HAE Hereditary angioedema
Conditions
Interventions
During the Treatment Period, study drug (ISIS 721744 or Placebo) will be
administered as a single-SC injection every 4 weeks. For Part A, approximately
18 eligible patients (HAE 1 or HAE 2) will be
Sponsors
Ionis Pharmaceuticals, Inc.
Eligibility
Age
18 Years to 99 Years
Inclusion 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 or more acute medication(s) to treat angioedema attacks.
Exclusion criteria
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. - History of acquired coagulopathies or bleeding diathesis. - Active infection with 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: 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 Lanadelumab within 10 weeks prior to Screening. c. Oligonucleotides (including small interfering RNA) within 4 months of Screening if single dose received, or within 12 months of Screening if multiple doses received.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoint is the time-normalized number of HAE attacks (per month) from Week 1 to Week 17. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary endpoints include the following: * The time-normalized number of HAE attacks (per month) from Week 5 to Week 17 * The time-normalized number of moderate or severe HAE attacks (per month) from Week 5 to Week 17 * The number of patients with a clinical response (defined as a * 50%, * 70%, or * 90% reduction from Baseline in HAE attack rate) by Week 17 * The number of HAE attacks requiring acute therapy from Week 5 to Week 17 * Cleaved high molecular weight kininogen (cHK) levels at Weeks 9 and 17 * PKK activity at Weeks 9 and 17 * Consumption of on-demand medication at Weeks 9 and 17 * Angioedema quality of life (AE-QoL) questionnaire score at Weeks 9 and 17 | — |
Countries
Netherlands
Outcome results
None listed