Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema

NCT ID: NCT06415448

Last Updated: 2025-07-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

AVAILABLE

Study Classification

EXPANDED_ACCESS

Brief Summary

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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

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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.

Conditions

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Hereditary Angioedema

Interventions

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Donidalorsen

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

Intervention Type DRUG

Other Intervention Names

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IONIS PKK-LRx ISIS 721744

Eligibility Criteria

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* Any patient who was withdrawn from the ISIS 721744-CS3 OLE study due to a serious adverse event (SAE) related to donidalorsen therapy or who voluntarily withdrew prior to 205 weeks of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

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ionis Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Central Contacts

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Ionis Pharmaceuticals Medical Information

Role: CONTACT

1-833-644-6647 (833-MI-IONIS)

Related Links

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Other Identifiers

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ISIS 721744-E01

Identifier Type: -

Identifier Source: org_study_id

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