Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

APPROVED_FOR_MARKETING

Study Classification

EXPANDED_ACCESS

Brief Summary

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The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR).

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Detailed Description

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Choosing to participate in an expanded access program is an important personal decision. Talk with your doctor and family members or friends about deciding to join a research study. To learn more about this study, please have your doctor contact the study research staff using the Contacts provided. For general information, see the links provided in More Information.

Conditions

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TTR-mediated Amyloidosis Amyloidosis, Hereditary Amyloid Neuropathies, Familial Familial Amyloid Polyneuropathies Amyloid Neuropathies Amyloidosis, Hereditary, Transthyretin-Related

Interventions

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patisiran (ALN-TTR02)

patisiran (ALN-TTR02) administered by intravenous (IV) infusion

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Male or female greater than or equal to 18 years of age
* Have a diagnosis of hATTR
* Meet Karnofsky performance status and Polyneuropathy Disability (PND) score requirements
* Have adequate complete blood counts, liver function tests and coagulation tests

Exclusion Criteria

* Participated in an interventional hATTR amyloidosis clinical trial involving RNA interference (RNAi) therapeutics within the last 12 months
* Are currently eligible to participate in or currently enrolled in an ongoing interventional hATTR amyloidosis clinical trial
* Have inadequate cardiac function
* Known primary amyloidosis (AL amyloidosis) or leptomeningeal amyloidosis
* Have known serious comorbidities or considered unfit for the program by the investigator
* Prior or planned liver or heart transplantation

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No