A Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy Volunteers

Transthyretin (TTR)-Mediated Amyloidosis Clinical Trial

Official title:

A Phase 1, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose, Safety, Tolerability and Pharmacokinetics Study of ALN-TTR02 in Japanese Healthy Volunteers

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02053454
• Other study ID #: ALN-TTR02-005
• Status: Completed
• Phase: Phase 1
• First received: January 24, 2014
• Last updated: May 22, 2015
• Start date: January 2014
• Est. completion date: June 2014

Study information

• Verified date: May 2015
• Source: Alnylam Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Health authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK) of patisiran (ALN-TTR02) in Japanese subjects

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: June 2014
• Est. primary completion date: March 2014
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Both
• Age group: 20 Years to 65 Years

Eligibility

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

Allocation: Randomized, Endpoint Classification: Safety Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Amyloidosis
• Transthyretin (TTR)-Mediated Amyloidosis

Intervention

Drug:
• patisiran (ALN-TTR02)
Ascending doses administered by intravenous (IV) infusion
• Sterile Normal Saline (0.9% NaCl)
Calculated volume to match active comparator

Locations

Country	Name	City	State
United Kingdom	Clinical Site	London

Sponsors (1)

Lead Sponsor	Collaborator
Alnylam Pharmaceuticals

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation		Up to 28 days	Yes
Secondary	Pharmacodynamics (PD) of ALN-TTR02 (serum concentrations of Transthyretin, Vitamin A, and Retinol Binding Protein)		Up to 90 days	No
Secondary	Observed maximum concentration (Cmax) of ALN-TTR02		Up to 90 days	No
Secondary	Time of observed maximum concentration (tmax) of ALN-TTR02		Up to 90 days	No
Secondary	Area under the plasma concentration versus time curve (AUC) of ALN-TTR02		Up to 90 days	No
Secondary	Terminal elimination half-life (t1/2) of ALN-TTR02		Up to 90 days	No
Secondary	Systemic clearance (CL) of ALN-TTR02		Up to 90 days	No
Secondary	Volume of distribution (V) of ALN-TTR02		Up to 90 days	No
Secondary	Renal clearance (CLR) of ALN-TTR02		Up to 90 days	No