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CompletedNCT01559077Updated Jan 15, 2013

Trial to Evaluate Safety, Tolerability, and Parmacokinetics of ALN-TTR02 in Healthy Volunteer Subjects

A Phase 1 interventional study of ALN-TTR02 and Sterile Normal Saline (0.9% NaCl) in TTR-mediated Amyloidosis, sponsored by Alnylam Pharmaceuticals. Completed at 2 sites in United Kingdom. Open to participants aged 18 Years to 45 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2013-01-15.

Sponsored by Alnylam Pharmaceuticals · Phase 1, Interventional, and Treatment

Phase
Phase 1
Study type
Interventional
Enrollment
17
Allocation
Randomized
Ages
18 Years to 45 Years
Sex
All
01

Study 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.

02

Conditions studied

  • TTR-mediated Amyloidosis

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Keywords

  • ATTR
03

In context

Amyloidosis

491 studies on the registry are indexed under Amyloidosis; 135 are open to participants now.

This study's enrollment of 17 is below the median of 40 across 304 interventional studies indexed under Amyloidosis.

Browse Amyloidosis studies →

Lead sponsor

Alnylam Pharmaceuticals is the lead sponsor of 82 studies on the registry; 20 are open to participants now.

Of its 17 completed or terminated interventional studies of FDA-regulated products, 14 (82%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 45 Years
Sexes eligible
All
Accepts healthy volunteers
Yes

Inclusion criteria

  • 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.

Exclusion criteria

Exclusion Criteria:

  • 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
05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Single (Participant)
Enrollment
17 participants (actual)

Study arms

  • Active comparator
    ALN-TTR02

    Drug: ALN-TTR02

  • Placebo comparator
    Sterile Normal Saline (0.9% NaCl)

    Drug: Sterile Normal Saline (0.9% NaCl)

Interventions

  • DrugALN-TTR02

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

  • DrugSterile Normal Saline (0.9% NaCl)

    Calculated volume to match active comparator

06

What researchers measure

Primary outcomes

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

    Time frame: Up to 28 days

Secondary outcomes

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

    Time frame: Up to 180 days

  2. 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)

    Time frame: Up to 56 days

07

Study locations

2 sites
  • Clinical Site
    Leeds, LS2 9LH, United Kingdom
  • Clinical Site
    London, NW10 7EW, United Kingdom
08

References and documents

Publications

  • 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. PubMed 23984729 ↗
09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jan 15, 2013, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT01559077
Lead sponsor
Alnylam Pharmaceuticals
Responsible party
Sponsor
First posted
Mar 21, 2012
Start date
Mar 2012
Primary completion
Jun 2012
Completion
Nov 2012
Last update
Jan 15, 2013

Study contacts

Jared Gollob, MD
study director · Alnylam Pharmaceuticals

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Jan 2013. You cannot join it, but the record below documents what was studied.

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