An Open-Label, Long Term Safety and Efficacy Study of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
- Conditions
- HAEHereditary angioedema1000533010002426
- Registration Number
- NL-OMON53420
- Lead Sponsor
- Ionis Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 11
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 placebo-controlled
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
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
4. Any clinically-significant abnormalities in screening laboratory values
5. 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.
6. Hypersensitivity to the active substance (donidalorsen) or to any of the
excipients
7. 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
8. Recent history of, or current drug or alcohol abuse
9. Participated in a prior donidalorsen study
10. 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
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The primary endpoint is the incidence and severity of treatment-emergent<br /><br>adverse events (TEAEs)</p><br>
- Secondary Outcome Measures
Name Time Method