Respiratory Functions, Thoracoabdominal Movements and Exercise Capacity in Neuromuscular Diseases

Condition(s) studied

Investigational drug(s) / intervention(s)

Respiratory Function Test: A spirometer (microQuark, COSMED) will be used to assess respiratory functions. During a forced expiratory maneuver after a deep, full inspiration, airway and lung volumes are measured. During the measurement, a deep inspiration followed by a strong, fast, and continuous expiration until unable to exhale anymore should be performed. The expiration time should be at least 6 seconds, and if necessary, extended up to 15 seconds. More than eight repetitions at one time are not recommended.

Structured light plethysmography: Participants' thoracoabdominal movements will be assessed using the PneumoCare device that measures with the SLP technique. The evaluation begins with positioning participants wearing a white tank top, t-shirt, or with a bare chest. In our study, measurements will be taken with participants seated, supported by their backs, and their chest area exposed. The participant's age, height, and weight information are entered into the system. After the participant is positioned, the lights of the SLP measuring device are set up 90-100 cm away to encompass the chest area. The light field, which is in a checkerboard pattern, rectangular or square, should align its upper edge with the participant's clavicles and its lower edge with the anterior-superior iliac spine. During this measurement, the participant is asked to look straight ahead and breathe normally for five minutes. At the end of the measurement, the three-dimensional movements of the chest and respiratory parameters are recorded.

6 Minute Walking Test: For Ambulatory Participants: Exercise capacity will be assessed using the Six-Minute Walk Test (6MWT). The test will be performed according to ATS/ERS guidelines . The test involves walking for six minutes at a submaximal level in a 20-meter corridor. Blood pressure, heart rate, respiratory rate, oxygen saturation, and dyspnea, general fatigue, and leg fatigue according to the Modified BORG Scale (MBS) will be assessed before and after the test. Participants can stop and rest during the test. In such cases, the test duration is paused, and the rest time is recorded, then the test resumes from where it was left off. The distance walked in meters at the end of the test is recorded.

6-Minute Peg Board Ring Test: For Non-Ambulatory Participants: The 6-Minute Pegboard Ring Test will be used to assess upper extremity exercise capacity . Pre- and post-test evaluations of heart rate, blood pressure, respiratory rate, and MBS for dyspnea and fatigue will be made. The board used for the test has a total of 6 holes spaced 10 cm apart horizontally and another set of 6 holes 20 cm below aligned with the top row. There are four 20 cm long iron rods suitable for the holes. A total of 20 rings suitable for the iron rods will be used. The iron rods will be placed in the designated holes according to the patients' shoulder width. An adjustable chair will be used to suit the patients' heights, and the distance between the chair and the board will be adjusted according to arm length. Patients will be asked to place the rings from top to bottom and bottom to top with both hands simultaneously. The total number of rings placed in six minutes will be recorded in units.

Hand Grip Strength: Hand grip strength will be measured using a digital hand dynamometer (CAMRY Digital Hand Dynamometer) . The device handle will be adjusted according to the participants' hand size. The test is conducted while seated, with the dominant upper extremity in 90 degrees of elbow flexion. Participants are asked to squeeze the dynamometer as strongly as possible. The value displayed on the dynamometer is recorded. The measurement is repeated three times, and the average of the three measurements is taken to determine the participant's hand grip strength. The measurement takes five minutes and does not need to be repeated.

Study summary

The clinical trial titled "Investigation of Respiratory Functions, Thoracoabdominal Movements, and Exercise Capacity in Neuromuscular Diseases" aims to evaluate the respiratory functions, thoracoabdominal movements, and exercise capacity in children with Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA) compared to healthy controls. The study will use spirometry, structured light plethysmography (SLP), the six-minute walk test, and the six-minute pegboard ring test to assess these functions. This trial will be conducted at the Lokman Hekim University Muscle and Nerve Diseases Application and Research Center from May 2024 to Dec 2025.

Eligibility

Primary outcome measure(s)

• Forced Vital Capacity (FVC) — Baseline (Day 1)
  Forced Vital Capacity (FVC) measures the maximum volume of air that a participant can exhale forcefully after taking a deep breath. This measure reflects lung capacity.
• Forced Expiratory Volume in 1 Second (FEV1) — Baseline (Day 1)
  Forced Expiratory Volume in 1 second (FEV1) represents the volume of air that can be forcefully exhaled in one second. This measure evaluates airway function and obstruction.
• Peak Expiratory Flow (PEF) — Baseline (Day 1)
  Peak Expiratory Flow (PEF) assesses the maximum speed of air expelled during expiration, indicating airway resistance.
• FEV1/FVC Ratio — Baseline (Day 1)
  The ratio of FEV1 to FVC, expressed as a percentage, evaluates airflow limitation severity.
• 6-Minute Walk Test (6MWT) — Baseline (Day 1)
  The 6-Minute Walk Test (6MWT) assesses lower-limb functional capacity by measuring the total distance a participant can walk in six minutes at their own pace on a flat, 20-meter corridor. The test evaluates endurance and physical capacity. Heart rate, blood pressure, and oxygen saturation will be measured immediately before and after the test to assess physiological responses.
• Thoracoabdominal movement — 8 min
  Detection of thoracoabdominal asyncrony and analysis of the kinematics of breathing
• Hand Grip Strength — 5 min
  Digital hand dynamometer
• 6-Minute Peg Board Ring Test (6PBRT) — Baseline (Day 1)
  The 6-Minute Peg Board Ring Test (6PBRT) evaluates upper-limb endurance and strength. Participants will move rings between pegs on a pegboard for six minutes, with the total number of rings successfully placed recorded as the outcome. This test is designed to assess upper-limb functional capacity and fatigue.

Trial sites (1)

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