A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects

Phase 1

Completed

• Conditions: Transthyretin-mediated Amyloidosis (ATTR Amyloidosis)
• Interventions: Drug: Sterile Normal Saline (0.9% NaCl); Drug: ALN-TTRSC02

• Registration Number: NCT02797847
• Lead Sponsor: Alnylam Pharmaceuticals

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2016-05-18
• Study Start: 2016-06-07
• Study First Submitted QC: 2016-06-08
• Study First Posted: 2016-06-14
• Primary Completion: 2018-01-12
• Study Completion: 2018-01-12
• Status Verified: 2018-09
• Last Update Submitted: 2018-09-20
• Last Update Posted: 2018-09-21

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 80

Inclusion Criteria

• 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.

Exclusion Criteria

• 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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Sterile normal saline 0.9% for SC administration	Sterile Normal Saline (0.9% NaCl)	-
ALN-TTRSC02	ALN-TTRSC02	-

Primary Outcome Measures

Name	Time	Method
Safety of ALN-TTRSC02, evaluated by the proportion of subjects experiencing adverse events (AEs)	Day 1 through to Day 314

Secondary Outcome Measures

Name	Time	Method
Profile of pharmacokinetics (PK) of ALN-TTRSC02	Day 1 through to Day 314	CLR
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

Trial Locations

Locations (1)

Clinical Trial Site; 🇬🇧 London, United Kingdom