A Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy Volunteers
Phase 1
Completed
- Conditions
- Transthyretin (TTR)-Mediated Amyloidosis
- Interventions
- Drug: Sterile Normal Saline (0.9% NaCl)
- Registration Number
- NCT02053454
- Lead Sponsor
- Alnylam Pharmaceuticals
- Brief Summary
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK) of patisiran (ALN-TTR02) in Japanese subjects
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 12
Inclusion Criteria
- Healthy Japanese adult males and females aged 20 to 65 years, inclusive (The subject was born in Japan and has lived outside of Japan for <10 years, and subject's biological parents and grandparents are fully Japanese and were born in Japan);
- Subjects who are healthy as determined by clinical assessments;
- Females subjects must be of non-childbearing potential;
- Males with partners of child-bearing potential, must agree to use appropriate contraception.
Exclusion Criteria
- Subjects with a history of serious mental illness;
- Subjects who have a clinically relevant medical or surgical history;
- Subjects with a positive screen for alcohol or drugs of abuse;
- Subjects with safety laboratory test results deemed clinically significant;
- Subjects with known hepatitis B surface antigen (HBsAg), hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) infection;
- Subjects who have received an investigational agent within the 3 months prior to study entry.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description patisiran (ALN-TTR02) patisiran (ALN-TTR02) - Sterile Normal Saline (0.9% NaCl) Sterile Normal Saline (0.9% NaCl) -
- Primary Outcome Measures
Name Time Method The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation Up to 28 days
- Secondary Outcome Measures
Name Time Method Pharmacodynamics (PD) of ALN-TTR02 (serum concentrations of Transthyretin, Vitamin A, and Retinol Binding Protein) Up to 90 days Time of observed maximum concentration (tmax) of ALN-TTR02 Up to 90 days Area under the plasma concentration versus time curve (AUC) of ALN-TTR02 Up to 90 days Systemic clearance (CL) of ALN-TTR02 Up to 90 days Renal clearance (CLR) of ALN-TTR02 Up to 90 days Observed maximum concentration (Cmax) of ALN-TTR02 Up to 90 days Volume of distribution (V) of ALN-TTR02 Up to 90 days Terminal elimination half-life (t1/2) of ALN-TTR02 Up to 90 days
Trial Locations
- Locations (1)
Clinical Site
🇬🇧London, United Kingdom