Clinical trial

Whole Transcriptome Profiling and Metabolic Phenotyping in Children With ROHHAD Syndrome

Recruiting now · Not applicable · 1 countries · Registry ID NCT02602769

Recruiting nowNot applicableObservational

What this study is about

Rapid onset Obesity, Hypoventilation, Hypothalamic dysfunction and Autonomic Dysregulation (ROHHAD) is a syndrome named in 2007. The hallmark of the syndrome is the rapid onset obesity and dysregulation of central ventilation. There is little information about the metabolic changes that lead to the rapid onset obesity in these children. The investigators would like to study the metabolic phenotype of these children to understand the disturbances in energy balance that lead to the rapid onset obesity.

A promising-looking record is not the same as confirmed eligibility. The study team must review the full criteria and current recruitment status.

Basic eligibility

Age2 Years to 20 Years
SexAll
Healthy volunteersNot accepted
ConditionChildhood Obesity, Morbid Obesity

Full registry criteria

Inclusion Criteria: * Children with ROHHAD syndrome Exclusion Criteria: * Children with known genetic causes of obesity

Treatments and study arms

Transcriptome profiling

Diagnostic Test

The investigators will obtain blood to extract peripheral mononuclear cells. These cells will be used to generate patient specific hypothalamic cells that will be used for transcriptome profiling.

Primary outcomes

Changes in the transcriptome profile of hypothalamic cells of children with ROHHAD syndrome compared to their unaffected first degree relatives.2 year

The investigators will perform whole transcriptome profiling of iPSC-derived hypothalamic neurons and compare the whole genome sequencing to identify the changes that may give rise to the disease.

Study locations

2 locations were listed when this page was built. The first 40 are shown.

Boston Children's Hospital🇺🇸 Boston, New York, United States
Columbia University Irving Medical Center🇺🇸 New York, New York, United States
Vidhu Thaker, MDContact