N/A
N=42
FACE for Children With Rare Diseases
Rare Diseases
Bottom Line
View on ClinicalTrials.gov: NCT04855734 ↗Enrolled (actual)
42
Serious AEs
0.0%
Results posted
May 2025
Primary outcome: Primary: Beck Anxiety Inventory — 10.4; 9.5; 11.1; 8.5 score on a scale — p=0.835
Study Design & Population
- Study type
- Interventional
- Phase
- N/A
- Interventions
- FAmily CEntered (FACE) pACP Intervention (Behavioral)
- Age
- Pediatric, Adult, Older Adult · 1+ yrs
- Sex
- All
- Sponsor
- Children's National Research Institute
- Primary completion
- Dec 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Beck Anxiety Inventory |
10.4; 9.5; 11.1; 8.5 | 0.835 |
| PRIMARY Functional Assessment of Chronic Illness Therapy (FACIT)- Spiritual Well-Being Scale, Version 4 |
24.0; 25.0; 26.0; 26.0; 11.0; 6.0 | 0.032 sig |
| PRIMARY Advance Care Plan Document for Children With Rare Diseases |
8; 12; 1; 0; 6; 12 | — |
| PRIMARY Days in Palliative Care Prior to Death |
— | — |
| PRIMARY Hospitalizations |
7; 7; 2; 5; 7; 8 | — |
| PRIMARY Place of Death |
— | — |
| SECONDARY Brief-Multidimensional Measure of Religion and Spirituality - Responses to Question 1 |
1; 0; 3; 1; 1; 3 | — |
| SECONDARY Family Appraisal of Caregiving Questionnaire for Palliative Care for Palliative Care (FACQ-PC) |
3.3; 3.0; 4.7; 4.6; 3.3; 3.5 | 0.284 |
| SECONDARY Hickman Role Stress Decisional Burden Scale |
75; 71.0; 75.0; 72.5 | — |
| SECONDARY Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted) Survey Responses to Question 2. |
2; 2; 1; 3; 3; 3 | — |
Summary
Children with ultra-rare or complex rare diseases are routinely excluded from research studies because of their conditions, creating a health disparity. However, new statistical techniques make it possible to study small samples of heterogeneous populations. We propose to study the palliative care needs of family caregivers of children with ultra-rare diseases and to pilot test a palliative care needs assessment and advance care planning intervention to facilitate discussions about the future medical care choices families are likely to be asked to make for their child.
Eligibility Criteria
Inclusion Criteria
Child inclusion criteria are
- ≥1.0 years and <18.0 years at enrollment;e
- unable to participate in end-of-life care decision-making;
- have a rare disease as operationally defined (See Human Subjects);
- not under a Do Not Resuscitate Order or Allow a Natural Death Order; and
- not in the Intensive Care Unit.
Family caregiver inclusion criteria are:
- ≥ 18.0 years at enrollment;
- legal guardian of child and child's caregiver;
- can speak and understand English; and
- not known to be developmentally delayed.
Exclusion Criteria
(1) Family caregiver is actively homicidal, suicidal, or psychotic at the time of enrollment.
Data sourced from ClinicalTrials.gov (NCT04855734). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.