Clinical trial NCT06716931: Investigating Exercise in Myotonic Dystrophy Type 2 (DM2)
Official title: Investigating Benefits of a Physical Therapist (PT)-Guided Exercise Program in Myotonic Dystrophy Type 2 (DM2)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Myotonic Dystrophy 2
Keywords: exercise
physical therapist
myotonic dystrophy type 2
electrical impedance myography
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
First posted: 2024-12-04
Enrollment: 24
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Manual Muscle Test (MMT)
timeFrame: From enrollment to the end of treatment at 6 months
measure: Six-minute walking test
timeFrame: From enrollment to the end of treatment at 6 months
Secondary outcomes:
measure: Quantitative myometry
timeFrame: From enrollment to the end of treatment at 6 months
measure: Muscle resistance/reactance measures in ohms
timeFrame: From enrollment to the end of treatment at 6 months
Other outcomes:
measure: Time up to go
timeFrame: From enrollment to the end of treatment at 6 months
measure: 10 meter walk testing
timeFrame: From enrollment to the end of treatment at 6 months
measure: Five times sit to stand
timeFrame: From enrollment to the end of treatment at 6 months
measure: Functional gait assessment (FGA)
timeFrame: From enrollment to the end of treatment at 6 months
Locations:
city: Boston
country: United States
facility: Neuromuscular Diagnostic Center. Massachusetts General Hospital
state: Massachusetts
Study officials:
References: