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

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

29 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-05-31

Study Completion Date

2014-01-31

Brief Summary

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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).

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Detailed Description

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Conditions

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TTR-mediated Amyloidosis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Patisiran (ALN-TTR02)

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.

Group Type EXPERIMENTAL

Patisiran

Intervention Type DRUG

Participants received a single dose of patisiran as an intravenous (IV) infusion on Day 0 and Day 28 (Q4W). Optional cohorts received an alternative dosing regimen (once every 3 weeks \[Q3W\]: Day 0 and Day 21) and an alternative premedication regimen.

Interventions

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Patisiran

Participants received a single dose of patisiran as an intravenous (IV) infusion on Day 0 and Day 28 (Q4W). Optional cohorts received an alternative dosing regimen (once every 3 weeks \[Q3W\]: Day 0 and Day 21) and an alternative premedication regimen.

Intervention Type DRUG

Other Intervention Names

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ALN-TTR02

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Maximum Eligible Age

85 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No