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

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

Status

Completed

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01148953

Enrollment

Registered

2010-06-23

Start date

2010-06-30

Completion date

2012-02-29

Last updated

2012-05-24

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Transthyretin Mediated Amyloidosis (ATTR)

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).

Interventions

DRUGALN-TTR01

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

DRUGSterile Normal Saline (0.9% NaCl)

Calculated volume to match active comparator

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

SINGLE (Subject)

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

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.

Design outcomes

Primary

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

Secondary

Measure	Time frame
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

Countries

France, Portugal, Sweden, United Kingdom

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Mar 10, 2026