An Open-Label, Long Term Safety and Efficacy Study of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
Overview
- Phase
- Phase 3
- Intervention
- Donidalorsen
- Conditions
- Hereditary Angioedema
- Sponsor
- Ionis Pharmaceuticals, Inc.
- Enrollment
- 154
- Locations
- 1
- Primary Endpoint
- Percentage of Participants with at Least One Treatment-emergent Adverse Event (TEAE), Graded by Severity
- Status
- Active, not recruiting
- Last Updated
- 4 months ago
Overview
Brief Summary
The purpose of this study is to evaluate the long-term safety and efficacy of donidalorsen in people with HAE and the effects of donidalorsen on the number of HAE attacks and their impact on quality of life (QoL).
Detailed Description
This is a Phase 3, multi-center, open-label, global study with donidalorsen in up to approximately 144 participants with HAE-1 (Type I) and HAE-2 (Type II). There are two groups in this study: 1) participants who roll over from another study of donidalorsen (open-label extension \[OLE\] participants), and 2) new participants who are not rolling over from another study of donidalorsen and were previously maintained on HAE prophylactic therapy with lanadelumab, berotralstat, or a C1-esterase inhibitor (C1-INH). The length of participation in the study is approximately 70 weeks for OLE participants and 76 weeks for other participants. Participants will receive donidalorsen in an Extended Treatment Period for up to an additional 104 weeks.
Investigators
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
OLE Participants
Group 1 and Group 2 participants will be administered donidalorsen by SC injection for up to 157 weeks.
Intervention: Donidalorsen
Outcomes
Primary Outcomes
Percentage of Participants with at Least One Treatment-emergent Adverse Event (TEAE), Graded by Severity
Time Frame: Up to approximately 70 weeks, plus 104 weeks for Group 1; up to approximately 76 weeks, plus 104 weeks for Group 2
Secondary Outcomes
- Time-normalized Number of Investigator-confirmed HAE Attacks (per Month)(Week 1 to Week 157 for Group 1 and Group 2)
- Percentage of Investigator-confirmed HAE Attack-free Participants(Week 1 to Week 157 for Group 1 and Group 2)
- Time-normalized Number of Moderate or Severe Investigator-confirmed HAE Attacks (per Month)(Week 1 to Week 157 for Group 1 and Group 2)
- Number of Investigator-confirmed HAE Attacks Requiring Acute Therapy(Week 1 to Week 157 for Group 1 and Group 2)
- Angioedema Quality of Life (AE-QoL) Questionnaire Total Score(Up to 157 weeks for Group 1 and Group 2)