CLINICAL TRIAL

Vutrisiran (ALN-TTRSC02) for Amyloidosis

Waitlist Available · 18+ · All Sexes · La Mesa, CA

This study is evaluating whether a drug called vutrisiran can treat hereditary transthyretin amyloidosis.

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About the trial for Amyloidosis

Eligible Conditions
Amyloidosis · Transthyretin Amyloidosis · Amyloidosis, Hereditary · Amyloidosis, Familial · Amyloid Neuropathies, Familial

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.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Vutrisiran (ALN-TTRSC02)
DRUG
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.
Patisiran
DRUG
Vutrisiran (ALN-TTRSC02)
DRUG

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.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
Any person aged 18 to 85 (inclusive) is eligible to participate in the study. show original
The text states that the person has an adequate polyneuropathy disability score. show original
This patient has an adequate neurologic impairment score. show original
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Odds of Eligibility
Unknown<50%
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Up to Month 18
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Up to Month 18.
View detailed reporting requirements
Trial Expert
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- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

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.

Change from Baseline in the Timed 10-Meter Walk Test (10-MWT) at Month 9
BASELINE, MONTH 9
Change from Baseline in the Modified Neurologic Impairment Score +7 (mNIS+7) at Month 9
BASELINE, MONTH 9
The mNIS+7 is a composite score that quantifies motor, sensory, and autonomic neurologic impairment due to injury of large and small nerves. The minimum and maximum values are 0 and 304, respectively. A higher score indicates a worse outcome.
Change from Baseline in Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Total Score at Month 9
BASELINE, MONTH 9
The Norfolk QoL-DN questionnaire is a standardized 35-item patient-reported outcomes measure that is sensitive to the different features of diabetic neuropathy - small fiber, large fiber, and autonomic nerve function. The minimum and maximum values are -4 and 136, respectively. A higher score indicates a worse outcome.
Change from Baseline in Norfolk QoL-DN Total Score at Month 18
BASELINE, MONTH 18
The Norfolk QoL-DN questionnaire is a standardized 35-item patient-reported outcomes measure that is sensitive to the different features of diabetic neuropathy - small fiber, large fiber, and autonomic nerve function. The minimum and maximum values are -4 and 136, respectively. A higher score indicates a worse outcome.
Change from Baseline in the Modified Body Mass Index (mBMI) at Month 18
BASELINE, MONTH 18
Change from Baseline in the Rasch-Built Overall Disability Scale (R-ODS) at Month 18
BASELINE, MONTH 18
The R-ODS is comprised of a 24-item linearly weighted scale that specifically captures activity and social participation limitations. The minimum and maximum values are 0 and 48, respectively. A higher score indicates a better outcome.
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Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer

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.

Anonymous Patient Answer
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