NCT05006924

Brief Summary

Myotonic Dystrophy type 1 (DM1) is a multisystem disease that causes muscle weakness and myotonia. As a result upper limb function might become impaired. In this study we will examine patients with DM1 and record their upper limb function. We will will use a battery of patient reported outcomes (PROs) and Outcome measures (OMs) in order to evalute which ones are suitable for use in clinical practise and research studies.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
89

participants targeted

Target at P50-P75 for all trials

Timeline
Completed

Started Dec 2021

Typical duration for all trials

Geographic Reach
1 country

3 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 10, 2021

Completed
6 days until next milestone

First Posted

Study publicly available on registry

August 16, 2021

Completed
4 months until next milestone

Study Start

First participant enrolled

December 10, 2021

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 31, 2024

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 31, 2024

Completed
Last Updated

April 27, 2025

Status Verified

April 1, 2025

Enrollment Period

2.7 years

First QC Date

August 10, 2021

Last Update Submit

April 23, 2025

Conditions

Myotonic Dystrophy 1

Keywords

Outcome measurePatient reported outcomeUpper limb functionarm- and hand functionSymptoms

Outcome Measures

Primary Outcomes (6)

  • Motor Function Measures (MFM)

    Functional test

    30-45 minutes

  • Nine Hole Peg Test

    Fine motor function test

    1-3 minutes

  • Myogrip/Dynamometer

    Measures hand strenght

    1-3 minutes

  • Myopinch

    Measures finger strenght

    1-3 minutes

  • ABILHAND Questionnaire

    A measure of manual ability for adults with upper limb impairments. The scale measures a person's ability to manage daily activities that require the use of the upper limbs, whatever the strategies involved. Consists of 22 questions (18 to be answered by adults). The categories are easy, difficult or unable to perform. The querionnaire gives a sum score where higher score equals better function.Score from 0 to 36

    5 min

  • ACTIVLIM Questionnaire

    A measure of activity limitations for patients with upper and/or lower limb impairments. The scale measures a patient's ability to perform daily activities requiring the use of the upper and/or the lower limbs, whatever the strategies involved. Consists of 22 questions (18 to be answered by adults). The categories are easy, difficult or unable to perform. The questionnaire gives a sum score where higher score equals better function. Score fror 0 to 36

    5 min

Secondary Outcomes (7)

  • Trunk Impairment Scale - modified Norwegian version

    8-15 minutes

  • Four Square Step Test

    1-3 minutes

  • PROMIS29

    5-10 minutes

  • Starkstein Apathy Scale

    5 minutes

  • Montreal Cognitive Assessment (MoCA)

    10 minutes

  • +2 more secondary outcomes

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Adults with Myotonic Dystrophy type 1 in Norway

You may qualify if:

  • Genetically confirmed Myotonic Dystrophy type 1

You may not qualify if:

  • Unable to answer or understand questionnaires due to language barriers or cognitive status
  • Unable to perform functional tests

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Haukeland University Hospital

Bergen, Norway

Location

Oslo University Hispital

Oslo, Norway

Location

Frambu Centre for Rare Disorders

Siggerud, Norway

Location

MeSH Terms

Conditions

Myotonic Dystrophy

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesMyotonic DisordersHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesNervous System DiseasesNeuromuscular DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Kristin L Ørstavik, PHD

    Oslo University Hospital

    PRINCIPAL INVESTIGATOR

  • Hilde S Robinson, PHD

    University of Oslo

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
CROSS SECTIONAL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
PI

Study Record Dates

First Submitted

August 10, 2021

First Posted

August 16, 2021

Study Start

December 10, 2021

Primary Completion

August 31, 2024

Study Completion

August 31, 2024

Last Updated

April 27, 2025

Record last verified: 2025-04

Data Sharing

IPD Sharing
Will not share

Locations

NO(3)