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

NCT02053454 | Completed Phase 1

• 1 other identifier: ALN-TTR02-005
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Transthyretin (TTR)-Mediated Amyloidosis

Keywords

RNAi therapeutic

Outcome Measures

Primary Outcomes (1)

• The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation

  Up to 28 days

Secondary Outcomes (8)

• 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

• Time of observed maximum concentration (tmax) of ALN-TTR02

  Up to 90 days

• Area under the plasma concentration versus time curve (AUC) of ALN-TTR02

  Up to 90 days

• Terminal elimination half-life (t1/2) of ALN-TTR02

  Up to 90 days

Study Arms (2)

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

patisiran (ALN-TTR02) DRUG

Ascending doses administered by intravenous (IV) infusion

patisiran (ALN-TTR02)

Sterile Normal Saline (0.9% NaCl) DRUG

Calculated volume to match active comparator

Sterile Normal Saline (0.9% NaCl)

Eligibility Criteria

• Age: 20 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Alnylam Pharmaceuticals: lead

Study Sites (1)

Clinical Site

London, NW10 7EW, United Kingdom

Location

MeSH Terms

Interventions

patisiran

Study Officials

• Jared Gollob, MD

  Alnylam Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 24, 2014
• First Posted: February 3, 2014
• Study Start: January 1, 2014
• Primary Completion: March 1, 2014
• Study Completion: June 1, 2014
• Last Updated: May 25, 2015

Record last verified: 2015-05

Locations

GB (1)