Myotonic Dystrophy 2DM2NCT06716931
Investigating Exercise in Myotonic Dystrophy Type 2 (DM2)
A study testing Exercise in people with Myotonic Dystrophy 2, run by Massachusetts General Hospital at 1 site in United States.
Trial summary
An exercise regimen (PRIME: Proximal Resistance In-House Movement Exercise) has been designed for patients with myotonic dystrophy type 2 (DM2). The hypothesis is that this patient-friendly physical therapist (PT)-guided exercise program associates with improved functional capacity and muscle composition in DM2 in… Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Myotonic Dystrophy 2
- What is being tested?
- Exercise
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- 4 Mar 2025to 1 Mar 2028
- Phase
- Interventional
- 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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with genetically confirmed myotonic dystrophy 2 (DM2) who are ≥18 and ≤ 70 years old.
- Patients are ambulatory without need of any assistance or device.
- Patients have a normal ECG within the 3 months prior to Screening visit (of note, patients with DM2 are recommended to have routine ECG as part of patient care. So, this ECG will be done as part of patient care).
- Body mass index (BMI) ≥20 and ≤30.
Who cannot join
- Subjects younger than 18 years old or older than 70 years old.
- Inability or unwillingness of the subject to give written informed consent.
- Subjects with pacemaker.
- Blood pressure <90/50 mmHg or >160 mmHg/90 mmHg at Screening visit
- Resting heart rate of <60 or >100 beats/minute at Screening visit.
- Any fall within the last six months.
- Any history of syncopal episodes or family history of sudden death.
- Patients who are pregnant or plan to become pregnant. A urine pregnancy test will be performed at visit 1 and 3 for women of childbearing age. If positive or if subject is planning to become pregnant, subject will be not eligible for the study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Neuromuscular Diagnostic Center. Massachusetts General Hospital
Boston, Massachusetts, 02114
Blanca Escauriaza, RN, 617-724-5297, bescauriaza@mgh.harvard.edu
Blanca Escauriaza, RN, 617-726-3642, bescauriaza@mgh.harvard.edu
About this study
Official title: Investigating Benefits of A Physical Therapist (PT)-Guided Exercise Program in Myotonic Dystrophy Type 2 (DM2)
An exercise regimen (PRIME: Proximal Resistance In-House Movement Exercise) has been designed for patients with myotonic dystrophy type 2 (DM2). The hypothesis is that this patient-friendly physical therapist (PT)-guided exercise program associates with improved functional capacity and muscle composition in DM2 in this two-period two-sequence cross-over study. Thus, participant will be randomized to one of the three possible groups. Participants in GROUP A will perform exercise routine virtually under the direct supervision of a physical therapist once a week and another session on their own for the first three months, then they will continue with same exercise routine at home for the last 3 months on their own. Participants in GROUP B will perform exercise routine virtually under the direct supervision of a physical therapist twice a week for the first three months, then they will continue with same exercise routine at home for the last 3 months on their own. Participants in GROUP C will perform exercise routine on their own during the first 3 months, then they will perform exercise routine virtually under the direct supervision of a physical therapist. Each group will include around 8 participants. Duration of the study is 6 months. In addition to exercise sessions, participants will have evaluation of their strength, motor function and muscle composition at three time points: initiation, 3 months and completion of the study at 6 months. Muscle composition will be assessed by electrical impedance myography which is a portable, non-invasive, painless and non-radiation tool that applies a weak high multifrequency electrical current to the examined muscle and allows to obtain information about its composition.
- Conditions
- Myotonic Dystrophy 2
- Being tested
- Exercise
- Keywords
- exercise, physical therapist, myotonic dystrophy type 2, electrical impedance myography
- Medical subject headings
- Myotonic Dystrophy, Motor Activity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ExerciseOther
A patient-friendly exercise routine has been designed for patients with myotonic dystrophy type 2. Benefits of this exercise regimen in different settings (at home versus clinic, self-directed versus physical therapist-directed) will be investigated.
In person exercise supervised by physical therapist and then self-directed exercise in Group A
ExerciseOther
Exercise virtually supervised by physical therapist and then self-directed for group B
ExerciseOther
Self-directed and virtually supervised exercise by physical therapist for Group C
| Study group | Role | What they receive |
|---|---|---|
| Group A | Active comparator | Exercise under the supervision of physical therapist in clinic during the first 3 months, then self-directed exercise routine for the last 3 months Receives: Other: Exercise |
| Group B | Active comparator | Exercise under the supervision of physical therapist virtually during the first 3 months, then self-directed exercise routine for the last 3 months Receives: Other: Exercise |
| Group C | Active comparator | Self-directed exercise routine for the first 3 months, then exercise routine under the supervision of physical therapist virtually during the last 3 months Receives: Other: Exercise |
What is being measured
The primary outcome is the main question the study is designed to answer.
Manual Muscle Test (MMT)
Score of manual muscle strength test scale. This is a strength exam at bedside of muscle groups
Measured: From enrollment to the end of treatment at 6 months
Six-minute walking test
Distance in meters that subject can walk in six minutes
Measured: From enrollment to the end of treatment at 6 months
Secondary outcomes (2)
Quantitative myometry
Muscle dynamometry of different muscle groups measured in kilograms
Measured: From enrollment to the end of treatment at 6 months
Muscle resistance/reactance measures in ohms
using electrical impedance myography, resistance and reactance measures in ohms will be obtained simultaneously from each examined muscle
Measured: From enrollment to the end of treatment at 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Dec 2024
- Study start4 Mar 2025
- Primary completion1 Dec 2027 (estimated)
- Study completion1 Mar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myotonic Dystrophy 2Studies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe 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 N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Paloma Gonzalez Perez, MD, PhD, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06716931
- Sponsor's protocol ID
- 2024P002567
- Acronym
- DM2
- Status last verified
- Oct 2026
- First submitted
- 30 Oct 2024
- Last updated
- 8 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Investigating Benefits of A Physical Therapist (PT)-Guided Exercise Program in Myotonic Dystrophy Type 2 (DM2)
- ClinicalTrials.gov ID
- NCT06716931
- Acronym / sponsor ID
- DM2 / 2024P002567
- Conditions
- Myotonic Dystrophy 2
- Keywords
- exercise, physical therapist, myotonic dystrophy type 2, electrical impedance myography
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Study start
- 4 Mar 2025 (actual)
- Primary completion
- 1 Dec 2027 (estimated)
- Study completion
- 1 Mar 2028 (estimated)
- First posted
- 4 Dec 2024 (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.
An exercise regimen (PRIME: Proximal Resistance In-House Movement Exercise) has been designed for patients with myotonic dystrophy type 2 (DM2). The hypothesis is that this patient-friendly physical therapist (PT)-guided exercise program associates with improved functional capacity and muscle composition in DM2 in this two-period two-sequence cross-over study. Thus, participant will be randomized to one of the three possible groups. Participants in GROUP A will perform exercise routine virtually under the direct supervision of a physical therapist once a week and another session on their own for the first three months, then they will continue with same exercise routine at home for the last 3 months on their own. Participants in GROUP B will perform exercise routine virtually under the direct supervision of a physical therapist twice a week for the first three months, then they will continue with same exercise routine at home for the last 3 months on their own. Participants in GROUP C will perform exercise routine on their own during the first 3 months, then they will perform exercise routine virtually under the direct supervision of a physical therapist. Each group will include around 8 participants. Duration of the study is 6 months. In addition to exercise sessions, participants will have evaluation of their strength, motor function and muscle composition at three time points: initiation, 3 months and completion of the study at 6 months. Muscle composition will be assessed by electrical impedance myography which is a portable, non-invasive, painless and non-radiation tool that applies a weak high multifrequency electrical current to the examined muscle and allows to obtain information about its composition.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Group A | Active comparator | Other: ExerciseExercise under the supervision of physical therapist in clinic during the first 3 months, then self-directed exercise routine for the last 3 months |
| Group B | Active comparator | Other: ExerciseExercise under the supervision of physical therapist virtually during the first 3 months, then self-directed exercise routine for the last 3 months |
| Group C | Active comparator | Other: ExerciseSelf-directed exercise routine for the first 3 months, then exercise routine under the supervision of physical therapist virtually during the last 3 months |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Patients with genetically confirmed myotonic dystrophy 2 (DM2) who are ≥18 and ≤ 70 years old.
- Patients are ambulatory without need of any assistance or device.
- Patients have a normal ECG within the 3 months prior to Screening visit (of note, patients with DM2 are recommended to have routine ECG as part of patient care. So, this ECG will be done as part of patient care).
- Body mass index (BMI) ≥20 and ≤30.
Key exclusion criteria (8 listed)
- Subjects younger than 18 years old or older than 70 years old.
- Inability or unwillingness of the subject to give written informed consent.
- Subjects with pacemaker.
- Blood pressure <90/50 mmHg or >160 mmHg/90 mmHg at Screening visit
- Resting heart rate of <60 or >100 beats/minute at Screening visit.
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 |
|---|---|---|
| ExerciseA patient-friendly exercise routine has been designed for patients with myotonic dystrophy type 2. Benefits of this exercise regimen in different settings (at home versus clinic, self-directed versus physical therapist-directed) will be investigated. In person exercise supervised by physical therapist and then self-directed exercise in Group A | Other | Group A |
| ExerciseExercise virtually supervised by physical therapist and then self-directed for group B | Other | Group B |
| ExerciseSelf-directed and virtually supervised exercise by physical therapist for Group C | Other | Group C |
Primary outcome
Manual Muscle Test (MMT)
Time frame: From enrollment to the end of treatment at 6 months
Score of manual muscle strength test scale. This is a strength exam at bedside of muscle groups
Six-minute walking test
Time frame: From enrollment to the end of treatment at 6 months
Distance in meters that subject can walk in six minutes
2 secondary outcomes
Quantitative myometry
Time frame: From enrollment to the end of treatment at 6 months
Muscle resistance/reactance measures in ohms
Time frame: From enrollment to the end of treatment at 6 months
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 8 Oct 2026.
- Individual participant dataSharing planned: Undecided
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Exercise in people with Myotonic Dystrophy 2, run by Massachusetts General Hospital at 1 site in United States.
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 4 inclusion and 8 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: looking for participants now, as of the sponsor's last update on 8 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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.
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 Blanca Escauriaza, RN (617-726-3642, bescauriaza@mgh.harvard.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Manual Muscle Test (MMT) (time frame: From enrollment to the end of treatment at 6 months). The study lists 2 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 8 Oct 2026.