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

May 22, 2015 updated by: Alnylam Pharmaceuticals

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

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

12

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

20 years to 65 years (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

Yes

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: DOUBLE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
ACTIVE_COMPARATOR: patisiran (ALN-TTR02)
Drug: patisiran (ALN-TTR02)
Ascending doses administered by intravenous (IV) infusion
ACTIVE_COMPARATOR: Sterile Normal Saline (0.9% NaCl)
Drug: Sterile Normal Saline (0.9% NaCl)
Calculated volume to match active comparator

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation
Time Frame: Up to 28 days
Up to 28 days

Secondary Outcome Measures

Outcome Measure
Time Frame
Pharmacodynamics (PD) of ALN-TTR02 (serum concentrations of Transthyretin, Vitamin A, and Retinol Binding Protein)
Time Frame: Up to 90 days
Up to 90 days
Observed maximum concentration (Cmax) of ALN-TTR02
Time Frame: Up to 90 days
Up to 90 days
Time of observed maximum concentration (tmax) of ALN-TTR02
Time Frame: Up to 90 days
Up to 90 days
Area under the plasma concentration versus time curve (AUC) of ALN-TTR02
Time Frame: Up to 90 days
Up to 90 days
Terminal elimination half-life (t1/2) of ALN-TTR02
Time Frame: Up to 90 days
Up to 90 days
Systemic clearance (CL) of ALN-TTR02
Time Frame: Up to 90 days
Up to 90 days
Volume of distribution (V) of ALN-TTR02
Time Frame: Up to 90 days
Up to 90 days
Renal clearance (CLR) of ALN-TTR02
Time Frame: Up to 90 days
Up to 90 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alnylam Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2014

Primary Completion (ACTUAL)

March 1, 2014

Study Completion (ACTUAL)

June 1, 2014

Study Registration Dates

First Submitted

January 24, 2014

First Submitted That Met QC Criteria

January 31, 2014

First Posted (ESTIMATE)

February 3, 2014

Study Record Updates

Last Update Posted (ESTIMATE)

May 25, 2015

Last Update Submitted That Met QC Criteria

May 22, 2015

Last Verified

May 1, 2015

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALN-TTR02-005

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Transthyretin (TTR)-Mediated Amyloidosis

Clinical Trials on Sterile Normal Saline (0.9% NaCl)

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Slovenia

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe