Myotonic Dystrophy 1 Clinical Trial
Official title:
Open Label Trial of Ranolazine in Myotonia Congenita, Paramyotonia Congenita, & Myotonic Dystrophy Type 1
The purpose of this study is to gather preliminary data to determine if ranolazine is a safe and effective treatment for the symptoms of myotonia congenital, paramyotonia congenita, and myotonic dystrophy type 1. The duration of the study is 5 weeks.
Recent advances in the understanding of myotonia congenita have identified potential areas that could possibly respond to treatment in a drug study. The drug ranolazine (trade name Ranexa) is a FDA-approved medication to treat chest pain in patients with heart disease. Ranolazine has been studied in mice with myotonia congenita. The data from this animal model suggest that ranolazine may improve the symptoms and signs of myotonia. All individuals that participate will be placed on active drug. The investigators want to see if this drug is safe to take without causing too many side effects for people with myotonia congenita, paramyotonia congenital and myotonic dystrophy type 1. Participants will go to The Ohio State University for study visits. Participants will take ranolazine for four weeks. Participants can expect a total of 4 study visits and 2 phone calls over the 5 week period. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
NCT02880735 -
Ventilatory Response After Non Invasive Ventilation in Type 1 Myotonic Dystrophy
|
N/A | |
| Recruiting |
NCT06378216 -
Myotonic Dystrophy Type 1 Congenital and Juvenile Form: From Diagnosis to Rehabilitation [MDCJ-NeuBeRe]
|
||
| Active, not recruiting |
NCT06089018 -
Observational Study of Digital Biomarkers of Myotonia and Gait in Adults and Children With Myotonic Dystrophy
|
||
| Completed |
NCT05662150 -
Low-frequency Repetitive Nerve Stimulation in Myotonic Dystrophy Type 1
|
N/A | |
| Active, not recruiting |
NCT04698551 -
NIPD on cffDNA for Triplet Repeat Diseases
|
||
| Completed |
NCT05027269 -
Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
|
Phase 1/Phase 2 | |
| Completed |
NCT04712422 -
Poor Neck Proprioception May Cause Balance Deficits in Myotonic Dystrophy 1
|
||
| Recruiting |
NCT05006924 -
Symptoms and Outcome Measures for Upper- Limb Function in Myotonic Dystrophy Type 1
|
||
| Recruiting |
NCT04656210 -
Myotonic Dystrophy - Vascular and Cognition
|
||
| Completed |
NCT04634682 -
Effect of MYODM on Quality of Life, Fatigue and Hypersomnia in Patients With Myotonic Dystrophy Type 1
|
N/A | |
| Withdrawn |
NCT06270186 -
Evaluation of Cognitive Functions in 20 Patients With Type 1 Myotonic Dystrophy With Virtual Reality Approach
|
N/A | |
| Not yet recruiting |
NCT06300307 -
Study of ATX-01 in Participants With DM1
|
Phase 1/Phase 2 | |
| Completed |
NCT02729597 -
Tracking the Brain in Myotonic Dystrophies: a 5-year Longitudinal Follow-up Study
|
N/A | |
| Recruiting |
NCT06138743 -
Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy
|
Phase 1 | |
| Not yet recruiting |
NCT05532813 -
Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease)
|
Phase 3 | |
| Active, not recruiting |
NCT04886518 -
Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1
|
Phase 2 | |
| Not yet recruiting |
NCT05865483 -
Profile of Dysphagia in Myotonic Dystrophy Type 1 (DM1)
|
||
| Completed |
NCT05036447 -
Myotonic Dystrophy Type 1 and Resistance Exercise
|
N/A | |
| Recruiting |
NCT03424460 -
Venous Thromboembolism in Myotonic Dystrophy Type 1
|
N/A | |
| Completed |
NCT04001920 -
Effects of a Multiple Component Training Program on Muscles in Adults With Myotonic Dystrophy Type 1
|
N/A |