CompletedNot Applicable

FACE for Children With Rare Diseases

United States42 participantsStarted 2021-07-06

Plain-language summary

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 can participate

Age range

1 Year – 99 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. ≥1.0 years and \<18.0 years at enrollment;e
. unable to participate in end-of-life care decision-making;
. have a rare disease as operationally defined (See Human Subjects);
. not under a Do Not Resuscitate Order or Allow a Natural Death Order; and
. not in the Intensive Care Unit.
. ≥ 18.0 years at enrollment;
. legal guardian of child and child's caregiver;
. can speak and understand English; and

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Beck Anxiety Inventory

Timeframe: Change from Baseline Anxiety at 3 Months Post-Enrollment

Functional Assessment of Chronic Illness Therapy (FACIT)- Spiritual Well-Being Scale, Version 4

Timeframe: Change from Baseline Spiritual Well-Being at 3 Months Post-Enrollment

Advance Care Plan Document for Children With Rare Diseases

Timeframe: Change from Baseline ACP Documentation in the Electronic Health Record at 12 weeks Post-Enrollment

Days in Palliative Care Prior to Death

Timeframe: 12 weeks Post-Enrollment

Hospitalizations

Timeframe: Change from Baseline to Frequency at 12 weeks Post-Enrollment

Place of Death

Timeframe: 12 weeks Post-Enrollment

Trial details

NCT IDNCT04855734

SponsorChildren's National Research Institute

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2023-12-31

Results submitted2025-03-27

View on ClinicalTrials.gov More Rare Diseases trials

Plain-language summary

Who can participate

Age range

1 Year – 99 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. ≥1.0 years and \<18.0 years at enrollment;e
. unable to participate in end-of-life care decision-making;
. have a rare disease as operationally defined (See Human Subjects);
. not under a Do Not Resuscitate Order or Allow a Natural Death Order; and
. not in the Intensive Care Unit.
. ≥ 18.0 years at enrollment;
. legal guardian of child and child's caregiver;
. can speak and understand English; and

What they're measuring

Beck Anxiety Inventory

Timeframe: Change from Baseline Anxiety at 3 Months Post-Enrollment

Functional Assessment of Chronic Illness Therapy (FACIT)- Spiritual Well-Being Scale, Version 4

Timeframe: Change from Baseline Spiritual Well-Being at 3 Months Post-Enrollment

Advance Care Plan Document for Children With Rare Diseases

Timeframe: Change from Baseline ACP Documentation in the Electronic Health Record at 12 weeks Post-Enrollment

Days in Palliative Care Prior to Death

Timeframe: 12 weeks Post-Enrollment

Hospitalizations

Timeframe: Change from Baseline to Frequency at 12 weeks Post-Enrollment

Place of Death

Timeframe: 12 weeks Post-Enrollment