DDrare: Database of Drug Development for Rare Diseases

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	EUCTR2014-005296-81-BE (EUCTR)	02/05/2016	19/11/2015	A clinical study to provide drisapersen (study medication) to patients withDuchenne disease (muscular disease) and to assess the efficacy, safety and tolerability.	A 24 week Randomized Double-Blind, Placebo-Controlled Study followed by 72 week open-label extension to assess the efficacy, safety and tolerability of drisapersen sodium in subjects with Duchenne Muscular Dystrophy - Drisapersen confirmatory study	Duchenne Muscular Dystrophy (DMD) 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: Drisapersen Product Code: Drisapersen INN or Proposed INN: Drisapersen Other descriptive name: DRISAPERSEN SODIUM	BioMarin Pharmaceutical Inc.	NULL	Not Recruiting			Female: no Male: yes	104	Phase 3	United States;Spain;Turkey;Russian Federation;Israel;United Kingdom;Italy;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Japan;Sweden
2	EUCTR2015-001955-54-DE (EUCTR)	13/01/2016	18/08/2015	A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the safety, tolerability and effect by giving the medication long term.	An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. - Drisapersen extension study	Duchenne Muscular Dystrophy (DMD) 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: Drisapersen Product Code: Drisapersen INN or Proposed INN: drisapersen Other descriptive name: DRISAPERSEN SODIUM Product Name: Drisapersen Product Code: Drisapersen INN or Proposed INN: drisapersen Other descriptive name: DRISAPERSEN SODIUM	BioMarin Pharmaceutical Inc.	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	Portugal;Belarus;United States;Taiwan;Spain;Ukraine;Russian Federation;Chile;Israel;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Uruguay;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Poland;Belgium;Brazil;Bulgaria;Norway;Germany;Japan
3	EUCTR2015-001955-54-BE (EUCTR)	06/01/2016	21/09/2015	A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the safety, tolerability and effect by giving the medication long term.	An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. - Drisapersen extension study	Duchenne Muscular Dystrophy (DMD) 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: Drisapersen Product Code: Drisapersen INN or Proposed INN: drisapersen Other descriptive name: DRISAPERSEN SODIUM	BioMarin Pharmaceutical Inc.	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	Portugal;Belarus;United States;Taiwan;Spain;Ukraine;Russian Federation;Chile;Israel;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Uruguay;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Poland;Belgium;Brazil;Bulgaria;Norway;Germany;Japan
4	EUCTR2015-001955-54-NL (EUCTR)	08/12/2015	09/07/2015	A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the safety, tolerability and effect by giving the medication long term.	An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. - Drisapersen extension study	Duchenne Muscular Dystrophy (DMD) 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: Drisapersen Product Code: Drisapersen	BioMarin Nederland BV	NULL	Not Recruiting			Female: no Male: yes	220	Phase 3	Portugal;United States;Belarus;Taiwan;Spain;Ukraine;Russian Federation;Chile;Israel;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Uruguay;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Poland;Belgium;Brazil;Bulgaria;Norway;Germany;Japan;Sweden
5	NCT02636686 (ClinicalTrials.gov)	November 27, 2015	9/12/2015	Extension Study of Drisapersen in DMD Subjects	An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of Drisapersen in Subjects With Duchenne Muscular Dystrophy.	Duchenne Muscular Dystrophy	Drug: Drisapersen	BioMarin Pharmaceutical	NULL	No longer available	5 Years	80 Years	Male			United States;Argentina;Australia;Belgium;Bulgaria;Czechia;France;Germany;Israel;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Russian Federation;Spain;Taiwan;Turkey;United Kingdom;Czech Republic
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	EUCTR2015-001955-54-ES (EUCTR)	24/11/2015	05/10/2015	A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the safety, tolerability and effect by giving the medication long term.	An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. - Drisapersen extension study	Duchenne Muscular Dystrophy (DMD) 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: Drisapersen Product Code: Drisapersen INN or Proposed INN: drisapersen Other descriptive name: DRISAPERSEN SODIUM	BioMarin Pharmaceutical Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	220		Portugal;Belarus;United States;Taiwan;Spain;Ukraine;Russian Federation;Chile;Israel;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Uruguay;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Poland;Belgium;Brazil;Bulgaria;Norway;Germany;Japan
7	NCT01890798 (ClinicalTrials.gov)	January 2014	27/6/2013	Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol	A Continued Access Protocol for Eligible US Subjects With Duchenne Muscular Dystrophy Who Previously Participated in an Approved Drisapersen Study	Muscular Dystrophies	Drug: Drisapersen	GlaxoSmithKline	NULL	Withdrawn	5 Years	N/A	Male	0	Phase 3	NULL
8	NCT01803412 (ClinicalTrials.gov)	May 1, 2013	28/2/2013	A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects	An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of Drisapersen in US and Canadian Subjects With Duchenne Muscular Dystrophy.	Muscular Dystrophies	Drug: Drisapersen	BioMarin Pharmaceutical	NULL	Terminated	5 Years	N/A	Male	53	Phase 3	United States;Canada
9	EUCTR2007-004819-54-SE (EUCTR)	19/03/2008	31/01/2008	A study to assess the effect and safety of multiple subcutaneous doses of drisapersen in patients with Duchenne Disease	A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of drisapersen in patients with Duchenne muscular dystrophy and to assess the potential for intravenous dosing as an alternative route of administration	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: Drisapersen Product Code: PRO051 INN or Proposed INN: PRO051 Product Name: Drisapersen Product Code: PRO051 INN or Proposed INN: PRO051	Prosensa Therapeutics BV	NULL	Not Recruiting			Female: no Male: yes	12	Phase 1;Phase 2	Belgium;Netherlands;Sweden
10	NCT01910649 (ClinicalTrials.gov)	March 2008	2/8/2012	A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Administration	A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for Intravenous Dosing as an Alternative Route of Administration	Muscular Dystrophies	Drug: Drisapersen	BioMarin Pharmaceutical	NULL	Terminated	5 Years	16 Years	Male	12	Phase 2	NULL
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	EUCTR2007-004819-54-BE (EUCTR)	22/01/2008	14/12/2007	A study to assess the effect and safety of multiple subcutaneous doses of drisapersen in patients with Duchenne Disease	A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of drisapersen in patients with Duchenne muscular dystrophy and to assess the potential for intravenous dosing as an alternative route of administration	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: Drisapersen Product Code: PRO051 INN or Proposed INN: PRO051 Product Name: Drisapersen Product Code: PRO051 INN or Proposed INN: PRO051	Prosensa Therapeutics BV	NULL	Not Recruiting			Female: no Male: yes	12	Phase 1;Phase 2	Belgium;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

EUCTR2014-005296-81-BE
(EUCTR)

02/05/2016

19/11/2015

A clinical study to provide drisapersen (study medication) to patients withDuchenne disease (muscular disease) and to assess the efficacy, safety and tolerability.

A 24 week Randomized Double-Blind, Placebo-Controlled Study followed by 72 week open-label extension to assess the efficacy, safety and tolerability of drisapersen sodium in subjects with Duchenne Muscular Dystrophy - Drisapersen confirmatory study

Duchenne Muscular Dystrophy (DMD)
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: Drisapersen
Product Code: Drisapersen
INN or Proposed INN: Drisapersen
Other descriptive name: DRISAPERSEN SODIUM

BioMarin Pharmaceutical Inc.

NULL

Not Recruiting

Female: no
Male: yes

104

Phase 3

United States;Spain;Turkey;Russian Federation;Israel;United Kingdom;Italy;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Japan;Sweden

EUCTR2015-001955-54-DE
(EUCTR)

13/01/2016

18/08/2015

A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the safety, tolerability and effect by giving the medication long term.

An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. - Drisapersen extension study

Product Name: Drisapersen
Product Code: Drisapersen
INN or Proposed INN: drisapersen
Other descriptive name: DRISAPERSEN SODIUM
Product Name: Drisapersen
Product Code: Drisapersen
INN or Proposed INN: drisapersen
Other descriptive name: DRISAPERSEN SODIUM

BioMarin Pharmaceutical Inc.

NULL

Not Recruiting

Female: no
Male: yes

220

Phase 3

Portugal;Belarus;United States;Taiwan;Spain;Ukraine;Russian Federation;Chile;Israel;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Uruguay;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Poland;Belgium;Brazil;Bulgaria;Norway;Germany;Japan

EUCTR2015-001955-54-BE
(EUCTR)

06/01/2016

21/09/2015

A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the safety, tolerability and effect by giving the medication long term.

An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. - Drisapersen extension study

Duchenne Muscular Dystrophy (DMD)
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: Drisapersen
Product Code: Drisapersen
INN or Proposed INN: drisapersen
Other descriptive name: DRISAPERSEN SODIUM

BioMarin Pharmaceutical Inc.

NULL

Not Recruiting

Female: no
Male: yes

220

Phase 3

EUCTR2015-001955-54-NL
(EUCTR)

08/12/2015

09/07/2015

A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the safety, tolerability and effect by giving the medication long term.

An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. - Drisapersen extension study

Product Name: Drisapersen
Product Code: Drisapersen

BioMarin Nederland BV

NULL

Not Recruiting

Female: no
Male: yes

220

Phase 3

Portugal;United States;Belarus;Taiwan;Spain;Ukraine;Russian Federation;Chile;Israel;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Uruguay;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Poland;Belgium;Brazil;Bulgaria;Norway;Germany;Japan;Sweden

NCT02636686
(ClinicalTrials.gov)

November 27, 2015

9/12/2015

Extension Study of Drisapersen in DMD Subjects

An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of Drisapersen in Subjects With Duchenne Muscular Dystrophy.

Duchenne Muscular Dystrophy

Drug: Drisapersen

BioMarin Pharmaceutical

NULL

No longer available

5 Years

80 Years

Male

United States;Argentina;Australia;Belgium;Bulgaria;Czechia;France;Germany;Israel;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Russian Federation;Spain;Taiwan;Turkey;United Kingdom;Czech Republic

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

EUCTR2015-001955-54-ES
(EUCTR)

24/11/2015

05/10/2015

A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the safety, tolerability and effect by giving the medication long term.

An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. - Drisapersen extension study

Product Name: Drisapersen
Product Code: Drisapersen
INN or Proposed INN: drisapersen
Other descriptive name: DRISAPERSEN SODIUM

BioMarin Pharmaceutical Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

220

NCT01890798
(ClinicalTrials.gov)

January 2014

27/6/2013

Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol

A Continued Access Protocol for Eligible US Subjects With Duchenne Muscular Dystrophy Who Previously Participated in an Approved Drisapersen Study

Muscular Dystrophies

Drug: Drisapersen

GlaxoSmithKline

NULL

Withdrawn

5 Years

N/A

Male

Phase 3

NULL

NCT01803412
(ClinicalTrials.gov)

May 1, 2013

28/2/2013

A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects

An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of Drisapersen in US and Canadian Subjects With Duchenne Muscular Dystrophy.

Muscular Dystrophies

Drug: Drisapersen

BioMarin Pharmaceutical

NULL

Terminated

5 Years

N/A

Male

Phase 3

United States;Canada

EUCTR2007-004819-54-SE
(EUCTR)

19/03/2008

31/01/2008

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

A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of drisapersen in patients with Duchenne muscular dystrophy and to assess the potential for intravenous dosing as an alternative route of administration

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: Drisapersen
Product Code: PRO051
INN or Proposed INN: PRO051
Product Name: Drisapersen
Product Code: PRO051
INN or Proposed INN: PRO051

Prosensa Therapeutics BV

NULL

Not Recruiting

Female: no
Male: yes

Phase 1;Phase 2

Belgium;Netherlands;Sweden

NCT01910649
(ClinicalTrials.gov)

March 2008

2/8/2012

A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Administration

A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for Intravenous Dosing as an Alternative Route of Administration

Muscular Dystrophies

Drug: Drisapersen

BioMarin Pharmaceutical

NULL

Terminated

5 Years

16 Years

Male

Phase 2

NULL

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

EUCTR2007-004819-54-BE
(EUCTR)

22/01/2008

14/12/2007

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

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: Drisapersen
Product Code: PRO051
INN or Proposed INN: PRO051
Product Name: Drisapersen
Product Code: PRO051
INN or Proposed INN: PRO051

Prosensa Therapeutics BV

NULL

Not Recruiting

Female: no
Male: yes

Phase 1;Phase 2

Belgium;Netherlands;Sweden