Spinal Muscular Atrophy Type INCT06864767
Impact of Motor and Oral Motor Function on Quality of Life in Children With SMA
An observational study of Spinal Muscular Atrophy Type I, run by Zeynep HOŞBAY at 1 site in Turkey (Türkiye).
Trial summary
The aim of this study is to investigate the impact of motor and oral motor functions on the quality of life in children aged 2-4 years diagnosed with Spinal Muscular Atrophy (SMA) Type I. Read more about this study
- Who is it for?
- Ages 2 to 4 years
- Conditions the study is for
- Spinal Muscular Atrophy Type I
- What is being tested?
- Neuro-sensory Motor Developmental Assessment, Functional Oral Intake Scale and Behavioral Pediatrics Feeding Assessment Scale
- Type of treatment
- Pill or oral medicine
- How many people
- 23 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- 30 May 2024to 30 Aug 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
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 2 to 4 years
- All sexes
- No healthy volunteers
- Child
Study population: The study was completed with 23 children between the ages of 2-4 who were followed up with the diagnosis of Spinal Muscular Atrophy (SMA) Type 1 and whose parents volunteered to participate in the study.
Who may be able to join
- Diagnosed with Spinal Muscular Atrophy Type I
- Being between the ages of 2-4
- Families volunteering to participate in the study
Who cannot join
- Accompanied by another chronic disease other than SMA
- The mother or father has a cognitive problem that prevents them from expressing themselves
- The mother or father is illiterate
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Impact of Motor and Oral Motor Function on Quality of Life in Children With Type I Spinal Muscular Atrophy
The aim of this study is to investigate the impact of motor and oral motor functions on the quality of life in children aged 2-4 years diagnosed with Spinal Muscular Atrophy (SMA) Type I. In the study, the Neuro-Sensory-Motor Developmental Assessment (NSMDA) was used to evaluate motor functions, the Functional Oral Intake Scale (FOIS) to assess oral motor function, the Behavioral Pediatric Feeding Assessment Scale (BPFAS) to evaluate children's attitudes towards feeding, and the Pediatric Quality of Life Inventory (PedsQL) Neuromuscular Module to assess quality of life.
- Conditions
- Spinal Muscular Atrophy Type I
- Being tested
- Neuro-sensory Motor Developmental Assessment, Functional Oral Intake Scale, Behavioral Pediatrics Feeding Assessment Scale, Pediatric Quality of Life Inventory
- Keywords
- Spinal Muscular Atrophy Type I, Oral Motor Function, Motor Function, Quality of Life
- Medical subject headings
- Spinal Muscular Atrophies of Childhood
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Neuro-sensory Motor Developmental AssessmentOther
The test used to assess children's development levels evaluates the child's development in six parameters: gross motor function, fine motor function, neurological status, infant movement patterns, postural development and sensory-motor function (tactile, proprioceptive, ocular and vestibular systems) on a scale of 1 to 5. High scores are interpreted as motor dysfunction.
Functional Oral Intake ScaleOther
It is a scale consisting of a total of 7 levels and two sections, developed by Crary et al. to indicate the functional oral intake of patients with dysphagia. A higher score indicates a better nutritional level.
Behavioral Pediatrics Feeding Assessment ScaleOther
The scale used to determine eating problems in children evaluates both the child's nutritional status and the parents' feelings about the child's nutritional status. It consists of 35 items in total and is scored from 1 to 5. High scores indicate problematic eating behaviors and habits.
Pediatric Quality of Life InventoryOther
The PedsQL assesses health-related quality of life in children ages 2 to 18 years with chronic illnesses from both the child's and parents' perspectives. It consists of 25 items and 3 categories (About My Child's Neuromuscular Disease, Communication, About Our Family Resources). The Neuromuscular Module was used to assess the quality of life of the children in our study and was answered by the parents only. The scale is scored from 0 to 4, with higher scores indicating better quality of life.
What is being measured
The primary outcome is the main question the study is designed to answer.
Neuro-sensory Motor Developmental Assessment
The test used to assess children's development levels evaluates the child's development in six parameters: gross motor function, fine motor function, neurological status, infant movement patterns, postural development and sensory-motor function (tactile, proprioceptive, ocular and vestibular systems) on a scale of 1 to 5. High scores are interpreted as motor dysfunction.
Measured: Once, beginning of the study
Functional Oral Intake Scale
It is a scale consisting of a total of 7 levels and two sections, developed by Crary et al. to indicate the functional oral intake of patients with dysphagia. A higher score indicates a better nutritional level.
Measured: Once, beginning of the study
Behavioral Pediatrics Feeding Assessment Scale
The scale used to determine eating problems in children evaluates both the child's nutritional status and the parents' feelings about the child's nutritional status. It consists of 35 items in total and is scored from 1 to 5. High scores indicate problematic eating behaviors and habits.
Measured: Once, beginning of the study
Secondary outcomes (1)
Pediatric Quality of Life Inventory
The PedsQL assesses health-related quality of life in children ages 2 to 18 years with chronic illnesses from both the child's and parents' perspectives. It consists of 25 items and 3 categories (About My Child's Neuromuscular Disease, Communication, About Our Family Resources). The Neuromuscular Module was used to assess the quality of life of the children in our study and was answered by the parents only. The scale is scored from 0 to 4, with higher scores indicating better quality of life.
Measured: Once, beginning of the study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2025
- Study start30 May 2024
- Primary completion15 Jun 2024
- Study completion30 Aug 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal Muscular Atrophy Type IStudies of the same condition, open and under way.
- 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.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Cross-sectional
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- Zeynep HOŞBAY
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Zeynep HOŞBAY, Biruni University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06864767
- Sponsor's protocol ID
- Oral Motor Functions in SMA
- Status last verified
- Mar 2025
- First submitted
- 4 Mar 2025
- Last updated
- 7 Mar 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Impact of Motor and Oral Motor Function on Quality of Life in Children With Type I Spinal Muscular Atrophy
- ClinicalTrials.gov ID
- NCT06864767
- Acronym / sponsor ID
- Oral Motor Functions in SMA
- Conditions
- Spinal Muscular Atrophy Type I
- Keywords
- Spinal Muscular Atrophy Type I, Oral Motor Function, Motor Function, Quality of Life
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Zeynep HOŞBAYUniversity, hospital or other
- Study start
- 30 May 2024 (actual)
- Primary completion
- 15 Jun 2024 (actual)
- Study completion
- 30 Aug 2024 (actual)
- First posted
- 7 Mar 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The aim of this study is to investigate the impact of motor and oral motor functions on the quality of life in children aged 2-4 years diagnosed with Spinal Muscular Atrophy (SMA) Type I. In the study, the Neuro-Sensory-Motor Developmental Assessment (NSMDA) was used to evaluate motor functions, the Functional Oral Intake Scale (FOIS) to assess oral motor function, the Behavioral Pediatric Feeding Assessment Scale (BPFAS) to evaluate children's attitudes towards feeding, and the Pediatric Quality of Life Inventory (PedsQL) Neuromuscular Module to assess quality of life.
Study design
- Observational model
- Other
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 2 to 4 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- The study was completed with 23 children between the ages of 2-4 who were followed up with the diagnosis of Spinal Muscular Atrophy (SMA) Type 1 and whose parents volunteered to participate in the study.
Key inclusion criteria (3 listed)
- Diagnosed with Spinal Muscular Atrophy Type I
- Being between the ages of 2-4
- Families volunteering to participate in the study
Key exclusion criteria (3 listed)
- Accompanied by another chronic disease other than SMA
- The mother or father has a cognitive problem that prevents them from expressing themselves
- The mother or father is illiterate
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Neuro-sensory Motor Developmental AssessmentThe test used to assess children's development levels evaluates the child's development in six parameters: gross motor function, fine motor function, neurological status, infant movement patterns, postural development and sensory-motor function (tactile, proprioceptive, ocular and vestibular systems) on a scale of 1 to 5. High scores are interpreted as motor dysfunction. | Other | – |
| Functional Oral Intake ScaleIt is a scale consisting of a total of 7 levels and two sections, developed by Crary et al. to indicate the functional oral intake of patients with dysphagia. A higher score indicates a better nutritional level. | Other | – |
| Behavioral Pediatrics Feeding Assessment ScaleThe scale used to determine eating problems in children evaluates both the child's nutritional status and the parents' feelings about the child's nutritional status. It consists of 35 items in total and is scored from 1 to 5. High scores indicate problematic eating behaviors and habits. | Other | – |
| Pediatric Quality of Life InventoryThe PedsQL assesses health-related quality of life in children ages 2 to 18 years with chronic illnesses from both the child's and parents' perspectives. It consists of 25 items and 3 categories (About My Child's Neuromuscular Disease, Communication, About Our Family Resources). The Neuromuscular Module was used to assess the quality of life of the children in our study and was answered by the parents only. The scale is scored from 0 to 4, with higher scores indicating better quality of life. | Other | – |
Primary outcome
Neuro-sensory Motor Developmental Assessment
Time frame: Once, beginning of the study
The test used to assess children's development levels evaluates the child's development in six parameters: gross motor function, fine motor function, neurological status, infant movement patterns, postural development and sensory-motor function (tactile, proprioceptive, ocular and vestibular systems) on a scale of 1 to 5. High scores are interpreted as motor dysfunction.
Functional Oral Intake Scale
Time frame: Once, beginning of the study
It is a scale consisting of a total of 7 levels and two sections, developed by Crary et al. to indicate the functional oral intake of patients with dysphagia. A higher score indicates a better nutritional level.
Behavioral Pediatrics Feeding Assessment Scale
Time frame: Once, beginning of the study
The scale used to determine eating problems in children evaluates both the child's nutritional status and the parents' feelings about the child's nutritional status. It consists of 35 items in total and is scored from 1 to 5. High scores indicate problematic eating behaviors and habits.
1 secondary outcome
Pediatric Quality of Life Inventory
Time frame: Once, beginning of the study
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Mar 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Spinal Muscular Atrophy Type I, run by Zeynep HOŞBAY at 1 site in Turkey (Türkiye).
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Turkey (Türkiye). Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Neuro-sensory Motor Developmental Assessment (time frame: Once, beginning of the study). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Mar 2025.