Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis

Alnylam Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: TTR-mediated Amyloidosis

Treatments: Drug: Patisiran

Study type: Interventional

Funder types: Industry

Identifiers

NCT01617967

ALN-TTR02-002

2012-000467-24 (EudraCT Number)

Details and patient eligibility

About

This was a multiple dose, dose escalation study designed to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of patisiran (ALN-TTR02) in participants with transthyretin (TTR) mediated amyloidosis (ATTR).

Enrollment: 29 patients

Sex: All

Ages: 18 to 85 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Body mass index must be between 17 kg/m^2 and ≤ 33 kg/m^2;
• Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must use appropriate contraception;
• Males agree to use appropriate contraception;
• Diagnosis of TTR amyloidosis;
• Adequate blood counts, liver and renal function;
• Willing to give written informed consent and are willing to comply with the study requirements.

Exclusion criteria

• Known human immunodeficiency virus (HIV) positive status or known or suspected systemic bacterial, viral, parasitic, or fungal infection;
• Received an investigational agent, other than tafamidis or diflunisal, within 30 days prior to first dose study drug administration;
• Prior liver transplant;
• Poor cardiac function;
• Considered unfit for the study by the Principal Investigator;
• Employee or family member of the sponsor or the clinical study site personnel.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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29 participants in 1 patient group

Patisiran (ALN-TTR02)

Experimental group

Description:

Two administrations of patisiran will be administered once every 4 weeks \[Q4W\]) in 4 sequential cohorts with escalating doses followed by optional cohorts with an alternative dosing regimen (once every 3 weeks \[Q3W\]), and an alternative premedication regimen.

Treatment:

Drug: Patisiran