ATTR Expanded Access Program (EAP) by Ionis
- Conditions
- Amyloidosis, Hereditary
- Registration Number
- NCT03400098
- Lead Sponsor
- Ionis Pharmaceuticals, Inc.
- 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.
Recruitment & Eligibility
- Status
- APPROVED_FOR_MARKETING
- Sex
- All
- Target Recruitment
- Not specified
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
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method