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A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT)

NCT ID: NCT06659640

Last Updated: 2025-11-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

120 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-11-11

Study Completion Date

2028-06-21

Brief Summary

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The purpose of this study is to:

* evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single ascending doses of ALN-6400 in healthy volunteers
* evaluate the efficacy, safety, tolerability and PD of multiple doses of ALN-6400 in adult patients with HHT

Detailed Description

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Conditions

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Hereditary Hemorrhagic Telangiectasia

Keywords

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siRNA RNAi therapeutic Plasminogen PLG HHT Osler-Weber-Rendu epistaxis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Part A: ALN-6400

Participants will be administered a single dose of ALN-6400.

Group Type EXPERIMENTAL

ALN-6400

Intervention Type DRUG

ALN-6400 will be administered subcutaneously (SC)

Part A: Placebo

Participants will be administered a single dose of placebo.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo will be administered subcutaneously (SC)

Part B: ALN-6400

Participants will be administered multiple doses of ALN-6400.

Group Type EXPERIMENTAL

ALN-6400

Intervention Type DRUG

ALN-6400 will be administered subcutaneously (SC)

Part B: Placebo

Participants will be administered multiple doses of placebo.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo will be administered subcutaneously (SC)

Interventions

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ALN-6400

ALN-6400 will be administered subcutaneously (SC)

Intervention Type DRUG

Placebo

Placebo will be administered subcutaneously (SC)

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Alnylam Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Alnylam Pharmaceuticals

Locations

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Clinical Trial Site

Cypress, California, United States

Site Status RECRUITING

Clinical Trial Site

Montreal, , Canada

Site Status RECRUITING

Countries

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United States Canada

Central Contacts

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Alnylam Clinical Trial Information Line

Role: CONTACT

Phone: 1-877-ALNYLAM

Email: [email protected]

Alnylam Clinical Trial Information Line

Role: CONTACT

Phone: 1-877-256-9526

Email: [email protected]

Other Identifiers

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2025-522510-23-00

Identifier Type: CTIS

Identifier Source: secondary_id

ALN-6400-001

Identifier Type: -

Identifier Source: org_study_id