Arimoclomol Early Access Program for Niemann-Pick Disease
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Description
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.
Eligibility and criteria
IRB Number:
20-017658
Eligible age range:
Clinical trial phase:
Phase IV
Official title:
What to expect
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.
Related specialties
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