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

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

Study Overview

• Status: Terminated
• Conditions: Gaucher Disease, Type 1; Gaucher Disease, Type 3
• Intervention / Treatment: Drug: Placebo oral capsule; Drug: Arimoclomol

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 Type: Interventional
• Enrollment (Actual): 39
• Phase: Phase 2

Contacts and Locations

Study Locations

• India
  • New Delhi, India, 110029
    • All India Institute of Medical Sciences
  • New Delhi, India, 110002
    • Maulana Azad Medical College
  • Delhi
    • New Delhi, Delhi, India, 110060
      • Sir Ganga Ram Hospital
  • Maharashtra
    • Mumbai, Maharashtra, India, 4000012
      • King Edward Memorial Hospital
    • Mumbai, Maharashtra, India, 400026
      • Jaslok Hospital and Reseach Centre
    • Pune, Maharashtra, India, 411011
      • KEM Hospital Research Centre
  • Tamilnadu
    • Vellore, Tamilnadu, India, 632004
      • Christian Medical College and Hospital.
  • West Bengal
    • Kolkata, West Bengal, India, 700017
      • Institute of Child Health

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 years to 60 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Arimoclomol I; Arimoclomol, oral capsule	Drug: Arimoclomol; Arimoclomol in 3 different dosages
Experimental: Arimoclomol II; Arimoclomol, oral capsule	Drug: Arimoclomol; Arimoclomol in 3 different dosages
Experimental: Arimoclomol III; Arimoclomol, oral capsule	Drug: Arimoclomol; Arimoclomol in 3 different dosages
Placebo Comparator: Placebo; Placebo oral capsule matching experimental arm	Drug: Placebo oral capsule; Matching placebo capsule

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Growth Endpoint	Change in weight curve (Khadilkar and Khadilkar, 2011) measured in kilograms, at every 6 months	6 months
Growth Endpoint	Change in length curve (Khadilkar and Khadilkar, 2011) measured in meters at every 6 months	6 months
Maturation Endpoint	Age at pubertal onset (Tanner Stage II) for subjects who had not reached puberty at screening	6 months
Maturation Endpoint	Tanner Staging at baseline, 6 months and at least every 12 months (Tanner stage I-IV)	6 - 12 months
Imaging Endpoint	Change in size of liver and spleen assessed by ultrasound	6 months

Collaborators and Investigators

• Sponsor: KemPharm Denmark A/S
• Investigators: Principal Investigator: Aabha Nagral, MD, Jaslok Hospital and Reseach Centre, Mumbai

Study record dates

Study Major Dates

• Study Start (Actual): June 6, 2018
• Primary Completion (Actual): April 1, 2020
• Study Completion (Actual): October 15, 2021

Study Registration Dates

• First Submitted: November 1, 2018
• First Submitted That Met QC Criteria: November 16, 2018
• First Posted (Actual): November 19, 2018

Study Record Updates

• Last Update Posted (Estimate): January 11, 2023
• Last Update Submitted That Met QC Criteria: January 10, 2023
• Last Verified: January 1, 2023

More Information

Terms related to this study

• Keywords: Gaucher Disease
• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Lipidoses; Lipid Metabolism, Inborn Errors; Gaucher Disease
• Other Study ID Numbers: OR-ARI-GAU-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No