NCT01559077

Brief Summary

The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR02 in healthy volunteer subjects.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
17

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Mar 2012

Shorter than P25 for phase_1

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2012

Completed
18 days until next milestone

First Submitted

Initial submission to the registry

March 19, 2012

Completed
2 days until next milestone

First Posted

Study publicly available on registry

March 21, 2012

Completed
2 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2012

Completed
5 months until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2012

Completed
Last Updated

January 15, 2013

Status Verified

January 1, 2013

Enrollment Period

3 months

First QC Date

March 19, 2012

Last Update Submit

January 14, 2013

Conditions

TTR-mediated Amyloidosis

Keywords

ATTR

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 (2)

  • Pharmacokinetics (PK) of ALN-TTR02 (Cmax, tmax, t1/2, AUC0-last, CL).

    Up to 180 days

  • Effect of ALN-TTR02 on transthyretin (TTR), vitamin A, and retinol binding protein (RBP) (Determination of % Lowering of TTR, vitamin A and RBP to pretreatment/Baseline Levels)

    Up to 56 days

Study Arms (2)

ALN-TTR02

ACTIVE COMPARATOR
Drug: ALN-TTR02

Sterile Normal Saline (0.9% NaCl)

PLACEBO COMPARATOR
Drug: Sterile Normal Saline (0.9% NaCl)

Interventions

ALN-TTR02DRUG

Dose levels between 0.01 and 0.5 mg/kg by intravenous (IV) infusion

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

Calculated volume to match active comparator

Sterile Normal Saline (0.9% NaCl)

Eligibility Criteria

Age18 Years - 45 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64)

You may qualify if:

  • Body mass index must be between 18.0 kg/m2 and ≤ 31.5 kg/m2;
  • Non-smokers for at least 3 months preceding screening;
  • Females subjects must be of non-childbearing potential e.g., post-menopausal or pre-menopausal with surgical sterilization;
  • Males agree to use appropriate contraception;
  • Medical history must be verified by either a personal physician or medical practitioner as appropriate;
  • Willing to give written informed consent and are willing to comply with the study requirements.

You may not qualify if:

  • Known hepatitis B surface antigen (HBsAg), hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) infection
  • Multiple drug allergies or know sensitivity to oligonucleotide
  • History of drug abuse and/or alcohol abuse
  • Receiving an investigational agent within 3 months prior to study drug administration
  • Subjects with safety laboratory test results deemed clinical significant by the Investigator;
  • Received prescription drugs within 4 weeks of first dosing
  • Subjects who have donated more than 500 mL of blood within the 3 months prior to ALN-PCS02 or placebo administration;
  • Subjects who have used prescription drugs within 4 weeks of first dosing
  • Considered unfit for the study by the Principal Investigator
  • Employee or family member of the sponsor or the clinical study site personnel

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Clinical Site

Leeds, LS2 9LH, United Kingdom

Location

Clinical Site

London, NW10 7EW, United Kingdom

Location

Related Publications (1)

  • Coelho T, Adams D, Silva A, Lozeron P, Hawkins PN, Mant T, Perez J, Chiesa J, Warrington S, Tranter E, Munisamy M, Falzone R, Harrop J, Cehelsky J, Bettencourt BR, Geissler M, Butler JS, Sehgal A, Meyers RE, Chen Q, Borland T, Hutabarat RM, Clausen VA, Alvarez R, Fitzgerald K, Gamba-Vitalo C, Nochur SV, Vaishnaw AK, Sah DW, Gollob JA, Suhr OB. Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med. 2013 Aug 29;369(9):819-29. doi: 10.1056/NEJMoa1208760.

    PMID: 23984729DERIVED

MeSH Terms

Interventions

patisiran

Study Officials

  • Jared Gollob, MD

    Alnylam Pharmaceuticals

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
SINGLE
Who Masked
PARTICIPANT
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 19, 2012

First Posted

March 21, 2012

Study Start

March 1, 2012

Primary Completion

June 1, 2012

Study Completion

November 1, 2012

Last Updated

January 15, 2013

Record last verified: 2013-01

Locations

GB(2)