We updated the design of this site on September 25th. Learn more.
Show more
ClinicalTrials.gov
ClinicalTrials.gov Menu

A Phase 1, Single- and Multi-Dose, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN-TTRSC (Revusiran) in Healthy Volunteers

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01814839
First Posted: March 20, 2013
Last Update Posted: February 5, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Alnylam Pharmaceuticals
  Purpose
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-TTRSC (revusiran) in healthy volunteer subjects.

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

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 1, Randomized, Double-blind, Placebo-controlled, Single- and Multi-Dose, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN-TTRSC in Healthy Volunteers

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 63 days ]

Secondary Outcome Measures:
  • Pharmacokinetics (PK) of ALN-TTRSC (revusiran) (Cmax, tmax, t1/2, AUC0-last, CL). [ Time Frame: Up to 90 days ]
  • Effect of ALN-TTRSC (revusiran) on transthyretin (TTR) (Determination of % Lowering of TTR to pretreatment/Baseline Levels). [ Time Frame: Up to 90 days ]
  • Effect of ALN-TTRSC (revusiran) on vitamin A (Determination of % Lowering of vitamin A to pretreatment/Baseline Levels). [ Time Frame: Up to 90 days ]
  • Effect of ALN-TTRSC (revusiran) on retinol binding protein (RBP) (Determination of % Lowering of RBP to pretreatment/Baseline Levels). [ Time Frame: Up to 90 days ]

Enrollment: 85
Study Start Date: March 2013
Study Completion Date: May 2015
Primary Completion Date: April 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: ALN-TTRSC (revusiran) Drug: ALN-TTRSC (revusiran)
Ascending doses of ALN-TTRSC (revusiran) by subcutaneous (SC) injection
Placebo Comparator: Sterile Normal Saline (0.9% NaCl) Drug: Sterile Normal Saline (0.9% NaCl)
Calculated volume to match active comparator

  Eligibility

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years to 55 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

  • Body mass index (BMI) must be ≥ 16.5 kg/m2 and ≤ 35.0 kg/m2;
  • Female subjects must be of non-childbearing potential; e.g., postmenopausal or pre-menopausal with surgical sterilization;
  • Male subjects agree to use appropriate contraception;
  • Adequate blood counts, liver and renal function;
  • Non-smokers for at least 3 months;
  • Willing to give written informed consent and are willing to comply with the study requirements;
  • 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 (cohorts 19 and 20).

Exclusion Criteria:

  • Known human immunodeficiency virus (HIV) positive status or known or suspected systemic bacterial, viral, parasitic, or fungal infection;
  • Subjects with a history of multiple drug allergies or intolerance to SC injection;
  • History of drug abuse and/or alcohol abuse;
  • Receiving an investigational agent within 3 months prior to study drug administration;
  • Considered unfit for the study by the Principal Investigator.
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01814839


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

Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01814839     History of Changes
Other Study ID Numbers: ALN-TTRSC-001
First Submitted: March 18, 2013
First Posted: March 20, 2013
Last Update Posted: February 5, 2016
Last Verified: February 2016

Keywords provided by Alnylam Pharmaceuticals:
RNAi therapeutic

Additional relevant MeSH terms:
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases