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Trial record 69 of 359 for:    transthyretin

A Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy Volunteers

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. Identifier: NCT02053454
Recruitment Status : Completed
First Posted : February 3, 2014
Last Update Posted : May 25, 2015
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Brief Summary:
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK) of patisiran (ALN-TTR02) in Japanese subjects

Condition or disease Intervention/treatment Phase
Transthyretin (TTR)-Mediated Amyloidosis Drug: patisiran (ALN-TTR02) Drug: Sterile Normal Saline (0.9% NaCl) Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 1, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose, Safety, Tolerability and Pharmacokinetics Study of ALN-TTR02 in Japanese Healthy Volunteers
Study Start Date : January 2014
Actual Primary Completion Date : March 2014
Actual Study Completion Date : June 2014

Arm Intervention/treatment
Active Comparator: patisiran (ALN-TTR02) Drug: patisiran (ALN-TTR02)
Ascending doses administered by intravenous (IV) infusion

Active Comparator: Sterile Normal Saline (0.9% NaCl) Drug: Sterile Normal Saline (0.9% NaCl)
Calculated volume to match active comparator

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

Secondary Outcome Measures :
  1. Pharmacodynamics (PD) of ALN-TTR02 (serum concentrations of Transthyretin, Vitamin A, and Retinol Binding Protein) [ Time Frame: Up to 90 days ]
  2. Observed maximum concentration (Cmax) of ALN-TTR02 [ Time Frame: Up to 90 days ]
  3. Time of observed maximum concentration (tmax) of ALN-TTR02 [ Time Frame: Up to 90 days ]
  4. Area under the plasma concentration versus time curve (AUC) of ALN-TTR02 [ Time Frame: Up to 90 days ]
  5. Terminal elimination half-life (t1/2) of ALN-TTR02 [ Time Frame: Up to 90 days ]
  6. Systemic clearance (CL) of ALN-TTR02 [ Time Frame: Up to 90 days ]
  7. Volume of distribution (V) of ALN-TTR02 [ Time Frame: Up to 90 days ]
  8. Renal clearance (CLR) of ALN-TTR02 [ Time Frame: Up to 90 days ]

Information from the National Library of Medicine

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Ages Eligible for Study:   20 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes

Inclusion Criteria:

  • Healthy Japanese adult males and females aged 20 to 65 years, inclusive (The 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);
  • Subjects who are healthy as determined by clinical assessments;
  • Females subjects must be of non-childbearing potential;
  • Males with partners of child-bearing potential, must agree to use appropriate contraception.

Exclusion Criteria:

  • Subjects with a history of serious mental illness;
  • Subjects who have a clinically relevant medical or surgical history;
  • Subjects with a positive screen for alcohol or drugs of abuse;
  • Subjects with safety laboratory test results deemed clinically significant;
  • Subjects with known hepatitis B surface antigen (HBsAg), hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) infection;
  • Subjects who have received an investigational agent within the 3 months prior to study entry.

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 identifier (NCT number): NCT02053454

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United Kingdom
Clinical Site
London, United Kingdom, NW10 7EW
Sponsors and Collaborators
Alnylam Pharmaceuticals
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Study Director: Jared Gollob, MD Alnylam Pharmaceuticals

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Responsible Party: Alnylam Pharmaceuticals Identifier: NCT02053454     History of Changes
Other Study ID Numbers: ALN-TTR02-005
First Posted: February 3, 2014    Key Record Dates
Last Update Posted: May 25, 2015
Last Verified: May 2015

Keywords provided by Alnylam Pharmaceuticals:
RNAi therapeutic

Additional relevant MeSH terms:
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Proteostasis Deficiencies
Metabolic Diseases