An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema
An Open-Label Extension Study of ISIS 721744 in Patients With Hereditary Angioedema
2 other identifiers
interventional
20
2 countries
7
Brief Summary
The purpose of this study is to evaluate the safety and efficacy of extended dosing of donidalorsen administered subcutaneously (SC), with alternative dosing and/or dose frequency with donidalorsen in participants with hereditary angioedema (HAE).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Apr 2020
Longer than P75 for phase_2
7 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 11, 2020
CompletedFirst Posted
Study publicly available on registry
March 13, 2020
CompletedStudy Start
First participant enrolled
April 1, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 24, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
January 24, 2025
CompletedMarch 24, 2025
March 1, 2025
4.8 years
March 11, 2020
March 21, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Percentage of Participants with at Least One Treatment-emergent Adverse Event (TEAE), Graded by Severity
Up to Week 221
Secondary Outcomes (4)
The Time-normalized HAE Attacks (per Month) by Treatment
Up to Week 221
Plasma Prekallikrein (PKK) Levels
Up to Week 221
Consumption of On-demand Medications
Up to Week 221
Angioedema Quality of Life (AE-QoL) Questionnaire Score
Up to Week 221
Study Arms (1)
Donidalorsen
EXPERIMENTALParticipants will be administered donidalorsen SC for up to 53 weeks. Participants will also be administered donidalorsen in the extended treatment period for an additional 156 weeks, up to week 209.
Interventions
Eligibility Criteria
You may qualify if:
- Satisfactory completion of ISIS 721744-CS2 (index study) through Week 17 with an acceptable safety and tolerability profile, per Sponsor and Investigator judgement
- Able and willing to participate in a 64-week study
- Females must be non-pregnant, non-lactating and either surgically sterile or post-menopausal
- 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 may not qualify if:
- \. 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (7)
Ionis Investigative Site
Scottsdale, Arizona, 85251, United States
Ionis Investigative Site
Santa Monica, California, 90404, United States
Ionis Investigative Site
Plymouth, Minnesota, 55446, United States
Ionis Investigative Site
Cincinnati, Ohio, 45231, United States
Ionis Investigative Site
Hershey, Pennsylvania, 17033, United States
Ionis Investigative Site
Dallas, Texas, 75231, United States
Ionis Investigative Site
Amsterdam, 1105 AZ, Netherlands
Related Publications (1)
Petersen RS, Bordone L, Riedl MA, Tachdjian R, Craig TJ, Lumry WR, Manning ME, Bernstein JA, Raasch J, Zuraw BL, Deng Y, Newman KB, Alexander VJ, Lui C, Schneider E, Cohn DM. A phase 2 open-label extension study of prekallikrein inhibition with donidalorsen for hereditary angioedema. Allergy. 2024 Mar;79(3):724-734. doi: 10.1111/all.15948. Epub 2023 Nov 27.
PMID: 38009241DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
- Expanded Access
- Yes
Study Record Dates
First Submitted
March 11, 2020
First Posted
March 13, 2020
Study Start
April 1, 2020
Primary Completion
January 24, 2025
Study Completion
January 24, 2025
Last Updated
March 24, 2025
Record last verified: 2025-03
Data Sharing
- IPD Sharing
- Will share
Ionis may share anonymized individual participant data, aggregated clinical data, and other types of data that support the results in this study. Data requests from qualified researchers will be considered once all three of the following criteria are met: (1) 12 months from marketing approval of the study drug in both the United States and European Union; (2) 18 months from conclusion of the study; and (3) 6 months from publication of study article. Access would be via a secure environment and is contingent upon approval of a research proposal and entry into an appropriate data use agreement. Requests to access data can be submitted via the website https://vivli.org/ourmember/ionis/.