Vutrisiran (ALN-TTRSC02) for Amyloidosis
This study is evaluating whether a drug called vutrisiran can treat hereditary transthyretin amyloidosis.
See full descriptionAbout the trial for Amyloidosis
Treatment Groups
This trial involves 2 different treatments. Vutrisiran (ALN-TTRSC02) 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 aged 18 and older. There are 5 eligibility criteria to participate in this trial as listed below.
Approximate Timelines
Measurement Requirements
This trial is evaluating whether Vutrisiran (ALN-TTRSC02) will improve 1 primary outcome and 8 secondary outcomes in patients with Amyloidosis. Measurement will happen over the course of Baseline, Month 9.
Patient Q & A Section
What is amyloidosis?
Amyloidosis refers to a group of conditions where abnormal amyloid material is deposited in an unrelated and usually peripheral body organ system. It occurs in normal aging but becomes important with age. \n\nThe prevalence of atrial amyloidosis increases with age. Amyloidosis can be an incidental finding in individuals without any prior history of an inflammatory or chronic lung disease.\n\nThe pathophysiology of amyloidosis depends on two crucial aspects. First of all it depends on the production of, or adherence of an abnormal substance to cardiorthracheal connective tissue of the lungs.
Can amyloidosis be cured?
Amyloidosis does not have a curative potential if it spreads to involve the heart (particularly the medullary region), to occur in association with AL amyloidosis, and if the underlying cause is not treated.
What are common treatments for amyloidosis?
Patients are commonly treated through standard therapy and supportive measures in addition to palliative measures. Treatment options include chemotherapy, blood-cell transplantation, amyloidogenic treatment, and dialysis. Allowing patients to make decisions for treatment may help to improve outcomes.
What causes amyloidosis?
Liver or kidney failure leads to protein breakdown from overabundance, and the resulting breakdown product, amyloid fibrils, accumulate in a specific subset of tissues that includes the lungs, heart, kidneys, and spleen. Ultimately, the accumulation of amyloid fibrils may restrict blood or lymphatic circulation. Over many years, these changes lead to the development of a specific amyloid deposition pattern that can be detected in these tissues. We speculate that this proteinaceous deposit is a hallmark of amyloidosis and that it may be an early clue for identifying the disease.
How many people get amyloidosis a year in the United States?
As with many diseases, amyloidosis is less common in areas with an immigrant population from Southeast Asia (the Philippines). This may be due in part to low amyloidosis screening rates, possibly related to lack of awareness from the local healthcare providers.
What are the signs of amyloidosis?
The early signs are frequently related to underlying disease processes and include loss of appetite, weight loss, and fatigue. As disease progresses, symptoms become more significant and are more pronounced in some patient populations.
What is the latest research for amyloidosis?
Since the advent of biopharmaceuticals, new treatment options for AL amyloidosis have been created that provide better quality of life. In addition, there is more efficient and new research for AL amyloidosis.
What is vutrisiran (aln-ttrsc02)?
This trial demonstrates compelling results that indicate that a single dose of vutrisiran achieves complete and durable responses in patients with AL amyloidosis, including amyloidosis-related end organ damage.
Is vutrisiran (aln-ttrsc02) safe for people?
Vutrisiran is highly tolerable, with no significant safety concern; the most common adverse events (>2%) were mild to moderate, and none were serious. There was no evidence of an increased risk of heart block, hypotension, or other serious adverse events compared with placebo or at the dose studied.
Does amyloidosis run in families?
It has been well known for quite long that familial cases of amyloidosis have been identified, but the incidence of familial amyloidosis has not been determined in clinical populations for a period as long. The prevalence of familial amyloidosis is probably high but remains undetected because of the low frequency of the disease.
What is the primary cause of amyloidosis?
A diagnosis of AL amyloidosis with trisomy 18 is made in a patient with other features of trisomy for 18. Diagnosis typically occurs in the 40-year-old adult age group; however, there are cases noted in children in the 1st and 2nd decades.