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

Silke Wiegand-Grefe, Prof. Dr.

Status: Active, not recruiting

Conditions: Rare Diseases

Treatments: Behavioral: WEP-CARE; Behavioral: CARE-FAM

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT04339465

U1111-1223-2871

Details and patient eligibility

About

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.

Full 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.

Enrollment: 687 patients

Sex: All

Ages: 1 day to 21 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Supportive Care

Allocation: Randomized

Interventional model: Factorial Assignment

Masking: Single Blind

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687 participants in 4 patient groups

CARE-FAM

Experimental group

Description:

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).

Treatment:

Behavioral: CARE-FAM

WEP-CARE

Experimental group

Description:

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.

Treatment:

Behavioral: WEP-CARE

CARE-FAM + WEP-CARE

Experimental group

Description:

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

Treatment:

Behavioral: WEP-CARE

Behavioral: CARE-FAM

Treatment as usual

No Intervention group

Description:

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.