Pediatric Palliative Care for 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

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

Compensation: Varies

Number of Visits: 6

480 Participants Needed

Trial Summary

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

The trial information does not specify whether participants need to stop taking their current medications.

What data supports the effectiveness of the treatment Family Centered pediatric palliative care for rare diseases?

Research shows that family-centered pediatric palliative care improves the quality of life for children with life-limiting illnesses and their families by enhancing emotional well-being and preparing parents for end-of-life care. This approach focuses on improving outcomes in physical, emotional, and cognitive areas, benefiting both the child and their family. ^{1 2 3 4 5}

Is pediatric palliative care safe for children with rare diseases?

Pediatric palliative care is generally focused on improving quality of life and addressing suffering for children with serious conditions, and it is considered safe as it aims to support both the child and their family. ^{1 2 6 7 8}

How is the Family Centered pediatric palliative care treatment different from other treatments for rare diseases?

Family Centered pediatric palliative care is unique because it focuses on improving the quality of life for children with life-threatening conditions and their families, rather than just treating the disease. It emphasizes support for both the child and family, helping them manage symptoms and make complex decisions, which is especially important for rare diseases with uncertain outcomes. ^{1 2 9 10 11}

What is the purpose of this trial?

The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare for future medical decision making. This trial will test the FACE-Rare intervention to see if investigators can identify and meet those needs; and if FACE-Rare effects family caregivers' quality of life and child healthcare utilization. Finally, investigators will determine if the intersectionality of child-sex, family-race, Federal poverty level, and social connection influences family quality of life and child health care utilization longitudinally.

Research Team

Maureen E Lyon, PhD

Principal Investigator

Children's National Research Institute

Eligibility Criteria

This trial is for family caregivers of children with various rare diseases, including genetic syndromes and chronic conditions. Caregivers must be in need of support for medical decision-making and coping with their child's illness.

Inclusion Criteria

Child not in the Intensive Care Unit

Family caregiver not known to be developmentally delayed

My child cannot make decisions about their end-of-life care.

Exclusion Criteria

Family caregiver or support person is actively homicidal, suicidal, or psychotic at the time of enrollment

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Intervention

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

2-8 weeks

3 sessions (in-person)

Follow-up

Participants are monitored for quality of life and healthcare utilization at multiple time points

12 months

Follow-up assessments at 3, 6, and 12 months

Treatment Details

Interventions

• Family Centered pediatric palliative care

Trial Overview The trial tests the FACE-Rare intervention, which aims to identify and meet the palliative care needs of these families, improve caregiver quality of life, and potentially reduce healthcare usage by the sick children.

Participant Groups

Group I: FACE-Rare Experimental Treatment 1 Intervention

FACE-Rare (CSNAT-P Sessions 1 \& 2 plus Next Steps: Respecting Choices Sessions 3). The CSNAT-Pediatric intervention consists of two assessment visits with the facilitator, 2-8 weeks apart, comprising conversations about sources for support in a tertiary children's hospital. The adapted Next Steps: Respecting Choices pediatric Advance Care Planning conversation engages families in a process for how to make future medical decisions consistent with the families' goals and values. The Respecting Choices structured and facilitated conversation has five stages. Stage 1: Assesses the family's understanding of illness. Stage 2: Explores experiences with hospitalization. Stage 3: Explores goals of care. Stage 4: Creates an Advance Care Plan; Stage 5: Questions for providers are written down. Stage 6: Follow-up plan and referrals, as needed.

Group II: Enhanced Treatment As Usual (TAU) Experimental Treatment 1 Intervention

Treatment as Usual Control (TAU): To minimize the burden to families, we have chosen an enhanced (palliative care information and resources) TAU comparison condition.

Who Is Running the Clinical Trial?

Children's National Research Institute

Lead Sponsor

Trials

227

Recruited

258,000+

Akron Children's Hospital

Collaborator

Trials

50

Recruited

5,009,000+

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Collaborator

Trials

2,103

Recruited

2,760,000+

Stanford University

Collaborator

Trials

2,527

Recruited

17,430,000+

References

1. England pubmed.ncbi.nlm.nih.gov

Family caregivers of children and adolescents with rare diseases: a novel palliative care intervention. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Palliative care in pediatrics. [2019]

3. United States pubmed.ncbi.nlm.nih.gov

Primary palliative care: Skills for all clinicians. [2021]

4. United States pubmed.ncbi.nlm.nih.gov

Evidence of improved quality of life with pediatric palliative care. [2015]

5. United States pubmed.ncbi.nlm.nih.gov

Integrating palliative care in pediatrics. [2019]

6. United States pubmed.ncbi.nlm.nih.gov

Pediatric palliative care patients: a prospective multicenter cohort study. [2022]

7. United States pubmed.ncbi.nlm.nih.gov

Specialized Pediatric Palliative Care in Neonates with Life-Limiting Illness: A Systematic Review. [2022]

8. England pubmed.ncbi.nlm.nih.gov

An estimation of the number of children requiring pediatric palliative care in Italy. [2021]

9. United States pubmed.ncbi.nlm.nih.gov

Pediatric palliative care for children with complex chronic medical conditions. [2014]

10. United States pubmed.ncbi.nlm.nih.gov

Providing Palliative Care in Rare Pediatric Diseases: A Case Series of Three Children with Congenital Disorder of Glycosylation. [2020]

11. United States pubmed.ncbi.nlm.nih.gov

Development of a state-wide pediatric palliative care service in Australia: referral and outcomes over two years. [2022]