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

Approved for marketing
Treatment IND/Protocol
Amyloidosis, Hereditary

Drug: Inotersen
Inotersen administered by subcutaneous (SC) injections in the abdomen, thigh, or upper arm

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

Eligibility Gender
All
Eligibility Age
Minimum: 18 Years
Ionis Pharmaceuticals, Inc.
NCT Number
MeSH Terms
Amyloidosis, Familial
Amyloidosis