Ignite Creation Date:
2024-05-06 @ 10:00 AM
Last Modification Date:
2024-10-26 @ 12:23 PM
Study NCT ID:
NCT03135730
Status:
RECRUITING
Last Update Posted:
2023-08-31
First Post:
2017-04-27
Brief Title:
International Rapid-onset Obesity With Hypothalamic Dysfunction Hypoventilation Autonomic Dysregulation ROHHAD Registry
Sponsor:
Debra Weese-Mayer
Organization:
Ann Robert H Lurie Childrens Hospital of Chicago
Study Overview
Official Title:
International Rapid-onset Obesity With Hypothalamic Dysfunction Hypoventilation and Autonomic Dysregulation ROHHAD REDCap Registry
Status:
RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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 patients 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
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 patients 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
Detailed Description:
ROHHAD is a rare devastating disorder in which affected children demonstrate abnormalities of respiratory control hypothalamicendocrine function and autonomic nervous system ANS dysregulation ANSD Affected children are seemingly normal until development of dramatic and rapid weight gain which occurs over a 6-month period between 15 and 10 years of age - heralding disease onset The ROHHAD phenotype appears to evolve with advancing age and with variable timing of successive features This study aims to develop a patient registry for ROHHAD which will provide crucial insight into disease development improving outcome in these children through improving early recognition of the disorder understanding the phenotypic spectrum and evolution of clinical course Data will be stored in REDCap Research Electronic Data Capture system a secure web application designed exclusively to support data capture for research studies in a secure manner The REDCap server is hosted securely at Northwestern University behind a firewall with virus protection and using Secure Socket Layer SSL authentication to encrypt communication between a user and the server Protected Health Information PHI will be labeled as such in the database and access to it will be restricted to the Principal Investigator PI and key personnel participating in the consent process and follow up contact of participants
Participants in the International ROHHAD REDCap Registry will be identified and recruited from CAMPs registry of new current and past ROHHAD referrals including ROHHAD patients referred for testing andor consultation Additionally patients may also be recruited via the internet using emails Facebook pages and mailing lists for family groups Anyone interested will be offered inclusion into the International ROHHAD Registry Participants will be able to participate remotely from their homes or locations where they have internet phone and computer access
De-identified data collected through the REDCap registry will be de-identified and analyzed Patients enrolled in this study will be offered participation in the NIH GRDR This is an optional part of the study and is not required for inclusion The Global Rare Disease Registry GRDR is established by the NIH Office of Rare Disease Research The goal of the GRDR is to establish a data repository of de-identified patient data aggregated in a standardized manner using Common Data Elements CDEs and standardized terminology De-identification of patients data will utilize the Global Unique Identifiers GUID system Lurie Childrens Hospital will retain ownership of all data shared with the GRDR The de-identified data in the GRDR will be available to all investigators to enable analyses across many rare diseases and to facilitate various biomedical studies including clinical trials in pursuit of developing drugs and therapeutics to improve the healthcare and the quality of life for the many millions of people who are diagnosed with rare diseases
Any patient agreeing to be part of the GRDR will have their data de-identified and this de-identified data exported and shared with the GRDR These participants are also given the option to be contacted for participation in clinical trials If this option is chosen any researcher accessing de-identified information through the GRDR and planning to conduct a clinical trial will be allowed to contact the CAMP project coordinator to ask that patients fitting the profile of needed participants be contacted and offered inclusion No de-identified information will be shared with researchers outside of CAMP rather the CAMP project coordinator will contact identified patients fitting participation criteria to share contact information and details for the clinical trial Interested patients will then be given to the option to contact researchers conducting clinical trials at their discretion
Participants in the International ROHHAD REDCap Registry will be identified and recruited from CAMPs registry of new current and past ROHHAD referrals including ROHHAD patients referred for testing andor consultation Additionally patients may also be recruited via the internet using emails Facebook pages and mailing lists for family groups Anyone interested will be offered inclusion into the International ROHHAD Registry Participants will be able to participate remotely from their homes or locations where they have internet phone and computer access
De-identified data collected through the REDCap registry will be de-identified and analyzed Patients enrolled in this study will be offered participation in the NIH GRDR This is an optional part of the study and is not required for inclusion The Global Rare Disease Registry GRDR is established by the NIH Office of Rare Disease Research The goal of the GRDR is to establish a data repository of de-identified patient data aggregated in a standardized manner using Common Data Elements CDEs and standardized terminology De-identification of patients data will utilize the Global Unique Identifiers GUID system Lurie Childrens Hospital will retain ownership of all data shared with the GRDR The de-identified data in the GRDR will be available to all investigators to enable analyses across many rare diseases and to facilitate various biomedical studies including clinical trials in pursuit of developing drugs and therapeutics to improve the healthcare and the quality of life for the many millions of people who are diagnosed with rare diseases
Any patient agreeing to be part of the GRDR will have their data de-identified and this de-identified data exported and shared with the GRDR These participants are also given the option to be contacted for participation in clinical trials If this option is chosen any researcher accessing de-identified information through the GRDR and planning to conduct a clinical trial will be allowed to contact the CAMP project coordinator to ask that patients fitting the profile of needed participants be contacted and offered inclusion No de-identified information will be shared with researchers outside of CAMP rather the CAMP project coordinator will contact identified patients fitting participation criteria to share contact information and details for the clinical trial Interested patients will then be given to the option to contact researchers conducting clinical trials at their discretion
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None