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Clinical Trials in Germany / NCT05392114
Active, not recruiting Phase 3

A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)

NCT05392114 · tracked via the Priya Life Science Germany tracker
Phase
Phase 3
Started
2022-07-13
Last updated
2025-12-12

Condition(s) studied

Hereditary Angioedema

Investigational drug(s) / intervention(s)

Donidalorsen →

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)

Trial sites (50)

FacilityCityRegionStatus
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

Other trials for the same condition

Official registry record

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.

View NCT05392114 on ClinicalTrials.gov ↗ ← All trials in Germany