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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01559077
First received: March 19, 2012
Last updated: January 14, 2013
Last verified: January 2013
History of Changes
  Purpose

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.


Condition Intervention Phase
TTR-mediated Amyloidosis
 Drug: ALN-TTR02
Drug: Sterile Normal Saline (0.9% NaCl)
 Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Subject)
Primary Purpose: Treatment
Official Title: A Phase 1, Randomized, Single-blind, Placebo-Controlled, Single Ascending Dose, Safety, Tolerability and Pharmacokinetics Study of ALN-TTR02 in Healthy Volunteers

Resource links provided by NLM:

MedlinePlus related topics: Amyloidosis
U.S. FDA Resources

Further study details as provided by Alnylam Pharmaceuticals:

Primary Outcome Measures:
  • The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation. [ Time Frame: Up to 28 days ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Pharmacokinetics (PK) of ALN-TTR02 (Cmax, tmax, t1/2, AUC0-last, CL). [ Time Frame: Up to 180 days ] [ Designated as safety issue: Yes ]
  • 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 ] [ Designated as safety issue: No ]

Enrollment: 17
Study Start Date: March 2012
Study Completion Date: November 2012
Primary Completion Date: June 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: ALN-TTR02 Drug: ALN-TTR02
Dose levels between 0.01 and 0.5 mg/kg by intravenous (IV) infusion
Placebo Comparator: Sterile Normal Saline (0.9% NaCl) Drug: Sterile Normal Saline (0.9% NaCl)
Calculated volume to match active comparator

  Eligibility

Ages Eligible for Study:   18 Years to 45 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   Yes
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01559077

Locations
United Kingdom
Clinical Site
Leeds, United Kingdom, LS2 9LH
Clinical Site
London, United Kingdom, NW10 7EW
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
Study Director: Jared Gollob, MD Alnylam Pharmaceuticals
  More Information

No publications provided

Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01559077     History of Changes
Other Study ID Numbers: ALN-TTR02-001
Study First Received: March 19, 2012
Last Updated: January 14, 2013
Health Authority: United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Alnylam Pharmaceuticals:
ATTR

Additional relevant MeSH terms:
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases

ClinicalTrials.gov processed this record on May 16, 2013