Gaucher’s disease type III – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Sat, 20 Jun 2026 04:03:28 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Gaucher’s disease type III – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study on the Effectiveness and Safety of Venglustat for Adults and Children with Gaucher Disease Type 3 https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-venglustat-for-adults-and-children-with-gaucher-disease-type-3/ Wed, 29 Apr 2026 15:03:03 +0000 https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-venglustat-for-adults-and-children-with-gaucher-disease-type-3/ This clinical trial is focused on studying *Gaucher disease Type 3*, a rare genetic disorder that affects various parts of the body, including the brain. The study aims to evaluate the effectiveness and safety of a medication called *venglustat* (also known by its code names GZ402671 and SAR402671) in both adults and children who have been managing their condition with a treatment known as *Enzyme Replacement Therapy* (ERT). The trial will compare *venglustat* to another treatment called *Cerezyme*, which is a form of ERT that contains the active substance *imiglucerase*. Participants in the study will receive either *venglustat* or *Cerezyme*, and some may receive a placebo.

The purpose of the study is to see how well *venglustat* works in improving symptoms of *Gaucher disease Type 3*, such as problems with movement and thinking skills. The study will look at changes in movement using a tool called the Scale for the Assessment and Rating of Ataxia (SARA) and changes in thinking skills using the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS). Participants will be monitored over a period of time to assess these changes and to ensure their safety while taking the medication.

Throughout the study, participants will have regular check-ups to monitor their health and any changes in their condition. The study will also track other health indicators, such as spleen and liver size, blood levels, and any side effects that may occur. The trial is designed to provide valuable information on whether *venglustat* can be a safe and effective treatment option for people with *Gaucher disease Type 3*.

]]> Study of Eliglustat alone or with Imiglucerase in children and adolescents aged 2-17 years with Gaucher disease type 1 and type 3 https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-eliglustat-and-imiglucerase-for-children-with-gaucher-disease-types-1-and-3/ Wed, 29 Apr 2026 14:29:54 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-eliglustat-and-imiglucerase-for-children-with-gaucher-disease-types-1-and-3/ This study focuses on Gaucher disease type 1 and type 3, a rare genetic disorder that affects various organs in the body. The study will test a medication called eliglustat, which is available in both capsule form and as a powder for oral suspension. Some participants will also receive imiglucerase, which is given through an intravenous infusion.

The purpose of this research is to evaluate how safe eliglustat is and how it moves through the body in young patients between 2 and 18 years of age. The study includes two groups of patients: one group will receive only eliglustat, while the other group will receive both eliglustat and imiglucerase. The treatment will continue for approximately one year.

During the study, doctors will monitor various aspects of the disease, including blood cell counts, liver and spleen size, lung function, and bone health. They will also track any side effects that may occur during treatment. The study will additionally look at how the treatment affects the quality of life of young patients.

]]> Study of Venglustat and Imiglucerase for Adult Patients with Gaucher Disease Type 3 https://clinicaltrials.eu/trial/study-of-venglustat-and-imiglucerase-for-adult-patients-with-gaucher-disease-type-3/ Wed, 29 Apr 2026 14:25:48 +0000 https://clinicaltrials.eu/trial/study-of-venglustat-and-imiglucerase-for-adult-patients-with-gaucher-disease-type-3/ This clinical trial is focused on studying Gaucher disease, specifically types 1 and 3. Gaucher disease is a genetic disorder where fatty substances accumulate in certain organs, particularly the spleen and liver. The study involves a treatment using a medication called venglustat, which is taken as a capsule, and another medication called Cerezyme (also known as imiglucerase), which is given through an intravenous infusion. Venglustat is also referred to by its code name, GZ402671 – SAR402671.

The purpose of the study is to evaluate the safety and tolerability of venglustat, both in combination with Cerezyme and as a standalone treatment, in adult patients with Gaucher disease type 3. The study is divided into four parts. Initially, it involves evaluating certain markers in the cerebrospinal fluid, which is the fluid surrounding the brain and spinal cord, to distinguish between Gaucher disease types 1 and 3. Following this, the study will assess the short-term and long-term effects of the combination treatment. Finally, the study will explore the effects of venglustat alone in patients who have shown stability with the combination treatment.

Throughout the study, participants will receive either the combination of venglustat and Cerezyme or venglustat alone, depending on the phase of the study. The study aims to monitor changes in specific markers related to the central nervous system and ensure the treatment is safe and well-tolerated by the participants. The study will not explain the use of a placebo, as it is not part of this trial.

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