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9 Trials

   

Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
delpacibart etedesiran (AOC 1001) / Avidity Biosci
HARBOR, NCT06411288: Global Study of Del-desiran for the Treatment of DM1

Recruiting
3
150
Europe, Canada, Japan, US
AOC 1001 (del-desiran), Placebo
Avidity Biosciences, Inc.
DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonia, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Steinert, Myotonic Muscular Dystrophy
10/26
04/27
HARBOR-OLE, NCT07008469: Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

Not yet recruiting
3
217
NA
Del-desiran (AOC 1001)
Avidity Biosciences, Inc.
Myotonic Dystrophy Type 1, DM1, Myotonic Dystrophy, Myotonia, Myotonic Dystrophy 1, Myotonic Disorders, Steinert Myotonic Dystrophy, Steinert's Disease
08/30
10/30
MARINA-OLE, NCT05479981: Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients

Active, not recruiting
2
37
US
Placebo, Saline, AOC 1001
Avidity Biosciences, Inc.
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
06/27
06/27
MARINA, NCT05027269: Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients

Completed
1/2
38
US
AOC 1001, Placebo
Avidity Biosciences, Inc.
DM1, Myotonic Dystrophy 1, Myotonic Dystrophy, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Myotonic Muscular Dystrophy
02/23
02/23
delpacibart zotadirsen (AOC-1044) / Eli Lilly
EXPLORE44OLE, NCT06244082: Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping

Active, not recruiting
2
39
US
AOC 1044
Avidity Biosciences, Inc.
DMD, Duchenne Muscular Dystrophy, Duchenne, Exon 44
04/27
07/27
EXPLORE44, NCT05670730: Study of AOC 1044 in Healthy Adult Volunteers and Participants with Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping

Completed
1/2
70
US
AOC 1044, Placebo, Saline
Avidity Biosciences, Inc.
Duchenne Muscular Dystrophy, Exon 44
11/24
11/24
delpacibart braxlosiran (AOC-1020) / Avidity Biosci
FORWARD, NCT07038200: A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD

Recruiting
3
200
US
AOC-1020, del-brax, Placebo, saline
Avidity Biosciences, Inc.
Facioscapulohumeral Muscular Dystrophy, FSHD, FSHD - Facioscapulohumeral Muscular Dystrophy, FSHD1, FSHD2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Facioscapulohumeral Muscular Dystrophy 1, Facioscapulohumeral Dystrophy, Facio-Scapulo-Humeral Dystrophy, Facioscapulohumeral Muscular Dystrophy 2, Facioscapulohumeral Muscular Dystrophy Type 1 (FSHD1), FSH Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Landouzy-Dejerine Syndrome
05/28
07/28
FORTITUDE-OLE, NCT06547216: Phase 2 Open-label Extension Study of AOC 1020 in Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

Enrolling by invitation
2
84
Europe, Canada, US
AOC 1020, delpacibart braxlosiran, del-brax
Avidity Biosciences, Inc.
FSHD, FSHD1, FSHD2, FMD, FMD2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Dystrophies, Facioscapulohumeral Muscular, Dystrophy, Facioscapulohumeral Muscular, Facioscapulohumeral Muscular Dystrophy 1, Facioscapulohumeral Muscular Dystrophy 2, Facio-Scapulo-Humeral Dystrophy, Atrophy, Facioscapulohumeral, Atrophies, Facioscapulohumeral, Facioscapulohumeral Atrophy, Muscular Dystrophies, Muscular Dystrophy, Facioscapulohumeral, FSH Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Dystrophies, Landouzy-Dejerine, Dystrophy, Landouzy-Dejerine, Landouzy-Dejerine Syndrome, Muscular Dystrophy, Landouzy Dejerine, Progressive Muscular Dystrophy, FSH
04/28
04/28
FORTITUDE, NCT05747924: Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

Active, not recruiting
1/2
90
Europe, Canada, US
AOC 1020, Placebo, Saline
Avidity Biosciences, Inc.
FSHD, FSHD1, FSHD2, FMD, FMD2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Dystrophies, Facioscapulohumeral Muscular, Dystrophy, Facioscapulohumeral Muscular, Facioscapulohumeral Muscular Dystrophy 1, Facioscapulohumeral Muscular Dystrophy 2, Facio-Scapulo-Humeral Dystrophy, Atrophy, Facioscapulohumeral, Atrophies, Facioscapulohumeral, Facioscapulohumeral Atrophy, Muscular Dystrophies, Muscular Dystrophy, Facioscapulohumeral, FSH Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Dystrophies, Landouzy-Dejerine, Dystrophy, Landouzy-Dejerine, Landouzy-Dejerine Syndrome, Muscular Dystrophy, Landouzy Dejerine, Progressive Muscular Dystrophy, FSH
10/26
04/27
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
delpacibart etedesiran (AOC 1001) / Avidity Biosci
HARBOR, NCT06411288: Global Study of Del-desiran for the Treatment of DM1

Recruiting
3
150
Europe, Canada, Japan, US
AOC 1001 (del-desiran), Placebo
Avidity Biosciences, Inc.
DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonia, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Steinert, Myotonic Muscular Dystrophy
10/26
04/27
HARBOR-OLE, NCT07008469: Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

Not yet recruiting
3
217
NA
Del-desiran (AOC 1001)
Avidity Biosciences, Inc.
Myotonic Dystrophy Type 1, DM1, Myotonic Dystrophy, Myotonia, Myotonic Dystrophy 1, Myotonic Disorders, Steinert Myotonic Dystrophy, Steinert's Disease
08/30
10/30
MARINA-OLE, NCT05479981: Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients

Active, not recruiting
2
37
US
Placebo, Saline, AOC 1001
Avidity Biosciences, Inc.
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
06/27
06/27
MARINA, NCT05027269: Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients

Completed
1/2
38
US
AOC 1001, Placebo
Avidity Biosciences, Inc.
DM1, Myotonic Dystrophy 1, Myotonic Dystrophy, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Myotonic Muscular Dystrophy
02/23
02/23
delpacibart zotadirsen (AOC-1044) / Eli Lilly
EXPLORE44OLE, NCT06244082: Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping

Active, not recruiting
2
39
US
AOC 1044
Avidity Biosciences, Inc.
DMD, Duchenne Muscular Dystrophy, Duchenne, Exon 44
04/27
07/27
EXPLORE44, NCT05670730: Study of AOC 1044 in Healthy Adult Volunteers and Participants with Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping

Completed
1/2
70
US
AOC 1044, Placebo, Saline
Avidity Biosciences, Inc.
Duchenne Muscular Dystrophy, Exon 44
11/24
11/24
delpacibart braxlosiran (AOC-1020) / Avidity Biosci
FORWARD, NCT07038200: A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD

Recruiting
3
200
US
AOC-1020, del-brax, Placebo, saline
Avidity Biosciences, Inc.
Facioscapulohumeral Muscular Dystrophy, FSHD, FSHD - Facioscapulohumeral Muscular Dystrophy, FSHD1, FSHD2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Facioscapulohumeral Muscular Dystrophy 1, Facioscapulohumeral Dystrophy, Facio-Scapulo-Humeral Dystrophy, Facioscapulohumeral Muscular Dystrophy 2, Facioscapulohumeral Muscular Dystrophy Type 1 (FSHD1), FSH Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Landouzy-Dejerine Syndrome
05/28
07/28
FORTITUDE-OLE, NCT06547216: Phase 2 Open-label Extension Study of AOC 1020 in Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

Enrolling by invitation
2
84
Europe, Canada, US
AOC 1020, delpacibart braxlosiran, del-brax
Avidity Biosciences, Inc.
FSHD, FSHD1, FSHD2, FMD, FMD2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Dystrophies, Facioscapulohumeral Muscular, Dystrophy, Facioscapulohumeral Muscular, Facioscapulohumeral Muscular Dystrophy 1, Facioscapulohumeral Muscular Dystrophy 2, Facio-Scapulo-Humeral Dystrophy, Atrophy, Facioscapulohumeral, Atrophies, Facioscapulohumeral, Facioscapulohumeral Atrophy, Muscular Dystrophies, Muscular Dystrophy, Facioscapulohumeral, FSH Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Dystrophies, Landouzy-Dejerine, Dystrophy, Landouzy-Dejerine, Landouzy-Dejerine Syndrome, Muscular Dystrophy, Landouzy Dejerine, Progressive Muscular Dystrophy, FSH
04/28
04/28
FORTITUDE, NCT05747924: Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

Active, not recruiting
1/2
90
Europe, Canada, US
AOC 1020, Placebo, Saline
Avidity Biosciences, Inc.
FSHD, FSHD1, FSHD2, FMD, FMD2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Dystrophies, Facioscapulohumeral Muscular, Dystrophy, Facioscapulohumeral Muscular, Facioscapulohumeral Muscular Dystrophy 1, Facioscapulohumeral Muscular Dystrophy 2, Facio-Scapulo-Humeral Dystrophy, Atrophy, Facioscapulohumeral, Atrophies, Facioscapulohumeral, Facioscapulohumeral Atrophy, Muscular Dystrophies, Muscular Dystrophy, Facioscapulohumeral, FSH Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Dystrophies, Landouzy-Dejerine, Dystrophy, Landouzy-Dejerine, Landouzy-Dejerine Syndrome, Muscular Dystrophy, Landouzy Dejerine, Progressive Muscular Dystrophy, FSH
10/26
04/27

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