Gaucher Disease Clinical Trials 2023
Browse 10 Gaucher Disease Medical Studies Across 18 Cities
2 Phase 3 Trial · 36 Gaucher Disease Clinics
LY3884961for Gaucher Disease
FLT201for Gaucher Disease
Venglustatfor Gaucher Disease
Aldurazyme (laronidase)for Myocardial Perfusion Imaging
LY3884961for Gaucher Disease
Imiglucerase GZ437843for Gaucher Disease
Imiglucerasefor Gaucher Disease
N-acetylcysteinefor Gaucher Disease
Enzyme Analysisfor Gaucher Disease
Treatmentfor Gaucher Disease
What Are Gaucher Disease Clinical Trials?
The condition is caused by the buildup of fatty substances in different organs of the body, such as the liver and spleen. Gaucher Disease leads to organ enlargement, thereby affecting their functioning. Moreover, the fatty substance can also build up in bone tissue which can cause the bones to weaken and increase the chances of fractures.
Gaucher Disease clinical trials are carried out to determine the impact of the disease on the patients and the best treatments to alleviate the condition. These clinical trials aim to improve the care and outcome of patients with GD.
Why Is Being Studied Through Clinical Trials Gaucher Disease?
Gaucher Disease (GD) is a rare condition with a prevalence rate of 1.75/100,000 individuals. The condition has a birth incidence of 0.58/100,000 people. Gaucher Disease can be divided into three types. Type 1 is more common in Western countries like Europe, the US, and Israel. However, GD types 2 and 3 are more common in non-western countries like the Middle East, China, Korea, and Japan.
Gaucher Disease clinical trials are underway for multiple reasons. These trials can provide valuable insights to clinicians and researchers to find the cure for the disease. Currently, no cure for GD is available. Moreover, through these clinical trials, researchers can determine the efficacy of novel treatments and ways to manage the symptoms.
What Are the Types of Treatments Available for Gaucher Disease?
Treatments available can only control the Gaucher disease symptoms, which include an enlarged spleen, enlarged liver, yellow spots in the patient's eyes, fatigue, and lung problems. Treatments depend on the type and severity of Gaucher's disease. These include:
- Transplantation of bone marrow
- Enzyme replacement therapy
- Blood transfusions
- Surgery or complete removal of the spleen
What Are Some Recent Breakthrough Clinical Trials for Gaucher Disease?
2005: Enzyme replacement therapy for Gaucher disease in Australia: The study was conducted to determine the effectiveness of ERT (enzyme replacement therapy) for patients with a severe type of Gaucher Disease. Forty-eight patients with GD received ERT for at least six months. Forty of these patients had Type 1 GD, while eight had Type 3. It was concluded that enzyme replacement therapy (ERT) provided beneficial results. Also, some evidence related to hematological complication reversal was collected. However, more evidence needs to be collected to examine ERT's effects on the quality of life of patients with GD.
2019: Safety and effectiveness of taliglucerase alfa in patients with Gaucher disease: The clinical trial featured 106 patients with Gaucher Disease. The trial was conducted to understand the efficacy and safety of TALIA (Taliglucerase Alfa). The study was carried out for five years.
The results consistently matched the known Taliglucerase Alfa effectiveness and safety. However, prior enzyme replacement therapy patients had a higher platelet count and hemoglobin concentration.
Who Are Some of the Key Opinion Leaders/Researchers/Institutions Conducting Gaucher Disease Clinical Trial Research?
It is a non-profit committed to serving GD patients through educational programs, quality patient services, financial support, and research. NGF aims to empower patients with GD to experience quality living.
The institute focuses on providing scientific advances and educational resources related to Gaucher disease to support the community of healthcare professionals and patients.