Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)

Ionis Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: FAP; TTR; Amyloidosis; Familial Amyloid Polyneuropathy; Transthyretin

Treatments: Drug: Inotersen

Study type: Interventional

Funder types: Industry

Identifiers

NCT02175004

2013-004561-13 (EudraCT Number)

ISIS 420915-CS3

Details and patient eligibility

About

This study evaluates the safety and tolerability of extended dosing with IONIS-TTR Rx in patients with Familial Amyloid Polyneuropathy.

Full description

Familial Amyloid Polyneuropathy (FAP) is a rare, hereditary disease caused by mutations in the transthyretin (TTR) protein. TTR is made by the liver and secreted into the blood. TTR mutations cause it to misfold and deposit in multiple organs causing FAP.

IONIS-TTR Rx is an antisense drug that is designed to decrease the amount of mutant and normal TTR made by the liver. It is predicted that decreasing the amount of TTR protein will result in a decrease in the formation of TTR deposits, and thus slow or stop disease progression.

This study evaluates the safety and tolerability of extended dosing with IONIS-TTR Rx in patients with Familial Amyloid Polyneuropathy.

Enrollment: 135 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Satisfactory completion of dosing & efficacy assessments in ISIS 420915-CS2

Exclusion criteria

• Any new condition or worsening of existing condition that could make the patient unsuitable for participation, or interfere with the patient participating in and/or completing the study

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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135 participants in 2 patient groups

Previous Placebo-Inotersen 300 mg

Experimental group

Description:

Participants received subcutaneous (SC) doses of 300 milligrams (mg) inotersen once weekly for up to 260 weeks. Participants who received inotersen-matching placebo in the previous study- ISIS 420915-CS2 (NCT01737398) were included in this group.

Treatment:

Drug: Inotersen

Previous Inotersen-Inotersen 300 mg

Experimental group

Description:

Participants received SC doses of 300 mg inotersen once weekly for up to 260 weeks. Participants who received inotersen in the previous study- ISIS 420915-CS2 were included in this group.

Treatment:

Drug: Inotersen