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New program aims to ease burden on families of kids with rare diseases

NCT ID NCT06938542

Symptom relief Sponsor: Children's National Research Institute Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only This study
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests a program called FACE-Rare, designed to support family caregivers of children with rare, life-limiting diseases. The program includes three sessions to help families prepare for future medical decisions and improve their quality of life. Researchers will compare this program to usual care in 480 families, tracking outcomes like caregiver stress, spiritual well-being, and the child's healthcare use over 12 months.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Family-centered pediatric palliative care (FACE-Rare intervention)
What this could lead to
If successful, this program could provide a model to better support families caring for children with rare diseases, improving their quality of life and helping them prepare for difficult medical decisions.
What could go wrong
This is an early-stage behavioral study, not testing a drug or cure. Results may not apply to all families, and improvements in quality of life may be modest or hard to measure.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

About 480 people

The number the study aims to enrol. It can still change while the study runs.

Started

Oct 2025

Expected to finish

Aug 2029

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 months to 99 years

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Child inclusion criteria: * ≥1.0 years and \<18.0 years at enrollment. * Unable to participate in end-of-life care decision-making. * Has a rare disease as operationally defined by NIH's Genetic and Rare Diseases Information Center (GARD). * Not under a Do Not Resuscitate Order or Allow a Natural Death Order. * Not in the Intensive Care Unit. Family caregiver inclusion criteria: * \> 18.0 years at enrollment. * Child's family caregiver/legal guardian. * Not known to be developmentally delayed. Support person inclusion criteria: * \> 18.0 years at enrollment. * Chosen by family caregiver. * Not known to be developmentally delayed. Exclusion Criteria: * Family caregiver or support person is actively homicidal, suicidal, or psychotic at the time of enrollment.

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Conditions

The condition(s) this trial relates to.

anxiety disorder CHARGE syndrome chromosome 17p13.1 deletion syndrome Cockayne syndrome congenital diaphragmatic hernia congenital microcephaly - severe encephalopathy - progressive cerebral atrophy syndrome Depression early-infantile DEE factor VII deficiency FOXG1 disorder hemophilia A infantile epileptic-dyskinetic encephalopathy KBG syndrome mitochondrial DNA depletion syndrome neurodegeneration with brain iron accumulation 5 Noonan syndrome Rare Diseases severe hemophilia A short bowel syndrome trisomy 13 Trisomy 13 Syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Children's National Hospital

    Washington D.C., District of Columbia, 20010, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.