MedTrace Logo

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

NCT02441491 · Status: WITHDRAWN · Phase: NA · Type: INTERVENTIONAL

Last updated 2024-04-02

No results posted yet for this study

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.

Conditions

  • ROHHAD Syndrome

Interventions

DRUG

Cyclophosphamide

Cyclophosphamide will be given by vein once a day for four straight days. Ten days after starting cyclophosphamide, filgrastim, a drug that helps normal blood cells to grow, by vein once every day to try to help your blood cells grow faster.

Sponsors & Collaborators

Principal Investigators

  • Allen Chen, MD, PhD · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Months
Max Age
22 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-03-31
Primary Completion
2024-03-31
Completion
2024-03-31

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02441491 on ClinicalTrials.gov