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

Alnylam Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Amyloidosis

Treatments: Drug: Patisiran

Study type: Interventional

Funder types: Industry

Identifiers

NCT02510261

2014-003877-40 (EudraCT Number)

ALN-TTR02-006

Details and patient eligibility

About

The purpose of this study is to evaluate the safety and efficacy of long-term dosing with ALN-TTR02 (patisiran) in participants with transthyretin (TTR) mediated amyloidosis (ATTR).

Enrollment: 211 patients

Sex: All

Ages: 18 to 85 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Have completed a patisiran study (i.e., completed the last efficacy visit in the parent study) and, in the opinion of the investigator, tolerated study drug
• Be willing and able to comply with the protocol-required visit schedule and visit requirements and provide written informed consent

Exclusion criteria

• Any new or uncontrolled condition that could make the participant unsuitable for participation

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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211 participants in 3 patient groups

Prior Placebo Group of Study 004

Experimental group

Description:

Participants who received placebo and completed parent study ALN-TTR02-004 (NCT01960348) were enrolled to receive 0.3 milligrams per kilogram (mg/kg) patisiran intravenously (IV) once every 3 weeks (Q3W) up to 65.5 months.

Treatment:

Drug: Patisiran

Prior Patisiran Group of Study 004

Experimental group

Description:

Participants who received patisiran and completed parent study ALN-TTR02-004 (NCT01960348) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 66.9 months.

Treatment:

Drug: Patisiran

Prior Patisiran Group of Study 003

Experimental group

Description:

Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months.

Treatment:

Drug: Patisiran