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
Related Research Topics
Explore scientific publications, clinical data analysis, treatment approaches, and expert-compiled information related to the mechanisms and outcomes of this trial. Click any topic for comprehensive research insights.
What are the molecular mechanisms of Inotersen in targeting TTR amyloid formation in hATTR patients?
How does Inotersen compare to standard-of-care treatments like tafamidis in managing hATTR amyloidosis?
Which biomarkers are used for patient selection and monitoring treatment response in the Inotersen EAP?
What are the known adverse events associated with Inotersen and how are they managed in clinical practice?
Are there any combination therapies or competitor drugs for hATTR amyloidosis currently under investigation alongside Inotersen?