Family-Centered Palliative Care for Children with Rare Diseases

(FACE-Rare Trial)

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

42 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

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.

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+