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

NCT02441491 | Withdrawn DMC

• 2 other identifiers: IRB00060912J1520
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief 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

Outcome Measures

Primary Outcomes (2)

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

  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.

  18 months

Secondary Outcomes (2)

• To calculate Area Under Curve (AUC) of high dose cyclophosphamide in obese children.

  predose, 0, 1, 2, 4, 8, 12 and 24 hrs from the completion of infusion.

• Neuropsychological function of patients with ROHHAD before and after high-dose cyclophosphamide measured using complementary age appropriate evaluation tools including Wechsler Preschool and Primary Scale of Intelligence RAN objects, and AIM.

  18 months

Study Arms (1)

Cyclophosphamide

EXPERIMENTAL

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, will be given by vein once every day to try to help your blood cells grow faster.

Drug: Cyclophosphamide

Interventions

Cyclophosphamide DRUG

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.

Also known as: Cytoxan

Cyclophosphamide

Eligibility Criteria

• Age: 12 Months - 22 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Johns Hopkins University: lead

Study Sites (1)

Sidney Kimmel Cancer Center

Baltimore, Maryland, 21287, United States

Location

Related Publications (1)

• Jacobson LA, Rane S, McReynolds LJ, Steppan DA, Chen AR, Paz-Priel I. Improved Behavior and Neuropsychological Function in Children With ROHHAD After High-Dose Cyclophosphamide. Pediatrics. 2016 Jul;138(1):e20151080. doi: 10.1542/peds.2015-1080. Epub 2016 Jun 16.

  PMID: 27313069 DERIVED

MeSH Terms

Interventions

Cyclophosphamide

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds

Study Officials

• Allen Chen, MD, PhD

  Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 8, 2015
• First Posted: May 12, 2015
• Study Start: March 1, 2015
• Primary Completion: March 31, 2024
• Study Completion: March 31, 2024
• Last Updated: April 2, 2024

Record last verified: 2024-04

Locations

US (1)