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NCT Number: NCT06716931

Investigating Exercise 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.

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Key information

Age range

18 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Neuromuscular Diagnostic Center. Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location status: Recruiting

Location contact

Zoe Sheitman, DPT

CONTACT

[email protected]

617-724-5297

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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

Primary outcomes

  1. 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

  2. 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

Secondary outcomes

  1. Quantitative myometry

    Time frame: From enrollment to the end of treatment at 6 months

    Muscle dynamometry of different muscle groups measured in kilograms

  2. Muscle resistance/reactance measures in ohms

    Time frame: From enrollment to the end of treatment at 6 months

    using electrical impedance myography, resistance and reactance measures in ohms will be obtained simultaneously from each examined muscle

Other outcomes

  1. Time up to go

    Time frame: From enrollment to the end of treatment at 6 months

    Time in seconds from standing up, walk 3 meters, come back and sit again.

  2. 10 meter walk testing

    Time frame: From enrollment to the end of treatment at 6 months

    Time in seconds during a 10-meter walk.

  3. Five times sit to stand

    Time frame: From enrollment to the end of treatment at 6 months

    Time in seconds to complete 5 times sit to stand.

  4. Functional gait assessment (FGA)

    Time frame: From enrollment to the end of treatment at 6 months

    Assessment of balance using a scale (0-30) while walking and performing motor tasks.

Study contacts

Contact information is provided by the study sponsor or research team.

Zoe Sheitman, DPT

CONTACT

[email protected]

617-726-3642

Sponsors and collaborators

Lead sponsor

Massachusetts General Hospital

Other

Registry information

Official study title

Investigating Benefits of A Physical Therapist (PT)-Guided Exercise Program in Myotonic Dystrophy Type 2 (DM2)

Acronym: DM2

Important dates

Study start
2025
Primary completion
2027
Study completion
2028
First posted
Dec 4, 2024
Registry last updated
Apr 15, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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