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New injection for rare protein disease passes first safety check

NCT ID NCT05661916

Disease control Sponsor: Alnylam Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage study tested a new drug called ALN-TTRSC04 in 96 healthy people to see if it is safe and how the body processes it. The drug aims to lower a harmful protein that causes transthyretin amyloidosis, a serious disease affecting nerves and the heart. Participants received a single injection, and researchers monitored side effects and drug levels in the blood.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

96 people

The number who actually took part.

Started

Jan 2023

Finished

Sep 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 65 years

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Body mass index (BMI) ≥18.0 kg/m\^2 and ≤30 kg/m\^2 assessed at Screening. Exclusion Criteria: * Has an estimated glomerular filtration rate (eGFR) \<90 mL/min/1.73m\^2 at screening. * Has alanine aminotransferase (ALT), aspartate aminotransferase (AST) or direct bilirubin above the upper limit of normal (ULN).

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Conditions

The condition(s) this trial relates to.

familial amyloid neuropathy hereditary amyloidosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Transthyretin-mediated amyloidosis

Contacts and locations

Locations

  • Clinical Trial Site

    London, SE1 1YR, United Kingdom

More trials for these conditions

Other studies related to the condition(s) this trial covers.