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Recruiting NCT06716931

Investigating Exercise in Myotonic Dystrophy Type 2 (DM2)

No phase Interventional Myotonic Dystrophy 2

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Exercise, Exercise, Exercise.
Who it may be relevant to
Registry conditions: Myotonic Dystrophy 2. Basic parameters: 18 years — 70 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Official title

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

Overview

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.

Interventions

  • Other 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
  • Other Exercise
    Exercise virtually supervised by physical therapist and then self-directed for group B
  • Other Exercise
    Self-directed and virtually supervised exercise by physical therapist for Group C

Primary outcome measures

  • Manual Muscle Test (MMT) [Time frame: From enrollment to the end of treatment at 6 months]
  • Six-minute walking test [Time frame: From enrollment to the end of treatment at 6 months]
Secondary outcome measures (2)
  • 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]

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Crossover
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 1 center
  • Neuromuscular Diagnostic Center. Massachusetts General Hospital — Boston

Identifiers

NCT: NCT06716931 · PGP-ZS

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗