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

TTR-mediated Amyloidosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01559077
• Other study ID #: ALN-TTR02-001
• Status: Completed
• Phase: Phase 1
• First received: March 19, 2012
• Last updated: January 14, 2013
• Start date: March 2012
• Est. completion date: November 2012

Study information

• Verified date: January 2013
• 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 determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR02 in healthy volunteer subjects.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 17
• Est. completion date: November 2012
• Est. primary completion date: June 2012
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Both
• Age group: 18 Years to 45 Years

Eligibility

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

Study Design

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

Related Conditions & MeSH terms

• Amyloidosis
• TTR-mediated Amyloidosis

Intervention

Drug:
• ALN-TTR02
Dose levels between 0.01 and 0.5 mg/kg 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	Leeds
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	Pharmacokinetics (PK) of ALN-TTR02 (Cmax, tmax, t1/2, AUC0-last, CL).		Up to 180 days	Yes
Secondary	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)		Up to 56 days	No