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

Alnylam Pharmaceuticals

Status and phase

Completed

Phase 1

Conditions

Transthyretin Mediated Amyloidosis (ATTR)

Treatments

Drug: Sterile Normal Saline (0.9% NaCl)

Drug: ALN-TTR01

Study type

Interventional

Funder types

Industry

Identifiers

NCT01148953

ALN-TTR01-001

2009-017383-16 (EudraCT Number)

Details and patient eligibility

About

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).

Enrollment

32 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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.