Donidalorsen Expanded Access Program for Patients With He... | NCT06415448

Studies
Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema

NCT06415448

Sponsor

Ionis Pharmaceuticals, Inc.

Status

Available

Last Update Posted

Jul 11, 2025Actual

Enrollment

Not provided

Phase

Not provided

Conditions

Hereditary Angioedema

Interventions

Donidalorsen

Countries

Not provided

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Jul 2025

Overall Recruitment Status or Expanded Access Status

Available

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Not provided

Primary Completion Date

Not provided

Completion Date

Not provided

First Submitted Date

May 10, 2024

First Submission Date that Met QC Criteria

May 10, 2024

First Posted Date

May 16, 2024Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jul 9, 2025

Last Update Posted Date

Jul 11, 2025Actual

Description Module

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.

Design Module

Study Type

Expanded Access

Number of References to an Expanded Access Study

Expanded Access Types

Intermediate-type Population

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

No data available

No data is available for this block.

Biospecimen

No data available

No data is available for this block.

Enrollment

Not provided

Arms/Interventions Module

Arm Groups

Not provided

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Donidalorsen	Drug	Donidalorsen administered once monthly or every other month by subcutaneous (SC) injections in the abdomen, thigh, or upper arm.		IONIS PKK-LRx ISIS 721744

Eligibility Module

Eligibility Criteria

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.

Accepts Healthy Volunteers

Not provided

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Ionis Pharmaceuticals Medical Information	Contact	1-833-644-6647 (833-MI-IONIS)		MedInfo@ionisph.com

Overall Officials

Not provided

Locations

Not provided

Label	URL
Related Info	View source

Condition Browse Module

MeSH Terms

ID	Term
D054179	Angioedemas, Hereditary

Ancestor Terms

ID	Term
D000799	Angioedema
D014652	Vascular Diseases
D002318	Cardiovascular Diseases
D000081208	Hereditary Complement Deficiency Diseases

Browse Leaves

Not provided

Browse Branches

Not provided

Intervention Browse Module

MeSH Terms

ID	Term
C000723381	donidalorsen
C000711268	IONIS-PKK-LRx

Ancestor Terms

Not provided

Browse Leaves

Not provided

Browse Branches

Not provided