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

Purpose

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

Condition

  • Hereditary Hemorrhagic Telangiectasia

Eligibility

Eligible Ages
Over 18 Years
Eligible Sex
All
Accepts Healthy Volunteers
Yes

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

Study Design

Phase
Phase 1/Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Triple (Participant, Care Provider, Investigator)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Part A: ALN-6400 		Participants will be administered a single dose of ALN-6400.
  • Drug: ALN-6400
    ALN-6400 will be administered subcutaneously (SC)
Placebo Comparator
Part A: Placebo 		Participants will be administered a single dose of placebo.
  • Drug: Placebo
    Placebo will be administered subcutaneously (SC)
Experimental
Part B: ALN-6400 		Participants will be administered multiple doses of ALN-6400.
  • Drug: ALN-6400
    ALN-6400 will be administered subcutaneously (SC)
Placebo Comparator
Part B: Placebo 		Participants will be administered multiple doses of placebo.
  • Drug: Placebo
    Placebo will be administered subcutaneously (SC)

Recruiting Locations

Clinical Trial Site
Cypress 5341256, California 5332921 90630

Clinical Trial Site
Boston 4930956, Massachusetts 6254926 02114

More Details

Status
Recruiting
Sponsor
Alnylam Pharmaceuticals

Study Contact

Alnylam Clinical Trial Information Line
1-877-ALNYLAM
clinicaltrials@alnylam.com