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New drug aims to tame rare bleeding disorder

NCT ID NCT06659640

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 This study
Running, but no longer taking on new participants.
Completed
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 17, 2026 · Updated 6 times

Summary

This study tests a new medicine called ALN-6400 for people with hereditary hemorrhagic telangiectasia (HHT), a condition that causes abnormal blood vessels and frequent nosebleeds. First, healthy volunteers will receive a single dose to check safety. Then, HHT patients will receive multiple doses to see if it reduces bleeding. The goal is to control the disease, not cure it.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

89 people

The number who actually took part.

Started

Nov 2024

Expected to finish

Jun 2028

An estimate. End dates often move.

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 years and older

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 - Part A: * Is a healthy adult volunteer Part B: * Is an adult patient with a clinical diagnosis of HHT Exclusion Criteria - Part A: * Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \> upper limit of normal (ULN) * Has known human immunodeficiency virus (HIV) infection; or known current or chronic hepatitis C virus or hepatitis B virus infection * Has an estimated glomerular filtration (eGFR) of \<90 mL/min/1.73m\^2 at screening Part B: * Has ALT or AST \>2×ULN * Has total bilirubin \>1.5×ULN * Has eGFR of \<30 mL/min/1.73m\^2 at screening Parts A and B: * Is not willing to comply with the contraceptive requirements during the study period Note: other protocol defined inclusion / exclusion criteria apply

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Conditions

The condition(s) this trial relates to.

Epistaxis hereditary hemorrhagic telangiectasia

As listed by the trial registrant

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

Hereditary hemorrhagic telangiectasia

Contacts and locations

Locations

  • Clinical Trial Site

    Birmingham, Alabama, 35249, United States

  • Clinical Trial Site

    Cypress, California, 90630, United States

  • Clinical Trial Site

    Gainesville, Florida, 32608, United States

  • Clinical Trial Site

    Indianapolis, Indiana, 46260, United States

  • Clinical Trial Site

    Boston, Massachusetts, 02114, United States

  • Clinical Trial Site

    Rochester, Minnesota, 55905, United States

  • Clinical Trial Site

    St Louis, Missouri, 63110, United States

  • Clinical Trial Site

    Chapel Hill, North Carolina, 27514, United States

  • Clinical Trial Site

    Camperdown, 2050, Australia

  • Clinical Trial Site

    Parkville, 3050, Australia

  • Clinical Trial Site

    Mount Royal, H3P 3P1, Canada

  • Clinical Trial Site

    Toronto, M5B 1W8, Canada

  • Clinical Trial Site

    Bordeaux, 33000, France

  • Clinical Trial Site

    Bron, 69500, France

  • Clinical Trial Site

    Homburg, 66421, Germany

  • Clinical Trial Site

    L'Hospitalet de Llobregat, 8907, Spain

More trials for these conditions

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