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

Phase 2RecruitingNCT06659640

Alnylam Pharmaceuticals120 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

120

Conditions

Hereditary Hemorrhagic Telangiectasia

Interventions

ALN-6400DRUG

ALN-6400 will be administered subcutaneously (SC)

PlaceboDRUG

Placebo will be administered subcutaneously (SC)

Eligibility

Sex: ALLMin age: 18 YearsHealthy volunteers:

Medical Language ↔ Plain English

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

Locations (8)

Clinical Trial Site

Cypress, California, United States

RECRUITING

Clinical Trial Site

Boston, Massachusetts, United States

RECRUITING

Clinical Trial Site

Mount Royal, Canada

Outcomes

Primary Outcomes

Part A: Frequency of Adverse Events

Time frame: Up to Week 36

Part B: Frequency of Adverse Events

Time frame: Up to Week 96

Secondary Outcomes

Part A: Concentrations of ALN-6400 in Plasma

Time frame: Predose and up to 2 days postdose

Part A: Change from Baseline in Plasminogen (PLG) in Plasma Protein Levels

Time frame: Predose and up to Week 36 postdose

Part B: Change from Baseline in Plasminogen (PLG) in Plasma Protein Levels

Time frame: Screening and up to Week 96 postdose

Part A: Change from Baseline in Plasminogen (PLG) in Plasma Activity Levels

Time frame: Predose and up to Week 36 postdose

Part B: Change from Baseline in Plasminogen (PLG) in Plasma Activity Levels

Time frame: Part B: Screening and up to Week 96 postdose

Part B: Change from Baseline in Intensity-adjusted Epistaxis Duration

Intensity-adjusted epistaxis duration will be assessed using a daily patient epistaxis diary.

Time frame: Baseline up to Week 96

Part B: Change from Baseline in Epistaxis Severity Score (ESS) Scale

Validated bleeding scale in HHT scored between 0-10, higher scores indicate worse bleeding.

Time frame: Baseline up to Week 96

Part B: Change from Baseline in Epistaxis Duration

Central Contacts

Alnylam Clinical Trial Information Line

CONTACT

1-877-ALNYLAM clinicaltrials@alnylam.com

Alnylam Clinical Trial Information Line

CONTACT

1-877-256-9526 clinicaltrials@alnylam.com

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.