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

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorDebra Weese-Mayer

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

Individuals with PHOX2B mutation-confirmed CCHS.

Disqualifiers

Individuals without PHOX2B mutation-confirmed CCHS.

Trial population

CCHS patients with confirmed PHOX2B mutations

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

Not listed

Trial groups

No trial groups listed

Trial outcomes

Primary outcomes

1

Longitudinal phenotype in CCHS

Collection of longitudinal disease history in 400 CCHS patients

Time frame
20 years

Secondary outcomes

1

Sensitive markers of disease progression by PHOX2B mutation/genotype

Evaluate patient reported outcome measures to determine sensitive markers of disease progression for use in intervention trials

Time frame
20 years
2

Integration of CCHS Registry and Global Rare Diseases Patient Registry (GRDR)

Develop a patient registry for CCHS for use with the CCHS Secure Health-hub Advancing Research Efforts" (CCHS-SHARE) and the global rare diseases patient registry and data repository (GRDR)

Time frame
20 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Debra Weese-Mayer

Lead sponsor

Ann & Robert H Lurie Children's Hospital of Chicago

Sponsor institution

Northwestern University

Collaborator

The Chicago Community Trust

Collaborator