Hereditary Angioedema (HAE) MedDRA version: 23.1 Level: PT Classification code 10019860 Term: Hereditary angioedema System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: ISIS 721744
Product Code: [ISIS 721744]
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: Donidalorsen
CAS Number: 2304701-45-7
Current Sponsor code: ISIS 721744
Sponsors
IONIS PHARMACEUTICALS, INC.
Eligibility
Sex/Gender
All
Inclusion criteria
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 =65 years) yes F.1.3.1 Number of subjects for this age range 5
Exclusion criteria
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 estrogencontaining medications with systemic absorption (such as oral contraceptive or hormonal replacement therapy) within 4 weeks prior to Screening b. Chronic prophylaxis with Takhzyro, Haegarda, Cinryze or Orladeyo within 5 half-lives prior to Screening (i.e., Takhzyro within 10 weeks prior to Screening, Haegarda/Cinryze 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is to evaluate the clinical efficacy of ISIS 721744 in patients with HAE.;Secondary Objective: Evaluate the effects of ISIS 721744 on the quality and pattern of HAE attacks and their impact on Quality of Life.;Primary end point(s): The primary endpoint is the time-normalized number of Investigator-confirmed HAE attacks (per month) from Week 1 to Week 25 compared to placebo.;Timepoint(s) of evaluation of this end point: The time-normalized number of Investigator-confirmed HAE attacks (per month) from W5 to W25 compared to placebo • The percentage of Investigator-confirmed HAE attack-free patients from W5 to W25 compared to placebo • The time-normalized number of moderate or severe Investigatorconfirmed HAE attacks (per month) from W5 to W25 compared to placebo • The number of patients with a clinical response defined as a = 50%, = 70%, or = 90% reduction from Baseline (i.e., screening rate) in Investigator-confirmed HAE attack rate between W5 to W25 compared to placebo • Percent of patients who are well-controlled based on the AECT at W25 • Change in AE-QoL questionnaire total score at W25 • The number of Investigator-confirmed HAE attacks requiring acute therapy from W5 to W25 compared to placebo. | — |
Countries
Belgium, Bulgaria, Canada, Denmark, France, Germany, Israel, Italy, Netherlands, Poland, Spain, United Kingdom, United States
Contacts
Public ContactIonis Clinical Trial Information
Ionis Pharmaceuticals
Outcome results
None listed