Gaucher’s disease type I – 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 I – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 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 on the Long-term Safety of FLT201 for Patients with Gaucher Disease Type 1 https://clinicaltrials.eu/trial/study-on-the-long-term-safety-of-flt201-for-patients-with-gaucher-disease-type-1/ Wed, 29 Apr 2026 14:29:51 +0000 https://clinicaltrials.eu/trial/study-on-the-long-term-safety-of-flt201-for-patients-with-gaucher-disease-type-1/ This clinical trial is focused on studying Gaucher Disease Type 1, a genetic disorder that affects the body’s ability to break down a certain type of fat, leading to its buildup in various organs. The study is investigating a treatment called FLT201, which is a special type of therapy using an adeno-associated viral vector. This vector is designed to deliver a modified gene that helps produce an enzyme called beta-glucocerebrosidase, which is deficient in people with Gaucher Disease. The treatment is given as a solution for infusion, meaning it is administered directly into the bloodstream.

The purpose of this study is to assess the long-term safety of FLT201 in individuals who have Gaucher Disease Type 1. Participants in the study will be monitored over an extended period to observe how their bodies respond to the treatment. This includes checking for any side effects and measuring changes in various health indicators, such as enzyme activity levels and organ sizes, using methods like MRI scans. The study will also track the presence of the treatment in the body over time.

Participants in this study are those who have previously received the FLT201 treatment. They will be required to attend regular follow-up visits to ensure their safety and to gather important data on the treatment’s effects. The study aims to provide valuable information on the safety and effectiveness of this innovative therapy for managing Gaucher Disease Type 1.

]]> Study on the Safety of LY3884961 for Patients with Type 1 Gaucher Disease https://clinicaltrials.eu/trial/study-on-the-safety-of-ly3884961-for-patients-with-type-1-gaucher-disease/ Wed, 29 Apr 2026 14:27:03 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-of-ly3884961-for-patients-with-type-1-gaucher-disease/ This clinical trial is focused on studying Type 1 Gaucher Disease, which is a condition that affects certain parts of the body but not the brain. The study will test a new treatment called LY3884961, which is given as a single dose through an intravenous infusion, meaning it is delivered directly into the bloodstream. The main goal of the study is to see how safe and tolerable this treatment is for patients with this type of Gaucher Disease.

During the study, participants will receive a single dose of LY3884961 and will be monitored for any side effects or changes in their health. This includes regular checks of vital signs, blood tests, and imaging tests like MRIs to look at the abdomen and bones. The study will also track changes in certain proteins and antibodies in the blood, which can help understand how the body is responding to the treatment.

The study will take place over several years, with participants being followed up regularly to monitor their health and any long-term effects of the treatment. The researchers will also look at how the treatment affects the size of the spleen, the number of platelets in the blood, and the activity of certain enzymes related to Gaucher Disease. This information will help determine if LY3884961 could be a beneficial treatment option for people with Type 1 Gaucher Disease.

]]> 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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