Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at San Diego, California and other locations
Dates
study started
estimated completion

Description

Summary

The purpose of this study is to evaluate the safety and efficacy of extended dosing of IONIS-PKK-LRx administered subcutaneously (SC), with alternative dosing and/or dose frequency with IONIS-PKK-LRx in participants with hereditary angioedema (HAE).

Official Title

An Open-Label Extension Study of ISIS 721744 in Patients With Hereditary Angioedema

Details

This is an open-label extension study of IONIS-PKK-LRx in up to 24 participants with HAE. The length of participation in the study is approximately 68 weeks, which includes an up to 4-week qualification period, a 52-week treatment period, and a 12-week post-treatment period.

Keywords

Hereditary Angioedema HAE Angioedema Angioedemas, Hereditary IONIS-PKK-LRx

Eligibility

You can join if…

Open to people ages 18 years and up

  1. Satisfactory completion of ISIS 721744-CS2 (index study) through Week 17 with an acceptable safety and tolerability profile, per Sponsor and Investigator judgement
  2. Able and willing to participate in a 64-week study
  3. Females must be non-pregnant, non-lactating and either surgically sterile or post-menopausal
  4. Males must be surgically sterile or abstinent* or if engaged in sexual relations with a female of child-bearing potential, participant is utilizing an acceptable contraceptive method Participants must have access to, and the ability to use, ≥ 1 acute medication(s) (e.g., plasma-derived or recombinant C1- inhibitor (C1-INH) concentrate or a bradykinin-2 [BK-2] antagonist) to treat angioedema attacks

You CAN'T join if...

  1. Have any new condition or worsening of an existing condition or change or anticipated change in medication, which in the opinion of the Investigator would make the participant unsuitable for enrollment, or could interfere with the participant participating in or completing the study

Locations

  • University of California San Diego (UCSD) not yet accepting patients
    San Diego California 92122 United States
  • AIRE Medical of Los Angeles accepting new patients
    Santa Monica California 90404 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Ionis Pharmaceuticals, Inc.
ID
NCT04307381
Phase
Phase 2
Study Type
Interventional
Last Updated