Status:

APPROVED_FOR_MARKETING

ATTR Expanded Access Program (EAP) by Ionis

Lead Sponsor:

Ionis Pharmaceuticals, Inc.

Conditions:

Amyloidosis, Hereditary

Eligibility:

All Genders

18+ years

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.

Eligibility Criteria

Inclusion

  • 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

  • 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

Key Trial Info

Start Date :

Trial Type :

EXPANDED_ACCESS

End Date :

Estimated Enrollment :

Patients enrolled

Trial Details

Trial ID

NCT03400098

Last Update

August 5 2019

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