ATTR Expanded Access Program (EAP) by Ionis

Amyloidosis, Hereditary Clinical Trial

Official title:

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

Clinical Trial Details — Status: Approved for marketing

Administrative data

• NCT number: NCT03400098
• Other study ID #: ISIS 420915-CS5
• Status: Approved for marketing

Study information

• Verified date: August 2019
• Source: Ionis Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial Summary

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

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 information / eligibility

• Status: Approved for marketing
• Enrollment: 0
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Amyloidosis
• Amyloidosis, Familial
• Amyloidosis, Hereditary

Intervention

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

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Ionis Pharmaceuticals, Inc.