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Monoclonal Antibodies

Donidalorsen for Hereditary Angioedema

Phase 3
Recruiting
Research Sponsored by Ionis Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
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.
Participants must have a documented diagnosis of HAE-1/HAE-2.
Must not have
Exposure to any of the following medications: Angiotensin-converting enzyme (ACE) inhibitors or any estrogen containing medications with systemic absorption.
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).
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 157 weeks for group 1 and group 2
Awards & highlights

Summary

This trial is testing donidalorsen, a medication for people with Hereditary Angioedema (HAE) Types I and II. The goal is to see if it can reduce the number of swelling attacks and improve quality of life. Donidalorsen works by targeting pathways in the body that cause these attacks. Donidalorsen treatment resulted in a significantly lower rate of angioedema attacks.

Who is the study for?
This trial is for individuals aged 12 or older with a confirmed diagnosis of Hereditary Angioedema (HAE) types 1 or 2. They must have completed a previous study through Week 25, be on stable HAE prophylaxis treatment, and able to manage acute attacks. Excluded are those with recent malignancies, certain medication exposures, hypersensitivity to donidalorsen, other angioedema types, drug/alcohol abuse history.
What is being tested?
The trial studies the long-term safety and effectiveness of donidalorsen in preventing HAE attacks. It aims to understand how this intervention affects the frequency of attacks and quality of life over an extended period for people living with HAE.
What are the potential side effects?
While specific side effects aren't listed here, participants will be monitored for any adverse reactions due to long-term use of donidalorsen. Potential side effects may include reactions at injection sites or allergic responses if sensitivity to the drug exists.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I can access and use medication for sudden swelling attacks.
Select...
I have been diagnosed with hereditary angioedema type 1 or 2.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have taken ACE inhibitors or estrogen medications.
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I have been diagnosed with a type of recurrent swelling not caused by HAE Type I/II.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 157 weeks for group 1 and group 2
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 157 weeks for group 1 and group 2 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Angioedema Quality of Life (AE-QoL) Questionnaire Total Score

Side effects data

From 2021 Phase 2 trial • 23 Patients • NCT04030598
33%
Application site rash
33%
Hyperhidrosis
33%
Vomiting
33%
Feeling hot
100%
80%
60%
40%
20%
0%
Study treatment Arm
Part B: Donidalorsen 80 mg
Part A: Placebo
Part A: Donidalorsen 80 mg

Trial Design

1Treatment groups
Experimental Treatment
Group I: OLE ParticipantsExperimental Treatment1 Intervention
Group 1 and Group 2 participants will be administered donidalorsen by SC injection for up to 157 weeks.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Donidalorsen
2021
Completed Phase 3
~150

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for Hereditary Angioedema (HAE) often target the bradykinin pathway, which is responsible for the swelling episodes characteristic of the condition. Donidalorsen, an antisense oligonucleotide, works by reducing the production of prekallikrein, a precursor to kallikrein. Kallikrein is an enzyme that increases the production of bradykinin, a peptide that causes blood vessels to become more permeable, leading to swelling. By lowering prekallikrein levels, Donidalorsen helps to reduce bradykinin production, thereby preventing or lessening the severity of HAE attacks. This targeted approach is significant for HAE patients as it addresses the root cause of their symptoms, potentially offering more effective and sustained relief.

Find a Location

Who is running the clinical trial?

Ionis Pharmaceuticals, Inc.Lead Sponsor
148 Previous Clinical Trials
15,365 Total Patients Enrolled

Media Library

Donidalorsen (Monoclonal Antibodies) Clinical Trial Eligibility Overview. Trial Name: NCT05392114 — Phase 3
Hereditary Angioedema Research Study Groups: OLE Participants
Hereditary Angioedema Clinical Trial 2023: Donidalorsen Highlights & Side Effects. Trial Name: NCT05392114 — Phase 3
Donidalorsen (Monoclonal Antibodies) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05392114 — Phase 3
~69 spots leftby Dec 2026