Clinical trial NCT04339465: Children Affected by Rare Disease and Their Families Network
Official title: Children Affected by Rare Disease and Their Families Network
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Rare Diseases
Keywords: rare disease
children and adolescents
e-mental health
early detection and early treatment of mental disorders
Lead sponsor: Universitätsklinikum Hamburg-Eppendorf
Sponsor class: OTHER
Collaborators: name: Techniker Krankenkasse
name: BARMER
name: DAK Gesundheit
name: KKH Kaufmännische Krankenkasse
name: BKK Mobil Oil
name: Achse e.V.
name: University of Ulm
name: Universitätsklinikum Hamburg-Eppendorf
name: University Hospital Schleswig-Holstein
name: University Medical Center Rostock
name: University Hospital Muenster
name: University Hospital, Essen
name: Ruhr University of Bochum
name: Universitätsklinikum Köln
name: University of Göttingen
name: Hannover Medical School
name: Evangelisches Klinikum Bethel
name: Charite University, Berlin, Germany
name: DRK Kliniken Berlin Westend
name: Universitätsklinikum Leipzig
name: University of Giessen
name: University Hospital Freiburg
name: Josefinum Augsburg
name: University Hospital Augsburg
name: University Hospital, Saarland
name: Jena University Hospital
name: Leibniz Universität, Center for Health Economics Research Hannover
name: aQua-Institut
First posted: 2020-04-09
Enrollment: 687
Sex: ALL
Minimum age (years): 1
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: FACTORIAL
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).
Primary purpose: SUPPORTIVE_CARE

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Mental health of parents (SCID)
timeFrame: Change from baseline of the study at 6,12 and 18 months after the randomization
Secondary outcomes:
measure: Sociodemographic information of the parents
timeFrame: At baseline of the study
measure: Health-related quality of life of the parents (EQ-5D)
timeFrame: Change from baseline of the study at 6,12 and 18 months after the randomization
measure: Health-related quality of life of the parents (ULQIE)
timeFrame: Change from baseline of the study at 6,12 and 18 months after the randomization
measure: Health-related quality of life of the parents (SF-12)
timeFrame: Change from baseline of the study at 6,12 and 18 months after the randomization
measure: Health-related quality of life of the chronically-ill children/adolescents (Kidscreen-27)
timeFrame: Change from baseline of the study at 6,12 and 18 months after the randomization
measure: Health-related quality of life of the chronically-ill children/adolescents (DCGM-37)
timeFrame: Change from baseline of the study at 6,12 and 18 months after the randomization
measure: Mental health of the parents (PHQ)
timeFrame: Change from baseline of the study at 6,12 and 18 months after the randomization
measure: Mental health of the parents (BSI)
timeFrame: Change from baseline of the study at 6,12 and 18 months after the randomization
measure: Mental health of the chronically-ill children/adolescents and the siblings (Kiddie-SADS-PL)
timeFrame: Change from baseline of the study at 6,12 and 18 months after the randomization
measure: Psychiatric disorders of the chronically-ill children/adolescents and the siblings (CBCL)
timeFrame: Change from baseline of the study at 6,12 and 18 months after the randomization
measure: Psychiatric disorders of the chronically-ill children/adolescents and the siblings (YSR)
timeFrame: Change from baseline of the study at 6,12 and 18 months after the randomization
measure: Coping of the parents (CHIP-D)
timeFrame: Change from baseline of the study at 6,12 and 18 months after the randomization
measure: Coping of the chronically-ill children/adolescents and the siblings (Kidcope)
timeFrame: Change from baseline of the study at 6,12 and 18 months after the randomization
measure: Social support of the parents, of the chronically-ill children/adolescents and of the siblings (OSSQ)
timeFrame: Change from baseline of the study at 6,12 and 18 months after the randomization
measure: Family functioning (GARF)
timeFrame: Change from baseline of the study at 6,12 and 18 months after the randomization
measure: Relationships between siblings (SRQ)
timeFrame: Change from baseline of the study at 6,12 and 18 months after the randomization
measure: Satisfaction with the relationship and parenting relationship of the parents (PFB)
timeFrame: Change from baseline of the study at 6,12 and 18 months after the randomization
measure: Eating behaviour of the chronically-ill children/adolescents (EDY-Q)
timeFrame: Change from baseline of the study at 6,12 and 18 months after the randomization
measure: Body-related eating behaviour of the chronically-ill children/adolescents (ChEDE-Q8)
timeFrame: Change from baseline of the study at 6,12 and 18 months after the randomization
measure: Elimination disorders of the chronically-ill children/adolescents (Anamnesebogen Enuresis/Funktionelle Harninkontinenz)
timeFrame: Change from baseline of the study at 6,12 and 18 months after the randomization
measure: Treatment costs of the parents (CSSRI-DE)
timeFrame: Change from baseline of the study at 6 months after the randomization
measure: Treatment costs of the chronically-ill children/adolescents and the siblings (CAMHSRI-DE)
timeFrame: Change from baseline of the study at 6 months after the randomization
measure: Treatment assessment (FBB-T)
timeFrame: Change from 6 months after randomization at 12 and 18 months.
measure: Patient satisfaction (ZUF-8) Patient satisfaction
timeFrame: Change from 6 months after randomization at 12 and 18 months.
Other outcomes:
Locations:
city: Augsburg
country: Germany
facility: Medical Center Klinikum Augsburg, Kinderklinik Augsburg, l. Klinik für Kinder- und Jugendliche
city: Berlin-Mitte
country: Germany
facility: University Medical Center Charité-Universitätsmedizin Berlin, Klinik für Kinder- und Jugendmedizin
city: Berlin
country: Germany
facility: Medical Center DRK Kliniken Berlin Westend, Klinik für Kinder- und Jugendmedizin
city: Bielefeld
country: Germany
facility: Medical Center Evangelisches Klinikum Bethel, Klinik für Kinder- und Jugendmedizin
city: Bochum
country: Germany
facility: University Medical Center Ruhr-Universität Bochum, Klinik für Kinder- und Jugendmedizin
city: Cologne
country: Germany
facility: University Medical Center Universitätsklinik Köln, Klinik für Kinder- und Jugendmedizin
city: Essen
country: Germany
facility: University Medical Center Universitätsklinikum Essen, Kinderklinik I, Neuropädiatrie
city: Freiburg
country: Germany
facility: University Medical Center Universitätsklinikum Freiburg, Zentrum für Allgemeine Kinder- und Jugendmedizin, Klinik l
city: Gießen
country: Germany
facility: University Medical Center Universitätsklinikum Gießen und Marburg GmbH, Standort Gießen, Kinderklinik, Abteilung für Kinderneurologie, Sozialpädiatrie u. Epileptologie
city: Göttingen
country: Germany
facility: University Medical Center Universitätsmedizin Göttingen, Klinik für Kinder- und Jugendmedizin
city: Hamburg
country: Germany
facility: University Medical Center Universitätsklinikum Hamburg-Eppendorf, Klinik für Kinder- und Jugendmedizin
city: Hannover
country: Germany
facility: University Medical Center Medizinische Hochschule Hannover, Klinik für Pädiatrische Nieren-, Leber- und Stoffwechselerkrankungen
city: Homburg
country: Germany
facility: University Medical Center Universitätsklinikum des Saarlandes, Homburg, Klinik für Allgemeine Pädiatrie und Neonatologie
city: Jena
country: Germany
facility: University Medical Center Universitätsklinik Jena, Klinik für Kinder- und Jugendmedizin
city: Leipzig
country: Germany
facility: University Medical Center Universitätsmedizin Leipzig, Universitätskinderklinik
city: Münster
country: Germany
facility: University Medical Center Universitätsklinikum Münster, Klinik für Kinder- und Jugendmedizin
city: Rostock
country: Germany
facility: University Medical Center Universitätsmedizin Rostock, Kinder- und Jugendklinik
Study officials:
References:
citation: Boettcher J, Filter B, Denecke J, Hot A, Daubmann A, Zapf A, Wegscheider K, Zeidler J, von der Schulenburg JG, Bullinger M, Rassenhofer M, Schulte-Markwort M, Wiegand-Grefe S. Evaluation of two family-based intervention programs for children affected by rare disease and their families - research network (CARE-FAM-NET): study protocol for a rater-blinded, randomized, controlled, multicenter trial in a 2x2 factorial design. BMC Fam Pract. 2020 Nov 20;21(1):239. doi: 10.1186/s12875-020-01312-9.