Children Affected by Rare Disease and Their Families Network

Rare Diseases Clinical Trial

— CARE-FAM-NET  

Official title:

Children Affected by Rare Disease and Their Families Network

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04339465
• Other study ID #: U1111-1223-2871
• Status: Active, not recruiting
• Phase: N/A
• Start date: January 1, 2019
• Est. completion date: December 31, 2022

Study information

• Verified date: September 2022
• Source: Universitätsklinikum Hamburg-Eppendorf
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical 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. 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 687
• Est. completion date: December 31, 2022
• Est. primary completion date: September 30, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Day to 21 Years

Eligibility

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.

Related Conditions & MeSH terms

• Rare Diseases

Intervention

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.
• 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

Locations

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

Sponsors (29)

Lead Sponsor

Collaborator

Silke Wiegand-Grefe, Prof. Dr.

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

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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.
Secondary	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 & Nu¨bling, 2002).	Change from 6 months after randomization at 12 and 18 months.