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Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema

Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema (HAE)

Status
APPROVED_FOR_MARKETING
Phases
Unknown
Study type
Expanded Access
Source
ClinicalTrials.gov
Registry ID
NCT06415448
Enrollment
Unknown
Registered
2024-05-16
Start date
Unknown
Completion date
Unknown
Last updated
2026-09-04

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hereditary Angioedema

Keywords

HAE, IONIS PKK-LRx, Donidalorsen, ISIS 721744

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.

Interventions

DRUGDonidalorsen

Donidalorsen administered once monthly or every other month by subcutaneous (SC) injections in the abdomen, thigh, or upper arm.

Sponsors

Ionis Pharmaceuticals, Inc.
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
ALL
Age
18 Years to No maximum

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 * Any patient who was withdrawn from the ISIS 721744-CS3 OLE study due to a serious adverse event (SAE) related to donidalorsen therapy or who voluntarily withdrew prior to 205 weeks of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.

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Sep 5, 2026