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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 belowSelect...
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:Select...
I have taken ACE inhibitors or estrogen medications.
Select...
I have been diagnosed with a type of recurrent swelling not caused by HAE Type I/II.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 157 weeks for group 1 and group 2
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 157 weeks for group 1 and group 2
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 • NCT0403059833%
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
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have been in a previous study for donidalorsen.I can access and use medication for sudden swelling attacks.You have had problems with drugs or alcohol in the recent past or are currently using them.I have taken ACE inhibitors or estrogen medications.I haven't taken any experimental drugs or biological treatments recently.I have been diagnosed with hereditary angioedema type 1 or 2.I plan to change my current medication to prevent swelling attacks.I have a new or worsening health condition, or a change in my medication.I haven't had cancer in the last 5 years, except for certain skin, cervical, breast, or prostate cancers.You have any important abnormal results in your screening blood tests.I haven't taken any oligonucleotide medications in the last 4 to 12 months.You are allergic to the main ingredient (donidalorsen) or any other ingredients in the treatment.I have been diagnosed with a type of recurrent swelling not caused by HAE Type I/II.I have been on a stable dose of my preventive medication for at least 12 weeks.I am 12 years old or older.You have completed a specific study and have shown good safety and tolerability.
Research Study Groups:
This trial has the following groups:- Group 1: OLE Participants
Awards:
This trial has 2 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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