Rare DiseasesCARE-FAM-NETNCT04339465
Children Affected by Rare Disease and Their Families Network
A study testing CARE-FAM and WEP-CARE in people with Rare Diseases, run by Silke Wiegand-Grefe, Prof. Dr. at 17 sites in Germany.
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. Read more about this study
- Who is it for?
- Ages 1 day to 21 years
- Conditions the study is for
- Rare Diseases
- What is being tested?
- CARE-FAM and WEP-CARE
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 687 participants
- Where
- 17 sitesGermany
- Study dates
- 1 Jan 2019to 31 Dec 2022
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 day to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
- 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.
Who cannot join
None listed.
Trial locations and contacts
17 sites in 1 country.
17 sites in 1 country
Germany17 sites
Medical Center Klinikum Augsburg, Kinderklinik Augsburg, l. Klinik für Kinder- und Jugendliche
Augsburg
Medical Center DRK Kliniken Berlin Westend, Klinik für Kinder- und Jugendmedizin
Berlin
Medical Center Evangelisches Klinikum Bethel, Klinik für Kinder- und Jugendmedizin
Bielefeld
University Medical Center Ruhr-Universität Bochum, Klinik für Kinder- und Jugendmedizin
Bochum
University Medical Center Universitätsklinik Köln, Klinik für Kinder- und Jugendmedizin
Cologne
University Medical Center Universitätsklinikum Essen, Kinderklinik I, Neuropädiatrie
Essen
University Medical Center Universitätsklinikum Freiburg, Zentrum für Allgemeine Kinder- und Jugendmedizin, Klinik l
Freiburg im Breisgau
University Medical Center Universitätsklinikum Gießen und Marburg GmbH, Standort Gießen, Kinderklinik, Abteilung für Kinderneurologie, Sozialpädiatrie u. Epileptologie
Giessen
University Medical Center Universitätsmedizin Göttingen, Klinik für Kinder- und Jugendmedizin
Göttingen
University Medical Center Universitätsklinikum Hamburg-Eppendorf, Klinik für Kinder- und Jugendmedizin
Hamburg, 20246
University Medical Center Medizinische Hochschule Hannover, Klinik für Pädiatrische Nieren-, Leber- und Stoffwechselerkrankungen
Hanover
University Medical Center Universitätsklinikum des Saarlandes, Homburg, Klinik für Allgemeine Pädiatrie und Neonatologie
Homburg
University Medical Center Universitätsklinik Jena, Klinik für Kinder- und Jugendmedizin
Jena
University Medical Center Universitätsmedizin Leipzig, Universitätskinderklinik
Leipzig
University Medical Center Charité-Universitätsmedizin Berlin, Klinik für Kinder- und Jugendmedizin
Mitte
University Medical Center Universitätsklinikum Münster, Klinik für Kinder- und Jugendmedizin
Münster
University Medical Center Universitätsmedizin Rostock, Kinder- und Jugendklinik
Rostock
About this study
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.
Read the 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.
- Conditions
- Rare Diseases
- Being tested
- CARE-FAM, WEP-CARE
- Keywords
- rare disease, children and adolescents, e-mental health, early detection and early treatment of mental disorders
- Medical subject headings
- Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CARE-FAMBehavioural
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-CAREBehavioural
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
| Study group | Role | What they receive |
|---|---|---|
| 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). Receives: 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. Receives: 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. Receives: 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Change from baseline of the study at 6,12 and 18 months after the randomization
Secondary outcomes (24)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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).
Measured: 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.
Measured: 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).
Measured: Change from baseline of the study at 6,12 and 18 months after the randomization
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).
Measured: 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).
Measured: 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).
Measured: 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).
Measured: 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).
Measured: 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).
Measured: 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).
Measured: 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).
Measured: 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).
Measured: 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).
Measured: 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).
Measured: 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).
Measured: 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).
Measured: 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).
Measured: 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).
Measured: 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).
Measured: 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).
Measured: Change from 6 months after randomization at 12 and 18 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Apr 2020
- Study start1 Jan 2019
- Primary completion30 Sep 2022 (estimated)
- Study completion31 Dec 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Rare DiseasesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Factorial
- 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).
- Masking
- Single blind
- Who is masked
- Investigator
- Enrolment
- 687 participants (actual)
Sponsor and investigators
- Lead sponsor
- Silke Wiegand-Grefe, Prof. Dr.
- Sponsor type
- University, hospital or other
- Collaborators
- Techniker Krankenkasse, BARMER, DAK Gesundheit, KKH Kaufmännische Krankenkasse, BKK Mobil Oil, Achse e.V., University of Ulm, Universitätsklinikum Hamburg-Eppendorf, University Hospital Schleswig-Holstein, University Medical Center Rostock, University Hospital Muenster, University Hospital, Essen, Ruhr University of Bochum, Universitätsklinikum Köln, University of Göttingen, Hannover Medical School, Evangelisches Klinikum Bethel, Charite University, Berlin, Germany, DRK Kliniken Berlin Westend, Universitätsklinikum Leipzig, University of Giessen, University Hospital Freiburg, Josefinum Augsburg, University Hospital Augsburg, University Hospital, Saarland, Jena University Hospital, Leibniz Universität, Center for Health Economics Research Hannover, aQua-Institut
- Responsible party
- Sponsor-investigator, Silke Wiegand-Grefe, Prof. Dr., Universitätsklinikum Hamburg-Eppendorf
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04339465
- Sponsor's protocol ID
- U1111-1223-2871
- Acronym
- CARE-FAM-NET
- Status last verified
- Sep 2022
- First submitted
- 1 Apr 2020
- Last updated
- 16 Sep 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: It is not yet known if there will be a plan to make IPD available.
Publications
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;21(1):85. doi: 10.1186/s13023-026-04233-5.
PubMed 41620754Boettcher 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.
PubMed 33218310
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Children Affected by Rare Disease and Their Families Network
- ClinicalTrials.gov ID
- NCT04339465
- Acronym / sponsor ID
- CARE-FAM-NET / U1111-1223-2871
- Conditions
- Rare Diseases
- Keywords
- rare disease, children and adolescents, e-mental health, early detection and early treatment of mental disorders
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 687 participants (actual)
- Lead sponsor
- Silke Wiegand-Grefe, Prof. Dr.University, hospital or other
- Collaborators
- Techniker Krankenkasse, BARMER, DAK Gesundheit, KKH Kaufmännische Krankenkasse, BKK Mobil Oil, Achse e.V., University of Ulm, Universitätsklinikum Hamburg-Eppendorf, University Hospital Schleswig-Holstein, University Medical Center Rostock, University Hospital Muenster, University Hospital, Essen, Ruhr University of Bochum, Universitätsklinikum Köln, University of Göttingen, Hannover Medical School, Evangelisches Klinikum Bethel, Charite University, Berlin, Germany, DRK Kliniken Berlin Westend, Universitätsklinikum Leipzig, University of Giessen, University Hospital Freiburg, Josefinum Augsburg, University Hospital Augsburg, University Hospital, Saarland, Jena University Hospital, Leibniz Universität, Center for Health Economics Research Hannover, aQua-Institut
- Study start
- 1 Jan 2019 (actual)
- Primary completion
- 30 Sep 2022 (estimated)
- Study completion
- 31 Dec 2022 (estimated)
- First posted
- 9 Apr 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Factorial
- Masking
- Single blind (investigator)
- Number of arms
- 4
- 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).
| Arm | Type | Receives |
|---|---|---|
| CARE-FAM | Experimental | Behavioral: CARE-FAMThe 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). |
| WEP-CARE | Experimental | Behavioral: WEP-CAREThe 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. |
| CARE-FAM + WEP-CARE | Experimental | Behavioral: CARE-FAM, Behavioral: WEP-CAREThe families will receive both the face-to-face intervention CARE-FAM and the online intervention 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. |
Who may qualify
- Ages
- 1 day to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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.
- 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.
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CARE-FAMCARE-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. | Behavioural | CARE-FAM, CARE-FAM + WEP-CARE |
| WEP-CAREWEP-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 | Behavioural | CARE-FAM + WEP-CARE, WEP-CARE |
Primary outcome
Mental health of parents (SCID)
Time frame: Change from baseline of the study at 6,12 and 18 months after the randomization
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.
24 secondary outcomes
Sociodemographic information of the parents
Time frame: At baseline of the study
Health-related quality of life of the parents (EQ-5D)
Time frame: Change from baseline of the study at 6,12 and 18 months after the randomization
Health-related quality of life of the parents (ULQIE)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: Change from baseline of the study at 6,12 and 18 months after the randomization
Mental health of the parents (PHQ)
Time frame: Change from baseline of the study at 6,12 and 18 months after the randomization
Mental health of the parents (BSI)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: Change from baseline of the study at 6,12 and 18 months after the randomization
Coping of the parents (CHIP-D)
Time frame: 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)
Time frame: 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)
Time frame: Change from baseline of the study at 6,12 and 18 months after the randomization
Family functioning (GARF)
Time frame: Change from baseline of the study at 6,12 and 18 months after the randomization
Relationships between siblings (SRQ)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: Change from baseline of the study at 6,12 and 18 months after the randomization
Treatment costs of the parents (CSSRI-DE)
Time frame: 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)
Time frame: Change from baseline of the study at 6 months after the randomization
Treatment assessment (FBB-T)
Time frame: Change from 6 months after randomization at 12 and 18 months.
Patient satisfaction (ZUF-8) Patient satisfaction
Time frame: Change from 6 months after randomization at 12 and 18 months.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2022.
- Derived from this study: PubMed 41620754Boettcher 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;21(1):85. doi: 10.1186/s13023-026-04233-5.
- Derived from this study: PubMed 33218310Boettcher 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.
- Individual participant dataSharing planned: Undecided. It is not yet known if there will be a plan to make IPD available.
Related trials for Rare Diseases
RecruitingPhase 1/2
A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia
X-linked Hypophosphatemia
Not yet recruitingObservational study
Functional Impact of Psychological Symptoms in Patients With Superior Semicircular Canal Dehiscence Syndrome (SSCD): a National Cross-sectional Study
Superior Semicircular Canal Dehiscence
RecruitingObservational study
Genetic Analysis of Uncommon Disease Presentations in Non-US Populations
Undiagnosed Diseases, Rare Diseases
RecruitingPhase 1
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Friedreich's Ataxia (FA)
RecruitingObservational study
National Ophthalmic Genotyping and Phenotyping Network (eyeGENE (Registered Trademark)), Stage 3 - Expansion of DNA and Data Repositories for Rare Inherited Ophthalmic Diseases
Inherited Ophthalmic Diseases, Hypopigmentation Disorder
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
17 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing CARE-FAM and WEP-CARE in people with Rare Diseases, run by Silke Wiegand-Grefe, Prof. Dr. at 17 sites in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 16 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mental health of parents (SCID) (time frame: Change from baseline of the study at 6,12 and 18 months after the randomization). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04339465, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2022.