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Antisense Oligonucleotide

Donidalorsen for Hereditary Angioedema

Phase 2

Waitlist Available

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- inhibitor (C1-INH) concentrate or a bradykinin-2 [BK-2] antagonist) to treat angioedema attacks

Females must be non-pregnant, non-lactating and either surgically sterile or post-menopausal

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to week 221

Awards & highlights

Study Summary

This trial will test the safety and effectiveness of a drug called Donidalorsen for treating hereditary angioedema (HAE). The drug will be given as a shot under the skin, and different doses and frequencies will be tested.

Who is the study for?

This trial is for individuals who have hereditary angioedema and completed a previous study (ISIS 721744-CS2) without safety concerns. Participants must be able to commit to a 64-week study, use effective contraception if of childbearing potential, and have access to acute medications for angioedema attacks.Check my eligibility

What is being tested?

The trial is testing the extended use of donidalorsen administered subcutaneously in various dosing patterns or frequencies. It aims to assess the long-term safety and effectiveness of this treatment approach in people with hereditary angioedema.See study design

What are the potential side effects?

While specific side effects are not listed here, participants from the initial study tolerated donidalorsen well. Potential side effects may include reactions at the injection site, allergic responses, or other issues as observed in earlier phases.

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.

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I am not pregnant, not breastfeeding, and cannot have children due to surgery or menopause.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to week 221

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to week 221

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to week 221 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Percentage of Participants with at Least One Treatment-emergent Adverse Event (TEAE), Graded by Severity

Secondary outcome measures

Angioedema Quality of Life (AE-QoL) Questionnaire Score

Consumption of On-demand Medications

Plasma Prekallikrein (PKK) Levels

+1 more

Side effects data

From 2021 Phase 2 trial • 23 Patients • NCT04030598

14%

Headache

Nausea

100%

80%

60%

40%

20%

Study treatment Arm

Part A: Donidalorsen 80 mg

Part A: Placebo

Part B: Donidalorsen 80 mg

Trial Design

1Treatment groups

Experimental Treatment

Group I: DonidalorsenExperimental Treatment1 Intervention

Participants will be administered donidalorsen SC for up to 53 weeks. Participants will also be administered donidalorsen in the extended treatment period for an additional 156 weeks, up to week 209.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Donidalorsen

2021

Completed Phase 3

~150

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

Ionis Pharmaceuticals, Inc.Lead Sponsor

146 Previous Clinical Trials

15,262 Total Patients Enrolled

Media Library

Donidalorsen (Antisense Oligonucleotide) Clinical Trial Eligibility Overview. Trial Name: NCT04307381 — Phase 2

Hereditary Angioedema Research Study Groups: Donidalorsen

Hereditary Angioedema Clinical Trial 2023: Donidalorsen Highlights & Side Effects. Trial Name: NCT04307381 — Phase 2

Donidalorsen (Antisense Oligonucleotide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04307381 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What can be said in regards to the safety profile of Donidalorsen?

"Donidalorsen was estimated to have a safety rating of 2, as there is limited evidence supporting its efficacy and some data backing up its safety."

Answered by AI

How many healthcare facilities are engaging in this research experiment?

"The current recruitment process involves 12 medical sites, some of which are located in Plymouth, Cincinnati and Dallas. To reduce the strain on participants' travel needs, it is wise to select a trial centre near you."

Answered by AI

Are any additional participants being sought for the trial?

"Data on clinicaltrials.gov confirms that this particular trial, which was published April 1st 2020 before being edited for the last time August 2nd 2022, is not currently recruiting any participants. Nonetheless, there are 22 other trials searching for patients at present."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema

2020. "An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04307381.

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