Summary

for people ages 18 years and up (full criteria)
study started
Jeffrey Ralph

Description

Summary

The purpose of this program is to provide expanded access to Inotersen for up to 100 Patients with Hereditary Transthyretin Amyloidosis (hTTR).

Official Title

Expanded Access Program for Inotersen (ISIS 420915) in Patients With Hereditary Transthyretin Amyloidosis (hATTR)

Details

The Program is intended to provided expanded access to Inotersen for eligible patients with hATTR who have limited or no available treatment options.

Keywords

Amyloidosis, Hereditary Amyloidosis, Familial Amyloidosis Inotersen

Eligibility

You can join if…

Open to people ages 18 years and up

  • 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

You CAN'T join if...

  • 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

Lead Scientist

  • Jeffrey Ralph
    Professor, Neurology. Authored (or co-authored) 16 research publications

Details

Status
not accepting new patients
Start Date
Sponsor
Ionis Pharmaceuticals, Inc.
ID
NCT03400098
Study Type
Expanded Access
Last Updated