Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3 (NCT03746587) | Clinical Trial Compass
TerminatedPhase 2
Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3
Stopped: COVID-19 pandemic made it impossible to assess the trial objective
India39 participantsStarted 2018-06-06
Plain-language summary
Multicenter, doubleblinded, randomized placebo-controlled study of arimoclomol in patients with Gaucher Disease Type 1 or 3
Who can participate
Age range4 Years – 60 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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