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

Alnylam Pharmaceuticals1 site in 1 country32 target enrollmentJune 2010

ConditionsTransthyretin Mediated Amyloidosis (ATTR)

InterventionsALN-TTR01 Sterile Normal Saline (0.9% NaCl)

DrugsALN-TTR01 Sterile Normal Saline (0.9% NaCl)

Overview

Phase: Phase 1
Intervention: ALN-TTR01
Conditions: Transthyretin Mediated Amyloidosis (ATTR)
Sponsor: Alnylam Pharmaceuticals
Enrollment: 32
Locations: 1
Primary Endpoint: The proportion of patients experiencing adverse events (AEs), serious adverse events (SAEs), and study drug discontinuation.
Status: Completed
Last Updated: 13 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

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).

Registry

clinicaltrials.gov

Start Date

June 2010

End Date

February 2012

Last Updated

13 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Alnylam Pharmaceuticals

Responsible Party

Sponsor

Eligibility Criteria

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.

Arms & Interventions

ALN-TTR01

Intervention: ALN-TTR01

Sterile Normal Saline (0.9% NaCl)

Intervention: Sterile Normal Saline (0.9% NaCl)

Outcomes

Primary Outcomes

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

Time Frame: Up to 28 Days

Secondary Outcomes

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