Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema

NCT06415448 | Unknown

• 1 other identifier: ISIS 721744-E01
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Hereditary Angioedema

Keywords

HAE; IONIS PKK-LRx; Donidalorsen; ISIS 721744

Interventions

Donidalorsen DRUG

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

Also known as: IONIS PKK-LRx, ISIS 721744

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

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• Any patient who was withdrawn from the ISIS 721744-CS3 OLE study due to a serious adverse event (SAE) related to donidalorsen therapy or who voluntarily withdrew prior to 205 weeks of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

Sponsors & Collaborators

• Ionis Pharmaceuticals, Inc.: lead

Related Links

• Related Info

MeSH Terms

Conditions

Angioedemas, Hereditary

Interventions

donidalorsen; IONIS-PKK-LRx

Condition Hierarchy (Ancestors)

Angioedema; Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Central Study Contacts

Ionis Pharmaceuticals Medical Information

CONTACT

1-833-644-6647 (833-MI-IONIS); MedInfo@ionisph.com

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 10, 2024
• First Posted: May 16, 2024
• Last Updated: July 11, 2025

Record last verified: 2025-07