A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema

Completed · Phase 2 · Has a placebo group

Conditions studied: Hereditary Angioedema

In brief

The purpose of this study was to evaluate the clinical efficacy, safety, and tolerability of donidalorsen in participants with hereditary angioedema (HAE) type 1 (HAE-1), HAE type 2 (HAE-2), or HAE with normal C1-inhibitor (C1-INH) and to evaluate the effect of donidalorsen on plasma prekallikrein (PKK) and other relevant biomarkers.

Key facts

Study ID

NCT04030598

Run by

Ionis Pharmaceuticals, Inc.

People needed

23

Starts

2020-01-07

Expected to finish

2021-03-01

Last updated by the study team

2023-04-03

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Documented diagnosis of HAE-1/HAE-2 (for inclusion in Part A) or HAE-nC1-INH (for inclusion in Part B)
• Participants must have experienced a minimum of 2 HAE attacks (assessed by the Angioedema Activity Score [AAS] and confirmed by the investigator) during the screening period
• Access to, and the ability to use, ≥ 1 acute medication(s) to treat angioedema attacks

You may not qualify if…

• Anticipated use of short-term prophylaxis for angioedema attacks for a pre-planned procedure during the screening or study periods
• Concurrent diagnosis of any other type of recurrent angioedema, including acquired or idiopathic angioedema
• Known history of or positive test for human immunodeficiency virus (HIV), hepatitis C, or chronic hepatitis B
• Malignancy within 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated
• Treatment with another investigational drug or biological agent within 1 month or 5 half-lives, whichever is longer, of screening
• Exposure to any of the following medications:
• Angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications with systemic absorption (such as oral contraceptive or hormonal replacement therapy) within 4 weeks prior to screening
• Chronic prophylaxis with Lanadelumab within 10 weeks prior to screening
• Oligonucleotides (including small interfering ribonucleic acid [RNA]) within 4 months of screening (if single dose received) or within 12 months of screening (if multiple doses received)

Where it is running

• Medical Research of Arizona — Scottsdale, Arizona, United States
• University of California San Diego (UCSD) — San Diego, California, United States
• AIRE Medical of Los Angeles — Santa Monica, California, United States
• Midwest Immunology Clinical — Plymouth, Minnesota, United States
• Bernstein Clinical Research Center, LLC — Cincinnati, Ohio, United States
• Penn State Hershey Medical Center — Hershey, Pennsylvania, United States
• AARA Research Center — Dallas, Texas, United States
• Amsterdam UMC, loc. AMC — Amsterdam, Netherlands

Full record on ClinicalTrials.gov

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