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

Alnylam Pharmaceuticals

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Hereditary Hemorrhagic Telangiectasia

Treatments: Drug: Placebo; Drug: ALN-6400

Study type: Interventional

Funder types: Industry

Identifiers

NCT06659640

ALN-6400-001

2025-522510-23-00 (EU Trial (CTIS) Number)

Details and patient eligibility

About

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

Enrollment: 120 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: Accepts Healthy Volunteers

Inclusion and exclusion criteria

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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120 participants in 4 patient groups, including a placebo group

Part A: ALN-6400

Experimental group

Description:

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

Treatment:

Drug: ALN-6400

Part A: Placebo

Placebo Comparator group

Description:

Participants will be administered a single dose of placebo.

Treatment:

Drug: Placebo

Part B: ALN-6400

Experimental group

Description:

Participants will be administered multiple doses of ALN-6400.

Treatment:

Drug: ALN-6400

Part B: Placebo

Placebo Comparator group

Description:

Participants will be administered multiple doses of placebo.

Treatment:

Drug: Placebo