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Completed

NCT Number: NCT01559077

Trial to Evaluate Safety, Tolerability, and Parmacokinetics of ALN-TTR02 in Healthy Volunteer Subjects

The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR02 in healthy volunteer subjects.

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Key information

Conditions

TTR-mediated Amyloidosis

Age range

18 year–45 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Clinical Site, Leeds, United Kingdom

Loading trial locations.

Who can participate

Healthy volunteers accepted: Yes

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

Inclusion criteria

  • Body mass index must be between 18.0 kg/m2 and ≤ 31.5 kg/m2;
  • Non-smokers for at least 3 months preceding screening;
  • Females subjects must be of non-childbearing potential e.g., post-menopausal or pre-menopausal with surgical sterilization;
  • Males agree to use appropriate contraception;
  • Medical history must be verified by either a personal physician or medical practitioner as appropriate;
  • Willing to give written informed consent and are willing to comply with the study requirements.

Exclusion criteria

  • Known hepatitis B surface antigen (HBsAg), hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) infection
  • Multiple drug allergies or know sensitivity to oligonucleotide
  • History of drug abuse and/or alcohol abuse
  • Receiving an investigational agent within 3 months prior to study drug administration
  • Subjects with safety laboratory test results deemed clinical significant by the Investigator;
  • Received prescription drugs within 4 weeks of first dosing
  • Subjects who have donated more than 500 mL of blood within the 3 months prior to ALN-PCS02 or placebo administration;
  • Subjects who have used prescription drugs within 4 weeks of first dosing
  • Considered unfit for the study by the Principal Investigator
  • Employee or family member of the sponsor or the clinical study site personnel

Treatment and study plan

ALN-TTR02

Drug

Dose levels between 0.01 and 0.5 mg/kg 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. Pharmacokinetics (PK) of ALN-TTR02 (Cmax, tmax, t1/2, AUC0-last, CL).

    Time frame: Up to 180 days

  2. Effect of ALN-TTR02 on transthyretin (TTR), vitamin A, and retinol binding protein (RBP) (Determination of % Lowering of TTR, vitamin A and RBP to pretreatment/Baseline Levels)

    Time frame: Up to 56 days

Sponsors and collaborators

Lead sponsor

Alnylam Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2012
Primary completion
2012
Study completion
2012
First posted
Mar 21, 2012
Registry last updated
Jan 15, 2013

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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