 Years

Male

Phase 2

Belgium;Italy;Netherlands;Sweden

EUCTR2013-003605-26-SE
(EUCTR)

26/11/2014

26/09/2014

A study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01)

A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy

Product Name: PRO044
Product Code: PRO044
INN or Proposed INN: PS188 (company code)
Other descriptive name: PS188

BioMarin Nederland B.V.

NULL

Not Recruiting

Female: no
Male: yes

Phase 2

Belgium;Netherlands;Sweden

EUCTR2009-013762-63-NL
(EUCTR)

16/04/2010

25/08/2009

A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy

Duchenne Muscular Dystrophy

Product Name: PRO044
Product Code: PRO044
INN or Proposed INN: h44AON188

Prosensa Therapeutics B.V

NULL

Not Recruiting

Female: no
Male: yes

Phase 1;Phase 2a

Belgium;Netherlands;Italy;Sweden

EUCTR2009-013762-63-IT
(EUCTR)

25/02/2010

27/04/2010

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

Product Name: PRO044
Product Code: PRO044

PROSENSA THERAPEUTICS BV

NULL

Not Recruiting

Female: no
Male: yes

Phase 1;Phase 2a

Belgium;Netherlands;Italy;Sweden

EUCTR2009-013762-63-SE
(EUCTR)

19/02/2010

03/11/2009

A study to assess the effect and safety of multiple subcutaneous and intravenous doses of PRO044 in patients with Duchenne Disease

A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous and intravenous doses of PRO044 in patients with Duchenne muscular dystrophy

Duchenne Muscular Dystrophy
MedDRA version: 14.1;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: PRO044
Product Code: PRO044
INN or Proposed INN: h44AON188

Prosensa Therapeutics B.V

NULL

Not Recruiting

Female: no
Male: yes

Phase 1;Phase 2a

Belgium;Netherlands;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

EUCTR2009-013762-63-BE
(EUCTR)

09/12/2009

03/11/2009

Duchenne Muscular Dystrophy

Product Name: PRO044
Product Code: PRO044
INN or Proposed INN: h44AON188

Prosensa Therapeutics B.V

NULL

Not Recruiting

Female: no
Male: yes

Phase 1;Phase 2

Belgium;Netherlands;Italy;Sweden

NCT01037309
(ClinicalTrials.gov)

December 2009

21/12/2009

Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)

A Phase I/IIa, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous and Intravenous Doses of PRO044 in Patients With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Drug: PRO044 SC;Drug: PRO044 IV

BioMarin Pharmaceutical

NULL

Completed

5 Years

16 Years

Male

Phase 1;Phase 2

Belgium;Italy;Netherlands;Sweden