Children Affected by Rare Disease and Their Families Network

Status

UNKNOWN

Phases

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT04339465

Acronym

CARE-FAM-NET

Enrollment

687

Registered

2020-04-09

Start date

2019-01-01

Completion date

2022-12-31

Last updated

2022-09-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Rare Diseases

Keywords

rare disease, children and adolescents, e-mental health, early detection and early treatment of mental disorders

Brief 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. The project at hand will test two innovative forms of care (CARE-FAM and WEP-CARE) at 17 sites in 12 federal states of Germany. The goal is to improve the mental health and quality of life of children affected by rare diseases and their relatives in a sustainable manner. If successful, these interventions will be introduced into regular care.

Detailed description

The central objective of the study at hand is to close the supply gap for families with children and adolescents affected by rare diseases. Two innovative forms of care (CARE-FAM and WEP-CARE) will be implemented and evaluated at the 18 participating study sites. Both interventions include psychological diagnostics, early detection and treatment of concomitant mental diseases. The study is a prospective, randomized controlled multicenter study (RCT) with a factorial design with four groups: CAREFAM (face to face), WEP-CARE (online), both interventions, control group (TAU = treatment as usual). Central psychosocial outcomes will be assessed at four time points (i.e., Baseline and after six, 12 and 18 months) from the perspectives of the parents, the affected child and the siblings (0 - 9 years only external assessment; from 10 years of age additional self-assessment) and the professionals.

Interventions

BEHAVIORALCARE-FAM

CARE-FAM is a family-based intervention for the diagnostic, early detection and early treatment of mental health issues of children affected by rare diseases, their siblings and their parents. CARE-FAM is a brief low-frequency intervention comprising six to eight sessions per family over a period of six months.

BEHAVIORALWEP-CARE

WEP-CARE is an online-intervention that addresses parents of children and adolescents affected by rare diseases. The program is based on principles of cognitive-behavioral writing therapy. Supported by trained professionals, the participants perform 12 standardized writing tasks on a secured internet platform

Study design

Allocation

RANDOMIZED

Intervention model

FACTORIAL

Primary purpose

SUPPORTIVE_CARE

Masking

SINGLE (Investigator)

Masking description

Assessors are blind regarding the randomization (group affiliation) of the families.

Intervention model description

The study is a prospective, randomized controlled multicenter study (RCT) with a factorial design with four groups: CAREFAM (face to face), WEP-CARE (online), both interventions, control group (TAU = treatment as usual).

Eligibility

Sex/Gender

ALL

Age

1 Days to 21 Years

Healthy volunteers

Inclusion criteria

1. Family with at least one child between 0 and 21 years with a rare disease or a suspected rare disease. 2. Consent to participate in the study. 3. Sufficient knowledge of the German language of parents and children. 4. Insured at the participating insurance companies.

Exclusion criteria

Severe psychiatric disorders and impairments with acute symptoms such as suicidal tendencies, severe depression, addictions, acute psychotic symptoms etc., which will not be sufficiently supplied by this new low-frequency intervention. Children and parents with acute treatment demand in the control group will be placed at psychotherapists. Nevertheless, they stay in the control group.

Design outcomes

Primary

Measure	Time frame	Description
Mental health of parents (SCID)	Change from baseline of the study at 6,12 and 18 months after the randomization	Proportion of parents without mental abnormities among the parents with initial mental abnormities, assessed by the external, independent Structured clinical interview for DSM-IV (SCID; Wittchen, Zaudig & Fydrich,1997) 18 months after.

Secondary

Countries

Germany

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 25, 2026

Measure	Time frame	Description
Health-related quality of life of the parents (EQ-5D)	Change from baseline of the study at 6,12 and 18 months after the randomization	Health-related quality of life of the parents, assessed from the perspective of the parents by the EQ-5D (Brooks, Rabin & Charro, 2003; Hinz, Klaiberg, Brahler & Konig, 2006) at the beginning of the study as well as six, 12 and 18 months after the randomization.
Health-related quality of life of the parents (ULQIE)	Change from baseline of the study at 6,12 and 18 months after the randomization	Health-related quality of life of the parents, assessed from the perspective of the parents by the Ulmer Lebensqualitäts-inventar für Eltern chronisch kranker Kinder (ULQIE; Goldbeck & Storck, 2002) at the beginning of the study as well as six, 12 and 18 months after the randomization.
Health-related quality of life of the parents (SF-12)	Change from baseline of the study at 6,12 and 18 months after the randomization	Health-related quality of life of the parents, assessed from the perspective of the parents by the Short Form 12 (SF-12; Bullinger & Kirchberger, 1998) at the beginning of the study as well as six, 12 and 18 months after the randomization.
Health-related quality of life of the chronically-ill children/adolescents (Kidscreen-27)	Change from baseline of the study at 6,12 and 18 months after the randomization	Health-related quality of life of the chronically-ill children/adolescents and of the siblings, assessed from the perspective of the child/adolescent (from 10 years of age) and from the perspective of the parents by the Kidscreen-27 (The KIDSCREEN Group Europe, 2006).
Health-related quality of life of the chronically-ill children/adolescents (DCGM-37)	Change from baseline of the study at 6,12 and 18 months after the randomization	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 Disabkids Chronic Generic Measure (DCGM-37; Bullinger, Schmidt, Petersen & The DISABKIDS Group, 2002) at the beginning of the study as well as six, 12 and 18 months after the randomization.
Mental health of the parents (PHQ)	Change from baseline of the study at 6,12 and 18 months after the randomization	Mental health of the parents, assessed from the perspective of the parents by the Patient Health Questionnaire (PHQ; Löwe, Spitzer, Zipfel & Herzog, 2002).
Mental health of the parents (BSI)	Change from baseline of the study at 6,12 and 18 months after the randomization	Mental health of the parents, assessed from the perspective of the parents by the Brief Symptom Inventory (BSI; Franke, 2000).
Mental health of the chronically-ill children/adolescents and the siblings (Kiddie-SADS-PL)	Change from baseline of the study at 6,12 and 18 months after the randomization	Mental health of the chronically-ill children/adolescents and the siblings, assessed from the perspective of the parents and from the perspective of the children/adolescents (from 10 years of age) by an external independent interview Diagnostic Interview Kiddie-Sads-Present and Lifetime Version (Kiddie-SADS-PL; Delmo, Weiffenbach, Gabriel, Stadler & Poustka, 2001).
Psychiatric disorders of the chronically-ill children/adolescents and the siblings (CBCL)	Change from baseline of the study at 6,12 and 18 months after the randomization	Psychiatric disorders of the chronically-ill children/adolescents and the siblings, assessed from the perspective of the parents by the Child Behaviour Checklist (CBCL; Döpfner, Pflück, Kinnen & Arbeitsgruppe Deutsche Child Behavior Checklist, 2014).
Psychiatric disorders of the chronically-ill children/adolescents and the siblings (YSR)	Change from baseline of the study at 6,12 and 18 months after the randomization	Psychiatric disorders of the chronically-ill children/adolescents and the siblings, assessed from the perspective of the children/adolescents (from 10 years of age) by the Youth Self Report (YSR; Döpfner, Pflück, Kinnen & Arbeitsgruppe Deutsche Child Behavior Checklist, 2014).
Coping of the parents (CHIP-D)	Change from baseline of the study at 6,12 and 18 months after the randomization	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).
Sociodemographic information of the parents	At baseline of the study	Sociodemographic information of the parents, assessed from the perspective of the parents by ad-hoc items at the beginning of the study.
Social support of the parents, of the chronically-ill children/adolescents and of the siblings (OSSQ)	Change from baseline of the study at 6,12 and 18 months after the randomization	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 Questionnaire (OSSQ; Dalgard, 2006).
Family functioning (GARF)	Change from baseline of the study at 6,12 and 18 months after the randomization	Family functioning, assessed from the perspective of the therapist by the Global Assessment of Relational Functioning (GARF; Saß, Wittchen, Zaudig & Houben, 2003).
Relationships between siblings (SRQ)	Change from baseline of the study at 6,12 and 18 months after the randomization	Sibling relationship, assessed from the perspective of the siblings (from 10 years of age) by the Sibling Relationship Questionnaire (SRQ; Fuhrmann & Burmester, 1985).
Satisfaction with the relationship and parenting relationship of the parents (PFB)	Change from baseline of the study at 6,12 and 18 months after the randomization	Satisfaction with the relationship and parenting relationship of the parents, assessed from the perspective of the parents by the Partnerschaftsfragebogen (PFB; Hahlweg, 2016).
Eating behaviour of the chronically-ill children/adolescents (EDY-Q)	Change from baseline of the study at 6,12 and 18 months after the randomization	Eating behaviour of the chronically-ill children/adolescents, assessed from the perspective of the parents and from the perspective of the chronically-ill children/adolescents (from 10 years of age) by the Eating Disorders in Youth - Questionnaire (EDY-Q; van Dyck & Hilbert, 2016).
Body-related eating behaviour of the chronically-ill children/adolescents (ChEDE-Q8)	Change from baseline of the study at 6,12 and 18 months after the randomization	Body-related eating behaviour of the chronically-ill children/adolescents, assesse from the persepective of the chronically-ill children/adolescents (from 10 years of age) by the Eating Disorder Examination - Questionnaire (Short Form) (ChEDE-Q8; Kliem, Schmidt, Vogel, Hiemisch, Kiess & Hilbert, 2017).
Elimination disorders of the chronically-ill children/adolescents (Anamnesebogen Enuresis/Funktionelle Harninkontinenz)	Change from baseline of the study at 6,12 and 18 months after the randomization	Elimination disorders of the chronically-ill children/adolescents, assessed from the perspective of the parents by the Anamnesebogen Enuresis/Funktionelle Harninkontinenz (von Gontard, 2010).
Treatment costs of the parents (CSSRI-DE)	Change from baseline of the study at 6 months after the randomization	Treatment costs of the parents, assessed from the perspective of an external rater by the German version of the Client Socioeconomic and Services Receipt Inventory (CSSRI-DE; Roick, Kilian, Matschinger, Bernert, Mory & Angermeyer, 2001).
Treatment costs of the chronically-ill children/adolescents and the siblings (CAMHSRI-DE)	Change from baseline of the study at 6 months after the randomization	Treatment costs of the chronically-ill children/adolescents and the siblings, assessed from the perspective of an external rater by the German version of the Children and adolescent mental health services receipt inventory (CAMHSRI-DE; Kilian, Losert, McDaid, Park, Knapp, Beecham, Kusakovskaja, Murauskiene & the CAMHEE Project, 2009).
Treatment assessment (FBB-T)	Change from 6 months after randomization at 12 and 18 months.	Treatment assessment of the parents and the chronically ill children/adolescents and their siblings, in self-assessment from the age of 10 years and by the therapist, assessed on the basis of the treatment assessment questionnaire (FBB-T; Mattejat & Remschmid, 1998).
Patient satisfaction (ZUF-8) Patient satisfaction	Change from 6 months after randomization at 12 and 18 months.	Patient satisfaction of the parents and the chronically ill children/adolescents and their siblings, assessed on the basis of the questionnaire on patient satisfaction (ZUF-8; Schmid & Nübling, 2002).
Coping of the chronically-ill children/adolescents and the siblings (Kidcope)	Change from baseline of the study at 6,12 and 18 months after the randomization	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).