Family-Centered Palliative Care for Children with Rare Diseases

(FACE-Rare Trial)

No longer recruiting at 1 trial location

Overseen By Maureen E Lyon, PhD

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Children's National Research Institute

Disqualifiers: Homicidal, Suicidal, Psychotic, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 6

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to support family caregivers of children with ultra-rare diseases by understanding their palliative care needs and assisting in planning future medical decisions. It introduces the FAmily CEntered (FACE) pACP Intervention, which involves structured sessions to reduce caregiver burden and guide families in discussing their child's medical care and future choices. Participants may either receive this intervention or continue with their usual care for comparison. Families with a child who has a rare disease affecting day-to-day decision-making and who are not currently in critical care settings might be a good fit for this trial. As an unphased trial, this study offers families a unique opportunity to explore innovative support strategies tailored to their specific needs.

Do I need to stop my current medications to join the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What prior data suggests that the FACE pACP Intervention is safe for family caregivers of children with rare diseases?

Research has shown that the FAmily CEntered (FACE) pACP Intervention, designed for families of children with very rare diseases, received positive feedback from participants in past studies. Families actively engaged with the program, which addresses needs such as financial and legal support. Notably, no reports of negative effects have emerged, indicating that the program is well-tolerated. Furthermore, because the program involves structured conversations and planning, it lacks the physical risks associated with medical treatments. This suggests it is a safe option for families planning future medical care for their children. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about the FACE-Rare intervention because it introduces a proactive approach to palliative care for children with rare diseases, which differs from the standard method of waiting until a medical crisis to discuss advanced care planning. This behavioral intervention uniquely combines the CSNAT Pediatric Approach and the Respecting Choices® Next Steps ACP, offering structured support and planning over three sessions. By engaging families early in conversations about their child's medical condition, values, and future healthcare decisions, FACE-Rare aims to reduce caregiver burden and align medical treatments with family goals, creating a more personalized and supportive care experience.

What evidence suggests that the FACE pACP Intervention is effective for family-centered palliative care in children with rare diseases?

Research has shown that the FAmily CEntered (FACE) pACP Intervention, which participants in this trial may receive, helps families of children with ultra-rare diseases communicate better and feel more supported. Studies have found that this program assists families in discussing medical decisions and meeting their emotional needs. The FACE-Rare program includes guided conversations to reduce caregiver stress and plan for future healthcare choices. Early results suggest that families using FACE-Rare feel more prepared for medical discussions and future care planning. This approach aims to ease tough conversations and ensure that medical care aligns with the family's values and goals. Meanwhile, participants in the Treatment-as-Usual (TAU) control arm will receive their normal standard of care, which typically defers advanced care planning discussions until a medical crisis. ^{1 2 4 6 7}

Who Is on the Research Team?

Maureen E Lyon, PhD

Principal Investigator

Children's National Research Institute

Are You a Good Fit for This Trial?

This trial is for English-speaking caregivers of children with ultra-rare diseases, aged between 1 and less than 18 years old. The child must not be in intensive care, under end-of-life orders, or unable to make decisions about their care.

Inclusion Criteria

You do not have an order that says you cannot be resuscitated or allowed to pass away naturally.

You can speak and understand English.

You are not experiencing significant delays in your physical, mental, or emotional development.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Intervention

FACE-Rare intervention consisting of 3 sessions: CSNAT Sessions 1 & 2 and Respecting Choices Session 3

Up to 5 weeks

3 sessions (in-person or virtual)

Follow-up

Participants are monitored for safety and effectiveness after the intervention, including quality of life and healthcare utilization assessments

12 weeks

Questionnaires at baseline and 3-month follow-up

What Are the Treatments Tested in This Trial?

Interventions

• FAmily CEntered (FACE) pACP Intervention

Trial Overview The FACE pACP Intervention is being tested to see if it helps family caregivers discuss future medical choices for their children with rare diseases. It's a pilot study using new methods to assess palliative care needs.

How Is the Trial Designed?

Group I: FACE-Rare Intervention Experimental Treatment 1 Intervention

FACE-Rare is a behavioral intervention that combines the CSNAT Pediatric Approach and the Respecting Choices® Next Steps ACP over 3 sessions. Sessions 1\&2: CSNAT is an evidence-based process of family caregiver assessment and support in specialized medical (palliative) care. The CSNAT tool is structured around 16 categories of family caregiver support. With the goal to decrease caregiver burden, this process consists of 5 stages wherein a nurse or practitioner works with the caregiver to create a shared support plan for the child. Session 3: Respecting Choices® Next Steps- This advanced care planning (pACP) conversation engages families in a process for how to make future medical decisions consistent with their goals and values. The interview is structured in 6 stages to achieve 2 main goals: to facilitate conversations with the family about their child's medical condition, history, fears, values, beliefs, and hopes; and to set the stage for the family's future healthcare decisions.

Group II: Treatment-as-Usual (TAU) Control Active Control 1 Intervention

To minimize the burden to families, we have chosen a Treatment-as-Usual (TAU) comparison condition, where patients will receive their normal standard of care. Both study arms will receive palliative (specialized medical) care information at enrollment and complete questionnaires before and after the intervention or TAU period. Current practice for minors with life-limiting illnesses is to defer initial discussions of advanced care planning (pACP) until a medical crisis, so this is what the TAU control arm condition will consist of.

Who Is Running the Clinical Trial?

Children's National Research Institute

Lead Sponsor

Trials

227

Recruited

258,000+

National Institute of Nursing Research (NINR)

Collaborator

Trials

623

Recruited

10,400,000+

Respecting Choices

Collaborator

Trials

2

Recruited

830+

National Institutes of Health (NIH)

Collaborator

Trials

2,896

Recruited

8,053,000+

Citations

1. spcare.bmj.com spcare.bmj.com/content/13/Suppl_4/A16.2

BOS5c.003 FACE-rare: a novel palliative care intervention ... Pediatric advance care planning (pACP) has been proven to improve communication and spiritual and emotional well-being. Few empirically validated interventions ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8040699/

Family caregivers of children and adolescents with rare ... Objective. To develop and pilot test a palliative care intervention for family caregivers of children with rare diseases (FAmily-CEntered ...

3. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0885392424005682

Effect of Family Centered Pediatric Advance Care Planning ... We pilot tested an intervention designed to support the palliative care needs of families of children with ultra-rare disorders. Families randomized to the FACE ...

4. publications.aap.org publications.aap.org/pediatrics/article/149/5/e2021054796/186767/An-Intervention-in-Congruence-for-End-of-Life

An Intervention in Congruence for End-of-Life Treatment ... We tested the efficacy of Family Centered Advance Care Planning for Teens with Cancer (FACE-TC) pediatric advance care planning (to increase ...

5. cdn.clinicaltrials.gov cdn.clinicaltrials.gov/large-docs/34/NCT04855734/Prot_SAP_000.pdf

Palliative Care Needs of Children with Rare Diseases and ... To evaluate the initial efficacy of FACE-Rare on secondary outcomes: plans and actions: completion of and documentation of advance care plans in ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11153326/

Advance Care Planning for Children With Rare Diseases Investigators assessed the initial efficacy of the FAmily CEntered (FACE) pediatric advance care planning (pACP), FACE-Rare, intervention on ...

7. withpower.com withpower.com/trial/phase-rare-diseases-6-2021-2689e

Family-Centered Palliative Care for Children with Rare ... 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 ...