High-intensity Interval Training in Patients With Spinal Muscular Atrophy

Spinal Muscular Atrophy Clinical Trial

Official title:

High-intensity Interval Training in Patients With Spinal Muscular Atrophy

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06368076
• Other study ID #: H-23065096
• Status: Recruiting
• Phase: N/A
• Start date: January 9, 2024
• Est. completion date: January 2025

Study information

• Verified date: April 2024
• Source: Rigshospitalet, Denmark
• Contact: Sophia Frølich, stud.med
• Phone: + 45 3545 3561
• Email: sophia.vera.froelich.01[@]regionh.dk
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Patients with spinal muscular atrophy who are wheelchair users often experience lower back - and gluteal pain, reduced sleep quality, constipation and reduced quality of life - symptoms that regular exercise could potentially alleviate. However, only very little research has been done on exercise for patients who are wheelchair users. The aim of this study is to explore the impact of cycle exercise on patients with spinal muscular atrophy.

Description:

Spinal muscular atrophy (SMA) is a neuromuscular disease affecting the motor neurons of the spinal cord that innervate the skeletal muscles, leading to progressive muscle atrophy and weakness. Consequently, many patients end up having to use a wheelchair from an early age. Besides the impaired motor function, patients with SMA can also experience a variety of symptoms associated with their disease, a sedentary lifestyle and physical inactivity such as lower back and leg pain, obstipation, reduced sleep quality and impacted quality of life. Training can help alleviate these symptoms in patients with neuromuscular diseases. However, there isn´t any validated way for patients with SMA to train as of today. Research has shown that high-intensity interval training (HIIT) has been well tolerated among patients with SBMA, a disease resembling SMA with patients tolerating this form for training well. The aim of this study is to investigate:(1) if HIIT could be exercise modality in SMA patients (2) and to see what positive effects exercise has for their impaired motor function and related challenges. The investigators will test the participants at baseline, which will be followed by a 8-week control period. After the control period the participants will be tested again before the intervention period. During the intervention period the participants will have to train 5 times a week for 10 minutes. After the intervention period, there will be a final test day, where the final results will be compared with those from the baseline.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: January 2025
• Est. primary completion date: August 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 15 Years and older

Eligibility

Inclusion Criteria:

• Spinal muscular atrophy
• Age: over 15 years

Exclusion Criteria:

• Competing disorders (as arthritis) or other muscle disorders as well as heart- or lung related issues.
• Current psychiatric treatment
• Unable to use the cycle ergometer due to contractures

Related Conditions & MeSH terms

• Atrophy
• Muscular Atrophy
• Muscular Atrophy, Spinal
• Spinal Muscular Atrophy

Intervention

Behavioral:
• Exercise
High intensity interval training on a bike or pedaltrainer from the wheelchair.

Locations

Country	Name	City	State
Denmark	Rigshospitalet	Copenhagen

Sponsors (1)

Lead Sponsor	Collaborator
Rigshospitalet, Denmark

Country where clinical trial is conducted

Denmark, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Questionnaire on fatigue	The Multidimensional Fatigue Inventory (MFI-20) will be used. It measures fatigue in 5 different domains comprised of 4 items each with values ranging from 1 to 5. A higher score indicates more fatigue. Each domain is scored individually, ranging from 4 points (best outcome) to 20 points (worst outcome).	5 minutes
Primary	Questionnaire on pain.	A visual pain score for leg and lower back pain with 3 questions with scores ranging from 1 to 10 each, 10 being the worst pain imaginable, will be used.	5 minutes
Primary	Questionnaire on constipation	A questionnaire made from the Danish definition of constipation will be used. Four questions will be answered "yes" or "no".	5 minutes
Primary	Questionnaire on quality of life	A QoL that has been used in former studies will be used. 16 questions with scores ranging from 1 (very unsatisfied) to 7 (very satisfied) will be scored.	10 minutes
Primary	Questionnaire on sleep quality	Minimum Score = 0 (better); Maximum Score = 21 (worse) Interpretation: TOTAL < 5 associated with good sleep quality TOTAL > 5 associated with poor sleep quality Minimum Score = 0 (better); Maximum Score = 21 (worse) Interpretation: TOTAL < 5 associated with good sleep quality TOTAL > 5 associated with poor sleep quality Minimum score is 0, maximum score is 21. Total over 5 is associated with good sleep quality and under 5 associated with poor sleep quality.	10 minutes
Secondary	Blood sample	Change in blood sample for blood lipids, mmol/l	2 minutes
Secondary	Blood sample	Change in blood sample for HbA1C, mmol/mol	2 minutes
Secondary	Blood sample	Change in blood sample for Creatine kinase, U/I. This will be monitored before, in the middle and after the exercise period as a safety measure.	2 minutes
Secondary	Blood sample	Change in blood sample for fasting blood glucose, in mmol/L	2 minutes
Secondary	Blood sample	Change in blood sample for fasting insulin, in pmol/L.	2 minutes
Secondary	Blood sample	Change in blood sample for fasting c-peptide, in pmol/L.	2 minutes
Secondary	Blood sample	Change in blood sample for ALT (liver parameter) in U/L	2 minutes
Secondary	Blood sample	Change in blood sample for AST (liver parameter) in U/L	2 minutes
Secondary	Blood sample	Change in blood sample for alkaline phosphatase (liver parameter) in U/L	2 minutes
Secondary	Blood sample	Change in blood sample for bilirubin (liver parameter) in umol/L	2 minutes
Secondary	MRI scan liver size	Assessment of liver size	5 minutes
Secondary	MR-elastography	Assessment of liver fibrosis (assessed by MR-elastography)	5 minutes
Secondary	MRI scan liver steatosis	Assessment of liver steatosis	5 minutes
Secondary	Ultrasound scan	Additional assessment of liver size, fibrosis and steatosis	15 minutes
Secondary	MRI scan	Assessment of full body muscle size	15 minutes
Secondary	MRI scan	Assessment of muscle degeneration and subsequent fat replacement (using Dixon method to visualise fat in an optimal way)	15 minutes
Secondary	Exercise test	Change in time to cycle X km in minutes	7 minutes
Secondary	Motorscore	Change in motor function score, using the Motor Function Measurement (MFM-32). Assesses motor function in 3 domains, 32 two items in total. A higher score in each domain indicates better function.	20 minutes