Early Access Program With Arimoclomol in US Patients With NPC

November 13, 2023 updated by: ZevraDenmark

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

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.

Study Overview

Status

Available

Conditions

Intervention / Treatment

Study Type

Expanded Access

Expanded Access Type

  • Intermediate-size Population

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • Available
        • University of Alabama Birmingham
        • Contact:
        • Contact:
        • Principal Investigator:
          • Joy Dean, MD
    • California
      • Oakland, California, United States, 94609
        • Available
        • UCSF Benioff Children's Hospital and Research Center/ UCSF
        • Principal Investigator:
          • Caroline Hastings, MD
        • Contact:
      • Orange, California, United States, 92868
        • Available
        • Children's Hospital of Orange County (CHOC)
        • Principal Investigator:
          • Raymond Wang, MD
        • Contact:
        • Contact:
    • Florida
    • Illinois
      • Chicago, Illinois, United States, 60612
    • Massachusetts
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Available
        • Mayo Clinic Children's Center
        • Contact:
        • Contact:
        • Principal Investigator:
          • Marc Patterson, MD, PhD
    • New York
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Available
        • Cincinnati Children's Hospital Medical Center
        • Principal Investigator:
          • Loren Pena, MD, PhD
        • Contact:
        • Contact:
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Available
        • Children's Hospital of Philadelphia
        • Principal Investigator:
          • Can Ficicioglu, MD, PhD
        • Contact:
        • Contact:
          • Genevieve Nesom, MPH, MBE
          • Phone Number: 267-426-1368
          • Email: NesomG@chop.edu
      • Pittsburgh, Pennsylvania, United States, 15224
        • Available
        • UPMC Children's Hospital of Pittsburgh
        • Principal Investigator:
          • Damara Ortiz, MD
        • Contact:
        • Contact:
    • Texas
      • Austin, Texas, United States, 78723
        • Available
        • Dell Children's Medical Center
        • Contact:
        • Contact:
        • Principal Investigator:
          • Kristina Julich, MD
      • Houston, Texas, United States, 77030
        • Available
        • UT Health / McGovern Medical School; Division of Medical Genetics
        • Contact:
        • Contact:
        • Principal Investigator:
          • Paul Hillman, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

7 months and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Description

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

ZevraDenmark

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

March 18, 2020

First Submitted That Met QC Criteria

March 18, 2020

First Posted (Actual)

March 20, 2020

Study Record Updates

Last Update Posted (Actual)

November 14, 2023

Last Update Submitted That Met QC Criteria

November 13, 2023

Last Verified

November 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • OR-ARI-EAP-NPC

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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