COVID-19 pandemic made it impossible to assess the trial objective

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

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.

Key Inclusion Criteria:

• Be able to understand and voluntarily sign informed consent
• A diagnosis GD, either Type 1 or Type 3
• For GD3 at least 1 neurological symptom
• Age ≥ 4 years and ≤ 60 years at the time of enrolment
• Plasma or serum chitotriosidase activity greater than 3 times the upper limit of normal.

Key Exclusion Criteria:

• Recipient of a liver transplant or planned liver transplantation during the course of the study.
• Splenectomy within 4 months of study entry or planned splenectomy during the course of the study.
• Severe liver damage.
• Severe renal insufficiency.
• Body weight < 10 kg.

Other inclusion and exclusion criteria may apply

Double-blinded, randomized, placebo controlled