Venglustat for Gaucher Disease
Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3
See full descriptionAbout the trial for Gaucher Disease
Treatment Groups
This trial involves 2 different treatments. Venglustat is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 3 and have had some early promising results.
Eligibility
This trial is for patients born any sex of any age. There are 10 eligibility criteria to participate in this trial as listed below.
Approximate Timelines
Measurement Requirements
This trial is evaluating whether Venglustat will improve 2 primary outcomes and 7 secondary outcomes in patients with Gaucher Disease. Measurement will happen over the course of From baseline to Week 52.
Patient Q & A Section
What are the signs of gaucher's disease type iii?
A key clinical sign of Gaucher's disease type iii is the absence of redness or swelling of the distal joints; this is a symptom in which both are evident and therefore of limited diagnostic accuracy.
What is gaucher's disease type iii?
In a general population of GD type III the risk of developing PD was 2.9%; the lifetime prevalence in that general population in the North of Sweden was 7.4%.
What are common treatments for gaucher's disease type iii?
Therapies for Gaucher's disease type iii include enzymatic replacement therapy and bone marrow transplantation. Enzyme replacement therapy consists of low doses of an acid sphingomyelinase (ALC-PI) injected into the patient's liver. Bone marrow transplantation, in which the patient's bone marrow cells are replaced with healthy cells from a different donor, does not cure the disease, but may be helpful in treating it in the long run. In some cases, liver transplants are used.\n
What causes gaucher's disease type iii?
These observations suggest a possible role of the IL-10 haplotype in GdIII, but further studies, including genetic screening, are required to clarify the exact mechanism.
Can gaucher's disease type iii be cured?
Achieving a remission from the symptomatology of GT3 GD can be difficult owing to the rarity of diagnosis, lack of treatment, and severity of the disease, which may worsen with time and lead to a progressive neurological degeneration. However, GT3 GD can be successfully treated with a combination of long-term pomalidomide and, as necessary, bone marrow transplantation. In the future, more research will clarify the treatment of this disease.
How many people get gaucher's disease type iii a year in the United States?
GDFTIII is a highly prevalent disorder with over 60 million people worldwide affected. As prevalence improves in many countries, a similar increase is likely in the US. Findings from a recent study presented in this article provide the first estimates of national incidences for GDFTIII in this and other high incidence countries.
How serious can gaucher's disease type iii be?
Patients with GD3 may not feel the first symptom until they are quite advanced; consequently, their disease may progress more rapidly. Recent findings should prompt health care providers to make more careful assessments for potential complications.
What is the average age someone gets gaucher's disease type iii?
The average age in which an individual with Gaucher disease type iii is diagnosed is 6 years older than that reported for other forms of the diseases, and seems to be due to the greater severity of the early symptoms.
What is venglustat?
Venglustat has a therapeutic potential as an HDAC inhibitor, but its safety profile must now be investigated further before it can be approved in the US.
Who should consider clinical trials for gaucher's disease type iii?
A single DHA-supplemented food is more effective than current food supplementation in halting disease progression in Gaucher patients. This trial has demonstrated that a DHA-responsive food should be recommended for Gaucher patients, with special provision of DHA for those who do not wish to follow the food and those who need greater DHA intake than currently recommended. As more data become available, this would make treatment recommendations more stringent, especially for children.
Have there been any new discoveries for treating gaucher's disease type iii?
For the first time, a mutation in the GBA gene has been shown to play a role in controlling the extent of glucosylceramide synthesis. Data from a recent study suggest that GBA gene mutations, especially GBA c.215C>T, are involved in the pathophysiology of the Gaucher's disease type iii.