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

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

Status

Terminated

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03746587

Enrollment

Registered

2018-11-19

Start date

2018-06-06

Completion date

2021-10-15

Last updated

2023-01-11

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Gaucher Disease, Type 1, Gaucher Disease, Type 3

Keywords

Gaucher Disease

Brief summary

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

Detailed 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.

Interventions

DRUGArimoclomol

Arimoclomol in 3 different dosages

DRUGPlacebo oral capsule

Matching placebo capsule

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

TRIPLE (Subject, Caregiver, Investigator)

Masking description

Double-blinded, randomized, placebo controlled

Intervention model description

Randomized

Eligibility

Sex/Gender

ALL

Age

4 Years to 60 Years

Healthy volunteers

Inclusion 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

Design outcomes

Primary

Measure	Time frame	Description
Primary Endpoint	6 months	The percentage change in serum chitotriosidase levels from baseline to 6 months

Secondary

Measure	Time frame	Description
Growth Endpoint	6 months	Change in weight curve (Khadilkar and Khadilkar, 2011) measured in kilograms, at every 6 months
Maturation Endpoint	6 months	Age at pubertal onset (Tanner Stage II) for subjects who had not reached puberty at screening
Imaging Endpoint	6 months	Change in size of liver and spleen assessed by ultrasound

Countries

India

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026