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Completed

NCT Number: NCT02053454

A Study of the Safety, Tolerability and Pharmacokinetics 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

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Key information

Conditions

Transthyretin (TTR)-Mediated Amyloidosis

Age range

20 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Clinical Site

London, NW10 7EW, United Kingdom

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

patisiran (ALN-TTR02)

Drug

Ascending doses administered by intravenous (IV) infusion

Sterile Normal Saline (0.9% NaCl)

Drug

Calculated volume to match active comparator

Primary outcomes

  1. The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation

    Time frame: Up to 28 days

Secondary outcomes

  1. Pharmacodynamics (PD) of ALN-TTR02 (serum concentrations of Transthyretin, Vitamin A, and Retinol Binding Protein)

    Time frame: Up to 90 days

  2. Observed maximum concentration (Cmax) of ALN-TTR02

    Time frame: Up to 90 days

  3. Time of observed maximum concentration (tmax) of ALN-TTR02

    Time frame: Up to 90 days

  4. Area under the plasma concentration versus time curve (AUC) of ALN-TTR02

    Time frame: Up to 90 days

  5. Terminal elimination half-life (t1/2) of ALN-TTR02

    Time frame: Up to 90 days

  6. Systemic clearance (CL) of ALN-TTR02

    Time frame: Up to 90 days

  7. Volume of distribution (V) of ALN-TTR02

    Time frame: Up to 90 days

  8. Renal clearance (CLR) of ALN-TTR02

    Time frame: Up to 90 days

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2014
Primary completion
2014
Study completion
2014
First posted
Feb 3, 2014
Registry last updated
May 25, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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