The Study of an Investigational Drug, ALN-TTR02, for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02.

Phase 1

• Conditions: Transthyretin mediated amyloidosis (ATTR); MedDRA version: 19.0Level: PTClassification code 10007509Term: Cardiac amyloidosisSystem Organ Class: 10007541 - Cardiac disorders; MedDRA version: 19.0Level: PTClassification code 10019889Term: Hereditary neuropathic amyloidosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2013-001644-65-SE
• Lead Sponsor: Alnylam Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-06-04
• Last Update Posted: 10 October 2016

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 28

Inclusion Criteria

1. Previously received and tolerated ALN-TTR02 in Study ALN-TTR02-002.
2. Adequate Karnofsky performance status, liver function, and renal function.

Additional inclusion criteria are utilized to assess entry in PK subgroup and for the cardiac subgroup.

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 14
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 14

Exclusion Criteria

1. Pregnant or nursing.
2. Has had a liver transplant.
3. Has a New York Heart Association heart failure classification >2.
4. Has unstable angina.
5. Has uncontrolled clinically significant cardiac arrhythmia.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified