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

Phase 2

Terminated

• Conditions: Gaucher Disease, Type 3; Gaucher Disease, Type 1
• Interventions: Drug: Placebo oral capsule; Drug: Arimoclomol

• Registration Number: NCT03746587
• Lead Sponsor: ZevraDenmark

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.

Study Timeline

• Study Start: 2018-06-06
• Study First Submitted: 2018-11-01
• Study First Submitted QC: 2018-11-16
• Study First Posted: 2018-11-19
• Primary Completion: 2020-04-01
• Study Completion: 2021-10-15
• Status Verified: 2023-01
• Last Update Submitted: 2023-01-10
• Last Update Posted: 2023-01-11

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 39

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo oral capsule	Placebo oral capsule matching experimental arm
Arimoclomol II	Arimoclomol	Arimoclomol, oral capsule
Arimoclomol I	Arimoclomol	Arimoclomol, oral capsule
Arimoclomol III	Arimoclomol	Arimoclomol, oral capsule

Primary Outcome Measures

Name	Time	Method
Primary Endpoint	6 months	The percentage change in serum chitotriosidase levels from baseline to 6 months

Secondary Outcome Measures

Name	Time	Method
Growth Endpoint	6 months	Change in length curve (Khadilkar and Khadilkar, 2011) measured in meters at every 6 months
Imaging Endpoint	6 months	Change in size of liver and spleen assessed by ultrasound
Maturation Endpoint	6 - 12 months	Tanner Staging at baseline, 6 months and at least every 12 months (Tanner stage I-IV)

Trial Locations

Locations (8)

Maulana Azad Medical College; 🇮🇳 New Delhi, India

King Edward Memorial Hospital; 🇮🇳 Mumbai, Maharashtra, India

Jaslok Hospital and Reseach Centre; 🇮🇳 Mumbai, Maharashtra, India

Christian Medical College and Hospital.; 🇮🇳 Vellore, Tamilnadu, India

KEM HOSPITAL Research Centre; 🇮🇳 Pune, Maharashtra, India

Institute of Child Health; 🇮🇳 Kolkata, West Bengal, India

All India Institute of Medical Sciences; 🇮🇳 New Delhi, India

Sir Ganga Ram Hospital; 🇮🇳 New Delhi, Delhi, India