Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema
- Conditions
- Hereditary Angioedema
- Registration Number
- NCT06415448
- Lead Sponsor
- Ionis Pharmaceuticals, Inc.
- Brief Summary
The purpose of the Expanded Access Program is to provide pre-approval access of donidalorsen to eligible patients with Hereditary Angioedema (HAE) who complete the ISIS 721744-CS3 clinical trial.
- Detailed Description
The Expanded Access Program (EAP) is intended to provide pre-approval access to donidalorsen for eligible patients with HAE who complete the ISIS 721744-CS3 clinical trial.
Recruitment & Eligibility
- Status
- AVAILABLE
- Sex
- All
- Target Recruitment
- Not specified
Inclusion Criteria
- Patients in the ISIS 721744-CS3 (Open-Label Extension [OLE] Study) who have completed 205 weeks of treatment.
- Female patients of childbearing potential, and male patients with partners of childbearing potential must be willing to use acceptable contraception, or refrain from sexual activity.
Exclusion Criteria
- Any patient who is pregnant or plans to become pregnant.
- Any patient who was withdrawn from the ISIS 721744-CS3 OLE study due to a serious adverse event (SAE) related to donidalorsen therapy or who voluntarily withdrew prior to 205 weeks of treatment.
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Related Research Topics
Explore scientific publications, clinical data analysis, treatment approaches, and expert-compiled information related to the mechanisms and outcomes of this trial. Click any topic for comprehensive research insights.
What molecular mechanisms does donidalorsen target in the bradykinin pathway for HAE treatment?
How does donidalorsen's prekallikrein inhibition compare to standard-of-care C1 esterase inhibitors in HAE?
Which biomarkers correlate with response to donidalorsen in patients with HAE type I or II?
What adverse events are associated with long-term donidalorsen use in HAE expanded access programs?
Are there other antisense oligonucleotides in development for HAE similar to Ionis Pharmaceuticals' donidalorsen?