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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
  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:


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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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 by Alnylam Pharmaceuticals

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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
Metabolic Diseases
Proteostasis Deficiencies

ClinicalTrials.gov processed this record on November 23, 2014