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
Qualifiers
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
Treatments tested in this trial
- Amlodipine
Treatment groups
20 Participants
are divided into 2 treatment groupsLocations
Sponsors and collaborators
Source ClinicalTrials.gov