Summary

Eligibility
for people ages 2 years and up (full criteria)
Location
at Oakland, California and other locations
Dates
study started
Principal Investigator
by Caroline Hastings, MD
Headshot of Caroline Hastings
Caroline Hastings

Description

Summary

NPC is a rare, relentlessly progressive, neurological disease and associated with serious morbidity and shortened life expectancy.

The purpose of this Expanded Access Program is to provide early access to arimoclomol for patients with Niemann-Pick Type C disease who, in the opinion and the clinical judgement of the treating physician, may benefit from treatment with arimoclomol.

Participants will receive treatment with arimoclomol until their doctor finds it does not help them anymore, they withdraw, or the study is stopped for any reason.

Official Title

Early Access Program With Arimoclomol for the Treatment of Niemann-Pick Disease Type C in the US

Keywords

Niemann-Pick Disease, Type C, Pick Disease of the Brain, Primary Progressive Aphasia, Frontotemporal Dementia, Niemann-Pick Diseases, Type A Niemann-Pick Disease, Type C Niemann-Pick Disease, Arimoclomol

Eligibility

Locations

  • UCSF Benioff Children's Hospital and Research Center/ UCSF not accepting new patients
    Oakland California 94609 United States
  • Children's Hospital of Orange County (CHOC) not accepting new patients
    Orange California 92868 United States

Lead Scientist at UCSF

  • Caroline Hastings, MD
    Dr. Caroline Hastings is a Professor of Pediatrics and is specialty boarded in Pediatric Hematology Oncology. She has been at Children's Hospital Oakland and now UCSF since completion of her training in 1992. Her longevity in the group has allowed for personal and professional growth and development of long-term relationships with her patients.

Details

Status
not accepting new patients
Start Date
Sponsor
ZevraDenmark
ID
NCT04316637
Study Type
Expanded Access
Last Updated