A Study of the Safety, Tolerability and Pharmacokinetics... | NCT02053454

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

NCT02053454

Sponsor

Alnylam Pharmaceuticals

Status

Completed

Last Update Posted

May 25, 2015Estimated

Enrollment

12Actual

Phase

Phase 1

Conditions

Transthyretin (TTR)-Mediated Amyloidosis

Interventions

patisiran (ALN-TTR02)
Sterile Normal Saline (0.9% NaCl)

Countries

United Kingdom

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

May 2015

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jan 2014

Primary Completion Date

Mar 2014Actual

Completion Date

Jun 2014Actual

First Submitted Date

Jan 24, 2014

First Submission Date that Met QC Criteria

Jan 31, 2014

First Posted Date

Feb 3, 2014Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

May 22, 2015

Last Update Posted Date

May 25, 2015Estimated

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

12Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
patisiran (ALN-TTR02)	Active Comparator		Drug: patisiran (ALN-TTR02)
Sterile Normal Saline (0.9% NaCl)	Active Comparator		Drug: Sterile Normal Saline (0.9% NaCl)

Interventions

Name	Type	Description	Arm Group Labels	Other Names
patisiran (ALN-TTR02)	Drug	Ascending doses administered by intravenous (IV) infusion	patisiran (ALN-TTR02)

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation		Up to 28 days

Secondary Outcomes

Measure	Description	Time Frame
Pharmacodynamics (PD) of ALN-TTR02 (serum concentrations of Transthyretin, Vitamin A, and Retinol Binding Protein)		Up to 90 days
Observed maximum concentration (Cmax) of ALN-TTR02		Up to 90 days

Other Outcomes

Not provided

Eligibility Module

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.

Accepts Healthy Volunteers

Yes

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

20 Years

Maximum Age

65 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided