Spinal Muscular AtrophyNCT05539456
Reliability and Validity of the Turkish Version of the PedsQL 3.0 Neuromuscular Module for 2-to 4- Year-old
An observational study of Spinal Muscular Atrophy and Quality of Life, run by Istanbul Medipol University Hospital at 1 site in Turkey (Türkiye).
Trial summary
The aim of the investigator's study was to investigate translating the PedsQL 3.0 Neuromuscular Module for 2-to 4- Year-old and using it in clinics reliably and validity with a Turkish version of the PedsQL Generic Core (Pediatric Quality of Life Questionnare) in children with Spinal Muscular Atrophy in Turkey Read more about this study
- Who is it for?
- Ages 2 to 4 years
- Conditions the study is for
- Spinal Muscular Atrophy, Quality of Life, Neuromuscular Diseases
- How many people
- 55 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- 1 Sep 2022to 1 Nov 2022
- 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: Children with spinal muscular atrophy between 2 to 4 years age.
Who may be able to join
- Having a child with spinal muscular atrophy
- Having a child between the ages of 2-4
- Accept to participate in the study
Who cannot join
- Lack of cooperation during work
- Presence of disease other than SMA, which will prevent participation in the study
Trial locations and contacts
1 site in 1 country.
Turkey (Türkiye)1 site
Seval Kutlutürk Yıkılmaz
Istanbul, Kavacık, 34810
About this study
Official title: Reliability and Validity of the Turkish Version of the PedsQL 3.0 Neuromuscular Module for 2-to 4- Year-old: a Quality-of-life Measure for Patients With Spinal Muscular Atrophy in Turkey
The aim of the investigator's study was to investigate translating the PedsQL 3.0 Neuromuscular Module for 2-to 4- Year-old and using it in clinics reliably and validity with a Turkish version of the PedsQL Generic Core (Pediatric Quality of Life Questionnare) in children with Spinal Muscular Atrophy in Turkey
Read the detailed description
The PedsQL 3.0 Neuromuscular Module for 2-to 4- year-old will be translated into Turkish by following the language translation steps. First of all, the English form will be translated into Turkish by two people who are fluent in English. Then, the forms translated by another expert in the field will be reviewed. The revised text will be translated from Turkish into English by another person with a good command of English and will be compared with the original scale by the lecturer. If a significant change in meaning is not detected, the scale will be applied to 10 parents. In case of incomprehensible questions, which are reported to be lacking in expression, the scale will be finalized after revision and corrections are made by the research team.
- Keywords
- Spinal Muscular Atrophy, Quality of Life, Neuromuscular Diseases
- Medical subject headings
- Muscular Atrophy, Spinal, Neuromuscular Diseases
What is being measured
The primary outcome is the main question the study is designed to answer.
PedsQLTM 3.0 neuromuscular module for 2-to 4- Year-old
The module encompasses three scales:
- About My/My Child's Neuromuscular Disease (17 items),
- Communication (3 items),
- About Our Family Resources (5 items).
The scale is comprised a parent proxy-report format for children ages 2 to 4 years.
Items in all forms are essentially identical but contain slightly different language for first or third person tense.
The participants are asked how much of a problem each item had been during the past month.
Responses are rated on a 5-point Likert scale across child self-report for children, teens, and parent proxy-reports (0 = never a problem, 1 = almost never a problem, 2 = sometimes a problem, 3 = often a problem, 4 = almost always a problem).
Items are linearly transformed to a 0 to 100 scale (0 = 100, 1 = 75, 2 = 50, 3 = 25, and 4 = 0) so that higher scores indicate better HRQOL.
Measured: 5-10 minutes
Secondary outcomes (1)
PedsQLTM 4.0 generic core scales
The 23-item PedsQLTM 4.0 Generic Core Scales encompass:
- Physical Functioning (8 items),
- Emotional Functioning (5 items),
- Social Functioning (5 items),
- School Functioning (5 items)
The formats, instructions, Likert scales, and scoring methods are the same as those of the PedsQLTM 3.0 Neuromuscular Module. To create the Psychosocial Health Summary Score, the mean is computed as the sum of the items divided by the number of items answered in the Emotional, Social, and School Functioning Subscales.
Measured: 5-10 minutes
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Sep 2022
- Study start1 Sep 2022
- Primary completion1 Oct 2022 (estimated)
- Study completion1 Nov 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal Muscular AtrophyStudies 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 55 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Istanbul Medipol University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Seval Kutlutürk Yıkılmaz, Principal investigator, Medipol University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05539456
- Sponsor's protocol ID
- E-10840098-772.02-213
- Status last verified
- Sep 2022
- First submitted
- 12 Sep 2022
- Last updated
- 19 Sep 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Reliability and Validity of the Turkish Version of the PedsQL 3.0 Neuromuscular Module for 2-to 4- Year-old: a Quality-of-life Measure for Patients With Spinal Muscular Atrophy in Turkey
- ClinicalTrials.gov ID
- NCT05539456
- Acronym / sponsor ID
- E-10840098-772.02-213
- Keywords
- Spinal Muscular Atrophy, Quality of Life, Neuromuscular Diseases
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 55 participants (estimated)
- Lead sponsor
- Istanbul Medipol University HospitalUniversity, hospital or other
- Study start
- 1 Sep 2022 (actual)
- Primary completion
- 1 Oct 2022 (estimated)
- Study completion
- 1 Nov 2022 (estimated)
- First posted
- 14 Sep 2022 (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 the investigator's study was to investigate translating the PedsQL 3.0 Neuromuscular Module for 2-to 4- Year-old and using it in clinics reliably and validity with a Turkish version of the PedsQL Generic Core (Pediatric Quality of Life Questionnare) in children with Spinal Muscular Atrophy in Turkey
Detailed description
The PedsQL 3.0 Neuromuscular Module for 2-to 4- year-old will be translated into Turkish by following the language translation steps. First of all, the English form will be translated into Turkish by two people who are fluent in English. Then, the forms translated by another expert in the field will be reviewed. The revised text will be translated from Turkish into English by another person with a good command of English and will be compared with the original scale by the lecturer. If a significant change in meaning is not detected, the scale will be applied to 10 parents. In case of incomprehensible questions, which are reported to be lacking in expression, the scale will be finalized after revision and corrections are made by the research team.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 2 to 4 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Children with spinal muscular atrophy between 2 to 4 years age.
Key inclusion criteria (3 listed)
- Having a child with spinal muscular atrophy
- Having a child between the ages of 2-4
- Accept to participate in the study
Key exclusion criteria (2 listed)
- Lack of cooperation during work
- Presence of disease other than SMA, which will prevent participation in the study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
PedsQLTM 3.0 neuromuscular module for 2-to 4- Year-old
Time frame: 5-10 minutes
The module encompasses three scales: 1. About My/My Child's Neuromuscular Disease (17 items), 2. Communication (3 items), 3. About Our Family Resources (5 items). The scale is comprised a parent proxy-report format for children ages 2 to 4 years. Items in all forms are essentially identical but contain slightly different language for first or third person tense. The participants are asked how much of a problem each item had been during the past month. Responses are rated on a 5-point Likert scale across child self-report for children, teens, and parent proxy-reports (0 = never a problem, 1 = almost never a problem, 2 = sometimes a problem, 3 = often a problem, 4 = almost always a problem). Items are linearly transformed to a 0 to 100 scale (0 = 100, 1 = 75, 2 = 50, 3 = 25, and 4 = 0) so that higher scores indicate better HRQOL.
1 secondary outcome
PedsQLTM 4.0 generic core scales
Time frame: 5-10 minutes
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2022.
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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.
Seval Kutlutürk Yıkılmaz principal investigator
Medipol University
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 and Quality of Life, run by Istanbul Medipol University Hospital 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 2 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: status not confirmed, as of the sponsor's last update on 19 Sep 2022. 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?
Results are not available in the source record.
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?
PedsQLTM 3.0 neuromuscular module for 2-to 4- Year-old (time frame: 5-10 minutes). 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 19 Sep 2022.