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 for controlling rare swelling disorder

NCT ID NCT04307381

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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 tested the long-term safety and effectiveness of donidalorsen, an injectable drug, in 20 people with hereditary angioedema (HAE), a condition that causes sudden, painful swelling attacks. Participants received different doses over 64 weeks. The main goal was to check for side effects, but researchers also tracked how often attacks occurred. This was an extension of an earlier study, meaning all participants had already received the drug before.

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 given as an injection under the skin)
What this could lead to
If successful, this could provide a long-term treatment option to reduce swelling attacks in people with hereditary angioedema.
What could go wrong
This is a small, early-phase extension study with only 20 participants. It focuses on safety and dosing, not proof of cure. Results may not apply to all patients.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

20 people

The number who actually took part.

Started

Mar 2020

Finished

Jan 2025

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

18 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. Satisfactory completion of ISIS 721744-CS2 (index study) through Week 17 with an acceptable safety and tolerability profile, per Sponsor and Investigator judgement 2. Able and willing to participate in a 64-week study 3. Females must be non-pregnant, non-lactating and either surgically sterile or post-menopausal 4. Males must be surgically sterile or abstinent\* or if engaged in sexual relations with a female of child-bearing potential, participant is utilizing an acceptable contraceptive method 5. Participants must have access to, and the ability to use, ≥ 1 acute medication(s) (e.g., plasma-derived or recombinant C1- inhibitor (C1-INH) concentrate or a bradykinin-2 \[BK-2\] antagonist) to treat angioedema attacks Exclusion Criteria: 1\. Have any new condition or worsening of an existing condition or change or anticipated change in medication, which in the opinion of the Investigator would make the participant unsuitable for enrollment, or could interfere with the participant participating in or completing the study

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

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

    Scottsdale, Arizona, 85251, United States

  • Ionis Investigative Site

    Santa Monica, California, 90404, United States

  • Ionis Investigative Site

    Plymouth, Minnesota, 55446, United States

  • Ionis Investigative Site

    Cincinnati, Ohio, 45231, United States

  • Ionis Investigative Site

    Hershey, Pennsylvania, 17033, United States

  • Ionis Investigative Site

    Dallas, Texas, 75231, United States

  • Ionis Investigative Site

    Amsterdam, 1105 AZ, Netherlands

More trials for these conditions

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