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

Phase 1

Completed

• Conditions: Transthyretin Mediated Amyloidosis (ATTR)
• Interventions: Drug: ALN-TTR01; Drug: Sterile Normal Saline (0.9% NaCl)

• Registration Number: NCT01148953
• Lead Sponsor: Alnylam Pharmaceuticals

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).

Detailed Description

Not available

Study Timeline

• Study Start: 2010-06
• Study First Submitted: 2010-06-21
• Study First Submitted QC: 2010-06-22
• Study First Posted: 2010-06-23
• Primary Completion: 2012-01
• Study Completion: 2012-02
• Status Verified: 2012-05
• Last Update Submitted: 2012-05-23
• Last Update Posted: 2012-05-24

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 32

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
ALN-TTR01	ALN-TTR01	-
Sterile Normal Saline (0.9% NaCl)	Sterile Normal Saline (0.9% NaCl)	-

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

Clinical Site; 🇬🇧 London, United Kingdom