A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALN-TTRSC04 in Healthy Subjects

Alnylam Pharmaceuticals

Status and phase: Completed; Phase 1

Conditions: Transthyretin-Mediated Amyloidosis

Treatments: Drug: ALN-TTRSC04; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT05661916

ALN-TTRSC04-001

Details and patient eligibility

About

The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single ascending doses of ALN-TTRSC04.

Enrollment: 96 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

• Body mass index (BMI) ≥18.0 kg/m^2 and ≤30 kg/m^2 assessed at Screening.

Exclusion criteria

• Has an estimated glomerular filtration rate (eGFR) <90 mL/min/1.73m^2 at screening.
• Has alanine aminotransferase (ALT), aspartate aminotransferase (AST) or direct bilirubin above the upper limit of normal (ULN).

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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96 participants in 2 patient groups, including a placebo group

ALN-TTRSC04

Experimental group

Description:

Participants will be administered a single dose of ALN-TTRSC04.

Treatment:

Drug: ALN-TTRSC04

Placebo

Placebo Comparator group

Description:

Participants will be administered a single dose of placebo.

Treatment:

Drug: Placebo