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Early Access Program With Arimoclomol in US Patients With NPC

NCT04316637 · Status: AVAILABLE · Type: EXPANDED_ACCESS

Last updated 2024-08-16

No results posted yet for this study

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.

Conditions

  • Niemann-Pick Disease, Type C

Interventions

DRUG

Arimoclomol

Participants receive prescribed arimoclomol by oral administration

Sponsors & Collaborators

  • ZevraDenmark

    lead INDUSTRY

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Countries

  • United States

Study Locations

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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 NCT04316637 on ClinicalTrials.gov