ATTR Expanded Access Program (EAP) by Ionis

Ionis Pharmaceuticals

Status

Conditions: Amyloidosis, Hereditary

Treatments: Drug: Inotersen

Study type: Expanded Access

Funder types: Industry

Identifiers

NCT03400098

ISIS 420915-CS5

Details and patient eligibility

About

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

Full description

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

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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