An Open-Label, Long Term Safety and Efficacy Study of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)

Phase 1

• Conditions: Hereditary Angioedema; MedDRA version: 23.1Level: PTClassification code: 10019860Term: Hereditary angioedema Class: 100000004850; Therapeutic area: Phenomena and Processes [G] - Genetic Phenomena [G05]; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: CTIS2023-509201-77-00
• Lead Sponsor: Ionis Pharmaceuticals Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-03-14
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 55

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 placebocontrolled 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 C1-esterase inhibitor prior to the Screening Period.

Exclusion Criteria

1. Have any new condition or worsening of an existing condition or change or anticipated change in medication De-novo Participants., 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 Any clinically-significant abnormalities in screening laboratory values., 4. 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., 5. Hypersensitivity to the active substance (donidalorsen) or to any of the excipients., 6. 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., 7. Recent history of, or current drug or alcohol abuse., 8. Participated in a prior donidalorsen study., 9. Exposure to any of the following medications: Angiotensin-converting enzyme (ACE) inhibitors or any estrogen containing medications with systemic absorption 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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified