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

Zevra Therapeutics

Status and phase: Terminated; Phase 2

Conditions: Gaucher Disease, Type 3; Gaucher Disease, Type 1

Treatments: Drug: Placebo oral capsule; Drug: Arimoclomol

Study type: Interventional

Funder types: Industry

Identifiers

NCT03746587

OR-ARI-GAU-01

Details and patient eligibility

About

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

Full 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.

Enrollment: 39 patients

Sex: All

Ages: 4 to 60 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

---

39 participants in 4 patient groups, including a placebo group

Arimoclomol I

Experimental group

Description:

Arimoclomol, oral capsule

Treatment:

Drug: Arimoclomol

Arimoclomol II

Experimental group

Description:

Arimoclomol, oral capsule

Treatment:

Drug: Arimoclomol

Arimoclomol III

Experimental group

Description:

Arimoclomol, oral capsule

Treatment:

Drug: Arimoclomol

Placebo

Placebo Comparator group

Description:

Placebo oral capsule matching experimental arm

Treatment:

Drug: Placebo oral capsule