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 | NCT05479981 (ClinicalTrials.gov) | August 4, 2022 | 27/7/2022 | Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients | A Phase 2 Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients | DM1;Muscular Dystrophies;Myotonic Dystrophy;Myotonic Dystrophy 1;Myotonic Disorders;Muscular Disorders, Atrophic;Muscular Diseases;Musculoskeletal Diseases;Neuromuscular Diseases;Nervous System Diseases;Genetic Diseases, Inborn;Heredodegenerative Disorders, Nervous System;Neurodegenerative Diseases | Drug: Placebo;Drug: AOC 1001 | Avidity Biosciences, Inc. | NULL | Enrolling by invitation | 18 Years | 66 Years | All | 44 | Phase 2 | United States |
| 2 | NCT05027269 (ClinicalTrials.gov) | October 28, 2021 | 24/8/2021 | Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients | A Randomized, Double-Blind, Placebo-Controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple-Doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients | DM1;Myotonic Dystrophy 1;Myotonic Dystrophy;Myotonic Dystrophy Type 1 (DM1);Dystrophy Myotonic;Myotonic Disorders;Steinert Disease;Myotonic Muscular Dystrophy | Drug: AOC 1001;Drug: Placebo | Avidity Biosciences, Inc. | NULL | Active, not recruiting | 18 Years | 65 Years | All | 44 | Phase 1/Phase 2 | United States |