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



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


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


You can join if…

Open to people ages 2 years and up

  • The patient has a confirmed diagnosis of NPC (NPC1 or NPC2)* and at least one neurological symptom.
  • The patient is two years of age or above.
  • The patient is a permanent resident of US.
  • If taking miglustat (Zavesca®), the patient must have been on the target dose for the past six weeks.
  • If the patient is a sexually active female of child-bearing potential (post-menarche), it is agreed to use highly effective contraception during the EAP and until three weeks after the last dose of arimoclomol.
  • Confirmed negative urine pregnancy test for sexually active female of child-bearing potential (post-menarche).
  • All sexually active male patients with female partners of child-bearing potential (postmenarche) agree to use a condom in addition to the birth control used by their partners during treatment and until three weeks after the last dose of arimoclomol.
  • If the patient has a history of seizures, the condition must be adequately controlled, i.e., the pattern of seizure activity must be stable, and the patient must be on a stable dose and regimen of antiepileptic medication during one month prior to screening.
  • Patient or parent/guardian must provide written informed consent to participate in EAP.
    • In line with Patterson et al. 2017

You CAN'T join if...

  • Severe liver insufficiency.
  • Renal insufficiency.
  • The patient has a known or suspected allergy or intolerance to arimoclomol or its constituents.
  • The patient is pregnant, planning to become pregnant (while on the EAP program) or is currently breastfeeding.
  • The patient will undergo treatment with another investigational drug*, whilst participating in the program or in the 4 weeks prior to commencing treatment with arimoclomol.
  • The patient is either eligible and able to participate in or is currently participating in an active interventional clinical trial within the indication.
  • The patient, in the opinion of the clinician, is unable to comply with the treatment or has a medical condition that would potentially increase the risk to the patient by participation.
  • The patient has a medical condition which hinders the clinician's assessment of arimoclomol safety and efficacy (e.g. certain epileptic conditions or severe cataplexy).
    • Including unlicensed product provided under an Early Access Program or equivalent compassionate use programs


  • 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.


not accepting new patients
Start Date
Study Type
Expanded Access
Last Updated