The Effect of Aerobic Exercise Training in Patients With Type III Spinal Muscular Atrophy

NCT05544994 | Unknown DMC

• 1 other identifier: Aerobic Training
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

In the last 10-15 years, a better understanding of the pathophysiology and molecular genetics of SMA has led to the emergence of previously unavailable pharmacological and genetic treatments.One of these new treatments, Nusinersen, targets SMN2, which is a slightly different copy of SMN1, and increases SMN protein levels. Preclinical studies have provided evidence that neuroprotection is strongly formed, with exercise significantly increasing motor neuron survival independent of SMN expression. In a limited number of clinical studies prior to Nusinersen treatment, it was reported that aerobic exercise training improved maximum oxygen uptake (VO2 max) without causing muscle damage, but still caused fatigue. The aim of this study is to determine the effect of aerobic exercise training on motor and respiratory functions, exercise capacity, fatigue and quality of life in SMA Type III patients who can walk and receive Nusinersen therapy. Twenty cases aged 10-50 years with genetically confirmed SMA diagnosis will be included in this study. The cases to be included in the study will be randomized into 2 groups as the training and control groups. In addition to the routine physiotherapy program, medium-intensity Aerobic Exercise Training will be given to the study group for 12 weeks. Before and 12 weeks after the training, the cases will be evaluated with the Six Minute Walking Test, Submaximal Exercise Test, SMN protein level, function and strength assessments, (FVC) value, fatigue and quality of life scales. In clinical trials, the supporting evidence for aerobic interventions in SMA is limited. Additional studies on aerobic intervention parameters (frequency, intensity and duration) are needed.The results of this study will determine the feasibility of aerobic exercise training and provide important guidance for the clinical management of SMA patients.

Conditions

Neuromuscular Diseases; Spinal Muscular Atrophy Type 3

Keywords

Spinal Muscular Atrophy Type 3; Aerobic Training; SMN protein level; Physiotherapy

Outcome Measures

Primary Outcomes (1)

• Functional Capacity

  Change from baseline functional capacity test at 12 weeks. Functional capacity will be assessed by the 6 minute walking test.The 6MWT, an objective evaluation of functional capacity, measures the maximum distance a person can walk in six minutes over a 25-meter linear course.

  Baseline and 12 weeks

Secondary Outcomes (10)

• Submaximal Exercise Test

  Baseline and 12 weeks

• Pulmonary Function

  Baseline and 12 weeks

• Motor Function

  Baseline and 12 weeks

• Motor Performance

  Baseline and 12 weeks

• Balance and Mobility

  Baseline and 12 weeks

Study Arms (2)

Aerobic Exercise Training Group

EXPERIMENTAL

Aerobic training will be performed 3 days a week for 12 weeks at 60% -%75 of their maximum hearth rate with 30 minutes total duration consisting of 5 min warm up and 5 min cool down period in treatment group. Home exercise program will be given. This home program will include stretching, breathing, normal joint movement for 3 to 5 days a week

Other: Aerobic Exercise Training; Other: Home exercise program

Control Group

ACTIVE COMPARATOR

Home exercise program will be given. This home program will include stretching, breathing, normal joint movement, for 3 or 5 days a week.

Other: Home exercise program

Interventions

Aerobic Exercise Training OTHER

Aerobic training will be performed 3 days a week for 12 weeks at 60%-%75 of their maximum hearth rate with 30 minutes total duration consisting of 5 min warm up and 5 min cool down period in treatment group.

Aerobic Exercise Training Group

Home exercise program OTHER

Home exercise program will be given. This home program will include stretching, breathing, normal joint movement, for 3 to 5 days a week.

Aerobic Exercise Training Group; Control Group

Eligibility Criteria

• Age: 10 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Have been diagnosed of SMA Type 3 confirmed by genetic analysis,
• Between the ages of 10-50,
• Being able to walk 25 m without assistive device

You may not qualify if:

• Using research drugs for SMA treatment other than Nusinersen treatment,
• Having a serious systemic disease that may prevent exercise,
• Have had a lower extremity injury and/or surgery in the last 6 months,
• To be applying a regular aerobic training program in the last 6 months

Sponsors & Collaborators

• Istanbul Medipol University Hospital: lead
• Istanbul University: collaborator

Study Sites (1)

Istanbul Faculty of Medicine, Department of Neurological Sciences

Istanbul, Fatih, Turkey (Türkiye)

RECRUITING

MeSH Terms

Conditions

Neuromuscular Diseases; Spinal Muscular Atrophies of Childhood

Condition Hierarchy (Ancestors)

Nervous System Diseases; Muscular Atrophy, Spinal; Spinal Cord Diseases; Central Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Motor Neuron Disease; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Sezan Mergen KILIÇ, MSc PT

  Istanbul University, Istanbul Faculty of Medicine,Department of Neurological Sciences

  PRINCIPAL INVESTIGATOR

• Fatma Karantay Mutluay,, Professor

  Medipol University, Health Sciences Faculty, Physiotherapy and Rehabilitation

  STUDY DIRECTOR

• Fatma Yeşim Parman, Professor

  Istanbul University, Istanbul Faculty of Medicine,Department of Neurological Sciences

  STUDY DIRECTOR

Central Study Contacts

Sezan Mergen KILIÇ, MSc PT

CONTACT

05324569628; sezanmer@hotmail.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Msc -Physiotherapist

Study Record Dates

• First Submitted: August 19, 2022
• First Posted: September 19, 2022
• Study Start: July 28, 2022
• Primary Completion: June 30, 2024
• Study Completion: September 30, 2024
• Last Updated: September 28, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share

Locations

TU (1)