A Study to Assess the Clinical Efficacy of IONIS-PKK-LRx in Participants With Hereditary Angioedema

• Status: Completed
• Trial end date: 2021-03-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to evaluate the clinical efficacy, safety, and tolerability of IONIS-PKK-LRx 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 IONIS-PKK-LRx on plasma prekallikrein (PKK) and other relevant biomarkers.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Ionis Pharmaceuticals, Inc.

Criteria

Inclusion Criteria:

• Documented diagnosis of HAE-1/HAE-2 (for inclusion in Part A) or HAE-nC1-INH (for inclusion in Part B)

• Participants must experience 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 Criteria:

• 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)