113. 筋ジストロフィー Muscular dystrophy Clinical trials / Disease details

臨床試験数 : 622 / 薬物数 : 485 - (DrugBank : 99) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 168

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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	NCT05185622 (ClinicalTrials.gov)	March 15, 2022	9/11/2021	A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dy ...	A Phase II Open-Label, Multiple Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pha ...	Duchenne Muscular Dystrophy	Drug: Vamorolone	ReveraGen BioPharma, Inc.	Santhera Pharmaceuticals	Not yet recruiting	2 Years	17 Years	Male	44	Phase 2	NULL
2	NCT05126758 (ClinicalTrials.gov)	March 15, 2022	4/11/2021	A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy(HOPE- ...	A Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Efficacy and Safety of ...	Muscular Dystrophies;Muscular Dystrophy, Duchenne;Muscular Disorders, Atrophic;Muscular Diseases;Neu ...	Biological: CAP-1002;Drug: Placebo	Capricor Inc.	NULL	Not yet recruiting	10 Years	N/A	Male	68	Phase 3	United States
3	EUCTR2018-001762-42-NO (EUCTR)	04/03/2022	02/12/2021	A research study to compare different doses of a new investigational medicinal product for treatment ...	A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High D ...	Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchen ...	Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN	Sarepta Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	154	Phase 3	United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Russian Federation;Colombia;Italy;Switzerla ...
4	EUCTR2019-003374-91-ES (EUCTR)	21/02/2022	20/10/2021	Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy	A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study t ...	Duchenne Muscular Dystrophy MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchen ...	Product Code: SRP-9001 INN or Proposed INN: Not available Other descriptive name: ADENO-ASSOCIATED VIR ...	Sarepta Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	120	Phase 3	France;United States;Taiwan;Hong Kong;Belgium;Spain;Germany;United Kingdom;Italy;Japan
5	EUCTR2021-004276-33-DK (EUCTR)	11/02/2022	01/10/2021	GNT0006 gene therapy trial in patients with limb-girdle muscular dystrophy	A phase 1-2 multicenter study (2 stages) to evaluate the safety and efficacy of intravenous GNT0006, ...	FKRP-related limb-girdle muscular dystrophy(LGMD R9) MedDRA version: 20.0;Level: PT;Classification c ...	Product Name: rAAV9-hFKRPco_miR-208a Product Code: GNT0006 INN or Proposed INN: not yet available Other ...	Atamyo Therapeutics	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	39	Phase 1;Phase 2	France;United States;Canada;Denmark;Norway;Germany;United Kingdom;Sweden
6	EUCTR2018-001762-42-GR (EUCTR)	01/02/2022	01/12/2021	A research study to compare different doses of a new investigational medicinal product for treatment ...	A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High D ...	Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchen ...	Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN	Sarepta Therapeutics, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	154	Phase 3	United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Russian Federation;Colombia;Italy;Switzerla ...
7	NCT04336826 (ClinicalTrials.gov)	December 29, 2021	3/4/2020	A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From =6 Months to <2 ...	An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From =6 Month ...	Nonsene Mutation Duchenne Muscular Dystrophy	Drug: Ataluren	PTC Therapeutics	NULL	Recruiting	6 Months	2 Years	Male	10	Phase 2	United States
8	NCT05195775 (ClinicalTrials.gov)	December 14, 2021	4/1/2022	Tadalafil as Adjuvant Therapy for DMD	Tadalafil as an Adjuvant to Therapy for Duchenne Muscular Dystrophy	Duchenne Muscular Dystrophy	Drug: Tadalafil	University of Florida	NULL	Recruiting	7 Years	13 Years	Male	25	Phase 2/Phase 3	United States
9	NCT05160415 (ClinicalTrials.gov)	December 2021	2/12/2021	A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy	A Phase 1b, Open-label Study of the Safety and Pharmacokinetics of EDG-5506 in Adults With Becker Mu ...	Becker Muscular Dystrophy	Drug: EDG-5506	Edgewise Therapeutics, Inc.	Medpace, Inc.	Recruiting	18 Years	55 Years	Male	8	Phase 1	United States
10	EUCTR2021-000122-10-GR (EUCTR)	25/11/2021	13/10/2021	A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD)	A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen ...	Duchenne Muscular Dystrophy(DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: D ...	Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN	NS Pharma, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	74	Phase 3	United States;Hong Kong;Taiwan;Greece;Spain;Ukraine;Turkey;Chile;Russian Federation;United Kingdom;I ...

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