Gaucher Disease Clinical Trials
Here are the 3 most popular medical studies for gaucher disease
Enzyme Analysis for Gaucher Disease
This trial is testing whether a registry of patients' medical records can be used to identify people with undiagnosed Gaucher disease, a rare genetic disorder. If successful, the study could help doctors better understand and treat the condition.
Popular filter options for gaucher disease trials
GD Clinical Trials
View 10 GD medical studies.
Persons with electronic health records suggestive of Gaucher disease for Gaucher Disease
This trial is divided into three phases. In Phase I, a screening algorithm will be used to identify patients who may have Gaucher disease. The top 50 patients from each healthcare system will be moved
Monoclonal Antibodies
LY3884961 for Gaucher Disease
This trial is a Phase 1/2, multicenter, open-label study of LY3884961, which is being evaluated for safety and tolerability in adults with peripheral manifestations of GD. Up to 3 dose levels of LY3884961 will be assessed in 3 dose-finding cohorts of 3 patients, and then up to 6 patients may be enrolled into an expansion cohort. For each enrolled patient, the study will last approximately 5 years, including up to a 45-day screening period. During the first 18 months after dosing, subjects will be evaluated for the effects of LY3884961 on safety, tolerability, immun
Substrate Reduction Therapy
Venglustat vs Imiglucerase for Gaucher Disease
This trial will compare the effects of two different treatments for Gaucher disease Type 3. One treatment is a daily pill, and the other is an infusion every two weeks. The trial will measure how well each treatment works in improving or stabilizing the neurological symptoms and overall disease stability.
Gaucher Disease Clinical Trials With No Placebo
View 10 gaucher disease medical studies that do not have a placebo group.
Persons with electronic health records suggestive of Gaucher disease for Gaucher Disease
This trial is divided into three phases. In Phase I, a screening algorithm will be used to identify patients who may have Gaucher disease. The top 50 patients from each healthcare system will be moved
Monoclonal Antibodies
LY3884961 for Gaucher Disease
This trial is a Phase 1/2, multicenter, open-label study of LY3884961, which is being evaluated for safety and tolerability in adults with peripheral manifestations of GD. Up to 3 dose levels of LY3884961 will be assessed in 3 dose-finding cohorts of 3 patients, and then up to 6 patients may be enrolled into an expansion cohort. For each enrolled patient, the study will last approximately 5 years, including up to a 45-day screening period. During the first 18 months after dosing, subjects will be evaluated for the effects of LY3884961 on safety, tolerability, immun
Substrate Reduction Therapy
Venglustat vs Imiglucerase for Gaucher Disease
This trial will compare the effects of two different treatments for Gaucher disease Type 3. One treatment is a daily pill, and the other is an infusion every two weeks. The trial will measure how well each treatment works in improving or stabilizing the neurological symptoms and overall disease stability.
Frequently Asked Questions
Introduction to gaucher disease
What are the top hospitals conducting gaucher disease research?
In the field of rare diseases, clinical trials play a crucial role in advancing research and improving treatment options. When it comes to Gaucher disease, several top hospitals are leading the way in groundbreaking studies. Located in Fairfax, Virginia, the Lysosomal Rare Disorders Research and Treatment Center is currently conducting two active clinical trials for Gaucher disease—the first recorded trial taking place as recently as 2022. Similarly, at Duke University Health System in Durham, North carolina, researchers are focusing their efforts on one active trial for this rare condition.
Heading west to Los Angeles brings us to Kaiser Permanente—a hospital with extensive experience in Gaucher disease research and treatment. With one ongoing trial and two all-time trials under its belt since initiating studies back in 2016, this institution continues to contribute significantly to our understanding of this challenging disorder. In Chicago's Ann and Robert H Lurie Children's Hospital of Chicago—one active clinical trial is underway for Gaucher disease while they have completed two previous investigations since embarking on their first mission against this condition also backin 2016
Finally arriving at Columbia University Irving Medical Center located right here within New york Citywe find another beacon of hope: one active clinical trial focused specifically on Gaucher disease—a testament to their commitment towards combating devastating illnesses such as these; which thus far has lasted only untilrecently initiated investigation dating from early2022.
These hospitals dedicatedly invest time and resources into assisting individuals affected by Gaucher disease while concurrently pushing boundaries through innovative research endeavors.By gaining insights into how this complex ailment manifests itselfand developing novel treatments or interventions that provide relief; researchers aim towards unlocking brighter futures for those living with such conditions- globally
Which are the best cities for gaucher disease clinical trials?
When it comes to gaucher disease clinical trials, several cities emerge as key players in the research and development of potential treatments. Fairfax, Virginia leads with 8 active trials investigating Venglustat, imiglucerase, LY3884961, and other promising options. Pittsburgh, Pennsylvania follows closely behind with 3 ongoing studies exploring FLT201, LY3884961, Treatment, and more. New Haven in Connecticut also contributes significantly with 3 active trials focusing on imiglucerase and Venglustat among others. These cities offer individuals battling gaucher disease access to cutting-edge clinical trials that pave the way for advancements in care and hope for improved outcomes.
Which are the top treatments for gaucher disease being explored in clinical trials?
Exciting progress is being made in the exploration of clinical trials for Gaucher disease, with several promising treatments at the forefront. Leading the charge is LY3884961, currently involved in two active trials and marking its debut in 2021. Joining the ranks are FLT201 and Venglustat—both first listed in 2022—with one active trial each dedicated to Gaucher disease. Also making waves is Aldurazyme (laronidase), newly listed in 2021, participating actively in a single trial. Finally, Imiglucerase GZ437843 rounds out this group with one ongoing study since it was introduced back in 2018. With these innovative approaches being explored further through clinical trials, hope shines brighter than ever for patients living with Gaucher disease.
What are the most recent clinical trials for gaucher disease?
Exciting advancements in the field of Gaucher disease treatment have emerged through recent clinical trials. LY3884961, a promising candidate currently in Phase 1 and Phase 2 studies, has shown potential for effective management of this rare genetic disorder. Similarly, Venglustat has reached Phase 3 trials, offering hope for patients with Gaucher disease. FLT201 and Aldurazyme (laronidase) are additional treatments that have entered Phase 1 trials to explore their efficacy and safety profiles. With these cutting-edge therapies under investigation, there is renewed optimism for the future of Gaucher disease management.
What gaucher disease clinical trials were recently completed?
Recently, several clinical trials focusing on Gaucher disease have reached completion, providing valuable insights for the treatment of this rare genetic disorder. These include a trial sponsored by Sanofi Genzyme that concluded in December 2021, investigating a potential therapy for Type 3 Gaucher disease. Another significant study completed in September 2021 was led by Shire (now Takeda) and explored the efficacy of an enzyme replacement therapy for patients with Type 1 Gaucher disease. Both trials contribute to our understanding of potential treatments and offer hope for individuals affected by this debilitating condition.