A Phase 1, Randomized, Single-Blind, Placebo Controlled, Single-Ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-TTRSC02 in Healthy Subjects

Alnylam Pharmaceuticals1 site in 1 country80 target enrollmentJune 7, 2016

Overview

Phase: Phase 1
Intervention: ALN-TTRSC02
Conditions: Transthyretin-mediated Amyloidosis (ATTR Amyloidosis)
Sponsor: Alnylam Pharmaceuticals
Enrollment: 80
Locations: 1
Primary Endpoint: Safety of ALN-TTRSC02, evaluated by the proportion of subjects experiencing adverse events (AEs)
Status: Completed
Last Updated: 7 years ago

The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC02 in healthy subjects.

Registry

clinicaltrials.gov

Start Date

June 7, 2016

End Date

January 12, 2018

Last Updated

7 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Sponsor

Responsible Party

Sponsor

•Male and female subjects, age 18 to 65 years, inclusive.
•Body mass index (BMI) ≥18.0 kg/m2 and ≤30 kg/m2 assessed at Screening.
•No clinically significant health concerns, as determined by medical history and physical examination, in the opinion of the Investigator.
•Women of child bearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception, willing and able to comply with the study requirements and to provide written informed consent.
•For Japanese cohorts, subjects of Japanese descent are defined as people carrying a Japanese passport, descendants of 4 Japanese grandparents, and have not been outside Japan for more than 5 years.

•Clinically relevant history or presence of respiratory, gastrointestinal, renal, cardiovascular, hepatic, hematological, lymphatic, neurological, psychiatric, musculoskeletal, genitourinary, immunological, and other inflammatory diseases, or dermatological or connective tissue diseases or disorders.
•Active serious mental illness or psychiatric disorder.
•Clinically significant ECG abnormalities. Abnormal for AST/ALT and any other clinical safety laboratory result considered clinically significant.
•Known history of allergic reaction to an oligonucleotide or GalNAc.
•History of intolerance to subcutaneous injection.

Intervention: ALN-TTRSC02

Intervention: Sterile Normal Saline (0.9% NaCl)

Safety of ALN-TTRSC02, evaluated by the proportion of subjects experiencing adverse events (AEs)

Time Frame: Day 1 through to Day 314

Profile of pharmacokinetics (PK) of ALN-TTRSC02(Day 1 through to Day 314)
Effect of ALN-TTRSC02 on serum Vitamin A levels as measured by reduction from baseline in serum Vitamin A(Screening through to Day 314)
Effect of ALN-TTRSC02 on serum TTR levels as measured by reduction from baseline in serum TTR(Day 1 through to Day 314)