International Rapid-onset Obesity with Hypothalamic Dysfunction, Hypoventilation & Autonomic Dysregulation (ROHHAD) Registry

NCT03135730 | Recruiting

• 1 other identifier: 2012-14981 and 2013-15230b
• Study Type: observational
• Target: 1,000
• Locations: 1 country
• Sites: 1

Brief Summary

The Center for Autonomic Medicine in Pediatrics (CAMP) has collaborated with doctors from around the world to build the first International ROHHAD (Rapid-onset Obesity with Hypothalamic Dysfunction, Hypoventilation and Autonomic Dysregulation) REDCap (Research Electronic Data Capture) Registry. This registry is an international collaboration with ROHHAD patients and their physicians recruited from around the world. The purpose of this IRB-approved research study is to gain a better understanding of the various health problems ROHHAD patients face with advancing age, and how these relate to each patient's specific medical course. With a better understanding of specific ROHHAD and associated clinical manifestations, 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 ROHHAD. The study aims to obtain detailed phenotypic information (information about health and well-being) on patients with ROHHAD. Participation would require filling out a confidential survey that asks questions regarding health 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 devastating disease, with the goal of advancing treatment.

Conditions

ROHHAD; Rapid-Onset Obesity with Hypothalamic Dysfunction, Hypoventilation & Autonomic Dysregulation

Outcome Measures

Primary Outcomes (1)

• longitudinal clinical history in 200 ROHHAD patients

  phenotype inclusive of control of breathing and autonomic regulation

  10 years

Secondary Outcomes (2)

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

  10 years

• patient registry for ROHHAD for use with the global rare diseases patient registry and data repository (GRDR)

  10 years

Interventions

ROHHAD GENETIC

Suspected and Confirmed ROHHAD cases

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with suspected or confirmed ROHHAD worldwide

You may qualify if:

• Clinical diagnosis of suspected ROHHAD
• Clinical diagnosis of confirmed ROHHAD

You may not qualify if:

• Clinical diagnosis not consistent with ROHHAD

Sponsors & Collaborators

• Debra Weese-Mayer: lead
• ROHHAD Fight Inc.: collaborator

Study Sites (1)

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

Chicago, Illinois, 60611, United States

RECRUITING

MeSH Terms

Conditions

Hypoventilation; Primary Dysautonomias

Condition Hierarchy (Ancestors)

Respiratory Insufficiency; Respiration Disorders; Respiratory Tract Diseases; Signs and Symptoms, Respiratory; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Autonomic Nervous System Diseases; Nervous System Diseases

Central Study Contacts

Casey Rand, BS

CONTACT

312.227.3300; Crand@LurieChildrens.org

Erin S Lonergan, BS

CONTACT

CAMP@LurieChildrens.org

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 20 Years
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Pediatric Autonomic Medicine, Northwestern University Feinberg School of Medicine Chief, Center for Autonomic Medicine in Pediatrics, Ann & Robert H. Lurie Children's Hospital of Chicago and Stanley Manne Children's Research Institute

Study Record Dates

• First Submitted: April 27, 2017
• First Posted: May 1, 2017
• Study Start: June 1, 2013
• Primary Completion (Estimated): December 31, 2034
• Study Completion (Estimated): December 31, 2039
• Last Updated: February 20, 2025

Record last verified: 2024-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)