A Phase 3 Double Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ISIS 721744 in Patients with Hereditary Angioedema (HAE)

Phase 3

Completed

• Conditions: HAE; Hereditary angioedema; 10005330; 10002426

• Registration Number: NL-OMON51690
• Lead Sponsor: Ionis Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 8

Inclusion Criteria

• Patients must be aged >= 12 years at the time of informed consent, and, as
applicable, assent
• Patients must have a documented diagnosis of HAE-1/HAE-2 based upon ALL of
the following:
a. Documented clinical history consistent with HAE (subcutaneous [SC] or
mucosal, non-pruritic swelling episodes without accompanying urticaria)
b. Diagnostic testing results that confirm HAE-1/HAE-2: C1-INH functional
level < 40% normal level. Patients with a functional level of 40% to 50% of
normal can be enrolled if their complement factor C4 level is below the lower
limit of normal (LLN) or if a known pathogenic mutation in the SERPING1 gene
has been demonstrated
c. At least 1 of the following: age at reported HAE onset <= 30 years; a family
history consistent with HAE-1/HAE-2; or complement component 1q within the
normal range
• Patients must:
a. Experience a minimum of 2 HAE attacks (confirmed by the Investigator) during
the Screening Period
b. Be willing to complete the PRO assessments throughout the study
• Patients must have access to, and the ability to use, >= 1 acute medication(s)
(e.g., plasma derived or recombinant C1-INH concentrate or a BK2-receptor
antagonist) to treat angioedema attacks

Exclusion Criteria

• Anticipated use of short-term prophylaxis for angioedema attacks for a
pre-planned procedure during the Screening, Treatment or Post-Treatment Periods
• 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)
• Anticipated change in the use of concurrent androgen prophylaxis used to
treat angioedema attacks
• Participation in a prior ISIS 721744 study
• Exposure to any of the following medications:
a. 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
b. Chronic prophylaxis with Takhzyro (lanadelumab), Haegarda (C1-esterase
inhibitor SQ), Cinryze and Ruconest (C1 esterase inhibitor) or Orladeyo
(berotralstat) within 5 half lives prior to Screening (i.e., Takhzyro within 10
weeks prior to Screening, Haegarda/Cinryze/Ruconest within 2 weeks prior to
screening, Orladeyo within 3 weeks prior to Screening)
c. Oligonucleotides (including small interfering ribonucleic acid) 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
• Study Design: Not specified