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Not recruitingRare disease

Study on the Effectiveness and Safety of Vutrisiran and Patisiran for Patients with Hereditary Transthyretin Amyloidosis

Verified siteRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis). This is a rare genetic disease where abnormal proteins build up in the body, leading to nerve damage and other health issues. The study is evaluating a treatment called ALN-TTRSC02, which is also known by its code name. This treatment is designed to help reduce the effects of the disease on the nervous system.

The purpose of the study is to assess how effective and safe ALN-TTRSC02 is for patients with hATTR Amyloidosis. Participants in the study will receive the treatment as a solution for injection. The study will also compare the effects of ALN-TTRSC02 with another treatment called Onpattro, which is given as a solution for infusion. Some participants may receive a placebo instead of the active treatment. The study will monitor changes in nerve function and quality of life over time.

Throughout the study, participants will undergo regular assessments to track their progress and any changes in their condition. The study aims to provide valuable information on how ALN-TTRSC02 can help manage the symptoms of hATTR Amyloidosis and improve the lives of those affected by this condition.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying age, diagnosis of hereditary transthyretin amyloidosis, and specific scores related to neuropathy and disability.

  2. Step 2

    Treatment allocation

    Participants are randomly assigned to receive either the study medication ALN-TTRSC02 or a comparator medication. The study is open-label, meaning both the participant and the study team know which treatment is being administered.

  3. Step 3

    Medication administration

    The study medication, vutrisiran (ALN-TTRSC02), is administered as a solution for injection. The comparator medication, patisiran, is given as a solution for infusion. The specific dosage and frequency are determined by the study protocol.

  4. Step 4

    Regular monitoring

    Participants undergo regular monitoring to assess the effect of the treatment on neurologic impairment and other health parameters. This includes evaluations of quality of life, walking ability, and body mass index.

  5. Step 5

    End of study evaluation

    At the end of the study, a final evaluation is conducted to measure changes from baseline in neurologic impairment and other health outcomes. The study is expected to conclude by May 30, 2026.

Who can join the trial?

6 criteria

  • Must be a male or female between the ages of 18 and 85, or older if that is the legal age of consent.
  • Must have a diagnosis of Hereditary Transthyretin Amyloidosis (hATTR amyloidosis), which is a condition caused by a change in the TTR gene.
  • Must have a Neuropathy Impairment Score between 5 and 130. This score measures nerve damage and must be confirmed during the second screening visit.
  • Must have a Polyneuropathy Disability score of 3b or less. This score assesses the level of disability due to nerve problems and must be confirmed during the second screening visit.
  • Must have a Karnofsky Performance Status (KPS) of 60% or higher. This score evaluates the ability to perform daily activities.
  • Must be willing and able to follow the study requirements and provide written consent to participate.
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Investigated drugs

ALN-TTRSC02 is a medication being studied for its effectiveness in treating patients with hereditary transthyretin amyloidosis (hATTR amyloidosis). This condition is a rare genetic disorder that affects the nerves and other organs. The medication aims to reduce neurologic impairment caused by the disease.

What is already known about the treatment

ALN-TTRSC02 – This medication is administered through subcutaneous injection and is currently being studied in clinical trials for its effectiveness in treating hereditary transthyretin amyloidosis, a rare genetic disorder. The main therapeutic indication is to address neurologic impairment associated with this condition. ALN-TTRSC02 works by targeting and reducing the production of transthyretin protein, which is responsible for the disease's progression. It is classified as an RNA interference therapeutic, aiming to silence specific genes involved in the disease process.

Investigated diseases

Hereditary Transthyretin Amyloidosis – This is a genetic disorder characterized by the buildup of abnormal protein deposits called amyloid in various tissues and organs. The disease primarily affects the nervous system and heart, leading to symptoms such as neuropathy, which involves nerve damage causing pain, weakness, and sensory loss. Over time, the accumulation of amyloid can impair the function of affected organs, leading to progressive neurological and cardiac issues. The condition is inherited in an autosomal dominant pattern, meaning a single copy of the altered gene in each cell is sufficient to cause the disorder. Symptoms typically begin in adulthood and can vary widely among individuals, even within the same family.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-508365-33-00Protocol codeALN-TTRSC02-002Estimated enrolment135 patientsSponsorAlnylam Pharmaceuticals Inc.

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