Clinical trials: Myotonic Dystrophy 1
Myotonic Dystrophy, Congenital, Myotonic Dystrophy, Type 1 (DM1)
Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)
The goal of this interventional study is to demonstrate the feasibility and tolerability of music and movement intervention for children with congenital DM1, while providing indications of its effectiveness in improving brain and…
- Conditions
- Myotonic Dystrophy, Congenital, Myotonic Dystrophy, Type 1 (DM1), Myotonic Dystrophy Type 1, Myotonic Dystrophy Type 1 (DM1)
- Trial name
- NCT06809049
- Testing
- Music and movement education
- Where
- Canada
- Volunteers
- 13 people
- Sponsor
- Hanns Lochmuller
Diastolic Dysfunction
Diastolic Function in Myotonic Dystrophy Type 1
Myotonic dystrophy type 1 (DM1) is a neuromuscular disorder in relation with an unstable expansion of CTG repeat. Patients with DM1 are at risk of arrhythmia and conduction disorders.
- Trial name
- NCT06029192
- Where
- France
- Volunteers
- 500 people
- Sponsor
- Centre d'Investigation Clinique et Technologique 805
Myotonic Dystrophy, Duchenne Muscular Dystrophy
Biomarker Development for Muscular Dystrophies
Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies.
- Conditions
- Myotonic Dystrophy, Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy
- Trial name
- NCT05019625
- Where
- 5 sites in United States
- Volunteers
- 465 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
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
Breast Cancer
Tolerability and Efficacy of Adjuvant T-DM1 in Patients with HER2 Positive Breast Cancer After Incomplete Pathological Response to Neoadjuvant Chemotherapy Including Anti-HER2 Agents.
Multicenter, retrospective-prospective, real-world observational study, with the aim of evaluating tolerability and efficacy in a population of patients treated according to clinical practice outside of studies randomized.
- Trial name
- ATD (NCT06272799)
- Testing
- T-DM1 adjuvant
- Where
- Italy
- Volunteers
- 160 people
- Sponsor
- Regina Elena Cancer Institute
Myotonic Dystrophy Type 1, Congenital DM1
Remote Assessments and Genetic Determinants of Congenital and Childhood Myotonic Dystrophy
Myotonic dystrophy type 1 (DM1) can affect people in many different ways, even in the same family. The symptoms that children experience can be different and more severe than adults.
- Conditions
- Myotonic Dystrophy Type 1, Congenital DM1, Juvenile DM1, Childhood DM1
- Trial name
- REACH-DM KIDS (NCT07630389)
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- University of Rochester
HER2-positive Breast Cancer
Adjuvant Therapy Choice for Non-pCR HER2 Positive Early Breast Cancer After Neoadjuvant Therapy
Chemotherapy combined with trastuzumab and patuzumab is the standard neoadjuvant therapy for HER2 positive breast cancer patients.
- Trial name
- nonPHER (NCT06693037)
- Where
- China
- Volunteers
- 2,092 people
- Sponsor
- Shu Wang
Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophy
Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry
Myotonic dystrophy (DM) and facioscapulohumeral muscular dystrophy (FSHD) are inherited disorders characterized by progressive muscle weakness and loss of muscle tissue.
- Conditions
- Myotonic Dystrophy, Facioscapulohumeral Muscular Dystrophy, Muscular Dystrophy, Myotonic Dystrophy Type 1
- Trial name
- NCT00082108
- Where
- United States
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- University of Rochester
Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy
Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension
This is a natural history study to improve the types of assessments and biological samples that will be used in clinical drug trials in both congenital myotonic dystrophy and childhood myotonic dystrophy.
- Conditions
- Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy, Myotonic Dystrophy
- Trial name
- TREAT-EXT (NCT06747884)
- Where
- 4 sites in United States, Brazil
- Volunteers
- 200 people
- Sponsor
- Virginia Commonwealth University
Myotonic Dystrophy
The United Kingdom National Registry for Myotonic Dystrophy
Myotonic dystrophy (dystrophia myotonica - DM) exists in two forms, usually referred to as DM1 (type 1) and DM2 (type 2). Both conditions are genetic disorders but each affects a different gene.
- Trial name
- NCT04003363
- Testing
- Patient Registry
- Where
- United Kingdom
- Volunteers
- 900 people
- Sponsor
- Newcastle University
Congenital Myotonic Dystrophy, CDM
DMCRN-02-001: Assessing Pediatric Endpoints in DM1
The overall goal of the study is to establish valid clinical endpoint assessments for children with congenital myotonic dystrophy type 1 and develop biomarkers for the condition.
- Trial name
- ASPIRE-DM1 (NCT05224778)
- Where
- 5 sites in United States, Italy
- Volunteers
- 50 people
- Sponsor
- Virginia Commonwealth University
Myotonic Dystrophy 1, DM1
The Spanish National Registry for Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1 (DM1) is a rare genetic neuromuscular condition that can affect multiple organs and varies widely in how it presents.
- Conditions
- Myotonic Dystrophy 1, DM1, Myotonic Dystrophy Type 1, Myotonic Dystrophy, Congenital
- Trial name
- DM1-Hub (NCT07385443)
- Testing
- Patient Registry
- Where
- 8 sites in Spain
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Fundació Institut Germans Trias i Pujol
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 1, Huntington Disease
NIPD on cffDNA for Triplet Repeat Diseases
The purprose of this study is to develop and validate an analytical NIPD test for triplet repeat disesases by NGS analysis from maternal blood, searching for the familial mutation in families at risk of having one of the…
- Conditions
- Myotonic Dystrophy 1, Huntington Disease, Fragile X Syndrome
- Trial name
- NCT04698551
- Testing
- Non invasive prenatal diagnosis
- Where
- France
- Volunteers
- 36 people
- Sponsor
- University Hospital, Montpellier
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
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
Myotonic Dystrophy Type 1 (DM1)
A Registered Observational Cohort Study of Myotonic Dystrophy Type 1
Myotonic dystrophy type 1 (DM1) is the most common form of muscular dystrophy.There is little phenotype and genetic data for Chinese DM1 patients.
- Trial name
- NCT06979024
- Testing
- Triplet-primed PCR or Long-read sequencing
- Where
- China
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- First Affiliated Hospital of Fujian Medical University
LGMD1B, LGMD1C
Long-Term Development of Muscular Dystrophy Outcome Assessments
This is a 24-month, observational study of up to 1000 participants with Limb Girdle Muscular Dystrophy (LGMD), Myotonic Dystrophy Type 2 (DM2), and late onset Pompe disease (LOPD).
- Conditions
- LGMD1B, LGMD1C, LGMD1D, LGMD1E
- Trial name
- GRASP-01-005 (NCT05989620)
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Virginia Commonwealth University
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 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
- 43 sites in United States, Australia and 9 more
- Volunteers
- 150 people
- Sponsor
- Dyne Therapeutics