Arimoclomol Early Access Program for Niemann-Pick Disease

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This study will provide early access to an investigational drug which does not yet have marketing authorization for human use, Arimoclomol, for a group of patients suffering from Niemann-Pick Disease Type C that are not eligible for or able to participate in other clinical trials.

Who Do I Contact?

If you are interested in participating in the study or want to learn more please contact our study team at RamosJF [at] chop.edu or 267-426-1368.

ClinicalTrials.gov Identifier

NCT04316637

View full trial information on ClinicalTrials.gov

Eligibility & Criteria

IRB #:
20-017658
Official Title:
Early Access Program with Arimoclomol for the Treatment of Niemann-Pick Disease Type C in the US
Study Phase:
Phase IV
Eligible Age Range:
2 - 99 Years
Gender:
All
Study Categories:

Visit Criteria

The study will involve the following procedures:

  1. Administration of study drug (Arimoclomol)
  2. Blood tests and urine tests
  3. Medical history interviews
  4. Questionnaire completion
  5. Physical exams/height/weight
  6. Review of medical records.
Leader
Can Ficicioglu

Can Ficicioglu, MD, PhD