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

Primary Purpose

TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial

Status
No longer available
Phase
Locations
Study Type
Expanded Access
Intervention
patisiran (ALN-TTR02)
Sponsored by
Alnylam Pharmaceuticals
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for TTR-mediated Amyloidosis focused on measuring RNAi therapeutic, FAP, Familial Amyloid Polyneuropathies, TTR, Transthyretin, Amyloidosis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All Sexes

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

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    October 18, 2016
    Last Updated
    July 14, 2022
    Sponsor
    Alnylam Pharmaceuticals
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    1. Study Identification

    Unique Protocol Identification Number
    NCT02939820
    Brief Title
    Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)
    Official Title
    Expanded Access Protocol of Patisiran for Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR Amyloidosis) With Polyneuropathy
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    July 2022
    Overall Recruitment Status
    No longer available
    Study Start Date
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Alnylam Pharmaceuticals

    4. Oversight

    5. Study Description

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

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related
    Keywords
    RNAi therapeutic, FAP, Familial Amyloid Polyneuropathies, TTR, Transthyretin, Amyloidosis

    7. Study Design

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    patisiran (ALN-TTR02)
    Intervention Description
    patisiran (ALN-TTR02) administered by intravenous (IV) infusion

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Eligibility Criteria
    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
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Medical Director
    Organizational Affiliation
    Alnylam Pharmaceuticals
    Official's Role
    Study Director

    12. IPD Sharing Statement

    Citations:
    PubMed Identifier
    31215818
    Citation
    Miah KM, Hyde SC, Gill DR. Emerging gene therapies for cystic fibrosis. Expert Rev Respir Med. 2019 Aug;13(8):709-725. doi: 10.1080/17476348.2019.1634547. Epub 2019 Jun 27.
    Results Reference
    derived
    Links:
    URL
    https://ghr.nlm.nih.gov/condition/transthyretin-amyloidosis
    Description
    https://ghr.nlm.nih.gov/condition/transthyretin-amyloidosis
    URL
    https://www.nlm.nih.gov/medlineplus/amyloidosis.html
    Description
    https://www.nlm.nih.gov/medlineplus/amyloidosis.html

    Learn more about this trial

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

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