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

Myotonic Dystrophy 1 Clinical Trial

— SOUL-DM1  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05006924
• Other study ID #: 255164
• Status: Recruiting
• Start date: October 2021
• Est. completion date: December 2026

Study information

• Verified date: August 2021
• Source: Oslo University Hospital
• Contact: Hanne L Fossmo, MSC
• Phone: +47 93630606
• Email: halufo[@]ous-hf.no
• Is FDA regulated: No
• Study type: Observational

Clinical 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. 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 80
• Est. completion date: December 2026
• Est. primary completion date: October 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Genetically confirmed Myotonic Dystrophy type 1

Exclusion Criteria:

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

• Unable to perform functional tests

Related Conditions & MeSH terms

• Myotonic Dystrophy
• Myotonic Dystrophy 1

Locations

Country	Name	City	State
Norway	Haukeland University Hospital	Bergen
Norway	Oslo University Hispital	Oslo
Norway	Frambu Centre for Rare Disorders	Siggerud

Sponsors (3)

Lead Sponsor	Collaborator
Oslo University Hospital	Haukeland University Hospital, University of Oslo

Country where clinical trial is conducted

Norway, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Motor Function Measures (MFM)	Functional test	30-45 minutes
Primary	Nine Hole Peg Test	Fine motor function test	1-3 minutes
Primary	Myogrip/Dynamometer	Measures hand strenght	1-3 minutes
Primary	Myopinch	Measures finger strenght	1-3 minutes
Primary	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
Primary	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	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.	8-15 minutes
Secondary	Four Square Step Test	Dynamic balance test	1-3 minutes
Secondary	PROMIS29	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.	5-10 minutes
Secondary	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	5 minutes
Secondary	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.	10 minutes
Secondary	Lung function test	Spirometry with FVC (Forced Vital Capasity)	10 minutes
Secondary	Lung function test	PEF (Peak Expiratory Flow).	10 minutes