Clinical trials: Myotonic Dystrophy 1
Myotonic Dystrophy 1
Observational Study of Digital Biomarkers of Myotonia and Gait in Adults and Children With Myotonic Dystrophy
The goal of this observational study is to assess movement in individuals with Myotonic Dystrophy Type 1 (DM1) and Type 2 (DM2) using digital biomarker tools.
- Trial name
- NCT06089018
- Where
- United States
- Volunteers
- 125 people, healthy volunteers welcome
- Sponsor
- Stanford University
Myotonic Dystrophy
Extracellular RNA Biomarkers of Myotonic Dystrophy
Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies.
- Trial name
- NCT05020002
- Where
- 3 sites in United States
- Volunteers
- 215 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Pulmonary Disease, Chronic Obstructive, Amyotrophic Lateral Sclerosis
European Home Mechanical Ventilation Registry
The European Home Mechanical Ventilation Registry (EHMVR) will enable a thorough evaluation of HMV by documenting the characteristics of HMV patients and their treatment.
- Conditions
- Pulmonary Disease, Chronic Obstructive, Amyotrophic Lateral Sclerosis, Spinal Cord Injury, Muscular Dystrophies
- Trial name
- EHMVR (NCT02315339)
- Where
- Germany
- Volunteers
- 37 people
- Sponsor
- ResMed
Myotonic Dystrophy
Open Label Study in Adolescents and Children With Myotonic Disorders
This is an open-label, multi-centre, single arm, interventional study to describe the steady-state PK, safety, and efficacy of mexiletine in paediatric patients (6 to <18 years of age) with myotonic disorders.
- Trial name
- NCT04624750
- Testing
- Mexiletine
- Where
- France
- Volunteers
- 12 people
- Sponsor
- Lupin Ltd.
Myotonic Dystrophy Type 1
Virtual Reality-training in Theory of Mind in the Childhood Form of Myotonic Dystrophy Type 1
The ability of an individual to conceive some alternative representations and to behave in a flexible manner would emerge from preschool age and drastically improve between the ages of 3 and 5 (Doebel and Zelazo, 2013).
- Trial name
- TOM-VR-DM1 (NCT05916677)
- Testing
- Virtual Reality-training in Theory of Mind, Traditional training in Theory of Mind
- Where
- France
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Institut de Myologie, France
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
Myotonic Dystrophy, Congenital Myotonic Dystrophy
Myotonic Dystrophy Family Registry
The Myotonic Dystrophy Family Registry (MDFR) is an online, patient-entered database that collects information on myotonic dystrophy (DM) to aid researchers in developing new, effective treatments and help identify participants…
- Conditions
- Myotonic Dystrophy, Congenital Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Dystrophy 2
- Trial name
- MDFR (NCT02398786)
- Testing
- Patient-entered data
- Where
- United States
- Volunteers
- 3,500 people
- Sponsor
- Myotonic Dystrophy Foundation
Non Invasive Prenatal Diagnosis
NIPD on CFTC for Triplet Repeat Diseases
The purpose of this study is to develop and validate an analytical and clinical NIPD test for triplet repeat diseases by isolated circulating fetal trophoblastic cells (CFTC) analysis from maternal blood, searching for the…
- Trial name
- DIACCIMEX (NCT03087526)
- Testing
- Non invasive prenatal diagnosis
- Where
- 8 sites in France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University Hospital, Montpellier
Myotonic Dystrophy Type 1
Children's Health Research Institute(CHRI), Stanford Lucile Packard Children Hospital (LPCH) Protocol on Myotonic Dystrophy
Study to focus on the defining and managing the neuropsychological abnormalities of myotonic dystrophy and to find out if the neuropsychological abnormalities have any correlation with changes seen on Magnetic Resonance Imaging.
- Trial name
- CHRI (NCT02269865)
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Stanford University
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
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
Type 1 Diabetes, Adolescent Development
Emoticare: Serious Game for the Adaptation to the Disease of Adolescents With Type 1 Diabetes Mellitus.
The intervention program targets adolescents with chronic Type 1 Diabetes Mellitus. Given the critical developmental stage and life transitions, necessitating robust personal resources, psychological support becomes imperative.
- Trial name
- NCT06331429
- Testing
- EmoTiCare
- Where
- Spain
- Volunteers
- 200 people
- Sponsor
- University of Valencia