WithdrawnNot Applicable

Treatment of Rapid Onset Obesity, Hypoventilation, Hypothalamic Dysfunction, and Autonomic Dysregulation (ROHHAD )

Stopped: Inadequate accrual

United States0Started 2015-03

Plain-language summary

ROHHAD (rapid onset obesity, hypothalamic dysfunction, hypoventilation, and autonomic dysregulation) syndrome is a rare pediatric disorder associated with a cancer called neuroblastoma and presumed to be driven by an attack of the immune system on specific area in the brain. Patients develop severe symptoms and often succumb to this disease. Based on the researchers' experience the investigators conduct a clinical trial to study intensive immunosuppression with high-dose cyclophosphamide in these patients. In addition to describing the symptomatic improvement, the investigators' trial seeks to define objective markers of disease activity.

Who can participate

Age range12 Months – 22 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Diagnosis of ROHHAD syndrome confirmed by two physicians. * Any symptomatic improvement in response to a course of rituximab (five weekly doses) as assessed by parents and/or treating physician * Normal brain MRI * Cared for at home by the family * Patients requiring bilevel positive airway pressure (BiPAP) support are eligible * Negative pregnancy test for post pubertal female patients Exclusion Criteria: * Cardiac ejection fraction \<40% or shortening fraction \<20%. * Inadequate pulmonary function, i.e. forced vital capacity or forced expiratory volume at one second \< 50% of predicted for children greater than 8 years of age, or oxygen saturation \<93% on pulse oximetry for younger children. * Ventilator dependent * Known chromosomal abnormality * Active cancer diagnosis. Neuroblastoma that requires only follow up is eligible. * Pregnancy

What they're measuring

Symptomatic improvement on the ROHHAD Symptoms Scale of children with ROHHAD syndrome after treatment with high-dose cyclophosphamide .

Timeframe: 18 months

Safety and toxicity of high-dose cyclophosphamide used to treat children with ROHHAD of as measured by NCI Common Terminology Criteria for Adverse Events (CTCAE) v4.0.

Timeframe: 18 months

Trial details

NCT IDNCT02441491

SponsorJohns Hopkins University

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2024-03-31

View on ClinicalTrials.gov More ROHHAD Syndrome trials

Plain-language summary

Who can participate

Age range12 Months – 22 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Symptomatic improvement on the ROHHAD Symptoms Scale of children with ROHHAD syndrome after treatment with high-dose cyclophosphamide .

Timeframe: 18 months

Safety and toxicity of high-dose cyclophosphamide used to treat children with ROHHAD of as measured by NCI Common Terminology Criteria for Adverse Events (CTCAE) v4.0.

Timeframe: 18 months