Dysautonomia International

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

Arm	Description	Assigned 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

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.