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Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3

Gaucher Disease, Type 1 Clinical Trial

Official title:
Multicentre Double-blinded, Randomized Placebo-controlled Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3

Clinical Trial Summary

Multicenter, doubleblinded, randomized placebo-controlled study of arimoclomol in patients with Gaucher Disease Type 1 or 3

Clinical Trial Description

Evaluating the response of 3 dose levels of arimoclomol on various pharmacodynamic biomarkers in blood and cerebrospinal fluid as indicators of enhanced GBA in Gaucher Disease Type 1 or 3. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT03746587
Study type Interventional
Source KemPharm Denmark A/S
Contact
Status Terminated
Phase Phase 2
Start date June 6, 2018
Completion date October 15, 2021
View Details
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