Back to resultsA Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy Volunteers
Primary Purpose
Transthyretin (TTR)-Mediated Amyloidosis
Status
Completed
Phase
Phase 1
Locations
United Kingdom
Study Type
Interventional
Intervention
patisiran (ALN-TTR02)
Sterile Normal Saline (0.9% NaCl)
Sponsored by
About this trial
This is an interventional treatment trial for Transthyretin (TTR)-Mediated Amyloidosis focused on measuring RNAi therapeutic
Eligibility Criteria
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.
Sites / Locations
- Clinical Site
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Active Comparator
Arm Label
patisiran (ALN-TTR02)
Sterile Normal Saline (0.9% NaCl)
Arm Description
Outcomes
Primary Outcome Measures
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation
Secondary Outcome Measures
Pharmacodynamics (PD) of ALN-TTR02 (serum concentrations of Transthyretin, Vitamin A, and Retinol Binding Protein)
Observed maximum concentration (Cmax) of ALN-TTR02
Time of observed maximum concentration (tmax) of ALN-TTR02
Area under the plasma concentration versus time curve (AUC) of ALN-TTR02
Terminal elimination half-life (t1/2) of ALN-TTR02
Systemic clearance (CL) of ALN-TTR02
Volume of distribution (V) of ALN-TTR02
Renal clearance (CLR) of ALN-TTR02
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02053454
Brief Title
A Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy Volunteers
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 Type
Interventional
2. Study Status
Record Verification Date
May 2015
Overall Recruitment Status
Completed
Study Start Date
January 2014 (undefined)
Primary Completion Date
March 2014 (Actual)
Study Completion Date
June 2014 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alnylam Pharmaceuticals
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK) of patisiran (ALN-TTR02) in Japanese subjects
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Transthyretin (TTR)-Mediated Amyloidosis
Keywords
RNAi therapeutic
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
12 (Actual)
8. Arms, Groups, and Interventions
Arm Title
patisiran (ALN-TTR02)
Arm Type
Active Comparator
Arm Title
Sterile Normal Saline (0.9% NaCl)
Arm Type
Active Comparator
Intervention Type
Drug
Intervention Name(s)
patisiran (ALN-TTR02)
Intervention Description
Ascending doses administered by intravenous (IV) infusion
Intervention Type
Drug
Intervention Name(s)
Sterile Normal Saline (0.9% NaCl)
Intervention Description
Calculated volume to match active comparator
Primary Outcome Measure Information:
Title
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation
Time Frame
Up to 28 days
Secondary Outcome Measure Information:
Title
Pharmacodynamics (PD) of ALN-TTR02 (serum concentrations of Transthyretin, Vitamin A, and Retinol Binding Protein)
Time Frame
Up to 90 days
Title
Observed maximum concentration (Cmax) of ALN-TTR02
Time Frame
Up to 90 days
Title
Time of observed maximum concentration (tmax) of ALN-TTR02
Time Frame
Up to 90 days
Title
Area under the plasma concentration versus time curve (AUC) of ALN-TTR02
Time Frame
Up to 90 days
Title
Terminal elimination half-life (t1/2) of ALN-TTR02
Time Frame
Up to 90 days
Title
Systemic clearance (CL) of ALN-TTR02
Time Frame
Up to 90 days
Title
Volume of distribution (V) of ALN-TTR02
Time Frame
Up to 90 days
Title
Renal clearance (CLR) of ALN-TTR02
Time Frame
Up to 90 days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
20 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jared Gollob, MD
Organizational Affiliation
Alnylam Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Clinical Site
City
London
ZIP/Postal Code
NW10 7EW
Country
United Kingdom
12. IPD Sharing Statement
Learn more about this trial
A Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy Volunteers
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