113. 筋ジストロフィー Muscular dystrophy Clinical trials / Disease details
臨床試験数 : 646 / 薬物数 : 471 - (DrugBank : 105) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 170
| 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 | ChiCTR2100050052 | 2021-09-01 | 2021-08-16 | Observational study of Lonafarnib in Chinese patients with progeria and progeroid laminopathy | Observational study of Lonafarnib in Chinese patients with progeria and progeroid laminopathy | Hutchinson-Gilford Progeria Syndrome and Progeroid Laminopathies | Experimental group:Treatment with Lonafarnib; | The Children's Hospital Zhejiang University School of Medicine | NULL | Pending | 1 | 99 | Both | Experimental group:25; | N/A | China |
| 2 | EUCTR2019-000305-79-ES (EUCTR) | 14/05/2019 | 12/04/2019 | An extension study to the A083-02 and A083-03 studies that evaluate the long-term effect of the ACE-083 investigational product in patients with Facioscapulohumeral Muscular Dystrophy (FSHD) previously participated in study A083-02 and also in patients with Charcot-Marie Tooth (CMT) disease type 1 and X who previously participated in study A083-03. | An Open-Label Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy (FSHD) Previously Enrolled in Study A083-02 and in Patients with Charcot-Marie Tooth (CMT) Disease Types 1 and X Previously Enrolled in Study A083-03 | Facioscapulohumeral Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ACE-083 Product Code: ACE-083 INN or Proposed INN: pending Other descriptive name: ACE-083 | Acceleron Pharma Inc. | NULL | Not Recruiting | Female: yes Male: yes | 150 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Canada;Spain | ||
| 3 | EUCTR2016-003257-15-ES (EUCTR) | 12/04/2018 | 04/01/2018 | A Phase 2 clinical trial that is randomized and controlled by a placebo (similar to the product under investigation but does not have any therapeutic effect) of ACE-083 in Patients with muscular dystrophy (on face, around shoulder blades, in upper arms). The study is a double-blind study, this means that neither you nor your doctor will know if you receive study drug or placebo | A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy | Facioscapulohumeral Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ACE-083 Product Code: ACE-083 INN or Proposed INN: pending Other descriptive name: ACE-083 | Acceleron Pharma Inc. | NULL | Not Recruiting | Female: yes Male: yes | 92 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Canada;Spain | ||
| 4 | EUCTR2015-004333-27-GB (EUCTR) | 19/01/2016 | 04/11/2015 | A clinical trial to test how the study medication (SMT C1100) works and how safe it is when given to children with Duchenne Muscular Dystrophy | A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation with SMT C1100 in Ambulatory Paediatric Male Subjects with Duchenne Muscular Dystrophy (C11005) - PoC Study to Assess Activity and Safety of SMT C1100 in Boys with DMD | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: SMT C1100 INN or Proposed INN: Pending | Summit (Oxford) Limited | NULL | Not Recruiting | Female: no Male: yes | 40 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;United Kingdom |