This is a Phase 1 clinical trial designed to evaluate the safety and tolerability of amlodipine, a calcium channel blocker, in adults with Myotonic Dystrophy Type 1 (DM1). Amlodipine is being studied to see if it can improve muscle strength, reduce stiffness (myotonia), and improve function by modifying calcium flow in muscle cells. All participants will receive amlodipine starting at 2.5 mg daily for 2 weeks, then 5 mg for 4 weeks. After that, participants will be randomly assigned to continue on 5 mg or increase to 10 mg for an additional 4 weeks. The main goals are to assess changes in blood pressure and any adverse events to determine whether the drug is safe in this population. The study will also explore how amlodipine affects muscle strength, mobility, fatigue, and daily function using clinical tests and questionnaires. Findings will inform a future phase 2 trial.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Evaluating 2 different doses of amlodipine, after an initial titration phase (2.5 mg daily), amlodipine is increased to the target doses of 5 mg and 10 mg
Number of Subjects Who Provided Informed Consent
The total number of participants who signed the informed consent document and were enrolled in the study.
Time frame: Baseline
Number of Completed Study Visits
The total number of completed study visits, averaged across all enrolled participants. A visit is considered completed if all required procedures for that visit were performed.
Time frame: Baseline to End of Study at week 10
Number of Subjects Who Completed the Study Without Drug Discontinuation
Number of participants who completed all study visits and remained on study drug throughout the study period without permanent discontinuation due to adverse events, withdrawal, or protocol deviations.
Time frame: Baseline to End of Study at week 10
Number of Subjects Who Completed the Study
Number of participants who completed all required study visits and procedures through the final study visit.
Time frame: Baseline to End of Study at week 10
Mean Change in Systolic Blood Pressure
Systolic blood pressure (SBP) will be measured using a standardized automated sphygmomanometer in a seated position after 5 minutes of rest. The mean change will be calculated as the difference between SBP at the final study visit and baseline
Time frame: Baseline to End of Study at week 10
Number of Participants With Non-Serious Adverse Events
Number of participants who experienced at least one non-serious adverse event, as determined by investigator assessment and participant report. Events will be recorded according to MedDRA coding guidelines.
Time frame: Baseline to End of Study at week 10
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