Gaucher Disease, Type 1Phase 2NCT03746587
Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3
A Phase 2 study testing Arimoclomol in people with Gaucher Disease, Type 1 and Gaucher Disease, Type 3, run by ZevraDenmark at 8 sites in India.
Trial summary
Multicenter, doubleblinded, randomized placebo-controlled study of arimoclomol in patients with Gaucher Disease Type 1 or 3 Read more about this study
- Who is it for?
- Ages 4 to 60 years
- Conditions the study is for
- Gaucher Disease, Type 1, Gaucher Disease, Type 3
- What is being tested?
- Arimoclomol
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 39 participants
- Where
- 8 sitesIndia
- Study dates
- 6 Jun 2018to 15 Oct 2021
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
India8 sites
Institute of Child Health
Kolkata, West Bengal, 700017
King Edward Memorial Hospital
Mumbai, Maharashtra, 4000012
Jaslok Hospital and Reseach Centre
Mumbai, Maharashtra, 400026
Sir Ganga Ram Hospital
New Delhi, National Capital Territory of Delhi, 110060
Maulana Azad Medical College
New Delhi, 110002
All India Institute of Medical Sciences
New Delhi, 110029
KEM HOSPITAL Research Centre
Pune, Maharashtra, 411011
Christian Medical College and Hospital.
Vellore, Tamil Nadu, 632004
About this study
Official title: 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
Read the 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.
- Conditions
- Gaucher Disease, Type 1, Gaucher Disease, Type 3
- Being tested
- Arimoclomol, Placebo oral capsule
- Keywords
- Gaucher Disease
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ArimoclomolDrug
Arimoclomol in 3 different dosages
Placebo oral capsuleDrug
Matching placebo capsule
| Study group | Role | What they receive |
|---|---|---|
| Arimoclomol I | Experimental | Arimoclomol, oral capsule Receives: Drug: Arimoclomol |
| Arimoclomol II | Experimental | Arimoclomol, oral capsule Receives: Drug: Arimoclomol |
| Arimoclomol III | Experimental | Arimoclomol, oral capsule Receives: Drug: Arimoclomol |
| Placebo | Placebo comparator | Placebo oral capsule matching experimental arm Receives: Drug: Placebo oral capsule |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary Endpoint
The percentage change in serum chitotriosidase levels from baseline to 6 months
Measured: 6 months
Secondary outcomes (5)
Growth Endpoint
Change in weight curve (Khadilkar and Khadilkar, 2011) measured in kilograms, at every 6 months
Measured: 6 months
Growth Endpoint
Change in length curve (Khadilkar and Khadilkar, 2011) measured in meters at every 6 months
Measured: 6 months
Maturation Endpoint
Age at pubertal onset (Tanner Stage II) for subjects who had not reached puberty at screening
Measured: 6 months
Maturation Endpoint
Tanner Staging at baseline, 6 months and at least every 12 months (Tanner stage I-IV)
Measured: 6 - 12 months
Imaging Endpoint
Change in size of liver and spleen assessed by ultrasound
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2018
- Study start6 Jun 2018
- Primary completion1 Apr 2020
- Study completion15 Oct 2021
Why it stopped: COVID-19 pandemic made it impossible to assess the trial objective
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher Disease, Type 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Randomized
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- ZevraDenmark
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Aabha Nagral, MD, Principal investigator, Jaslok Hospital and Reseach Centre, Mumbai
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03746587
- Sponsor's protocol ID
- OR-ARI-GAU-01
- Status last verified
- Jan 2023
- First submitted
- 1 Nov 2018
- Last updated
- 11 Jan 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 11 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicentre Double-blinded, Randomized Placebo-controlled Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3
- ClinicalTrials.gov ID
- NCT03746587
- Acronym / sponsor ID
- OR-ARI-GAU-01
- Conditions
- Gaucher Disease, Type 1, Gaucher Disease, Type 3
- Keywords
- Gaucher Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: COVID-19 pandemic made it impossible to assess the trial objective
- Enrolment
- 39 participants (actual)
- Lead sponsor
- ZevraDenmarkIndustry (pharma, biotech, devices)
- Study start
- 6 Jun 2018 (actual)
- Primary completion
- 1 Apr 2020 (actual)
- Study completion
- 15 Oct 2021 (actual)
- First posted
- 19 Nov 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 4
- Model description
- Randomized
| Arm | Type | Receives |
|---|---|---|
| Arimoclomol I | Experimental | Drug: ArimoclomolArimoclomol, oral capsule |
| Arimoclomol II | Experimental | Drug: ArimoclomolArimoclomol, oral capsule |
| Arimoclomol III | Experimental | Drug: ArimoclomolArimoclomol, oral capsule |
| Placebo | Placebo comparator | Drug: Placebo oral capsulePlacebo oral capsule matching experimental arm |
Who may qualify
- Ages
- 4 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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 (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ArimoclomolArimoclomol in 3 different dosages | Drug | Arimoclomol I, Arimoclomol II, Arimoclomol III |
| Placebo oral capsuleMatching placebo capsule | Drug | Placebo |
Primary outcome
Primary Endpoint
Time frame: 6 months
The percentage change in serum chitotriosidase levels from baseline to 6 months
5 secondary outcomes
Growth Endpoint
Time frame: 6 months
Growth Endpoint
Time frame: 6 months
Maturation Endpoint
Time frame: 6 months
Maturation Endpoint
Time frame: 6 - 12 months
Imaging Endpoint
Time frame: 6 months
Results and safety
The study was stopped early (reason given: COVID-19 pandemic made it impossible to assess the trial objective). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jan 2023.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Aabha Nagral, MD principal investigator
Jaslok Hospital and Reseach Centre, Mumbai
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Arimoclomol in people with Gaucher Disease, Type 1 and Gaucher Disease, Type 3, run by ZevraDenmark at 8 sites in India.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 11 Jan 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in India. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: COVID-19 pandemic made it impossible to assess the trial objective). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary Endpoint (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jan 2023.