Donidalorsen: Donidalorsen will be administered by SC injection.
Study 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).
Eligibility
Sex
ALL
Min age
12 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria
1. Participants and, as applicable, legally authorized representatives (i.e., parent(s)/legal guardian), must provide written and signed informed consent form (ICF).
2. Participants must have access to, and the ability to use, ≥ 1 acute medication(s) (e.g., plasma-derived or recombinant C1-INH concentrate or a bradykinin receptor (BK) 2-receptor antagonist) to treat angioedema attacks.
Open-Label Extension Participants ONLY:
3. Satisfactory completion of ISIS 721744-CS5 (randomized placebo-controlled index study) through Week 25 or participants who are allowed to exit ISIS 721744-CS5 study per protocol with an acceptable safety and tolerability profile.
New (not previously on donidalorsen) Participants ONLY:
4. Participants must be aged ≥ 12 years at the time of informed consent and, as applicable, assent.
5. Participants must have a documented diagnosis of HAE-1/HAE-2.
6. Participants must be on a stable dose (≥ 12 weeks) of prophylaxis treatment with lanadelumab or berotralstat or SC C1-esterase inhibitor prior to the Screening Period.
Exclusion Criteria
Open-Label Extension Participants:
1. Have any new condition or worsening of an existing condition or change or anticipated change in medication.
New (not previously on donidalorsen) Participants ONLY:
2. Concurrent diagnosis of any other type of recurrent angioedema, including acquired, idiopathic angioedema or HAE with normal C1-INH (also known as HAE Type III).
3. Anticipated change in the use of concurrent androgen or tranexamic acid prophylaxis used to prevent angioedema attacks.
4. Any clinically-significant abnormalities in screening laboratory values.
5. Malignancy within 5 years of Screening, except for non-melanoma skin cancers, cervical in situ carcinoma, breast ductal carcinoma in situ, or stage 1 prostate carcinoma that has been successfully treated.
6. Hypersensitivity to the active substance (donidalorsen) or to any of the excipients.
7. Treatment with another investigational drug (non-oligonucleotide) or biological agent within 1 month of Screening or 5 half-lives of investigational agent, whichever is longer.
8. Recent history of, or current drug or alcohol abuse.
9. Participated in a prior donidalorsen study.
10. Exposure to any of the following medications:
1. Angiotensin-converting enzyme (ACE) inhibitors or any estrogen containing medications with systemic absorption.
2. Oligonucleotides (including small interfering ribonucleic acid \[siRNA\]) within 4 months of Screening if single dose received, or within 12 months of Screening if multiple doses received. This exclusion does not apply to vaccines.
Primary outcome measure(s)
Percentage of Participants with at Least One Treatment-emergent Adverse Event (TEAE), Graded by Severity — Up to approximately 70 weeks, plus 104 weeks for Group 1; up to approximately 76 weeks, plus 104 weeks for Group 2
Trial sites (50)
Facility
City
Region
Status
Ionis Investigative Site
Paradise Valley
Arizona
Ionis Investigative Site
La Jolla
California
Ionis Investigative Site
Santa Monica
California
Ionis Investigative Site
Walnut Creek
California
Ionis Investigative Site
Tampa
Florida
Ionis Investigative Site
Kansas City
Kansas
Ionis Investigative Site
Boston
Massachusetts
Ionis Investigative Site
Ann Arbor
Michigan
Ionis Investigative Site
Detroit
Michigan
Ionis Investigative Site
St Louis
Missouri
Ionis Investigative Site
Mooresville
North Carolina
Ionis Investigative Site
Cincinnati
Ohio
Ionis Investigative Site
Columbus
Ohio
Ionis Investigative Site
Hershey
Pennsylvania
Ionis Investigative Site
Dallas
Texas
Ionis Investigative Site
Murray
Utah
Ionis Investigative Site
Edegem
Belgium
Ionis Investigative Site
Sofia
Bulgaria
Ionis Investigative Site
Sofia
Bulgaria
Ionis Investigative Site
Ottawa
Ontario
Ionis Investigative Site
Edmonton
Canada
Ionis Investigative Site
La Tronche
France
Ionis Investigative Site
Marseille
France
Ionis Investigative Site
Paris
France
Ionis Investigative Site
Berlin
Germany
Ionis Investigative Site
Frankfurt
Germany
Ionis Investigative Site
München
Germany
Ionis Investigative Site
Ashkelon
Israel
Ionis Investigative Site
Haifa
Israel
Ionis Investigative Site
Tel Aviv
Israel
Ionis Investigative Site
Catania
Italy
Ionis Investigative Site
Naples
Italy
Ionis Investigative Site
Padova
Italy
Ionis Investigative Site
Palermo
Italy
Ionis Investigative Site
San Donato Milanese
Italy
Ionis Investigative Site
Amsterdam
Netherlands
Ionis Investigative Site
Groningen
Netherlands
Ionis Investigative Site
Krakow
Poland
Ionis Investigative Site
San Juan
Puerto Rico
Ionis Investigative Site
Barcelona
Spain
+ 10 more sites — see the full list on the official registry below.
More Ionis Pharmaceuticals, Inc. trials in Germany
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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