Clinical trial NCT04855734: FACE for Children With Rare Diseases
Official title: Palliative Care Needs of Children With Rare Diseases and Their Families
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Rare Diseases
Keywords: palliative care
psychosocial interventions
rare diseases
family interventions
family-centered care
patient-centered care
pediatrics
adolescent
end-of-life care
public health
mental health
spirituality
children with rare diseases
behavioral interventions
decisional burden
role stress
family caregiver
person-centered care
chronic conditions
complex health needs
caregiver burden
respecting choices
evidence-based practice
randomized clinical trial (RCT)
anxiety
Lead sponsor: Children's National Research Institute
Sponsor class: OTHER
Collaborators: name: Respecting Choices
name: National Institutes of Health (NIH)
name: National Institute of Nursing Research (NINR)
First posted: 2021-04-22
Enrollment: 42
Sex: ALL
Minimum age (years): 1
Maximum age (years): 99
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Investigators propose a pilot, two-arm, intent-to-treat, single-blinded, single-site, controlled RCT. Family/child dyads (N=30) will be enrolled and randomized to either the FACE-Rare intervention or treatment as usual (TAU) at a 1:1 ratio.
Primary purpose: SUPPORTIVE_CARE

Brief 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.

Detailed description: A rare disease is a condition affecting fewer than 200,000 persons. Pediatric patients with rare diseases experience high mortality with 30% not living to see their 5th birthday. Families are likely to be asked to make complex medical decisions for their child. Pediatric advance care planning involves preparation and skill development to help make future medical care choices. Children with rare disorders are a heterogeneous group often with co-morbidities, resulting in their exclusion from research, thereby creating a health disparity for this vulnerable population. Available research on families of children with rare diseases lacks scientific rigor. Although desperately needed, there are few empirically validated interventions to address these issues. We propose to close a gap in our knowledge of families' needs for support in a heterogeneous group of children with rare diseases; and to test an advance care planning intervention.

The FAmily CEntered (FACE) pediatric advance care planning intervention, proven successful with cancer and HIV, is adapted to families with children who have rare diseases. Theoretically informed and developed by the PI, Dr. Lyon, and colleagues, the proposed intervention will use Respecting Choices Next Steps Pediatric Advance Care Planning™ for families whose child is unable to participate in health care decision-making. Our consultation with families of children with rare disorders and the National Organization for Rare Disorders (NORD) revealed that basic palliative care needs should be addressed first, before an advance care planning intervention. For the study to be able to meet this request, all families randomized to the intervention will first complete the Carer Support Needs Assessment Tool (CSNAT)© which our investigative team adapted for use in pediatrics. In the CSNAT Approach, facilitators assess caregivers' prioritized palliative care needs and develop Shared Action Plans for palliative care support. Thus, we propose an innovative 3-session FACE-Rare intervention, integrating two evidence-based approaches. We will evaluate FACE-Rare using a scientifically rigorous intent-to-treat, single-blinded, randomized controlled trial design. Family/child pairs or dyads (N=30 dyads) will be randomized to FACE-Rare (CSNAT Sessions 1 \& 2 plus Respecting Choices Sessions 3) or control (Treatment As Usual) groups. Both groups will receive palliative care information. All families will complete questionnaires at baseline and 3-months follow-up. Investigators will evaluate the initial efficacy of FACE-Rare on family quality of life (psychological, spiritual). We will estimate how religiousness and caregiver appraisal influence families' quality of life. We will also explore health care utilization by the children during the study and family satisfaction.

If the aims of this pilot trial are achieved, a future, large, multi-site trial will test the full theoretical model to improve care for children with rare diseases and their families through family engaged pediatric Advance Care Planning. The ultimate goal is to minimize suffering and enhance the quality of life of family caregivers of children with rare diseases; and through this process to improve the palliative care of their children.

Eligibility criteria: Inclusion Criteria:

Child inclusion criteria are

1. ≥1.0 years and \<18.0 years at enrollment;e
2. unable to participate in end-of-life care decision-making;
3. have a rare disease as operationally defined (See Human Subjects);
4. not under a Do Not Resuscitate Order or Allow a Natural Death Order; and
5. not in the Intensive Care Unit.

Family caregiver inclusion criteria are:

1. ≥ 18.0 years at enrollment;
2. legal guardian of child and child's caregiver;
3. can speak and understand English; and
4. not known to be developmentally delayed.

Exclusion Criteria:

(1) Family caregiver is actively homicidal, suicidal, or psychotic at the time of enrollment.

Primary outcomes:
measure: Beck Anxiety Inventory
timeFrame: Change from Baseline Anxiety at 3 Months Post-Enrollment
measure: Functional Assessment of Chronic Illness Therapy (FACIT)- Spiritual Well-Being Scale, Version 4
timeFrame: Change from Baseline Spiritual Well-Being at 3 Months Post-Enrollment
measure: Advance Care Plan Document for Children With Rare Diseases
timeFrame: Change from Baseline ACP Documentation in the Electronic Health Record at 12 weeks Post-Enrollment
measure: Days in Palliative Care Prior to Death
timeFrame: 12 weeks Post-Enrollment
measure: Hospitalizations
timeFrame: Change from Baseline to Frequency at 12 weeks Post-Enrollment
measure: Place of Death
timeFrame: 12 weeks Post-Enrollment
Secondary outcomes:
measure: Brief-Multidimensional Measure of Religion and Spirituality - Responses to Question 1
timeFrame: Baseline and 3-month follow-up.
measure: Family Appraisal of Caregiving Questionnaire for Palliative Care for Palliative Care (FACQ-PC)
timeFrame: Change from Baseline appraisal of Caregiving at 12 weeks Post-Enrollment
measure: Hickman Role Stress Decisional Burden Scale
timeFrame: Up to 5 Weeks Post-Enrollment
measure: Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted) Survey Responses to Question 2.
timeFrame: Baseline and 3-month follow-up
Other outcomes:
measure: Satisfaction Questionnaire (Process Measure)
timeFrame: Up to 5 Weeks Post-Baseline
measure: Quality of Communication Questionnaire
timeFrame: Up to 5 Weeks Post-Baseline
measure: Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS) "How Often do You go to Religious Services?"
timeFrame: Baseline to 3 month follow-up
measure: Brief Multidimensional Measure of Religiousness/Spirituality (Brief MMRS): "To What Extent do You Consider Yourself a Religious Person?"
timeFrame: Baseline and 3 month follow-up
measure: Brief Multidimensional Measurement of Religiousness/Spirituality (BMMRS-adapted): To What Extent do You Consider Yourself a Spiritual Person?
timeFrame: Baseline to 3 month outcomes.
Locations:
city: Washington
country: United States
facility: Children's National Hospital
state: District of Columbia
Study officials:
affiliation: Children's National Research Institute
name: Maureen E Lyon, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Lyon ME, Wiener L. Special Issue: Psychosocial Considerations for Children and Adolescents Living with a Rare Disease. Children (Basel). 2022 Jul 21;9(7):1099. doi: 10.3390/children9071099.
citation: Sandquist M, Davenport T, Monaco J, Lyon ME. The Transition to Adulthood for Youth Living with Rare Diseases. Children (Basel). 2022 May 12;9(5):710. doi: 10.3390/children9050710.
citation: Fratantoni K, Livingston J, Schellinger SE, Aoun SM, Lyon ME. Family-Centered Advance Care Planning: What Matters Most for Parents of Children with Rare Diseases. Children (Basel). 2022 Mar 21;9(3):445. doi: 10.3390/children9030445.
citation: Aoun SM, Stegmann R, Deleuil R, Momber S, Cuddeford L, Phillips MB, Lyon ME, Gill FJ. "It Is a Whole Different Life from the Life I Used to Live": Assessing Parents' Support Needs in Paediatric Palliative Care. Children (Basel). 2022 Mar 1;9(3):322. doi: 10.3390/children9030322.
citation: Lyon ME, Thompkins JD, Fratantoni K, Fraser JL, Schellinger SE, Briggs L, Friebert S, Aoun S, Cheng YI, Wang J. Family caregivers of children and adolescents with rare diseases: a novel palliative care intervention. BMJ Support Palliat Care. 2022 Nov;12(e5):e705-e714. doi: 10.1136/bmjspcare-2019-001766. Epub 2019 Jul 25.
citation: Aoun SM, Gill FJ, Phillips MB, Momber S, Cuddeford L, Deleuil R, Stegmann R, Howting D, Lyon ME. The profile and support needs of parents in paediatric palliative care: comparing cancer and non-cancer groups. Palliat Care Soc Pract. 2020 Sep 25;14:2632352420958000. doi: 10.1177/2632352420958000. eCollection 2020.
citation: - Kreicbergs U (Discussant), Handberg C, Udo C, Thompkins J (presenter) Lyon ME (organizer). Symposium: Lessons Learned during the COVID-19 and Beyond Pandemic for Children Living with Rare Diseases and their Siblings. Lyon Presentation: Family Identified Palliative Care Needs of FAmily Caregivers of Children Living with Rare Diseases during COVID-19-United States 7th Public Health Palliative Care International Conference. September 21, 2022. Bruges, Belgium.
citation: - Lyon, ME, Fraser J, Thompkins J (presenter). FACE Rare: A novel palliative care intervention for family caregivers of children and adolescents living with a rare disease. Podium Presentation. University of Pittsburgh's National Center on Family Support's Second Biennial Conference on Caregiving Research. Building Bridges: Advancing Family Caregiving Research Across the Lifespan, National Center on Caregiving. Pittsburg, PA. October 1, 2022.
citation: Lyon ML. Detwiler K, Torres C, Guerrera MF, Thompkins J. FACE-Rare: A Novel Intervention for Family Caregivers of Children Living with Rare Diseases. BMJ Supportive & Palliative Care 2023;13(Suppl 4):A16.