Neuropsychological Profiles of Children With Duchenne Muscular Dystrophy and Its Effects on Motor Functions

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

• Ages 7 to 16 years
• Men only
• Healthy volunteers accepted
• Child

Study population: Children with Duchenne Muscular Dystrophy

Trial locations and contacts

1 site in 1 country.

About this study

Official title: Determination of Neuropsychological Profiles of Children With Duchenne Muscular Dystrophy and Investigation of Its Effects on Motor Functions

Conditions

Duchenne Muscular Dystrophy

Being tested

Modified Mini Mental Test:, The Controlled Oral Word Association Test, The Central Nervous System Vital Signs, Conners' Parent Rating Scale-48 (Parent Report), Child Behavior Checklist 6-18 ages (Parent Report)

Keywords

Duchenne Muscular Dystrophy (DMD), neuropsychological profile, cognitive status, neurodevelopmental status, motor function, balance

Medical subject headings

Muscular Dystrophy, Duchenne

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

• Modified Mini Mental Test:Other

  To assess the cognitive function of children

• The Controlled Oral Word Association TestOther

  To assess the cognitive function of children

• The Central Nervous System Vital SignsOther

  To assess the cognitive function of children

• Conners' Parent Rating Scale-48 (Parent Report)Other

  To assess attention-deficit/hyperactivity disorder

• Child Behavior Checklist 6-18 ages (Parent Report)Other

  To assesses both child adaptive behaviors and problem behaviors.

• Strengths and Difficulties Questionnaire (Self-Reported)Other

  It is a brief emotional and behavioural screening questionnaire for children and young people.

• Brooke Lower Extremity Functional ClassificationOther

  To determine the functional status of the lower extremity

• Motor Function Measurement-32 ItemsOther

  To assessment of motor function and progression of weakness in neuromuscular disorders

• Four Square Step TestOther

  A test of dynamic balance and coordination that clinically assesses the participant's ability to step over objects forward, sideways, and backwards.

• Six Minutes Walk TestOther

  It evaluates the walking function and physical capacity of children at the submaximal level.

• Timed Performance TestOther

  10 meters Walk\& Run Test, Gower's(from a supine to a standing position), Right\& Left Leg Standing, Ascent/Descent of 4 Steps

What is being measured

The primary outcome is the main question the study is designed to answer.

1. Modified Mini Mental Test

   The Mini Mental Test developed for adults was adapted to the pediatric population by making minor modifications. Test; It evaluates verbal responses including attention, orientation, memory and language skills, ability to obey verbal and written orders, write spontaneous sentences, and copy a complex drawing. The highest score that can be obtained from this test is 35, the lowest score is 0.

   Measured: only baseline

2. The Controlled Oral Word Association Test

   This test requires the individual to name as many words as possible that begin with a given letter, i.e. K, A and S. Sixty seconds are allotted for each letter. Individuals cannot use proper names or numbers and cannot use words with different tenses or endings once the root word has been given

   Measured: only baseline

3. The Central Nervous System Vital Signs

   Central Nervous System Vital Signs is a reimbursable assessment procedure that utilizes computerized neuropsychological tests to evaluate the neurocognitive status of patients and covers a range of mental processes from simple motor performance, attention, memory, to executive functions.

   Measured: only baseline

4. Conners' Parent Rating Scale-48 (Parent Report)

   The Conners' Parent Rating Scale-48 contains 48 items wherein the frequency of each item is rated on a 4-point Likert scale ranging from not at all (0)-3 very much (3). The test has adequate psychometric properties and is widely used for clinical and research purposes with the attention deficit/hyperactivity disorder population

   Measured: only baseline

5. Child Behavior Checklist 6-18 ages (Parent Report)

   The Child Behavior Checklist/6-18 assesses both child adaptive behaviors and problem behaviors. There are 112 items that assess problem behaviors and 20 items that assess adaptive behavior. Response format for problem behaviors is from 0 ("not true") to 2 ("very true"). The problem behavior items load onto two broad-band scales (Internalizing and Externalizing) and eight narrow-band scales (Rule Breaking, Aggressive Behavior, Withdrawn-Depressed, Somatic Complaints, Anxious Depressed, Social Problems, Thought Problems, and Attention Problems). The adaptive behavior items load onto three scales: Activities, Social Competence, and School Competence. A Total Competence and Total Behavior Problems score are also provided.

   Measured: only baseline

6. Strengths and Difficulties Questionnaire (Self-Reported)

   The Strengths and Difficulties Questionnaire is a brief emotional and behavioural screening questionnaire for children and young people. The tool can capture the perspective of children. The 25 items in the test comprise 5 scales of 5 items each. The scales include: emotional symptoms subscale, conduct problems subscale, hyperactivity/inattention subscale, peer relationships problem subscale, prosocial behaviour subscale.

   Measured: only baseline

7. Brooke Lower Extremity Functional Classification

   It was developed using the classification method based on "Vignos et al." to determine the functional status of the lower extremity. It consists of 10 different levels, ranging from Level 1 (walks independently and climbs stairs) to Level 10 (bound to bed).

   Measured: only baseline

8. Motor Function Measurement-32 Items

   The Motor Function Measure is a scale designed for the assessment of motor function and progression of weakness in neuromuscular disorders. It is applicable to both ambulant and non-ambulant patients with a wide range of severity. The scale exists in two versions, one with 32 items for patients over 6 years of age (MFM-32), the other with 20 items for children aged from 2 to 6 years (MFM-20). Concerning the development of the scale, factor analysis identified three functional dimensions: D1 = standing position and transfers (13 items; 8 items in the short version), D2 = axial and proximal motor function (12 items; 8 in the short version), and D3 = distal motor function (7 items; 4 in the short version).

   Measured: only baseline

9. Four Square Step Test

   It is a valid and reliable test that has been used frequently in children in recent years to evaluate dynamic balance. Sticks, each 90 cm long, are placed on the floor to form 4 squares and the squares are numbered from 1 to 4. For the test to be completed successfully, the child must quickly move from one square to the next without touching the sticks. Performance is determined by measuring the test completion time in seconds. Shorter completion time means better dynamic balance.

   Measured: only baseline

10. Six Minutes Walk Test

    The 6-minutes walk test, which is valid and reliable for DMD patients, will evaluate the walking function and physical capacity of children at the submaximal level. The distance the child walks for 6 minutes in a 25 m corridor will be recorded in meters. A physiotherapist will walk with the children during the test and track the time with a stopwatch. The test is simple and considered an important outcome measure for children with DMD.

    Measured: only baseline

11. 10 meters Walk& Run Test

    A 10-meter distance was marked on an unobstructed, flat surface using tape. To limit the impact of acceleration and deceleration on gait speed, start and finish lines were placed 30 centimeter before and after the 10-meter distance. Participants were instructed to begin with toes on the start line and walk or run as fast as possible, without compromising safety, to the finish line.

    Measured: only baseline

12. Gower's(from a supine to a standing position)

    Children lied down on a mat with straight position and asked them to stand up as fast as possible. Time was started when he moved and stopped when he was upright position.

    Measured: only baseline

13. Right& Left Leg Standing Test

    Children had to maintain a one-legged stance for as long as they could with their eyes open, and allowing them to freely-move their arms. Children were verbally encouraged to maintain the one-legged standing position for as long as possible during test.

    Measured: only baseline

14. Ascent/Descent of 4 Steps

    The children were asked to climb up the 4-step ladder with double-sided handrails as fast as possible. The time was started when his feet lifted from the ground and when both feet touched the ground, the time was stopped and recorded in seconds. After climbing the ladder, they were asked to descend as fast as possible, the time was started when the foot was lifted, and the time was stopped when both feet touched the ground and recorded in seconds.

    Measured: only baseline

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1. First posted22 Dec 2022
2. Study start11 May 2022
3. Primary completion1 Mar 2023 (estimated)
4. Study completion1 Mar 2023 (estimated)

Resources

• Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
• Publications (1)Papers the sponsor has linked to this study.
• Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
• More trials run by Hacettepe UniversityThe sponsor's other registered studies.
• What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.