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Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C

NCT02612129 · Status: COMPLETED · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 50

Last updated 2024-11-29

Study results available
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Summary

A prospective, randomized, double-blind, placebo controlled therapeutic study in participants with confirmed diagnosis of Niemann-Pick disease type C (NPC).

The purpose of this study is to assess the efficacy and safety of arimoclomol (compared to placebo) when it is administered as an add-on therapy to the participant's current prescribed best routine clinical care; participant's routine clinical care may, or may not, include miglustat.

The CT-ORZY-NPC-002 study has been expanded to include an open label paediatric sub-study including participants aged 6 to \<24 months at study enrolment.

Conditions

  • Niemann-Pick Disease, Type C

Interventions

DRUG

arimoclomol

DRUG

Placebo

Sponsors & Collaborators

  • ZevraDenmark

    lead INDUSTRY

Principal Investigators

  • Karl-Eugen Mengel · SphinCS GmbH, Hochheim, Germany

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
2 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-06-14
Primary Completion
2018-06-25
Completion
2024-10-31

Countries

  • United States
  • Denmark
  • France
  • Germany
  • Italy
  • Poland
  • Spain
  • Switzerland
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

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 NCT02612129 on ClinicalTrials.gov