Myotonic dystrophy (DM)
A natural history investigating clinical outcomes and quality of life in children with Myotonic Dystrophy (ANIQ)
Protocol ID: 78905
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: Sarah Ismail, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Natural history study of congenital and childhood DM1 in participants ages 0-24
Status: Recruiting soon
Stanford outcome validation with functional measures in myotonic dystrophy (MYOCAP/Actimyo)
Protocol ID: 62522
NCT: N/A
PI: Dr. Tina Duong
Study Coordinator: Sarah Ismail, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Outcome validation with functional measures in myotonic dystrophy
Status: Recruiting
Stanford study of central nervous system involvement in DM1 (DDC)
Protocol ID: 74320
NCT: N/A
PI: Dr. John W. Day
Study Coordinator: DIGBI, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Purpose: Characterization of neurological symptoms and biomarkers of DM1
Status: Recruiting
Recruitment Notes: Adults with DM1 and healthy individuals
Global Study of Del-desiran for the Treatment of DM1 (HARBOR)
Protocol ID: 74759
NCT06411288
PI: Dr. John W. Day
Study Coordinator: Rabia Farooquee, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avidity Biosciences
Purpose: Assessing the safety and efficacy of Delpacibart etedesiran in people with DM1
Status: Active, no longer recruiting
A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1 (HARBOR OLE)
Protocol ID: 79538
NCT07008469
PI: Dr. John W. Day
Study Coordinator: Natalia Wieczorek, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Avidity Biosciences
Purpose: Assessing the long-term safety and efficacy of Delpacibart etedesiran in people with DM1
Status: Recruiting by invitation only
Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
Protocol ID: 65435
NCT: NCT03981575
PI: Dr. Jacinda Sampson
Study Coordinator: Sarah Ismail, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Investigator Initiated
Status: Active, no longer recruiting
A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple-Ascending Dose Study of PGN-EDODM1 in Adult Participants with Myotonic Dystrophy Type 1 (FREEDOM2-DM1)
Protocol ID: 75989
NCT06667453
PI: Dr. Jacinda Sampson
Study Coordinator: Wendy Ao, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: PepGen Inc.
Purpose: Assessing the safety and efficacy of PGN-EDODM1 in adults with DM1
Status: On Hold
Recruitment Notes: Participants must have a diagnosis of DM1 and be 16-60 years old
A Phase 1/2, Randomized, Double-blind, Placebo‑controlled Single- and Multiple‑dose Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VX‑670 in Adult Subjects with Myotonic Dystrophy Type 1 (GALILEO)
Protocol ID: 73893
NCT06185764
PI: Dr. Jacinda Sampson
Study Coordinator: Sonia Pathak, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Vertex Pharmaceuticals
Purpose: Evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses
Status: Active, no longer recruiting
Recruitment Notes: Participants must have a diagnosis of DM1 and be 18-60 years old
An Open-label Extension Study Evaluating the Long-term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects with Myotonic Dystrophy Type I (GALILEO OLE)
Protocol ID: 81692
NCT: TBD
PI: Dr. Jacinda Sampson
Study Coordinator: Sonia Pathak, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Vertex Pharmaceuticals
Purpose: Evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses
Status: Recruiting soon by invitation only
A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents with Congenital Myotonic Dystrophy (REACH CDM)
Protocol ID: 61610
NCT03692312
PI: Dr. John W. Day
Study Coordinator: Susan Thomas, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: AMO Pharmaceuticals
Purpose: Long term follow up study determining safety and efficacy of Tideglusib in congenital DM1
Status: Active, no longer recruiting
A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants with Myotonic Dystrophy Type 1 (ACHIEVE)
Protocol ID: 83950
NCT05481879
PI: Dr. John W. Day
Study Coordinator: Sean Doker, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Dyne Therapeutics
Purpose: To evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1).
Status: Active, no longer recruiting
A Phase 3, Randomized, Double-Blind, 48-Week Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of DYNE-101 Administered to Participants with Myotonic Dystrophy Type 1 (HARMONIA)
Protocol ID: 84048
NCT07486934
PI: Dr. Jacinda Sampson
Study Coordinator: Anita Chacko, NeuromuscularResearch@stanford.edu, 650-725-4341
Sponsor: Dyne Therapeutics, Inc
Purpose: To assess the efficacy, safety, and tolerability of DYNE-101 for the treatment of myotonic dystrophy 1 (DM1).
Status: Recruiting by invitation only
