Quality of Life in Children and Young People with HHT
Official title: Health-Related Quality of Life and Clinical Utilization in Pediatric and Young Adult Patients With Hereditary Hemorrhagic Telangiectasia
Run by Children's Hospital Medical Center, Cincinnati · NCT07474428 · Data updated 2026-11-12
This study looks at how HHT affects daily life for kids and young adults ages 2 to 25 and asks caregivers and patients to fill out quality-of-life surveys.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 2 Years to 25 Years |
| Sex | All |
| Enrollment | 70 (estimated) |
| Sponsor | Children's Hospital Medical Center, Cincinnati Academic / other |
| Locations | 1 site (Cincinnati, Ohio) |
| Status | Recruiting |
| Started | September 1, 2026 |
| Main results expected | December 2026 |
| Study ends | August 2027 |
| Registry record updated | October 22, 2026 |
| Trial ID | NCT07474428 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Children and young adults aged 2–25 with definite HHT receiving care at Cincinnati Children’s Hospital Medical Center, with English-language survey capability and consent/assent as appropriate.
✕Probably not a fit
Individuals without a definite HHT diagnosis or outside the age range, or those unable to complete surveys in English, may not directly benefit.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This observational study describes health-related quality of life (HR-QoL) in pediatric and young adult patients with hereditary hemorrhagic telangiectasia (HHT) treated at Cincinnati Children's Hospital Medical Center. Participants complete validated questionnaires covering physical, emotional, social, and disease-specific aspects of functioning over the prior 30 days. A retrospective chart review will examine disease severity and healthcare utilization, including procedures and imaging. The primary goal is to describe mean QoL scores in this population, with secondary aims exploring associations between QoL, disease severity, and healthcare utilization.
Why it matters
Potential benefit: If successful, findings could help clinicians understand how HHT affects daily life in youth and guide patient-centered care and resource planning.
How similar studies have gone: Previous observational work in pediatric hematology/vascular conditions has used QoL questionnaires to capture patient- and caregiver-reported outcomes, though this exact HHT pediatric dataset is limited.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * • Patient aged 2-25 years with a confirmed (either genetic or clinical) diagnosis of definite HHT * Parent or legal guardian willing and able to complete the caregiver survey for patients aged 2-17. * For patients \>18, willing and able to complete the patient survey. * Receipt of care through CCHMC (at least one visit with genetic counselor or director of HHT Center) * Ability to complete survey in English * For self-report: patient age greater than or equal to 8 years old that assent to survey. Exclusion Criteria: * Patients that are older than 25 years old. * Patients that are younger than 2 years old. * Individuals without definite HHT diagnosis.
Where it's running
-
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States Recruiting
Study contacts
- Study coordinator: Katie Wusik, Master of Genetic Counseling
- Email: katie.wusik@cchmc.org
- Phone: (513) 636-3200
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.