A Longitudinal Study on Family Adaptation and Relationship Dynamics in Pediatric Rare Diseases (NCT07497581) | Clinical Trial Compass
Not Yet RecruitingNot Applicable
A Longitudinal Study on Family Adaptation and Relationship Dynamics in Pediatric Rare Diseases
Switzerland240 participantsStarted 2026-04-01
Plain-language summary
Rare diseases in children can affect not only the child's health but also the well-being and relationships within the entire family. Parents often experience stress, uncertainty, and emotional strain, which may in turn influence their child's mental health and quality of life. However, little is known about how families adapt over time to living with a rare disease or how daily experiences and family interactions shape this process.
This study aims to better understand how children with rare diseases and their caregivers adjust psychologically and emotionally over time. It will examine how factors such as parental stress, uncertainty, coping strategies, and family communication are linked to the mental health and quality of life of both children and parents.
The study will include children and adolescents (ages 1-18) with a diagnosed rare disease and their caregivers. Participants will complete online questionnaires at four time points over one year. A subgroup of families will also take part in a two-week smartphone-based assessment, where parents report their daily experiences, such as stress, emotions, and worries, several times per day. Some children and adolescents will additionally participate in interviews to share their own perspectives.
The main outcomes of interest are the child's mental health and quality of life. The study will also assess parental well-being and family functioning to understand how these factors influence each other over time.
By combining long-term and daily data, this study will provide a detailed picture of how families cope with rare diseases in everyday life. The findings may help improve psychological support and guide the development of targeted interventions for families affected by rare pediatric conditions.
Who can participate
Age range
1 Year – 18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
For children and adolescents:
* Diagnosed with an RD listed in Table 2.
* Age between 8 and 18 years.
* Sufficient knowledge of the German language.
* Declaration of informed consent must be signed by at least one legal guardian for all participants under 18; adolescents aged 14 - 17 additionally provide their own written consent.
* Lives at home (defined as sleeping inside of the home for more than four days per week, including weekends).
For parents:
* The child is affected by an RD listed in Table 2.
* The child's age is between 1 and 18 years.
* Sufficient knowledge of the German or English language.
* Availability of the signed declaration of informed consent.
* Only one child with a diagnosed RD.
* Up to two caregivers may participate per child. A stepparent may be included if they reside with the child, defined as spending at least three nights per week (including weekends) in the same household.
* Requirement ESM: Child lives at home (defined as sleeping inside of the home for more than four days per week, including weekends).
Exclusion Criteria:
For children and adolescents:
* The child is affected by an RD other than listed in Table 2.
* Age under 8 or over 18 years.
* Not sufficient knowledge of the German language.
* Lack of informed consent from at least one legal guardian (required for all participants under 18); and, for adolescents aged 14 - 17, absence of adolescent's own written consent.
* Lives outside of the home (defined as slee…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Child Mental Health and Health-Related Quality of Life
Timeframe: Baseline, 3 months, 6 months, and 12 months