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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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23 January 2023 |
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Main ID: |
NCT03746587 |
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Date of registration:
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01/11/2018 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3
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Scientific title:
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Multicentre Double-blinded, Randomized Placebo-controlled Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3 |
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Date of first enrolment:
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June 6, 2018 |
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Target sample size:
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39 |
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Recruitment status: |
Terminated |
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URL:
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https://clinicaltrials.gov/show/NCT03746587 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Investigator).
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Phase:
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Phase 2
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Countries of recruitment
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India
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Contacts
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Name:
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Aabha Nagral, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Jaslok Hospital and Reseach Centre, Mumbai |
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Key inclusion & exclusion criteria
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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
Age minimum:
4 Years
Age maximum:
60 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Gaucher Disease, Type 1
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Gaucher Disease, Type 3
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Intervention(s)
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Drug: Placebo oral capsule
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Drug: Arimoclomol
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Primary Outcome(s)
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Primary Endpoint
[Time Frame: 6 months]
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Secondary Outcome(s)
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Maturation Endpoint
[Time Frame: 6 - 12 months]
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Maturation Endpoint
[Time Frame: 6 months]
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Imaging Endpoint
[Time Frame: 6 months]
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Growth Endpoint
[Time Frame: 6 months]
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Secondary ID(s)
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OR-ARI-GAU-01
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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