RecruitingNCT03088020

International Congenital Central Hypoventilation Syndrome (CCHS) Registry and CCHS SHARE

International Congenital Central Hypoventilation Syndrome (CCHS) REDCap Registry and CCHS Secure Health-hub Advancing Research Efforts


Sponsor

Debra Weese-Mayer

Enrollment

1,000 participants

Start Date

Jun 24, 2013

Study Type

OBSERVATIONAL

Conditions

Summary

The Center for Autonomic Medicine in Pediatrics (CAMP), in collaboration with leading CCHS clinicians, scientists, and patient advocacy groups around the world has built the first International CCHS (Congenital Central Hypoventilation Syndrome REDCap (Research Electronic Data Capture) Registry. This registry is an international collaboration to capture CCHS natural history data with CCHS patients and their physicians recruited from around the world. This registry is part of a CCHS natural history study that includes the CCHS Secure Health-hub Advancing Research Efforts (CCHS-SHARE), a natural history data platform shared with the broader CCHS research and patient community to house extensive longitudinal, de-identified data. Inclusion of registry data in CCHS-SHARE is optional. The purpose of this IRB-approved research study is to gain a better understanding of the natural history of CCHS, including the various clinical manifestations of CCHS with advancing age, and as related to each patient's specific PHOX2B mutation. With a better understanding of CCHS natural history, we will be able to better anticipate healthcare needs and to provide more accurate guidelines to healthcare providers world-wide in caring for patients with CCHS. The study aims to obtain detailed phenotypic information (information about health and well-being) on patients with CCHS and their families. Participation would require filling out a confidential survey that asks questions regarding phenotype and past medical history. Involvement in the project is completely voluntary and there is no compensation for taking part. However, this project will help us learn more about this disease, with the goal of advancing treatment.


Eligibility

Plain Language Summary

Simplified for easier understanding

This international registry collects detailed medical information from patients with Congenital Central Hypoventilation Syndrome (CCHS) — a rare genetic condition where the brain does not automatically regulate breathing, particularly during sleep, meaning many patients require a breathing machine (ventilator) at night to stay safe. The registry aims to improve understanding of the condition's progression and the best approaches to long-term care. **You may be eligible if...** - You (or your child) have been confirmed to have CCHS through genetic testing showing a disease-causing mutation in the PHOX2B gene, which is the defining marker for this syndrome - Patients of any age may participate **You may NOT be eligible if...** - You do not have a confirmed PHOX2B gene mutation consistent with a diagnosis of CCHS Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Locations(1)

Ann & Robert H. Lurie Children's Hospital of Chicago and the Stanley Manne Children's Research Institute

Chicago, Illinois, United States

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NCT03088020


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