Rare DiseasesNCT04382820
Psychosocial Situation of Children With Rare (Congenital) Pediatric Surgical Diseases and Their Families
An observational study of Rare Diseases, run by Universitätsklinikum Hamburg-Eppendorf at 1 site in Germany.
Trial summary
Families of children with rare diseases (i.e., not more than 5 out of 10.000 people are affected) are often highly burdened with fears, insecurities and concerns regarding the affected child and his/her siblings. Read more about this study
- Who is it for?
- Ages 1 day to 21 yearsHealthy volunteers can take part
- Conditions the study is for
- Rare Diseases
- How many people
- 167 participants
- Where
- 1 siteGermany
- Study dates
- 1 Apr 2020to 1 Apr 2021
- How long will I take part?
- 1 Month
- 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 1 day to 21 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Study population: Clinical study participants for the diagnostic study are patients who have sought treatment at the University Medical Center Hamburg-Eppendorf due to a rare pediatric surgical disease. Participants in the healthy control sample are matched to the clinical sample in terms of age and gender. Included are families of children aged 0-21 years, who have undergone a surgical procedure at the University Medical Center Hamburg-Eppendorfin the first 3 years of life that does not cause chronic complaints; such as hernia surgery or testicular relocation.
Who may be able to join
- Family with at least one child between 0 and 21 years with a rare pediatric surgical disease.
- Consent to participate in the study.
- Sufficient knowledge of the German language of parents and children.
- Family with at least one child between 0 and 21 years who have undergone a surgical procedure in the first 3 years of life that does not cause chronic complaints
- Consent to participate in the study.
- Sufficient knowledge of the German language of parents and children.
Who cannot join
- Severe acute physical, mental and/or cognitive impairment of the child, so that the questionnaire survey does not appear possible and/or unreasonable at this stage.
- Families of children with a congenital or chronic disease.
Trial locations and contacts
1 site in 1 country.
About this study
Families of children with rare diseases (i.e., not more than 5 out of 10.000 people are affected) are often highly burdened with fears, insecurities and concerns regarding the affected child and his/her siblings. The aim of the present research project is to examine the psychosocial burden of the children with rare (congenital) pediatric surgical diseases and their family in order to draw attention to a possible psychosocial care gap in this population.
Read the detailed description
The central objective of the cross-sectional study is to show the psychosocial supply gap for families with children and adolescents affected by rare diseases in the field of pediatric surgery. Among the rare diseases that are included are diaphragmatic hernia, anorectal malformations, esophageal atresia, Hirschsprung's disease and biliary atresia. In order to have a comparative sample, additional data of a matched control group are collected. Central standardized psychosocial outcomes will be assessed from the perspectives of the parents, the affected child and the siblings.
- Conditions
- Rare Diseases
- Keywords
- rare diseases, pediatric surgery, children and adolescent, quality of life, psychological distress
- Medical subject headings
- Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Families of rare chronically ill children | Clinical study participants for the diagnostic study are patients who have sought treatment at the University Medical Center Hamburg-Eppendorf due to a rare pediatric surgical disease. Every family receives a comprehensive psychosocial diagnostic in the form of standardized instruments. | |
| Families in the comparative control group | Participants in the healthy control sample are matched to the clinical sample in terms of age and gender. Included are families of children aged 0-21 years, who have undergone a surgical procedure in the first 3 years of life that does not cause chronic complaints; such as hernia surgery or testicular relocation. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Quality of life of the parents (ULQIE)
Quality of life (QoL) of the parents, assessed from the perspective of the parents by the "Ulmer Lebensqualitätsinventar für Eltern chronisch kranker Kinder" (ULQIE; Goldbeck \& Storck, 2002). The instrument consists of 29 items, which are answered on a five-point Likert scale (0 to 4). Higher scores indicate greater QoL.
Measured: 4 minutes
Mental health of the parents (BSI)
Mental health of the parents, assessed from the perspective of the parents by the "Brief Symptom Inventory" (BSI; Franke, 2000). The instrument consists of 53 items, which are answered on a five-point Likert scale (0 to 4). Higher BSI scores indicate greater psychological distress.
Measured: 5 minutes
Health-related quality of life of the chronically-ill children/adolescents (Peds-QL 4.0)
Health-related quality of life of the chronically-ill children/adolescents, assessed from the perspective of the child/adolescent (from 10 years of age) and from the perspective of the parents by the "Pediatric Quality of Life Inventory 4.0" (Peds-QL 4.0; (Varni, Seid, \& Kurtin, 2001). Items will be linearly transformed to a scale of 0 to 100, with higher scores indicating better HRQoL.
Measured: 4 minutes
Psychiatric disorders of the chronically-ill children/adolescents and the siblings (SDQ)
Psychiatric disorders of the chronically-ill children/adolescents and the siblings assessed perspective of the child/adolescent (from 10 years of age) and from the perspective of the parents by the "Strengths and Difficulties Questionnaire" (SDQ; Klasen, Woerner, Rothenberger, \& Goodman, 2003). Items are rated on a three-point Likert scale (0 to 2). Higher scores represent greater psychopathology or greater prosocial behavior.
Measured: 4 minutes
Secondary outcomes (8)
Coping of the parents (CHIP-D)
Coping of the parents, assessed from the perspective of the parents by the German version of the "Coping Health Inventory for Parents" (CHIP-D; McCubbin, McCubbin, Cauble \& Goldbeck, 2001). Items are rated on a four-point Likert scale (0 to 3). Higher scores represent greater use of the respective coping pattern.
Measured: 3 minutes
Coping of the chronically-ill children/adolescents and the siblings (Kidcope)
Coping of the chronically-ill children/adolescents and the siblings, assessed from the perspective of the children/adolescents (from 10 years of age) by the "Kidcope Checklist" (Kidcope; Spirito, Stark \& Williams, 1988). Items related to frequency of the coping strategies are rated on a 4 point scale (0 = "Not at all" to 3 = "Almost all the time"), and items related to efficacy are rated on a 5 point scale (0 = "Not at all" to 4 = "Very much").
Measured: 3 minutes
Social support of the parents, of the chronically-ill children/adolescents and of the siblings (OSSS)
Social support of the parents, of the chronically-ill children/adolescents and of the siblings, assessed from the perspective of the parents, of the chronically-ill children/adolescents and from the sibling, respectively, by the "Oslo Social Support Scale" (OSSS; Dalgard, 2006). The sum score ranges from 3 to 14. The higher the sum score, the stronger the social support.
Measured: 3 minutes
Sociodemographic information of the parents
Sociodemographic information of the parents, assessed from the perspective of the parents by ad-hoc items.
Measured: 2 minutes
Relationships between siblings (SRQ)
Sibling relationship, assessed from the perspective of the siblings (from 10 years of age) by the "Sibling Relationship Questionnaire" (Bojanowski, Riestock, Nisslein, Weschenfelder-Stachwitz, \& Lehmkuhl, 2015). Each item is scored on a 5-point Likert scale
(1 to 5).
Measured: 3 minutes
Family relationships, family dynamics and functionality (FB-A)
Family relationships, family dynamics and functionality, assessed from the perspective of the parents, of the chronically-ill children/adolescents and from the sibling, respectively by "Familienbögen" (FB-A; Cierpka \& Frevert, 1994). Items are rated on a four-point Likert scale (0 to 3)
Measured: 3 minutes
Interpersonal problems (IIP-32)
Interpersonal problems assessed from the perspective of the parents by the "Inventory of Interpersonal Problems-32" (IIP-32; Thomas, Brähler, \& Strauß, 2011). Items are rated on a four-point Likert scale from 0 (not at all) to 4 (extremely). It provides an overall score and eight subscale scores.
Measured: 3 minutes
Familial predispositions (FaBel)
familial predispositions assessed from the perspective of the parents by the "Familien-Belastungs-Fragebogen" (FaBel; Ravens-Sieberer, 2001). Items are rated on a four-point Likert scale ranging from 1 (is not right at all) to 4 (is completely right).
Measured: 3 minutes
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 May 2020
- Study start1 Apr 2020
- Primary completion1 Apr 2021
- Study completion1 Apr 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Rare DiseasesStudies of the same condition, open and under way.
- More trials run by Universitätsklinikum Hamburg-EppendorfThe 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
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 167 participants (actual)
- Target duration
- 1 Month
Sponsor and investigators
- Lead sponsor
- Universitätsklinikum Hamburg-Eppendorf
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Johannes Boettcher, Universitätsklinikum Hamburg-Eppendorf
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04382820
- Sponsor's protocol ID
- PV7161
- Status last verified
- Nov 2021
- First submitted
- 4 May 2020
- Last updated
- 9 Dec 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: It is not yet known if there will be a plan to make IPD available.
Publications
Boettcher J, Hohmann S, Daubmann A, Denecke J, Muntau AC, Wiegand-Grefe S, Zapf H. The Family Stress Model in families of children with rare diseases: a cross-sectional multilevel path analysis for understanding family dynamics. Front Public Health. 2025 Nov 18;13:1713613. doi: 10.3389/fpubh.2025.1713613. eCollection 2025.
PubMed 41341441Boettcher J, Nazarian R, Fuerboeter M, Liedtke A, Wiegand-Grefe S, Reinshagen K, Boettcher M. Mental Health of Siblings of Children with Rare Congenital Surgical Diseases during the COVID-19 Pandemic. Eur J Pediatr Surg. 2022 Oct;32(5):422-428. doi: 10.1055/s-0041-1740978. Epub 2021 Dec 31.
PubMed 34972234
Study at a glance
From the registry record, last updated by the sponsor on 9 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Psychosocial Situation of Children With Rare (Congenital) Pediatric Surgical Diseases and Their Families
- ClinicalTrials.gov ID
- NCT04382820
- Acronym / sponsor ID
- PV7161
- Conditions
- Rare Diseases
- Keywords
- rare diseases, pediatric surgery, children and adolescent, quality of life, psychological distress
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 167 participants (actual)
- Lead sponsor
- Universitätsklinikum Hamburg-EppendorfUniversity, hospital or other
- Study start
- 1 Apr 2020 (actual)
- Primary completion
- 1 Apr 2021 (actual)
- Study completion
- 1 Apr 2021 (actual)
- First posted
- 11 May 2020 (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.
Families of children with rare diseases (i.e., not more than 5 out of 10.000 people are affected) are often highly burdened with fears, insecurities and concerns regarding the affected child and his/her siblings. The aim of the present research project is to examine the psychosocial burden of the children with rare (congenital) pediatric surgical diseases and their family in order to draw attention to a possible psychosocial care gap in this population.
Detailed description
The central objective of the cross-sectional study is to show the psychosocial supply gap for families with children and adolescents affected by rare diseases in the field of pediatric surgery. Among the rare diseases that are included are diaphragmatic hernia, anorectal malformations, esophageal atresia, Hirschsprung's disease and biliary atresia. In order to have a comparative sample, additional data of a matched control group are collected. Central standardized psychosocial outcomes will be assessed from the perspectives of the parents, the affected child and the siblings.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Target follow-up
- 1 Month
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Families of rare chronically ill children | – | –Clinical study participants for the diagnostic study are patients who have sought treatment at the University Medical Center Hamburg-Eppendorf due to a rare pediatric surgical disease. Every family receives a comprehensive psychosocial diagnostic in the form of standardized instruments. |
| Families in the comparative control group | – | –Participants in the healthy control sample are matched to the clinical sample in terms of age and gender. Included are families of children aged 0-21 years, who have undergone a surgical procedure in the first 3 years of life that does not cause chronic complaints; such as hernia surgery or testicular relocation. |
Who may qualify
- Ages
- 1 day to 21 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- Clinical study participants for the diagnostic study are patients who have sought treatment at the University Medical Center Hamburg-Eppendorf due to a rare pediatric surgical disease. Participants in the healthy control sample are matched to the clinical sample in terms of age and gender. Included are families of children aged 0-21 years, who have undergone a surgical procedure at the University Medical Center Hamburg-Eppendorfin the first 3 years of life that does not cause chronic complaints; such as hernia surgery or testicular relocation.
Key inclusion criteria (6 listed)
- Family with at least one child between 0 and 21 years with a rare pediatric surgical disease.
- Consent to participate in the study.
- Sufficient knowledge of the German language of parents and children.
- Family with at least one child between 0 and 21 years who have undergone a surgical procedure in the first 3 years of life that does not cause chronic complaints
- Consent to participate in the study.
Key exclusion criteria (2 listed)
- Severe acute physical, mental and/or cognitive impairment of the child, so that the questionnaire survey does not appear possible and/or unreasonable at this stage.
- Families of children with a congenital or chronic disease.
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
Quality of life of the parents (ULQIE)
Time frame: 4 minutes
Quality of life (QoL) of the parents, assessed from the perspective of the parents by the "Ulmer Lebensqualitätsinventar für Eltern chronisch kranker Kinder" (ULQIE; Goldbeck \& Storck, 2002). The instrument consists of 29 items, which are answered on a five-point Likert scale (0 to 4). Higher scores indicate greater QoL.
Mental health of the parents (BSI)
Time frame: 5 minutes
Mental health of the parents, assessed from the perspective of the parents by the "Brief Symptom Inventory" (BSI; Franke, 2000). The instrument consists of 53 items, which are answered on a five-point Likert scale (0 to 4). Higher BSI scores indicate greater psychological distress.
Health-related quality of life of the chronically-ill children/adolescents (Peds-QL 4.0)
Time frame: 4 minutes
Health-related quality of life of the chronically-ill children/adolescents, assessed from the perspective of the child/adolescent (from 10 years of age) and from the perspective of the parents by the "Pediatric Quality of Life Inventory 4.0" (Peds-QL 4.0; (Varni, Seid, \& Kurtin, 2001). Items will be linearly transformed to a scale of 0 to 100, with higher scores indicating better HRQoL.
Psychiatric disorders of the chronically-ill children/adolescents and the siblings (SDQ)
Time frame: 4 minutes
Psychiatric disorders of the chronically-ill children/adolescents and the siblings assessed perspective of the child/adolescent (from 10 years of age) and from the perspective of the parents by the "Strengths and Difficulties Questionnaire" (SDQ; Klasen, Woerner, Rothenberger, \& Goodman, 2003). Items are rated on a three-point Likert scale (0 to 2). Higher scores represent greater psychopathology or greater prosocial behavior.
8 secondary outcomes
Coping of the parents (CHIP-D)
Time frame: 3 minutes
Coping of the chronically-ill children/adolescents and the siblings (Kidcope)
Time frame: 3 minutes
Social support of the parents, of the chronically-ill children/adolescents and of the siblings (OSSS)
Time frame: 3 minutes
Sociodemographic information of the parents
Time frame: 2 minutes
Relationships between siblings (SRQ)
Time frame: 3 minutes
Family relationships, family dynamics and functionality (FB-A)
Time frame: 3 minutes
Interpersonal problems (IIP-32)
Time frame: 3 minutes
Familial predispositions (FaBel)
Time frame: 3 minutes
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 9 Dec 2021.
- Derived from this study: PubMed 41341441Boettcher J, Hohmann S, Daubmann A, Denecke J, Muntau AC, Wiegand-Grefe S, Zapf H. The Family Stress Model in families of children with rare diseases: a cross-sectional multilevel path analysis for understanding family dynamics. Front Public Health. 2025 Nov 18;13:1713613. doi: 10.3389/fpubh.2025.1713613. eCollection 2025.
- Derived from this study: PubMed 34972234Boettcher J, Nazarian R, Fuerboeter M, Liedtke A, Wiegand-Grefe S, Reinshagen K, Boettcher M. Mental Health of Siblings of Children with Rare Congenital Surgical Diseases during the COVID-19 Pandemic. Eur J Pediatr Surg. 2022 Oct;32(5):422-428. doi: 10.1055/s-0041-1740978. Epub 2021 Dec 31.
- Individual participant dataSharing planned: Undecided. It is not yet known if there will be a plan to make IPD available.
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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 Rare Diseases, run by Universitätsklinikum Hamburg-Eppendorf at 1 site in Germany.
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 6 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: finished, as of the sponsor's last update on 9 Dec 2021. 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 Germany. 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?
Quality of life of the parents (ULQIE) (time frame: 4 minutes). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04382820, which also lists 2 linked publications.
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 9 Dec 2021.