Pediatric Palliative Care for Rare Diseases

(FACE-Rare Trial)

Not yet 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

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 rare diseases by improving their quality of life and assisting with future medical decisions through a program called FACE-Rare. It will test two approaches: the FACE-Rare intervention, which involves guided conversations about care and planning, and an enhanced usual care option with additional resources. Good candidates for the trial include families with children who have rare diseases (as defined by the NIH) and face difficulties in making end-of-life care decisions. As an unphased trial, this study offers a unique opportunity to contribute to research that could significantly enhance support for families facing similar challenges.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that the FACE-Rare program is safe for families in pediatric palliative care. Studies have found it practical, well-received, and safe. Participants reported feeling more purposeful, finding more meaning in life, and experiencing greater peace. These results suggest that the treatment is easy to handle, with no major safety issues. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about the Family Centered pediatric palliative care trial because it offers a more personalized approach to managing rare diseases in children. Unlike traditional treatments that primarily focus on symptom management, this approach includes structured conversations, known as FACE-Rare, that guide families in creating an Advance Care Plan tailored to their values and goals. This method not only supports families emotionally but also empowers them to make informed decisions about their child's care. Additionally, the Enhanced Treatment As Usual (TAU) arm provides families with palliative care resources, aiming to alleviate the burden of navigation and decision-making. These approaches are designed to improve the quality of life for both patients and their families, a critical aspect often overlooked in standard treatments.

What evidence suggests that the FACE-Rare intervention is effective for improving family caregivers' quality of life and child healthcare utilization?

Research has shown that the FACE-Rare program, a treatment option in this trial, is promising and practical for families with children who have rare diseases. Previous studies indicate that the program is not only feasible and safe but also enhances family caregivers' sense of purpose, meaning, and peace. It aids families in making future medical decisions that align with their values and goals, which is crucial for managing rare diseases. The program also addresses important issues like financial and legal concerns, which 83% of families consider a priority. Overall, early evidence suggests that FACE-Rare could significantly improve the quality of life for both families and children. ^{1 3 5 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 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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

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

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

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06938542

Palliative Care Needs of Children With Rare Diseases and ... 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 ...

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

BOS5c.003 FACE-rare: a novel palliative care intervention ... FACE-Rare families prioritized financial, legal, or work issues, 83%, over other palliative care needs for their child and themselves. Video clips will be ...

4. jpsmjournal.com jpsmjournal.com/article/S0885-3924(24)00568-2/fulltext

Effect of Family Centered Pediatric Advance Care Planning ... FACE-Rare is feasible, acceptable, safe, and demonstrates initial efficacy. This promising intervention improved life purpose, meaning, and peace for family ...

5. publications.aap.org publications.aap.org/pediatrics/article/153/6/e2023064557/197230/Advance-Care-Planning-for-Children-With-Rare

Advance Care Planning for Children With Rare Diseases We pilot tested an intervention designed to support the palliative care needs of families of children with rare disorders. Families randomized ...

6. reporter.nih.gov reporter.nih.gov/search/6DkRNCAh8UWmaTg0bDkyIw/project-details/10041054

Palliative Care Needs of Children with Rare Diseases and their ... 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 ...

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

Effect of Family Centered Pediatric Advance Care Planning ... FACE-Rare is feasible, acceptable, safe, and demonstrates initial efficacy. This promising intervention improved life purpose, meaning, and peace for family ...