Calcium Channel Blocker in Myotonic Dystrophy Type 1
ConditionMyotonic Dystrophy 1
Trial statusNot yet recruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-65
SponsorUniversity of Rochester
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Male or Female between the ages 18 and 65, inclusive.
A genetically confirmed diagnosis of DM1, having a repeat expansion in the DMPK gene with at least 100 CTG repeats.
Grip strength less than 50% predicted based on age, height, and sex.
Video hand opening time is 4 sec or greater for at least one hand.
Disqualifiers
Congenital DM1 as defined by symptom onset in the first 4 weeks of life.
Abnormal liver function tests (LFTs): alanine aminotransferase (ALT), or aspartate aminotransferase (AST) >3 x upper limit of normal. Total bilirubin > 1.5 mg/dL, or INR > 1.3, or evidence of current active or chronic infection with hepatitis C, hepatitis B or other hepatobiliary conditions other than DM1 (or attributed to DM1) that cause abnormal liver laboratory parameters (e.g., hemochromatosis, Wilson's disease, autoimmune hepatitis)
Current or recent infection requiring antibiotic treatment within 2 weeks prior to screening.
Abnormal vital signs, including systolic blood pressure < 90 mmHg and diastolic blood pressure < 60 mmHg.
Trial design
Design model
Single group
Treatments tested in this trial
Amlodipine
DrugEvaluating 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
Treatment groups
20 Participants
are divided into 2 treatment groupsGroup A: Cohort AExperimental treatment 1 intervention
Group B: Cohort BExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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
Baseline2
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 103
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 104
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 10Secondary outcomes
1
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 102
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 10Locations
Sponsors and contacts
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Source ClinicalTrials.gov
This trial is not recruiting at the moment. You can still explore other options: