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

• Status: Unknown status
• Trial end date: 2021-06-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Orphazyme

Criteria

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