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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

32 participants

Study Classification

INTERVENTIONAL

Study Start Date

2010-06-30

Study Completion Date

2012-02-29

Brief Summary

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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).

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Detailed Description

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Conditions

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Transthyretin Mediated Amyloidosis (ATTR)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Participants

Study Groups

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ALN-TTR01

Group Type ACTIVE_COMPARATOR

ALN-TTR01

Intervention Type DRUG

Dose levels between 0.01 and 1.0 mg/kg by intravenous (IV) infusion

Sterile Normal Saline (0.9% NaCl)

Group Type PLACEBO_COMPARATOR

Sterile Normal Saline (0.9% NaCl)

Intervention Type DRUG

Calculated volume to match active comparator

Interventions

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ALN-TTR01

Dose levels between 0.01 and 1.0 mg/kg by intravenous (IV) infusion

Intervention Type DRUG

Sterile Normal Saline (0.9% NaCl)

Calculated volume to match active comparator

Intervention Type DRUG

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No