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

Primary Purpose

Gaucher Disease, Type 1, Gaucher Disease, Type 3

Status
Terminated
Phase
Phase 2
Locations
India
Study Type
Interventional
Intervention
Arimoclomol
Placebo oral capsule
Sponsored by
ZevraDenmark
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Gaucher Disease, Type 1 focused on measuring Gaucher Disease

Eligibility Criteria

4 Years - 60 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Sir Ganga Ram Hospital
  • King Edward Memorial Hospital
  • Jaslok Hospital and Reseach Centre
  • KEM HOSPITAL Research Centre
  • Christian Medical College and Hospital.
  • Institute of Child Health
  • Maulana Azad Medical College
  • All India Institute of Medical Sciences

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Experimental

Placebo Comparator

Arm Label

Arimoclomol I

Arimoclomol II

Arimoclomol III

Placebo

Arm Description

Arimoclomol, oral capsule

Arimoclomol, oral capsule

Arimoclomol, oral capsule

Placebo oral capsule matching experimental arm

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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

Full Information

First Posted
November 1, 2018
Last Updated
January 10, 2023
Sponsor
ZevraDenmark
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1. Study Identification

Unique Protocol Identification Number
NCT03746587
Brief Title
Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3
Official Title
Multicentre Double-blinded, Randomized Placebo-controlled Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3
Study Type
Interventional

2. Study Status

Record Verification Date
January 2023
Overall Recruitment Status
Terminated
Why Stopped
COVID-19 pandemic made it impossible to assess the trial objective
Study Start Date
June 6, 2018 (Actual)
Primary Completion Date
April 1, 2020 (Actual)
Study Completion Date
October 15, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
ZevraDenmark

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Gaucher Disease, Type 1, Gaucher Disease, Type 3
Keywords
Gaucher Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Model Description
Randomized
Masking
ParticipantCare ProviderInvestigator
Masking Description
Double-blinded, randomized, placebo controlled
Allocation
Randomized
Enrollment
39 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Arimoclomol I
Arm Type
Experimental
Arm Description
Arimoclomol, oral capsule
Arm Title
Arimoclomol II
Arm Type
Experimental
Arm Description
Arimoclomol, oral capsule
Arm Title
Arimoclomol III
Arm Type
Experimental
Arm Description
Arimoclomol, oral capsule
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo oral capsule matching experimental arm
Intervention Type
Drug
Intervention Name(s)
Arimoclomol
Intervention Description
Arimoclomol in 3 different dosages
Intervention Type
Drug
Intervention Name(s)
Placebo oral capsule
Intervention Description
Matching placebo capsule
Primary Outcome Measure Information:
Title
Primary Endpoint
Description
The percentage change in serum chitotriosidase levels from baseline to 6 months
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Growth Endpoint
Description
Change in weight curve (Khadilkar and Khadilkar, 2011) measured in kilograms, at every 6 months
Time Frame
6 months
Title
Growth Endpoint
Description
Change in length curve (Khadilkar and Khadilkar, 2011) measured in meters at every 6 months
Time Frame
6 months
Title
Maturation Endpoint
Description
Age at pubertal onset (Tanner Stage II) for subjects who had not reached puberty at screening
Time Frame
6 months
Title
Maturation Endpoint
Description
Tanner Staging at baseline, 6 months and at least every 12 months (Tanner stage I-IV)
Time Frame
6 - 12 months
Title
Imaging Endpoint
Description
Change in size of liver and spleen assessed by ultrasound
Time Frame
6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Aabha Nagral, MD
Organizational Affiliation
Jaslok Hospital and Reseach Centre, Mumbai
Official's Role
Principal Investigator
Facility Information:
Facility Name
Sir Ganga Ram Hospital
City
New Delhi
State/Province
Delhi
ZIP/Postal Code
110060
Country
India
Facility Name
King Edward Memorial Hospital
City
Mumbai
State/Province
Maharashtra
ZIP/Postal Code
4000012
Country
India
Facility Name
Jaslok Hospital and Reseach Centre
City
Mumbai
State/Province
Maharashtra
ZIP/Postal Code
400026
Country
India
Facility Name
KEM HOSPITAL Research Centre
City
Pune
State/Province
Maharashtra
ZIP/Postal Code
411011
Country
India
Facility Name
Christian Medical College and Hospital.
City
Vellore
State/Province
Tamilnadu
ZIP/Postal Code
632004
Country
India
Facility Name
Institute of Child Health
City
Kolkata
State/Province
West Bengal
ZIP/Postal Code
700017
Country
India
Facility Name
Maulana Azad Medical College
City
New Delhi
ZIP/Postal Code
110002
Country
India
Facility Name
All India Institute of Medical Sciences
City
New Delhi
ZIP/Postal Code
110029
Country
India

12. IPD Sharing Statement

Plan to Share IPD
Undecided

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

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