Children Affected by Rare Disease and Their Families Network

NCT04339465 | Unknown DMC

• 1 other identifier: U1111-1223-2871
• Study Type: interventional
• Target: 687
• Locations: 1 country
• Sites: 17

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.

Conditions

Rare Diseases

Keywords

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

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (24)

• Sociodemographic information of the parents

  At baseline of the study

• 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 (ULQIE)

  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)

  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)

  Change from baseline of the study at 6,12 and 18 months after the randomization

Study Arms (4)

CARE-FAM

EXPERIMENTAL

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

WEP-CARE

EXPERIMENTAL

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

CARE-FAM + WEP-CARE

EXPERIMENTAL

The families will receive both the face-to-face intervention CARE-FAM and the online intervention WEP-CARE.

Behavioral: CARE-FAM; Behavioral: WEP-CARE

Treatment as usual

NO INTERVENTION

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.

Interventions

CARE-FAM BEHAVIORAL

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.

CARE-FAM; CARE-FAM + WEP-CARE

WEP-CARE BEHAVIORAL

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

CARE-FAM + WEP-CARE; WEP-CARE

Eligibility Criteria

• Age: 1 Day - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

Sponsors & Collaborators

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

Study Sites (17)

Medical Center Klinikum Augsburg, Kinderklinik Augsburg, l. Klinik für Kinder- und Jugendliche

Augsburg, Germany

Location

Medical Center DRK Kliniken Berlin Westend, Klinik für Kinder- und Jugendmedizin

Berlin, Germany

Location

Medical Center Evangelisches Klinikum Bethel, Klinik für Kinder- und Jugendmedizin

Bielefeld, Germany

Location

University Medical Center Ruhr-Universität Bochum, Klinik für Kinder- und Jugendmedizin

Bochum, Germany

Location

University Medical Center Universitätsklinik Köln, Klinik für Kinder- und Jugendmedizin

Cologne, Germany

Location

University Medical Center Universitätsklinikum Essen, Kinderklinik I, Neuropädiatrie

Essen, Germany

Location

University Medical Center Universitätsklinikum Freiburg, Zentrum für Allgemeine Kinder- und Jugendmedizin, Klinik l

Freiburg im Breisgau, Germany

Location

University Medical Center Universitätsklinikum Gießen und Marburg GmbH, Standort Gießen, Kinderklinik, Abteilung für Kinderneurologie, Sozialpädiatrie u. Epileptologie

Giessen, Germany

Location

University Medical Center Universitätsmedizin Göttingen, Klinik für Kinder- und Jugendmedizin

Göttingen, Germany

Location

University Medical Center Universitätsklinikum Hamburg-Eppendorf, Klinik für Kinder- und Jugendmedizin

Hamburg, 20246, Germany

Location

University Medical Center Medizinische Hochschule Hannover, Klinik für Pädiatrische Nieren-, Leber- und Stoffwechselerkrankungen

Hanover, Germany

Location

University Medical Center Universitätsklinikum des Saarlandes, Homburg, Klinik für Allgemeine Pädiatrie und Neonatologie

Homburg, Germany

Location

University Medical Center Universitätsklinik Jena, Klinik für Kinder- und Jugendmedizin

Jena, Germany

Location

University Medical Center Universitätsmedizin Leipzig, Universitätskinderklinik

Leipzig, Germany

Location

University Medical Center Charité-Universitätsmedizin Berlin, Klinik für Kinder- und Jugendmedizin

Mitte, Germany

Location

University Medical Center Universitätsklinikum Münster, Klinik für Kinder- und Jugendmedizin

Münster, Germany

Location

University Medical Center Universitätsmedizin Rostock, Kinder- und Jugendklinik

Rostock, Germany

Location

Related Publications (2)

• Boettcher J, Lucke T, Zapf H, Daubmann A, Denecke J, Kersting M, Kalhoff H, Beblo S, Schmidt R, Thiele A, Hohmann S, Kiess W, Muntau AC, Hilbert A, Wiegand-Grefe S. Prevalence of avoidant/restrictive food intake disorder in children and adolescents with rare diseases. Orphanet J Rare Dis. 2026 Jan 31. doi: 10.1186/s13023-026-04233-5. Online ahead of print.

  PMID: 41620754 DERIVED

• 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.

  PMID: 33218310 DERIVED

MeSH Terms

Conditions

Rare Diseases

Condition Hierarchy (Ancestors)

Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: INVESTIGATOR
• Masking Details: Assessors are blind regarding the randomization (group affiliation) of the families.
• Purpose: SUPPORTIVE CARE
• Intervention Model: FACTORIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Prof. Dr.

Model Details: 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).

Study Record Dates

• First Submitted: April 1, 2020
• First Posted: April 9, 2020
• Study Start: January 1, 2019
• Primary Completion: September 30, 2022
• Study Completion: December 31, 2022
• Last Updated: September 16, 2022

Record last verified: 2022-09

Locations

DE (17)