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Myotonic Dystrophy 1SOUL-DM1NCT05006924

Symptoms and Outcome Measures for Upper- Limb Function in Myotonic Dystrophy Type 1

An observational study of Myotonic Dystrophy 1, run by Oslo University Hospital at 3 sites in Norway.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Myotonic Dystrophy 1
How many people
89 participants
Where
3 sitesNorway
Study dates
10 Dec 2021to 31 Aug 2024
Phase
Observational
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Study population: Adults with Myotonic Dystrophy type 1 in Norway

Who may be able to join

  • Genetically confirmed Myotonic Dystrophy type 1

Who cannot join

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

Trial locations and contacts

3 sites in 1 country.

Norway3 sites

  • Haukeland University Hospital

    Bergen

  • Oslo University Hispital

    Oslo

  • Frambu Centre for Rare Disorders

    Siggerud

Other trials in Norway

About this study

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.

Keywords
Outcome measure, Patient reported outcome, Upper limb function, arm- and hand function, Symptoms
Medical subject headings
Myotonic Dystrophy

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Motor Function Measures (MFM)

    Functional test

    Measured: 30-45 minutes

  2. Nine Hole Peg Test

    Fine motor function test

    Measured: 1-3 minutes

  3. Myogrip/Dynamometer

    Measures hand strenght

    Measured: 1-3 minutes

  4. Myopinch

    Measures finger strenght

    Measured: 1-3 minutes

  5. 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

    Measured: 5 min

  6. 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

    Measured: 5 min

Secondary outcomes (7)

  1. Trunk Impairment Scale - modified Norwegian version

    Motor impairment of trunk measured by functional testing. Six subdomains measuring stability and mobility of trunkal movement. Min score 0 - Max score 16. Higher score indicates better function.

    Measured: 8-15 minutes

  2. Four Square Step Test

    Dynamic balance test

    Measured: 1-3 minutes

  3. PROMIS29

    This a compostie measurement that is a Questionnaire. Patient reported outcome measure. Generic health related quality of life assesses each of the 7 PROMIS domains (depresseion, anxiety, physical function, pain, fatigue, sleep disturbance and ability to participate in social roles). The PROMIS 29 questionnaire contains 4 questions from each of seven PROMIS contents (physical function, depression, anxiety, fatigue, sleep disorder, participation in social activities and pain interaction) and 1 question from the intensity of pain. Each item has 5 answer options (from 1 to 5), only pain intensity has 11 answer options (from 0 to 10). From the sum of the answers to each question in the domain, the total raw score for each domain is calculated, resulting in seven domain scores, each between 4 and 20.

    Measured: 5-10 minutes

  4. Starkstein Apathy Scale

    Questionnaire to be filled in under guidance of interviewer. Patient reported outcome measure of apathy. 14 questions, each of which is scored on a 4-point scale of 0-3, and apathy is rated as severer as the total score (0-42) increases

    Measured: 5 minutes

  5. Montreal Cognitive Assessment (MoCA)

    Cognitive assessment screening questionnaire. To be filled out in an interview setting. Score from 0 to 30, where a higher score indicates a better cognitive function. A score of 26 or more is considered normal.

    Measured: 10 minutes

  6. Lung function test

    Spirometry with FVC (Forced Vital Capasity)

    Measured: 10 minutes

  7. Lung function test

    PEF (Peak Expiratory Flow).

    Measured: 10 minutes

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Aug 2021
  2. Study start10 Dec 2021
  3. Primary completion31 Aug 2024
  4. Study completion31 Aug 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Cross-sectional
Enrolment
89 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05006924
Sponsor's protocol ID
255164
Acronym
SOUL-DM1
Status last verified
Apr 2025
First submitted
10 Aug 2021
Last updated
27 Apr 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

An observational study of Myotonic Dystrophy 1, run by Oslo University Hospital at 3 sites in Norway.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 1 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 27 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in Norway. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Motor Function Measures (MFM) (time frame: 30-45 minutes). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05006924.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Apr 2025.

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