Hereditary AngioedemaNCT06415448
Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema
A study testing Donidalorsen in people with Hereditary Angioedema, run by Ionis Pharmaceuticals, Inc..
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hereditary Angioedema
- What is being tested?
- Donidalorsen
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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- Any patient who was withdrawn from the ISIS 721744-CS3 OLE study due to a serious adverse event (SAE) related to donidalorsen therapy or who voluntarily withdrew prior to 205 weeks of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.
About this study
Official title: Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema (HAE)
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.
Read the 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.
- Conditions
- Hereditary Angioedema
- Being tested
- Donidalorsen
- Keywords
- HAE, IONIS PKK-LRx, Donidalorsen, ISIS 721744
- Medical subject headings
- Angioedemas, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DonidalorsenDrug
Also called IONIS PKK-LRx, ISIS 721744
Donidalorsen administered once monthly or every other month by subcutaneous (SC) injections in the abdomen, thigh, or upper arm.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 May 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary AngioedemaStudies of the same condition, open and under way.
- More trials run by Ionis Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06415448
- Sponsor's protocol ID
- ISIS 721744-E01
- Status last verified
- Sep 2026
- First submitted
- 10 May 2024
- Last updated
- 4 Sep 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema (HAE)
- ClinicalTrials.gov ID
- NCT06415448
- Acronym / sponsor ID
- ISIS 721744-E01
- Conditions
- Hereditary Angioedema
- Keywords
- HAE, IONIS PKK-LRx, Donidalorsen, ISIS 721744
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Ionis Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- First posted
- 16 May 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DonidalorsenAlso: IONIS PKK-LRx, ISIS 721744Donidalorsen administered once monthly or every other month by subcutaneous (SC) injections in the abdomen, thigh, or upper arm. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Sep 2026.
- Related InfoLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Donidalorsen in people with Hereditary Angioedema, run by Ionis Pharmaceuticals, Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 4 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Sep 2026.