Palliative Care Needs of Children With Rare Diseases and Their Families

Part of paid clinical trials in Washington D.C., District of Columbia.

Sponsor
Children's National Research Institute
Study ID
NCT06938542
Status
Enrolling By Invitation

Conditions

  • Arthrogryposis Congenita Multiplex With Intestinal Atresia
  • Asparagine Synthetase Deficiency
  • Beta-Propeller Protein-Associated Neurodegeneration
  • Brain Injury of Prematurity With Periventricular Leukomalacia
  • CHARGE Syndrome
  • Chromosome 17p13.3 Microdeletion Syndrome
  • Chromosome 1q43-1q44 Deletion
  • Cockayne Syndrome
  • Congenital Diaphragmatic Hernia
  • Early Infantile Epileptic Encephalopathy
  • End-Stage Renal Disease With Cloacal Anomaly
  • FOXG1 Syndrome
  • KBG Syndrome
  • Mitochondrial Depletion Disorder
  • Noonan Syndrome
  • Severe Factor VII Deficiency
  • Severe Hemophilia A
  • Short Bowel Syndrome
  • Trisomy 13 Syndrome

Eligibility Criteria

Sex
ALL
Age
12 Months - 99 Years
Healthy Volunteers
Accepted

Interventions

  • Family Centered pediatric palliative care for family caregivers of children with rare diseases. — BEHAVIORAL
    Child with rare disease who is unable to participate in medical decision making/family caregiver/support person triads will be randomized at a 1:1 ratio to one of two study arms, either the 3 session FACE-Rare intervention or the enhanced Treatment as Usual. Assessments will be completed at baseline, 3, 6 and 12 month outcomes.

Study Details

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.

Key Dates

Start date
Oct 2, 2025
Status verified
Jan 2026
Primary completion
Feb 2, 2029
Completion
Aug 2, 2029

Study Design

Enrollment
480 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
SUPPORTIVE_CARE

Arms

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

Primary Outcome Measure

Family Appraisal of Caregiving for Palliative Care (FACQ-PC) [ Time Frame: Baseline, 3-, 6-, and 12 month post-intervention ]

Locations (1)

FacilityCityStateZIPSite coordinators
Children's National HospitalWashington D.C.District of Columbia20010-

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