Myotonic Dystrophy 1Phase 1CAP DM1NCT07075965
Calcium Channel Blocker in Myotonic Dystrophy Type 1
A Phase 1 study testing Amlodipine in people with Myotonic Dystrophy 1, run by University of Rochester.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Myotonic Dystrophy 1
- What is being tested?
- Amlodipine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- No sites listed yet
- Study dates
- 7 Dec 2026to 1 Oct 2030
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
- Ambulatory and able to walk 10 meters.
- Willingness to discontinue anti-myotonia drugs at least 2 weeks prior to screening.
- Highly effective method of contraception in women with childbearing potential.
Who cannot join
- 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.
- Treatment with concomitant medications with potential interactions with amlodipine, these may include but are not limited to sildenafil, cyclosporin, atorvastatin at high dosages (80 mg daily), simvastatin (at dosages higher than 20 mg daily) or strong inhibitors of CYP3A4.
- A history of syncope.
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- A history of symptomatic hypotension.
- Initiation or change in doses of concomitant medications, including herbal supplements, if in the opinion of the Investigator, may impact the results of the study.
- Women of childbearing potential must have a negative pregnancy test, cannot be planning a pregnancy, and cannot be breastfeeding at any time during the study.
- Known history of substance and/or alcohol abuse within one year prior to screening.
- The presence of comorbidities that, in the opinion of the Investigator, may influence study results, including, but not limited to, uncontrolled diabetes, generalized or mononeuropathy of the upper extremities, or cervical radiculopathy resulting in weakness and atrophy.
- Concurrent treatment with calcium channel blocker.
- Known ischemic or non-ischemic moderate or severe heart failure as defined by symptoms suggestive of heart failure or reduced left ventricular ejection fraction (LVEF) on echocardiogram < 55%.
- Moderate or severe aortic stenosis or other obstruction of the left ventricle outflow tract.
- Known sensitivity or allergy to amlodipine.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Calcium Channel Blocker in Myotonic Dystrophy Type 1 (CAP DM1)
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.
- Conditions
- Myotonic Dystrophy 1
- Being tested
- Amlodipine
- Medical subject headings
- Myotonic Dystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AmlodipineDrug
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
| Study group | Role | What they receive |
|---|---|---|
| Cohort A | Experimental | Amlodipine 5 mg Receives: Drug: Amlodipine |
| Cohort B | Experimental | Amlodipine 10 mg Receives: Drug: Amlodipine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Subjects Who Provided Informed Consent
The total number of participants who signed the informed consent document and were enrolled in the study.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Baseline to End of Study at week 10
Secondary outcomes (2)
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
Measured: 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.
Measured: Baseline to End of Study at week 10
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jul 2025
- Study start7 Dec 2026 (estimated)
- Primary completion1 Oct 2030 (estimated)
- Study completion1 Oct 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myotonic Dystrophy 1Studies of the same condition, open and under way.
- More trials run by University of RochesterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Rochester
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Johanna Hamel, University of Rochester
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07075965
- Sponsor's protocol ID
- STUDY00010646
- Acronym
- CAP DM1
- Status last verified
- Dec 2025
- First submitted
- 10 Jul 2025
- Last updated
- 9 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Calcium Channel Blocker in Myotonic Dystrophy Type 1 (CAP DM1)
- ClinicalTrials.gov ID
- NCT07075965
- Acronym / sponsor ID
- CAP DM1 / STUDY00010646
- Conditions
- Myotonic Dystrophy 1
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- University of RochesterUniversity, hospital or other
- Study start
- 7 Dec 2026 (estimated)
- Primary completion
- 1 Oct 2030 (estimated)
- Study completion
- 1 Oct 2030 (estimated)
- First posted
- 20 Jul 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort A | Experimental | Drug: AmlodipineAmlodipine 5 mg |
| Cohort B | Experimental | Drug: AmlodipineAmlodipine 10 mg |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
- Ambulatory and able to walk 10 meters.
Key exclusion criteria (15 listed)
- 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.
- Treatment with concomitant medications with potential interactions with amlodipine, these may include but are not limited to sildenafil, cyclosporin, atorvastatin at high dosages (80 mg daily), simvastatin (at dosages higher than 20 mg daily) or strong inhibitors of CYP3A4.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AmlodipineEvaluating 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 | Drug | Cohort A, Cohort B |
Primary outcome
Number of Subjects Who Provided Informed Consent
Time frame: Baseline
The total number of participants who signed the informed consent document and were enrolled in the study.
Number of Completed Study Visits
Time frame: Baseline to End of Study at week 10
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.
Number of Subjects Who Completed the Study Without Drug Discontinuation
Time frame: Baseline to End of Study at week 10
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.
Number of Subjects Who Completed the Study
Time frame: Baseline to End of Study at week 10
Number of participants who completed all required study visits and procedures through the final study visit.
2 secondary outcomes
Mean Change in Systolic Blood Pressure
Time frame: Baseline to End of Study at week 10
Number of Participants With Non-Serious Adverse Events
Time frame: Baseline to End of Study at week 10
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Dec 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Amlodipine in people with Myotonic Dystrophy 1, run by University of Rochester.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 9 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jeanne M Dekdebrun, MS (585-276-4611, Jeanne_Dekdebrun@URMC.Rochester.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Subjects Who Provided Informed Consent (time frame: Baseline). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Dec 2025.