AOC 1001 ( DrugBank: - )

---

1 disease

告示番号	疾患名（ページ内リンク）	臨床試験数
113	筋ジストロフィー	2

113. 筋ジストロフィー

---

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