Investigating Exercise in Myotonic Dystrophy Type 2 (DM2)

NCT ID: NCT06716931

Last Updated: 2025-09-29

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: NA
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-03-04
• Study Completion Date: 2026-03-01

Brief 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 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 in clinic 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 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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Group A

Exercise under the supervision of physical therapist in clinic during the first 3 months, then self-directed exercise routine for the last 3 months

Group Type: ACTIVE_COMPARATOR

Exercise

Intervention Type: OTHER

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

Group B

Exercise under the supervision of physical therapist virtually during the first 3 months, then self-directed exercise routine for the last 3 months

Group Type: ACTIVE_COMPARATOR

Exercise

Intervention Type: OTHER

Exercise virtually supervised by physical therapist and then self-directed for group B

Group C

Self-directed exercise routine for the first 3 months, then exercise routine under the supervision of physical therapist virtually during the last 3 months

Group Type: ACTIVE_COMPARATOR

Exercise

Intervention Type: OTHER

Self-directed and virtually supervised exercise by physical therapist for Group C

Interventions

Exercise

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

Intervention Type: OTHER

Exercise

Exercise virtually supervised by physical therapist and then self-directed for group B

Intervention Type: OTHER

Exercise

Self-directed and virtually supervised exercise by physical therapist for Group C

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

• 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.

Exclusion Criteria

• 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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Massachusetts General Hospital

OTHER

Sponsor Role: lead

Responsible Party

Paloma Gonzalez Perez, MD, PhD

Neurologist

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Neuromuscular Diagnostic Center. Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Zoe Sheitman, DPT

Role: CONTACT

zsheitman@mgh.harvard.edu

617-726-3642

Facility Contacts

Zoe Sheitman, DPT

Role: primary

zsheitman@mgh.harvard.edu

6177245297

Other Identifiers

PGP-ZS

Identifier Type: -

Identifier Source: org_study_id