Study Stopped
COVID-19 pandemic made it impossible to assess the trial objective
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
1 other identifier
interventional
39
1 country
8
Brief Summary
Multicenter, doubleblinded, randomized placebo-controlled study of arimoclomol in patients with Gaucher Disease Type 1 or 3
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Jun 2018
Typical duration for phase_2
8 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
June 6, 2018
CompletedFirst Submitted
Initial submission to the registry
November 1, 2018
CompletedFirst Posted
Study publicly available on registry
November 19, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 1, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
October 15, 2021
CompletedJanuary 11, 2023
January 1, 2023
1.8 years
November 1, 2018
January 10, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Primary Endpoint
The percentage change in serum chitotriosidase levels from baseline to 6 months
6 months
Secondary Outcomes (5)
Growth Endpoint
6 months
Growth Endpoint
6 months
Maturation Endpoint
6 months
Maturation Endpoint
6 - 12 months
Imaging Endpoint
6 months
Study Arms (4)
Arimoclomol I
EXPERIMENTALArimoclomol, oral capsule
Arimoclomol II
EXPERIMENTALArimoclomol, oral capsule
Arimoclomol III
EXPERIMENTALArimoclomol, oral capsule
Placebo
PLACEBO COMPARATORPlacebo oral capsule matching experimental arm
Interventions
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- ZevraDenmarklead
Study Sites (8)
King Edward Memorial Hospital
Mumbai, Maharashtra, 4000012, India
Jaslok Hospital and Reseach Centre
Mumbai, Maharashtra, 400026, India
KEM HOSPITAL Research Centre
Pune, Maharashtra, 411011, India
Sir Ganga Ram Hospital
New Delhi, National Capital Territory of Delhi, 110060, India
Christian Medical College and Hospital.
Vellore, Tamil Nadu, 632004, India
Institute of Child Health
Kolkata, West Bengal, 700017, India
Maulana Azad Medical College
New Delhi, 110002, India
All India Institute of Medical Sciences
New Delhi, 110029, India
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Aabha Nagral, MD
Jaslok Hospital and Reseach Centre, Mumbai
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR
- Masking Details
- Double-blinded, randomized, placebo controlled
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 1, 2018
First Posted
November 19, 2018
Study Start
June 6, 2018
Primary Completion
April 1, 2020
Study Completion
October 15, 2021
Last Updated
January 11, 2023
Record last verified: 2023-01