Investigating Exercise in Myotonic Dystrophy Type 2 (DM2)

Last updated: December 3, 2024
Sponsor: Massachusetts General Hospital
Overall Status: Active - Recruiting

Phase

N/A

Condition

Muscular Dystrophy

Treatment

Exercise

Clinical Study ID

NCT06716931
PGP-ZS
  • Ages 18-70
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Patients with genetically confirmed myotonic dystrophy 2 (DM2) who are ≥18 and ≤ 70years 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

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 beperformed at visit 1 and 3 for women of childbearing age. If positive or if subjectis planning to become pregnant, subject will be not eligible for the study.

Study Design

Total Participants: 24
Treatment Group(s): 1
Primary Treatment: Exercise
Phase:
Study Start date:
December 01, 2024
Estimated Completion Date:
March 01, 2026

Connect with a study center

  • Neuromuscular Diagnostic Center. Massachusetts General Hospital

    Boston, Massachusetts 02114
    United States

    Active - Recruiting

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