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L'essai clinique NCT06716931 (DM2) pour Myotonic Dystrophy 2 est en recrutement. Consultez la vue en carte du Radar des Essais Cliniques et les outils de découverte par IA pour tous les détails, ou posez vos questions ici.
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Investigating Exercise in Myotonic Dystrophy Type 2 (DM2)

En recrutement
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L'étude clinique NCT06716931 (DM2) est un essai interventionnel pour Myotonic Dystrophy 2. Son statut actuel est : en recrutement. L'étude a débuté le 4 mars 2025 et vise à recruter 24 participants. Dirigé par Massachusetts General Hospital, l'essai devrait être terminé d'ici le 1 mars 2026. Les données du site ClinicalTrials.gov ont été mises à jour pour la dernière fois le 29 septembre 2025.
Résumé succinct
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.
Titre officiel

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

Conditions
Myotonic Dystrophy 2
Autres identifiants de l'essai
  • DM2
  • PGP-ZS
Numéro NCT
Date de début (réel)
2025-03-04
Dernière mise à jour publiée
2025-09-29
Date de fin (estimée)
2026-03-01
Inscription (estimée)
24
Type d'essai
Interventionnel
PHASE
N/A
Statut
En recrutement
Mots clés
exercise
physical therapist
myotonic dystrophy type 2
electrical impedance myography
Objectif principal
Traitement
Plan d'attribution
Randomisé
Modèle d'intervention
Croisé
Masquage
Aucun (ouvert)
Bras / Interventions
Groupe de participants/BrasIntervention/Traitement
Comparateur actifGroup A
Exercise under the supervision of physical therapist in clinic during the first 3 months, then self-directed exercise routine for the last 3 months
EXERCICE
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
Comparateur actifGroup B
Exercise under the supervision of physical therapist virtually during the first 3 months, then self-directed exercise routine for the last 3 months
EXERCICE
Exercise virtually supervised by physical therapist and then self-directed for group B
Comparateur actifGroup C
Self-directed exercise routine for the first 3 months, then exercise routine under the supervision of physical therapist virtually during the last 3 months
EXERCICE
Self-directed and virtually supervised exercise by physical therapist for Group C
Critère principal d'évaluation
Critères d'évaluationDescription de critèresPériode
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
Critère secondaire d'évaluation
Critères d'évaluationDescription de critèresPériode
Quantitative myometry
Muscle dynamometry of different muscle groups measured in kilograms
From enrollment to the end of treatment at 6 months
Muscle resistance/reactance measures in ohms
using electrical impedance myography, resistance and reactance measures in ohms will be obtained simultaneously from each examined muscle
From enrollment to the end of treatment at 6 months
Critères d'éligibilité

Âges éligibles
Adulte, Adulte âgé
Âge minimum
18 Years
Sexes éligibles
Tous
  • 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.

  • 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.
Massachusetts General Hospital logoMassachusetts General Hospital
Partie responsable de l'essai
Paloma Gonzalez Perez, MD, PhD, Investigateur principal, Neurologist, Massachusetts General Hospital
Contact central de l'essai
Contact: Zoe Sheitman, DPT, 617-726-3642, [email protected]
1 Centres de l'essai dans 1 pays

Massachusetts

Neuromuscular Diagnostic Center. Massachusetts General Hospital, Boston, Massachusetts, 02114, United States
Zoe Sheitman, DPT, Contact, 6177245297, [email protected]
En recrutement