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

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

Interventions

OTHER

Aerobic Exercise Training

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.

OTHER

Home exercise program

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

Sponsors & Collaborators

• Istanbul University

  collaborator OTHER

• Istanbul Medipol University Hospital

  lead OTHER

Principal Investigators

• Sezan Mergen KILIÇ, MSc PT · Istanbul University, Istanbul Faculty of Medicine,Department of Neurological Sciences

• Fatma Karantay Mutluay,, Professor · Medipol University, Health Sciences Faculty, Physiotherapy and Rehabilitation

• Fatma Yeşim Parman, Professor · Istanbul University, Istanbul Faculty of Medicine,Department of Neurological Sciences

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Model

PARALLEL

Eligibility

Min Age

10 Years

Max Age

50 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2022-07-28

Primary Completion

2024-06-30

Completion

2024-09-30

Countries

• Turkey (Türkiye)

Study Locations