Status:
COMPLETED
A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema
Lead Sponsor:
Ionis Pharmaceuticals, Inc.
Conditions:
Hereditary Angioedema
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
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 wi...
Detailed Description
This was a randomized, double-blind, placebo-controlled study in 23 participants conducted concurrently in 2 parts (Part A and Part B); participants were allocated into Part A or Part B according to t...
Eligibility Criteria
Inclusion
- 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
Exclusion
- 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)
Key Trial Info
Start Date :
January 7 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 1 2021
Estimated Enrollment :
23 Patients enrolled
Trial Details
Trial ID
NCT04030598
Start Date
January 7 2020
End Date
March 1 2021
Last Update
April 3 2023
Active Locations (8)
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1
Medical Research of Arizona
Scottsdale, Arizona, United States, 85251
2
University of California San Diego (UCSD)
San Diego, California, United States, 92122
3
AIRE Medical of Los Angeles
Santa Monica, California, United States, 90404
4
Midwest Immunology Clinical
Plymouth, Minnesota, United States, 55446