Investigating Exercise in Myotonic Dystrophy Type 2 (DM2)

NCT06716931 | Recruiting

• 1 other identifier: PGP-ZS
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

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 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

Keywords

exercise; physical therapist; myotonic dystrophy type 2; electrical impedance myography

Outcome Measures

Primary Outcomes (2)

• Manual Muscle Test (MMT)

  Score of manual muscle strength test scale. This is a strength exam at bedside of muscle groups

  From enrollment to the end of treatment at 6 months

• Six-minute walking test

  Distance in meters that subject can walk in six minutes

  From enrollment to the end of treatment at 6 months

Secondary Outcomes (2)

• Quantitative myometry

  From enrollment to the end of treatment at 6 months

• Muscle resistance/reactance measures in ohms

  From enrollment to the end of treatment at 6 months

Other Outcomes (4)

• Time up to go

  From enrollment to the end of treatment at 6 months

• 10 meter walk testing

  From enrollment to the end of treatment at 6 months

• Five times sit to stand

  From enrollment to the end of treatment at 6 months

Study Arms (3)

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

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

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

Other: Exercise

Interventions

Exercise 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 A

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Massachusetts General Hospital: lead

Study Sites (1)

Neuromuscular Diagnostic Center. Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

RECRUITING

MeSH Terms

Conditions

Myotonic Dystrophy; Motor Activity

Interventions

Exercise

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Myotonic Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Behavior

Intervention Hierarchy (Ancestors)

Motor Activity; Movement; Musculoskeletal Physiological Phenomena; Musculoskeletal and Neural Physiological Phenomena

Central Study Contacts

Zoe Sheitman, DPT

CONTACT

617-726-3642; zsheitman@mgh.harvard.edu

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Neurologist

Study Record Dates

• First Submitted: October 30, 2024
• First Posted: December 4, 2024
• Study Start: March 4, 2025
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): March 1, 2028
• Last Updated: April 15, 2026

Record last verified: 2026-04

Locations

US (1)