Children Affected by Rare Disease and Their Families Network (CARE-FAM-NET)

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Rare Diseases
• Intervention / Treatment: Behavioral: CARE-FAM; Behavioral: WEP-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.

• Study Type: Interventional
• Enrollment (Actual): 687
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Germany
  • Augsburg, Germany
    • Medical Center Klinikum Augsburg, Kinderklinik Augsburg, l. Klinik für Kinder- und Jugendliche
  • Berlin, Germany
    • Medical Center DRK Kliniken Berlin Westend, Klinik für Kinder- und Jugendmedizin
  • Berlin-Mitte, Germany
    • University Medical Center Charité-Universitätsmedizin Berlin, Klinik für Kinder- und Jugendmedizin
  • Bielefeld, Germany
    • Medical Center Evangelisches Klinikum Bethel, Klinik für Kinder- und Jugendmedizin
  • Bochum, Germany
    • University Medical Center Ruhr-Universität Bochum, Klinik für Kinder- und Jugendmedizin
  • Cologne, Germany
    • University Medical Center Universitätsklinik Köln, Klinik für Kinder- und Jugendmedizin
  • Essen, Germany
    • University Medical Center Universitätsklinikum Essen, Kinderklinik I, Neuropädiatrie
  • Freiburg, Germany
    • University Medical Center Universitätsklinikum Freiburg, Zentrum für Allgemeine Kinder- und Jugendmedizin, Klinik l
  • Gießen, Germany
    • University Medical Center Universitätsklinikum Gießen und Marburg GmbH, Standort Gießen, Kinderklinik, Abteilung für Kinderneurologie, Sozialpädiatrie u. Epileptologie
  • Göttingen, Germany
    • University Medical Center Universitätsmedizin Göttingen, Klinik für Kinder- und Jugendmedizin
  • Hamburg, Germany, 20246
    • University Medical Center Universitätsklinikum Hamburg-Eppendorf, Klinik für Kinder- und Jugendmedizin
  • Hannover, Germany
    • University Medical Center Medizinische Hochschule Hannover, Klinik für Pädiatrische Nieren-, Leber- und Stoffwechselerkrankungen
  • Homburg, Germany
    • University Medical Center Universitätsklinikum des Saarlandes, Homburg, Klinik für Allgemeine Pädiatrie und Neonatologie
  • Jena, Germany
    • University Medical Center Universitätsklinik Jena, Klinik für Kinder- und Jugendmedizin
  • Leipzig, Germany
    • University Medical Center Universitätsmedizin Leipzig, Universitätskinderklinik
  • Münster, Germany
    • University Medical Center Universitätsklinikum Münster, Klinik für Kinder- und Jugendmedizin
  • Rostock, Germany
    • University Medical Center Universitätsmedizin Rostock, Kinder- und Jugendklinik

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 day to 21 years (ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: SUPPORTIVE_CARE
• Allocation: RANDOMIZED
• Interventional Model: FACTORIAL
• Masking: SINGLE

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: CARE-FAM; The face-to-face intervention 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. Following a preliminary talk, 2 sessions with the parents, 1 session with each affected child and each sibling and 3 sessions with the whole family will take place. This low-frequency approach (sessions every 2 to 3 weeks) allows families to integrate the intervention into their daily life. Upon request, the sessions will take place at the family's home (home-treatment).	Behavioral: CARE-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.
EXPERIMENTAL: WEP-CARE; The online intervention WEP-CARE 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. The 12 writing tasks will be conducted with a weekly frequency and participants will receive personalized feedback. WEP-CARE aims at enhancing mental health problems and the coping strategies of the family.	Behavioral: WEP-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
EXPERIMENTAL: CARE-FAM + WEP-CARE; The families will receive both the face-to-face intervention CARE-FAM and the online intervention WEP-CARE.	Behavioral: CARE-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.; Behavioral: WEP-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
NO_INTERVENTION: Treatment as usual; The treatment as usual implies that families of the control group receive the treatment that is customary in regular care. Thus, these families normally don't receive any post-treatment. If, however, a member of a control group family appears to have an urgent need for treatment (every family receives a comprehensive diagnostic investigation at the beginning of the study), the respective family will be placed in the ambulatory care system.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mental health of parents (SCID)	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.	Change from baseline of the study at 6,12 and 18 months after the randomization

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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).	Change from baseline of the study at 6,12 and 18 months after the randomization
Sociodemographic information of the parents	Sociodemographic information of the parents, assessed from the perspective of the parents by ad-hoc items at the beginning of the study.	At baseline of the study
Health-related quality of life of the parents (EQ-5D)	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.	Change from baseline of the study at 6,12 and 18 months after the randomization
Health-related quality of life of the parents (ULQIE)	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.	Change from baseline of the study at 6,12 and 18 months after the randomization
Health-related quality of life of the parents (SF-12)	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.	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 (Kidscreen-27)	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).	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 (DCGM-37)	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.	Change from baseline of the study at 6,12 and 18 months after the randomization
Mental health of the parents (PHQ)	Mental health of the parents, assessed from the perspective of the parents by the "Patient Health Questionnaire" (PHQ; Löwe, Spitzer, Zipfel & Herzog, 2002).	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 (Kiddie-SADS-PL)	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).	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 (CBCL)	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).	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 (YSR)	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).	Change from baseline of the study at 6,12 and 18 months after the randomization
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).	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 (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).	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 (OSSQ)	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).	Change from baseline of the study at 6,12 and 18 months after the randomization
Family functioning (GARF)	Family functioning, assessed from the perspective of the therapist by the "Global Assessment of Relational Functioning" (GARF; Saß, Wittchen, Zaudig & Houben, 2003).	Change from baseline of the study at 6,12 and 18 months after the randomization
Relationships between siblings (SRQ)	Sibling relationship, assessed from the perspective of the siblings (from 10 years of age) by the "Sibling Relationship Questionnaire" (SRQ; Fuhrmann & Burmester, 1985).	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 (PFB)	Satisfaction with the relationship and parenting relationship of the parents, assessed from the perspective of the parents by the "Partnerschaftsfragebogen" (PFB; Hahlweg, 2016).	Change from baseline of the study at 6,12 and 18 months after the randomization
Eating behaviour of the chronically-ill children/adolescents (EDY-Q)	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).	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 (ChEDE-Q8)	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).	Change from baseline of the study at 6,12 and 18 months after the randomization
Elimination disorders of the chronically-ill children/adolescents (Anamnesebogen Enuresis/Funktionelle Harninkontinenz)	Elimination disorders of the chronically-ill children/adolescents, assessed from the perspective of the parents by the "Anamnesebogen Enuresis/Funktionelle Harninkontinenz" (von Gontard, 2010).	Change from baseline of the study at 6,12 and 18 months after the randomization
Treatment costs of the parents (CSSRI-DE)	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).	Change from baseline of the study at 6 months after the randomization
Treatment costs of the chronically-ill children/adolescents and the siblings (CAMHSRI-DE)	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).	Change from baseline of the study at 6 months after the randomization
Treatment assessment (FBB-T)	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).	Change from 6 months after randomization at 12 and 18 months.
Patient satisfaction (ZUF-8) Patient satisfaction	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).	Change from 6 months after randomization at 12 and 18 months.

Collaborators and Investigators

• Sponsor: Silke Wiegand-Grefe, Prof. Dr.
• Collaborators: Charite University, Berlin, Germany; Hannover Medical School; University Hospital, Essen; University Hospital Muenster; Ruhr University of Bochum; BARMER; University Hospital Schleswig-Holstein; University of Ulm; University of Giessen; University Hospital Freiburg; University Hospital, Saarland; Universitätsklinikum Hamburg-Eppendorf; Jena University Hospital; University of Göttingen; Universitätsklinikum Leipzig; Universitätsklinikum Köln; University Medical Center Rostock; University Hospital Augsburg; Techniker Krankenkasse; Evangelisches Klinikum Bethel; DAK Gesundheit; BKK Mobil Oil; Josefinum Augsburg; KKH Kaufmännische Krankenkasse; aQua-Institut; Leibniz Universität, Center for Health Economics Research Hannover; Achse e.V.; DRK Kliniken Berlin Westend

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (ACTUAL): January 1, 2019
• Primary Completion (ANTICIPATED): September 30, 2022
• Study Completion (ANTICIPATED): December 31, 2022

Study Registration Dates

• First Submitted: April 1, 2020
• First Submitted That Met QC Criteria: April 6, 2020
• First Posted (ACTUAL): April 9, 2020

Study Record Updates

• Last Update Posted (ACTUAL): September 16, 2022
• Last Update Submitted That Met QC Criteria: September 14, 2022
• Last Verified: September 1, 2022

More Information

Terms related to this study

• Keywords: rare disease; children and adolescents; e-mental health; early detection and early treatment of mental disorders
• Additional Relevant MeSH Terms: Pathologic Processes; Disease Attributes; Rare Diseases
• Other Study ID Numbers: U1111-1223-2871

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED
• IPD Plan Description: It is not yet known if there will be a plan to make IPD available.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No