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ATTR Expanded Access Program (EAP) by Ionis

Primary Purpose

Amyloidosis, Hereditary

Status
Approved for marketing
Phase
Locations
Study Type
Expanded Access
Intervention
Inotersen
Sponsored by
Ionis Pharmaceuticals, Inc.
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Amyloidosis, Hereditary

Eligibility Criteria

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

Inclusion Criteria:

  • Male or female at least 18 years of age with a diagnosis of hATTR
  • Symptoms consistent with polyneuropathy
  • Meet Polyneuropathy Disability (PND) Stage I-III requirements

Exclusion Criteria:

  • Known Primary Amyloidosis, Leptomeningeal Amyloidosis or Monoclonal Gammopathy of Undetermined Significance or Multiple Myeloma
  • Have inadequate cardiac function
  • Have low platelet counts
  • Have inadequate renal function

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    January 8, 2018
    Last Updated
    August 1, 2019
    Sponsor
    Ionis Pharmaceuticals, Inc.
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    1. Study Identification

    Unique Protocol Identification Number
    NCT03400098
    Brief Title
    ATTR Expanded Access Program (EAP) by Ionis
    Official Title
    Expanded Access Program for Inotersen (ISIS 420915) in Patients With Hereditary Transthyretin Amyloidosis (hATTR)
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    August 2019
    Overall Recruitment Status
    Approved for marketing
    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
    Ionis Pharmaceuticals, Inc.

    4. Oversight

    5. Study Description

    Brief Summary
    The purpose of this program is to provide expanded access to Inotersen for up to 100 Patients with Hereditary Transthyretin Amyloidosis (hTTR).
    Detailed Description
    The Program is intended to provided expanded access to Inotersen for eligible patients with hATTR who have limited or no available treatment options.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Amyloidosis, Hereditary

    7. Study Design

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    Inotersen
    Intervention Description
    Inotersen administered by subcutaneous (SC) injections in the abdomen, thigh, or upper arm

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Eligibility Criteria
    Inclusion Criteria: Male or female at least 18 years of age with a diagnosis of hATTR Symptoms consistent with polyneuropathy Meet Polyneuropathy Disability (PND) Stage I-III requirements Exclusion Criteria: Known Primary Amyloidosis, Leptomeningeal Amyloidosis or Monoclonal Gammopathy of Undetermined Significance or Multiple Myeloma Have inadequate cardiac function Have low platelet counts Have inadequate renal function

    12. IPD Sharing Statement

    Learn more about this trial

    ATTR Expanded Access Program (EAP) by Ionis

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