Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

New drug shows promise in preventing hereditary swelling attacks

NCT ID NCT05392114

Disease control Sponsor: Ionis Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests the long-term safety and effectiveness of donidalorsen, a drug given as a shot under the skin, to prevent attacks of hereditary angioedema (HAE). HAE causes sudden, painful swelling in various body parts. The trial involves 154 participants who previously completed an earlier study. Researchers will track side effects, how well the drug reduces attack frequency, and its impact on quality of life over an extended period.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
donidalorsen (a drug injected under the skin to prevent swelling attacks)
What this could lead to
If successful, this could provide a new long-term option to prevent painful swelling attacks in people with hereditary angioedema, improving daily life.
What could go wrong
This is an open-label extension study, so there is no placebo group for comparison. Long-term safety and effectiveness are still being confirmed, and side effects may emerge over time.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

154 people

The number who actually took part.

Started

Jul 2022

Expected to finish

Mar 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria 1. Participants and, as applicable, legally authorized representatives (i.e., parent(s)/legal guardian), must provide written and signed informed consent form (ICF). 2. Participants must have access to, and the ability to use, ≥ 1 acute medication(s) (e.g., plasma-derived or recombinant C1-INH concentrate or a bradykinin receptor (BK) 2-receptor antagonist) to treat angioedema attacks. Open-Label Extension Participants ONLY: 3. Satisfactory completion of ISIS 721744-CS5 (randomized placebo-controlled index study) through Week 25 or participants who are allowed to exit ISIS 721744-CS5 study per protocol with an acceptable safety and tolerability profile. New (not previously on donidalorsen) Participants ONLY: 4. Participants must be aged ≥ 12 years at the time of informed consent and, as applicable, assent. 5. Participants must have a documented diagnosis of HAE-1/HAE-2. 6. Participants must be on a stable dose (≥ 12 weeks) of prophylaxis treatment with lanadelumab or berotralstat or SC C1-esterase inhibitor prior to the Screening Period. Exclusion Criteria Open-Label Extension Participants: 1. Have any new condition or worsening of an existing condition or change or anticipated change in medication. New (not previously on donidalorsen) Participants ONLY: 2. Concurrent diagnosis of any other type of recurrent angioedema, including acquired, idiopathic angioedema or HAE with normal C1-INH (also known as HAE Type III). 3. Anticipated change in the use of concurrent androgen or tranexamic acid prophylaxis used to prevent angioedema attacks. 4. Any clinically-significant abnormalities in screening laboratory values. 5. Malignancy within 5 years of Screening, except for non-melanoma skin cancers, cervical in situ carcinoma, breast ductal carcinoma in situ, or stage 1 prostate carcinoma that has been successfully treated. 6. Hypersensitivity to the active substance (donidalorsen) or to any of the excipients. 7. Treatment with another investigational drug (non-oligonucleotide) or biological agent within 1 month of Screening or 5 half-lives of investigational agent, whichever is longer. 8. Recent history of, or current drug or alcohol abuse. 9. Participated in a prior donidalorsen study. 10. Exposure to any of the following medications: 1. Angiotensin-converting enzyme (ACE) inhibitors or any estrogen containing medications with systemic absorption. 2. Oligonucleotides (including small interfering ribonucleic acid \[siRNA\]) within 4 months of Screening if single dose received, or within 12 months of Screening if multiple doses received. This exclusion does not apply to vaccines.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Hereditary angioedema are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

hereditary angioedema hereditary angioedema type 1 hereditary angioedema type 2

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hereditary angioedema

Contacts and locations

Locations

  • Ionis Investigative Site

    Paradise Valley, Arizona, 85253, United States

  • Ionis Investigative Site

    La Jolla, California, 92037, United States

  • Ionis Investigative Site

    Santa Monica, California, 90404, United States

  • Ionis Investigative Site

    Walnut Creek, California, 94598, United States

  • Ionis Investigative Site

    Tampa, Florida, 33613, United States

  • Ionis Investigative Site

    Kansas City, Kansas, 66103, United States

  • Ionis Investigative Site

    Boston, Massachusetts, 02114, United States

  • Ionis Investigative Site

    Ann Arbor, Michigan, 48109, United States

  • Ionis Investigative Site

    Detroit, Michigan, 48202, United States

  • Ionis Investigative Site

    St Louis, Missouri, 63141, United States

  • Ionis Investigative Site

    Mooresville, North Carolina, 28117, United States

  • Ionis Investigative Site

    Cincinnati, Ohio, 45236, United States

  • Ionis Investigative Site

    Columbus, Ohio, 43235, United States

  • Ionis Investigative Site

    Hershey, Pennsylvania, 17033, United States

  • Ionis Investigative Site

    Dallas, Texas, 75231, United States

  • Ionis Investigative Site

    Murray, Utah, 84107, United States

  • Ionis Investigative Site

    Edegem, 2650, Belgium

  • Ionis Investigative Site

    Sofia, 1431, Bulgaria

  • Ionis Investigative Site

    Sofia, 1680, Bulgaria

  • Ionis Investigative Site

    Ottawa, Ontario, K1H 1E4, Canada

  • Ionis Investigative Site

    Edmonton, AB T6G 1Z1, Canada

  • Ionis Investigative Site

    La Tronche, 38700, France

  • Ionis Investigative Site

    Marseille, 13385, France

  • Ionis Investigative Site

    Paris, 75012, France

  • Ionis Investigative Site

    Berlin, 12203, Germany

  • Ionis Investigative Site

    Frankfurt, 60590, Germany

  • Ionis Investigative Site

    München, 81675, Germany

  • Ionis Investigative Site

    Ashkelon, 7830604, Israel

  • Ionis Investigative Site

    Haifa, 3104802, Israel

  • Ionis Investigative Site

    Tel Aviv, 6423906, Israel

  • Ionis Investigative Site

    Catania, 95123, Italy

  • Ionis Investigative Site

    Naples, 80131, Italy

  • Ionis Investigative Site

    Padova, 35128, Italy

  • Ionis Investigative Site

    Palermo, 90146, Italy

  • Ionis Investigative Site

    San Donato Milanese, 20097, Italy

  • Ionis Investigative Site

    Amsterdam, 1105, Netherlands

  • Ionis Investigative Site

    Groningen, 9713 AP, Netherlands

  • Ionis Investigative Site

    Krakow, 31-501, Poland

  • Ionis Investigative Site

    San Juan, PR 00927, Puerto Rico

  • Ionis Investigative Site

    Barcelona, 08035, Spain

  • Ionis Investigative Site

    Barcelona, 08907, Spain

  • Ionis Investigative Site

    Madrid, 28046, Spain

  • Ionis Investigative Site

    Seville, 41013, Spain

  • Ionis Investigative Site

    Valencia, 46026, Spain

  • Ionis Investigative Site

    Altındağ, 06230, Turkey (Türkiye)

  • Ionis Investigative Site

    Bornova, 35100, Turkey (Türkiye)

  • Ionis Investigative Site

    Istanbul, 34093, Turkey (Türkiye)

  • Ionis Investigative Site

    Birmingham, B9 5SS, United Kingdom

  • Ionis Investigative Site

    Bristol, BS10 5NB, United Kingdom

  • Ionis Investigative Site

    London, E1 2ES, United Kingdom

More trials for these conditions

Other studies related to the condition(s) this trial covers.