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RNAi Therapeutics

Vutrisiran vs Patisiran for Amyloidosis

Phase 3

Waitlist Available

Research Sponsored by Alnylam Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Has a diagnosis of hATTR amyloidosis with transthyretin (TTR) mutation

Male or female of 18 to 85 years of age (inclusive)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to month 18

Awards & highlights

Study Summary

This trial is studying vutrisiran, which is given as a shot under the skin, to see how well it works compared to patisiran, which is given as a drip into a vein, for people with hereditary ATTR amyloidosis. People will get vutrisiran or patisiran once every 3 months or 3 weeks, respectively, for 18 months. The trial will use the placebo arm of the APOLLO study as an external comparator for the primary and most other efficacy endpoints during the 18 Month Treatment Period. Following the 18 Month Treatment Period, all participants will be randomized to receive

Who is the study for?

This trial is for adults aged 18-85 with hereditary transthyretin amyloidosis, a condition where abnormal proteins build up in the body. Participants should have specific levels of neurologic impairment and physical functioning, and not have received other TTR-lowering treatments or had a liver transplant.Check my eligibility

What is being tested?

The study compares Vutrisiran, given as an injection under the skin every three months, to Patisiran, administered intravenously every three weeks. After 18 months, participants may receive Vutrisiran less frequently in an extension period.See study design

What are the potential side effects?

Potential side effects include reactions at the injection site for Vutrisiran or infusion-related reactions for Patisiran. Both drugs could potentially cause liver function abnormalities and may affect nerve function.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have hATTR amyloidosis with a TTR mutation.

Select...

I am between 18 and 85 years old.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to month 18

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to month 18

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to month 18 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change From Baseline in the Modified Neurologic Impairment Score +7 (mNIS+7) at Month 9 Between the Vutrisiran Group (HELIOS-A) and the External Placebo Comparator Group [APOLLO (NCT01960348)]

Secondary outcome measures

Change From Baseline in Norfolk QoL-DN Total Score at Month 18 Between the Vutrisiran Group (HELIOS-A) and the External Placebo Comparator Group [APOLLO (NCT01960348)]

Change From Baseline in Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Total Score at Month 9 Between the Vutrisiran Group (HELIOS-A) and the External Placebo Comparator Group [APOLLO (NCT01960348)]

Change From Baseline in the 10-MWT at Month 18 Between the Vutrisiran Group (HELIOS-A) and the External Placebo Comparator Group [APOLLO (NCT01960348)]

+5 more

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: Vutrisiran + Vutrisiran (HELIOS-A)Experimental Treatment1 Intervention

Participants will receive vutrisiran 25 mg subcutaneous (SC) injection once every 3 months (q3M) for 18 months during the Treatment Period followed by vutrisiran SC injection once every 6 months (q6M) or q3M during the Randomized Treatment Extension (RTE) Period.

Group II: Patisiran + Vutrisiran (HELIOS-A)Active Control2 Interventions

Participants will receive patisiran 0.3 mg/kg intravenous (IV) infusion once every 3 weeks (q3w) for 18 months during the Treatment Period followed by vutrisiran SC injection q6M or q3M during the RTE Period.

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

Alnylam PharmaceuticalsLead Sponsor

72 Previous Clinical Trials

14,654 Total Patients Enrolled

29 Trials studying Amyloidosis

11,014 Patients Enrolled for Amyloidosis

Medical DirectorStudy DirectorAlnylam Pharmaceuticals

2,777 Previous Clinical Trials

8,063,316 Total Patients Enrolled

12 Trials studying Amyloidosis

3,424 Patients Enrolled for Amyloidosis

Media Library

Vutrisiran (RNAi Therapeutics) Clinical Trial Eligibility Overview. Trial Name: NCT03759379 — Phase 3

Amyloidosis Research Study Groups: Vutrisiran + Vutrisiran (HELIOS-A), Patisiran + Vutrisiran (HELIOS-A)

Amyloidosis Clinical Trial 2023: Vutrisiran Highlights & Side Effects. Trial Name: NCT03759379 — Phase 3

Vutrisiran (RNAi Therapeutics) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03759379 — Phase 3

Frequently Asked Questions

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

Are there still slots available for people who want to participate in this trial?

"This particular clinical trial, which was first announced on February 14th 2019, is not presently looking for patients. However, there are other 101 medical trials that are actively recruiting."

Answered by AI

Have other research groups conducted similar investigations?

"Vutrisiran (ALN-TTRSC02) has been under clinical scrutiny since 2019. The first official study took place that year and was sponsored by Alnylam Pharmaceuticals. After the initial study of 164 patients, Vutrisiran (ALN-TTRSC02) received Phase 3 drug approval. There are currently 4 live trials involving Vutrisiran (ALN-TTRSC02), which are located in 27 cities across 42 countries."

Answered by AI

Does this research program allow for geriatric patients?

"The age range for this particular trial is 18 to 85. However, there are other studies for minors and seniors outside of this age bracket."

Answered by AI

Is Vutrisiran (ALN-TTRSC02) a new medication?

"First appearing in 2019 at a clinical trial site, Vutrisiran (ALN-TTRSC02) has completed 8 trials as of now. Currently, 4 more trials are underway, 2 of which are based in Montréal while the other 2 are in Pennsylvania."

Answered by AI

Is this trial popular in North America?

"16 sites across North America are currently recruiting patients for this clinical trial. While the locations of some of the centres (Montréal, Vancouver and Philadelphia) have been mentioned, please be aware that there are many other cities with active recruitment. To help reduce the amount of travel needed, please try and select the site that is closest to your home."

Answered by AI

Has Vutrisiran (ALN-TTRSC02) undergone FDA approval?

"Vutrisiran (ALN-TTRSC02) is estimated to be a 3 on a scale of 1-3 in regards to safety. This is due to the fact that it is a Phase 3 trial, meaning that there is some data to support both its efficacy and safety."

Answered by AI

Recent research and studies

AmyloidJournal

Efficacy and Safety of Vutrisiran for Patients with Hereditary Transthyretin-mediated Amyloidosis with Polyneuropathy: A Randomized Clinical Trial

Adams, David, Ivailo L. Tournev, Mark S. Taylor, Teresa Coelho, Violaine Planté-Bordeneuve, John L. Berk, Alejandra González-Duarte, et al.. 2022. “Efficacy and Safety of Vutrisiran for Patients with Hereditary Transthyretin-mediated Amyloidosis with Polyneuropathy: A Randomized Clinical Trial”. Amyloid. Informa UK Limited. doi:10.1080/13506129.2022.2091985.

clinicaltrials.govStudy

HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

2019. "HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03759379.

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