Clinical trials: DM1
Myotonic Dystrophy 1
Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy
The purpose of this study is to determine whether Tideglusib is safe and efficacious in the treatment of adolescents and adults with congenital and juvenile-onset Myotonic Dystrophy.
- Trial name
- NCT02858908
- Testing
- Tideglusib
- Where
- United Kingdom
- Volunteers
- 16 people
- Sponsor
- AMO Pharma Limited
Myotonic Dystrophy 1
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared…
- Trial name
- FREEDOM2-DM1 (NCT06667453)
- Testing
- PGN-EDODM1
- Where
- 12 sites in Canada, New Zealand and 2 more
- Volunteers
- 24 people
- Sponsor
- PepGen Inc
Prostate Cancer
A Trial of MLN2704 in Subjects With Metastatic Androgen Independent Prostate Cancer
This is the first study of MLN2704 administered to humans. The purpose of the study is to determine the highest dose of MLN2704 that can be given safely to patients with prostate cancer, and to identify any side effects…
- Trial name
- NCT00052000
- Testing
- MLN2704 (DM1 conjugated monoclonal antibody MLN591)
- Where
- 2 sites in United States
- Volunteers
- 29 people
- Sponsor
- Millennium Pharmaceuticals, Inc.
Congenital Myotonic Dystrophy
Tideglusib: Expanded Access Use in Congenital Myotonic Dystrophy
This treatment plan is limited to a single patient with Congenital Myotonic Dystrophy, who is ineligible or otherwise unable to participate in ongoing clinical trials.
- Trial name
- NCT07119775
- Testing
- Tideglusib
- Sponsor
- AMO Pharma Limited
Congenital Myotonic Dystrophy
Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1).
- Trial name
- NCT03692312
- Testing
- Tideglusib
- Where
- 14 sites in United States, Australia and 3 more
- Volunteers
- 56 people
- Sponsor
- AMO Pharma Limited
Myotonic Dystrophy
The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2
A Randomized, Double-blind, Placebo-Controlled, Multi-Center Study to Investigate the Efficacy and Safety of Once Daily Mexiletine PR During 26 Weeks of Treatment in Patients with Myotonic Dystrophy Type 1 and Type 2 (HERCULES…
- Trial name
- NCT06523400
- Testing
- Mexiletine granules for prolonged-release oral suspension
- Where
- 7 sites in Belgium, Denmark and 4 more
- Volunteers
- 176 people
- Sponsor
- Lupin Ltd.
Myotonic Dystrophy
An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 Who Have Completed MEX-DM-302 Study.
This is an open-label extension study intended to evaluate the long-term safety and efficacy of mexiletine PR in patients with myotonic dystrophy type 1 and type 2 (DM1 and DM2) who have completed the parent study MEX-DM-302.
- Trial name
- NCT06549400
- Testing
- Mexiletine granules for prolonged-release oral suspension
- Where
- 6 sites in Belgium, Denmark and 4 more
- Volunteers
- 176 people
- Sponsor
- Lupin Ltd.
Myotonic Dystrophy
A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1
This is a Phase 1/Phase 2 open-label single arm, multicenter, and multinational study with SAR446268 for treatment of male and female participants 10 to 55 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1).
- Trial name
- BrAAVe (NCT06844214)
- Testing
- SAR446268
- Where
- 10 sites in United States, Argentina and 4 more
- Volunteers
- 32 people
- Sponsor
- Sanofi
DM1, Myotonic Dystrophy
Global Study of Del-desiran for the Treatment of DM1
A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy…
- Conditions
- DM1, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonia
- Trial name
- HARBOR (NCT06411288)
- Testing
- AOC 1001 (del-desiran)
- Where
- 34 sites in United States, Canada and 8 more
- Volunteers
- 159 people
- Sponsor
- Avidity Biosciences, Inc.
Congenital Myotonic Dystrophy
Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who…
- Trial name
- REACH CDM X (NCT05004129)
- Testing
- Tideglusib
- Where
- 14 sites in United States, Australia and 2 more
- Volunteers
- 76 people
- Sponsor
- AMO Pharma Limited
Myotonic Dystrophy Type 1 (DM1), DM1
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1).
- Conditions
- Myotonic Dystrophy Type 1 (DM1), DM1, Myotonic Dystrophy, Steinert Disease
- Trial name
- NCT07486934
- Testing
- zeleciment basivarsen (DYNE-101)
- Where
- 44 sites in United States, Australia and 10 more
- Volunteers
- 150 people
- Sponsor
- Dyne Therapeutics
Metastatic Breast Cancer
A Study of Trastuzumab-MCC-DM1 Administered Intravenously to Patients With HER2-Positive Metastatic Breast Cancer Who Have Previously Received a Trastuzumab-Containing Regimen
This is a phase I, multicenter, open-label, dose-escalation study of single-agent trastuzumab-MCC-DM1 administered by intravenous (IV) infusion in patients with HER2-positive metastatic breast cancer (MBC) who have previously…
- Trial name
- NCT00932373
- Testing
- trastuzumab-MCC-DM1
- Volunteers
- 54 people
- Sponsor
- Genentech, Inc.
Metastatic Breast Cancer
A Study of Trastuzumab Emtansine (Trastuzumab-MCC-DM1) Administered Intravenously to Patients With Human Epidermal Growth Factor Receptor 2 (HER2)-Positive Metastatic Breast Cancer
This was a multi-institutional, open-label, single-arm, Phase II study of trastuzumab emtansine (T-DM1) administered by intravenous (IV) infusion to patients with human epidermal growth factor receptor 2 (HER2)-positive…
- Trial name
- NCT00509769
- Testing
- Trastuzumab emtansine [Kadcyla]
- Where
- 40 sites in United States
- Volunteers
- 112 people
- Sponsor
- Genentech, Inc.
Myotonic Dystrophy Type 1, DM1
Global Open-Label Extension Study of Del-desiran for the Treatment of DM1
A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy…
- Conditions
- Myotonic Dystrophy Type 1, DM1, Myotonic Dystrophy, Myotonia
- Trial name
- HARBOR-OLE (NCT07008469)
- Testing
- Del-desiran (AOC 1001)
- Where
- 35 sites in United States, Canada and 8 more
- Volunteers
- 199 people
- Sponsor
- Avidity Biosciences, Inc.
Myotonic Dystrophy 1
An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)
The purpose of this study is to learn about the long-term safety and tolerability of PGN-EDODM1 in participants with myotonic dystrophy type 1 (DM1) who have completed a prior study with PGN-EDODM1.
- Trial name
- NCT07220603
- Testing
- PGN-EDODM1
- Where
- 3 sites in Canada
- Volunteers
- 48 people
- Sponsor
- PepGen Inc