Transthyretin (TTR)-Mediated Amyloidosis
Conditions
Keywords
RNAi therapeutic
Brief summary
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK) of patisiran (ALN-TTR02) in Japanese subjects
Interventions
Ascending doses administered by intravenous (IV) infusion
DRUGSterile Normal Saline (0.9% NaCl)
Calculated volume to match active comparator
Sponsors
Alnylam Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
20 Years to 65 Years
Healthy volunteers
Yes
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation | Up to 28 days |
Secondary
| Measure | Time frame |
|---|---|
| Observed maximum concentration (Cmax) of ALN-TTR02 | 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 |
| Renal clearance (CLR) of ALN-TTR02 | Up to 90 days |
| Systemic clearance (CL) of ALN-TTR02 | Up to 90 days |
| Volume of distribution (V) of ALN-TTR02 | Up to 90 days |
| Pharmacodynamics (PD) of ALN-TTR02 (serum concentrations of Transthyretin, Vitamin A, and Retinol Binding Protein) | Up to 90 days |
| Terminal elimination half-life (t1/2) of ALN-TTR02 | Up to 90 days |
Countries
United Kingdom
Outcome results
None listed