Trial to Evaluate Safety and Tolerability of ALN-TTR01 in Transthyretin (TTR) Amyloidosis

May 23, 2012 updated by: Alnylam Pharmaceuticals

A Phase 1, Randomized, Single-Blind, Placebo-Controlled, Dose Escalation Trial to Evaluate the Safety and Tolerability of a Single Dose of Intravenous ALN-TTR01 in Patients With TTR Amyloidosis

The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR01 in patients with transthyretin (TTR) mediated amyloidosis (ATTR).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

32

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Le Kremlin Bicêtre, France, 94275
        • Clinical Site
  • Portugal
      • Porto, Portugal, 4099-001
        • Clinical Site
  • Sweden
      • Umeå, Sweden, SE 901 85
        • Clinical Site
  • United Kingdom
      • London, United Kingdom, SE1 1YR
        • Clinical Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Diagnosis of TTR amyloidosis
  • Adequate blood counts, liver and renal function
  • Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must use an adequate method of birth control
  • Males agree to use appropriate contraception
  • Willing and able to comply with protocol-required visit schedule and visit requirements and provide written informed consent.

Exclusion Criteria:

  • Known human immunodeficiency virus (HIV) positive status
  • Receiving antibiotics for bacterial infection within 7 days of screening
  • Known or suspected systemic viral, parasitic or fungal infection
  • Receiving an investigational agent within 30 days prior to study drug administration
  • Poor cardiac function
  • Considered unfit for the study by the Principal Investigator
  • Known sensitivity to oligonucleotides
  • Employee or family member of the sponsor or the clinical study site personnel.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: SINGLE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
PLACEBO_COMPARATOR: Sterile Normal Saline (0.9% NaCl)
Drug: Sterile Normal Saline (0.9% NaCl)
Calculated volume to match active comparator
ACTIVE_COMPARATOR: ALN-TTR01
Drug: ALN-TTR01
Dose levels between 0.01 and 1.0 mg/kg by intravenous (IV) infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
The proportion of patients experiencing adverse events (AEs), serious adverse events (SAEs), and study drug discontinuation.
Time Frame: Up to 28 Days
Up to 28 Days

Secondary Outcome Measures

Outcome Measure
Time Frame
Pharmacokinetics (PK) of ALN-TTR01 (Cmax, tmax, t1/2, AUC0-last, CL)
Time Frame: Up to 70 days
Up to 70 days
Effect of ALN-TTR01 on Circulating TTR Levels (Determination of % Lowering of TTR to Pretreatment/Baseline TTR Level)
Time Frame: up to 70 days
up to 70 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alnylam Pharmaceuticals

Investigators

  • Study Director: Jared Gollob, MD, Alnylam Pharmaceuticals Inc

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2010

Primary Completion (ACTUAL)

January 1, 2012

Study Completion (ACTUAL)

February 1, 2012

Study Registration Dates

First Submitted

June 21, 2010

First Submitted That Met QC Criteria

June 22, 2010

First Posted (ESTIMATE)

June 23, 2010

Study Record Updates

Last Update Posted (ESTIMATE)

May 24, 2012

Last Update Submitted That Met QC Criteria

May 23, 2012

Last Verified

May 1, 2012

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALN-TTR01-001
  • 2009-017383-16 (EUDRACT_NUMBER)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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