Spina BifidaNCT06723951
QUALAS Validation in Dutch
An observational study of Spina Bifida, run by Erasmus Medical Center at 2 sites in Netherlands.
Trial summary
Rationale Patients with spina bifida often experience a decreased quality of life compared to patients without spina bifida. The Quality of Life Assessment for Spina Bifida (QUALAS) is a validated questionnaire to assess the quality of life in children, teenagers and adults with spina bifida, accounting for specific… Read more about this study
- Who is it for?
- Ages 8 years and olderHealthy volunteers can take part
- Conditions the study is for
- Spina Bifida
- What is being tested?
- QUALAS survey
- How many people
- 300 participants
- Where
- 2 sitesNetherlands
- Study dates
- 9 Dec 2024to 31 Mar 2025
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 8 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: Parents of children and children and adults with spina bifida that are patients at the Urology department of the two hospitals will be approached to participate. This questionnaire quantifies the quality of life in patients with spina bifida, with a special interest in bladder and bowel dysfunction. A group of children and adults without spina bifida will function as a control group. These children and adults will be approached at the ENT and paediatric clinics.
Who may be able to join
- Patient group:
- Male or female patients aged 8 -12 years (QUALAS-C)
- Male or female patients aged 13 - 17 years (QUALAS-T)
- Adult patients (QUALAS-A)
- Has spina bifida
- Adult or child and at least one parent fluent in the Dutch language
Show all 14Show fewer
- Signed informed consent
- Control group:
- Male or female children aged 8 -12 years (QUALAS-C)
- Male or female adolescents aged 13 - 17 years (QUALAS-T)
- Adults (QUALAS-A)
- Has no spina bifida
- Adult or child and at least one parent fluent in the Dutch language
- Signed informed consent
Who cannot join
- Has a neurogenic disease other than spina bifida
- Has had surgery in the last month
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Netherlands2 sites, 2 recruiting
- Not yet recruiting
UMCG
Groningen
Rianne L.M. Lammers, 0031503612167, rianne_lammers@hotmail.com
- Recruiting
Erasmus MC
Rotterdam, 3015 GD
Lisette 't Hoen, MD, PhD, 0031107030073, l.thoen@erasmusmc.nl
About this study
Official title: Validation of the Quality of Life Assessment in Spina Bifida for Children and Adolescents in Dutch
Rationale Patients with spina bifida often experience a decreased quality of life compared to patients without spina bifida. The Quality of Life Assessment for Spina Bifida (QUALAS) is a validated questionnaire to assess the quality of life in children, teenagers and adults with spina bifida, accounting for specific factors, such as bladder and bowel dysfunction. This questionnaire enables us to assess the current spina bifida related quality of life and identify if bladder and bowel dysfunction is of influence.
Objective(s) To validate the Quality of Life Assessment for Spina Bifida in Children, Teenagers and Adults in Dutch.
Read more
Study type A multicenter prospective validation cohort study.
Study population Children aged 8 - 17 years and adults who are diagnosed with spina bifida. As a control group children aged 8 - 17 years and adults without spina bifida will be approached at the Ear-Nose-Throat (ENT) and paediatric clinics.
Methods The main study parameter is the validation of the Dutch version of the questionnaire. Patients with spina bifida are asked to fill out the QUALAS at two time-points. They are also asked to fill out an extra questionnaire at two time-points, in order to validate the QUALAS. Children and adults in the control groups are asked to fill out the two questionnaires only once.
Burden and risks No (extra) outpatient visit is required. The questions in the questionnaires are similar to the medical history during an outpatient visit. The burden is therefore minimal for these patients. Participation in this study will not influence patient treatment.
Recruitment and consent Parents of children and children and adults who meet the inclusion criteria and none of the exclusion criteria will be informed orally about this study by their treating urologist during a regular visit. A patient information and consent form will be handed over to the parents and children over eight years of age and adults. Parents of children and children and adults at the ENT clinic will be approached by the local urologist for participation for this study. If they are willing to participate they will be asked to sign informed consent and fill out the questionnaire.
Read the detailed description
Introduction and rationale Patients with spina bifida often face challenges in different areas. It is common to have a dedicated team of medical specialists for yearly follow-up. This team includes paediatricians, neurosurgeons, orthopaedics, urologists and rehabilitation specialists. Patients with spina bifida may experience symptoms in each of these areas which will have an impact of these patient's quality of life. From the urologist perspective urinary incontinence and faecal incontinence have a high impact on quality of life.
Questionnaires to evaluate quality of life in patients with spina bifida do not incorporate these specific symptoms. The QUAlity of Life Assessment in Spina bifida in Children (QUALAS-C), QUAlity of Life Assessment in Spina bifida in Teenagers (QUALAS-T) and The QUAlity of Life Assessment in Spina bifida in Adults (QUALAS-A) have been developed to take these symptoms into account when assessing quality of life. These questionnaires are validated in the United States. For children aged 8-12 years the QUALAS-C version is available. For children between 13-17 years of age the QUALAS-T version is available. For adults the QUALAS-A version is available.
Currently, no versions of the QUALAS-C, QUALAS-T, QUALAS-A questionnaires are available in Dutch.
With the QUALAS-C, QUALAS-T and QUALAS-A the investigators will be able to assess spina bifida related quality of life and monitor the development over time. This will enable clinicians to offer improved standardized care.
Objective(s) The aim of this study is to validate the QUALAS questionnaire for children, teenagers and adults in Dutch.
Methods This is a multicenter prospective cohort validation study of the QUALAS questionnaire. This is a spina bifida related quality of life questionnaire. The investigators will validate the QUALAS using the validated KIDSCREEN-27 for children 8-17 years of age and the World Health Organization Quality of Life instrument (WHOQOL-BREF) and (International Consultation on Incontinence Questionnaire) ICIQ for adults questionnaires to determine spina bifida related quality of life in a standardized manner.
All patients are subject to treatment at the department of Paediatric Urology in Erasmus MC-Sophia or University Medical Center Groningen. Participation in this study will not interfere with their treatment.
For this study two different phases are distinguished:
- Phase 1: The QUALAS will be translated following a standardized translation process. This includes three forward translations by Dutch native speakers, one backward translation by a English native speaker and face-to-face testing in the target population.
- Phase 2: Children of 8 years and older and adults are asked to fill out the questionnaires, QUALAS and KIDSCREEN for children 8-17 years of age and the WHOQOL-BREF and ICIQ for adults, and repeat filling out the QUALAS questionnaire one week later. The team asks all parents to fill out the questionnaire in order to secure a constant answer during the study. Children of 8 years and older and adults in the control group are asked to fill out the QUALAS and KIDSCREEN-27 for children 8-17 years of age and the WHOQOL-BREF and ICIQ for adults. Internal consistency, criterion validity, construct validity an reproducibility will be determined.
- Conditions
- Spina Bifida
- Being tested
- QUALAS survey
- Medical subject headings
- Spinal Dysraphism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
QUALAS surveyOther
Also called KIDSCREEN survey for children and teenagers, World Health Organization Quality of Life instrument (WHOQOL-BREF) and (International Consultation on Incontinence Questionnaire) ICIQ for adults
Participants will be asked to complete the surveys at one (controls) and two (patients) timepoints.
| Study group | Role | What they receive |
|---|---|---|
| Children with spina bifida | • Male or female aged 8 -12 years with spina bifida Receives: Other: QUALAS survey | |
| Control group children | • Male or females aged 8 -12 years without spina bifida Receives: Other: QUALAS survey | |
| Teenagers with spina bifida | • Males or female aged 13 - 17 years with spina bifida Receives: Other: QUALAS survey | |
| Control group teenagers | • Males or females aged 13 - 17 years without spina bifida Receives: Other: QUALAS survey | |
| Adults with spina bifida | Adults with spina bifida Receives: Other: QUALAS survey | |
| Control group adults | Adults without spina bifida Receives: Other: QUALAS survey |
What is being measured
The primary outcome is the main question the study is designed to answer.
Internal consistency of QUALAS
Internal consistency is the main study endpoint. This will be calculated with Cronbach's alpha. Values between 0.70 and 0.95 reflect good consistency.
QUALAS questionnaire:
Scores range from 0-100 with higher scores indicating better quality of life.
Measured: 3 months
Secondary outcomes (3)
Reproducibility
Reproducibility will be determined to assess the ability to reproduce similar answers during the retest period. This will be calculated using Intraclass Correlation Coefficient (ICC), where ICC scores over 0.7 are acceptable. The limits of agreement (LOA), which is calculated by mean change in scores ± 1.96 SD are determined as well.
Measured: 2 weeks
Content validity
Content validity is subjectively determined by examining whether the questionnaire is measuring the quality of life related to spina bifida.
Measured: 3 months
Construct validity
Construct validity is determined by testing the following hypotheses:
- The control group will have higher scores in the QUALAS than patients with spina bifida.
- Patients who score higher in the KIDSCREEN-27 or WHOQOL-BREF/ICIQ will have higher scores in the QUALAS.
The KIDSCREEN-27: Each item is scored on a 5-point Likert scale ranging from 1 meaning "not at all" to 5 meaning "very much". Certain items (1, 9, 10 and 11) were reversed when scoring the questionnaire. Reverse scoring means that the numerical scoring scale runs in the opposite direction. The total KIDSCREEN score was calculated by summing up all the responses. Higher scores of KIDSCREEN indicated better QoL and social support.
The WHOQOL-BREF questionnaire: 26 items are scored on a 5-point Likert scale. Scores range from 0-100 with higher scores indicating better quality of life
The ICIQ questionnaire: 3 items are scored with a total sum range of 0-21, higher score indicate worse quality of life.
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2024
- Study start9 Dec 2024 (estimated)
- Primary completion28 Feb 2025 (estimated)
- Study completion31 Mar 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spina BifidaStudies of the same condition, open and under way.
- More trials run by Erasmus Medical CenterThe 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.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 300 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Erasmus Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- University Medical Center Groningen
- Responsible party
- Principal investigator, Lisette 't Hoen, Erasmus Medical Center
- Investigators
- Lisette 't Hoen, MD, PhD, Principal investigator, Erasmus Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06723951
- Sponsor's protocol ID
- EMCSophia2
- Status last verified
- Dec 2024
- First submitted
- 4 Dec 2024
- Last updated
- 12 Dec 2024
- Results posted
- No
- Data monitoring committee
- 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 12 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Validation of the Quality of Life Assessment in Spina Bifida for Children and Adolescents in Dutch
- ClinicalTrials.gov ID
- NCT06723951
- Acronym / sponsor ID
- EMCSophia2
- Conditions
- Spina Bifida
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- Erasmus Medical CenterUniversity, hospital or other
- Collaborators
- University Medical Center Groningen
- Study start
- 9 Dec 2024 (estimated)
- Primary completion
- 28 Feb 2025 (estimated)
- Study completion
- 31 Mar 2025 (estimated)
- First posted
- 9 Dec 2024 (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.
Rationale Patients with spina bifida often experience a decreased quality of life compared to patients without spina bifida. The Quality of Life Assessment for Spina Bifida (QUALAS) is a validated questionnaire to assess the quality of life in children, teenagers and adults with spina bifida, accounting for specific factors, such as bladder and bowel dysfunction. This questionnaire enables us to assess the current spina bifida related quality of life and identify if bladder and bowel dysfunction is of influence.
Objective(s) To validate the Quality of Life Assessment for Spina Bifida in Children, Teenagers and Adults in Dutch.
Study type A multicenter prospective validation cohort study.
Study population Children aged 8 - 17 years and adults who are diagnosed with spina bifida. As a control group children aged 8 - 17 years and adults without spina bifida will be approached at the Ear-Nose-Throat (ENT) and paediatric clinics.
Methods The main study parameter is the validation of the Dutch version of the questionnaire. Patients with spina bifida are asked to fill out the QUALAS at two time-points. They are also asked to fill out an extra questionnaire at two time-points, in order to validate the QUALAS. Children and adults in the control groups are asked to fill out the two questionnaires only once.
Burden and risks No (extra) outpatient visit is required. The questions in the questionnaires are similar to the medical history during an outpatient visit. The burden is therefore minimal for these patients. Participation in this study will not influence patient treatment.
Recruitment and consent Parents of children and children and adults who meet the inclusion criteria and none of the exclusion criteria will be informed orally about this study by their treating urologist during a regular visit. A patient information and consent form will be handed over to the parents and children over eight years of age and adults. Parents of children and children and adults at the ENT clinic will be approached by the local urologist for participation for this study. If they are willing to participate they will be asked to sign informed consent and fill out the questionnaire.
Detailed description
Introduction and rationale Patients with spina bifida often face challenges in different areas. It is common to have a dedicated team of medical specialists for yearly follow-up. This team includes paediatricians, neurosurgeons, orthopaedics, urologists and rehabilitation specialists. Patients with spina bifida may experience symptoms in each of these areas which will have an impact of these patient's quality of life. From the urologist perspective urinary incontinence and faecal incontinence have a high impact on quality of life.
Questionnaires to evaluate quality of life in patients with spina bifida do not incorporate these specific symptoms. The QUAlity of Life Assessment in Spina bifida in Children (QUALAS-C), QUAlity of Life Assessment in Spina bifida in Teenagers (QUALAS-T) and The QUAlity of Life Assessment in Spina bifida in Adults (QUALAS-A) have been developed to take these symptoms into account when assessing quality of life. These questionnaires are validated in the United States. For children aged 8-12 years the QUALAS-C version is available. For children between 13-17 years of age the QUALAS-T version is available. For adults the QUALAS-A version is available.
Currently, no versions of the QUALAS-C, QUALAS-T, QUALAS-A questionnaires are available in Dutch.
With the QUALAS-C, QUALAS-T and QUALAS-A the investigators will be able to assess spina bifida related quality of life and monitor the development over time. This will enable clinicians to offer improved standardized care.
Objective(s) The aim of this study is to validate the QUALAS questionnaire for children, teenagers and adults in Dutch.
Methods This is a multicenter prospective cohort validation study of the QUALAS questionnaire. This is a spina bifida related quality of life questionnaire. The investigators will validate the QUALAS using the validated KIDSCREEN-27 for children 8-17 years of age and the World Health Organization Quality of Life instrument (WHOQOL-BREF) and (International Consultation on Incontinence Questionnaire) ICIQ for adults questionnaires to determine spina bifida related quality of life in a standardized manner.
All patients are subject to treatment at the department of Paediatric Urology in Erasmus MC-Sophia or University Medical Center Groningen. Participation in this study will not interfere with their treatment.
For this study two different phases are distinguished:
- Phase 1: The QUALAS will be translated following a standardized translation process. This includes three forward translations by Dutch native speakers, one backward translation by a English native speaker and face-to-face testing in the target population.
- Phase 2: Children of 8 years and older and adults are asked to fill out the questionnaires, QUALAS and KIDSCREEN for children 8-17 years of age and the WHOQOL-BREF and ICIQ for adults, and repeat filling out the QUALAS questionnaire one week later. The team asks all parents to fill out the questionnaire in order to secure a constant answer during the study. Children of 8 years and older and adults in the control group are asked to fill out the QUALAS and KIDSCREEN-27 for children 8-17 years of age and the WHOQOL-BREF and ICIQ for adults. Internal consistency, criterion validity, construct validity an reproducibility will be determined.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Children with spina bifida | – | Other: QUALAS survey• Male or female aged 8 -12 years with spina bifida |
| Control group children | – | Other: QUALAS survey• Male or females aged 8 -12 years without spina bifida |
| Teenagers with spina bifida | – | Other: QUALAS survey• Males or female aged 13 - 17 years with spina bifida |
| Control group teenagers | – | Other: QUALAS survey• Males or females aged 13 - 17 years without spina bifida |
| Adults with spina bifida | – | Other: QUALAS surveyAdults with spina bifida |
| Control group adults | – | Other: QUALAS surveyAdults without spina bifida |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Parents of children and children and adults with spina bifida that are patients at the Urology department of the two hospitals will be approached to participate. This questionnaire quantifies the quality of life in patients with spina bifida, with a special interest in bladder and bowel dysfunction. A group of children and adults without spina bifida will function as a control group. These children and adults will be approached at the ENT and paediatric clinics.
Key inclusion criteria (14 listed)
- Male or female patients aged 8 -12 years (QUALAS-C)
- Male or female patients aged 13 - 17 years (QUALAS-T)
- Adult patients (QUALAS-A)
- Has spina bifida
- Adult or child and at least one parent fluent in the Dutch language
Key exclusion criteria (2 listed)
- Has a neurogenic disease other than spina bifida
- Has had surgery in the last month
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 |
|---|---|---|
| QUALAS surveyAlso: KIDSCREEN survey for children and teenagers, World Health Organization Quality of Life instrument (WHOQOL-BREF) and (International Consultation on Incontinence Questionnaire) ICIQ for adultsParticipants will be asked to complete the surveys at one (controls) and two (patients) timepoints. | Other | Adults with spina bifida, Children with spina bifida, Control group adults, Control group children, Control group teenagers, Teenagers with spina bifida |
Primary outcome
Internal consistency of QUALAS
Time frame: 3 months
Internal consistency is the main study endpoint. This will be calculated with Cronbach's alpha. Values between 0.70 and 0.95 reflect good consistency. QUALAS questionnaire: Scores range from 0-100 with higher scores indicating better quality of life.
3 secondary outcomes
Reproducibility
Time frame: 2 weeks
Content validity
Time frame: 3 months
Construct validity
Time frame: 3 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Dec 2024.
- 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.
Lisette 't Hoen, MD, PhD principal investigator
Erasmus Medical Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Spina Bifida, run by Erasmus Medical Center at 2 sites in Netherlands.
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 14 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: looking for participants now, as of the sponsor's last update on 12 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Netherlands. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Lisette 't Hoen, MD, PhD (0031107030073, l.thoen@erasmusmc.nl), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Internal consistency of QUALAS (time frame: 3 months). 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 12 Dec 2024.