Donidalorsen for Hereditary Angioedema
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to evaluate the safety and effectiveness of donidalorsen, a treatment for children with hereditary angioedema (HAE) Types I and II. HAE causes sudden swelling attacks without itching, often affecting the skin or mucous areas like the throat. The trial will test donidalorsen in three groups based on the child's weight to ensure proper dosing. Children diagnosed with HAE who experience recurring swelling episodes without hives may be suitable candidates. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to the potential availability of a new treatment.
Do I have to stop taking my current medications for the trial?
The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Is there any evidence suggesting that donidalorsen is likely to be safe for humans?
Research has shown that donidalorsen is generally safe for people with hereditary angioedema (HAE). Studies have found that it reduces the number of HAE attacks, indicating its effectiveness. Importantly, safety data do not reveal any major negative effects on heart health or vital signs. In teenagers, donidalorsen was safe and improved their quality of life. Long-term use also proved effective and was preferred by many patients. Current studies indicate that donidalorsen has a strong safety record in humans.12345
Why do researchers think this study treatment might be promising for hereditary angioedema?
Donidalorsen is unique because it offers a new approach for treating hereditary angioedema by targeting the kinin-kallikrein pathway, which is crucial in the disease's pathology. Unlike conventional treatments, which often focus on managing symptoms or acute attacks, donidalorsen aims to prevent these attacks from happening in the first place. Researchers are excited about its potential to provide more consistent relief and improve the quality of life for patients, as it might reduce the frequency and severity of these unpredictable and debilitating episodes.
What evidence suggests that donidalorsen might be an effective treatment for hereditary angioedema?
Research has shown that donidalorsen greatly reduces the number of hereditary angioedema (HAE) attacks. In one study, patients experienced 97% fewer attacks over four years. Other studies demonstrated a 62% decrease in attacks compared to before treatment. Many patients even reported no attacks after switching from their previous treatments. These findings suggest that donidalorsen could be a strong option for managing HAE. Participants in this trial will receive donidalorsen based on their weight group, with treatment administered over one year.45678
Are You a Good Fit for This Trial?
This trial is for children aged 2 to under 12 years who weigh at least 9 kg and have hereditary angioedema (HAE) type I or II, confirmed by specific blood tests or genetic testing.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive donidalorsen for one year to evaluate safety, efficacy, and pharmacokinetics
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Donidalorsen
Trial Overview
The study is testing donidalorsen, a new medication, to see how safe it is and how children's bodies process it when used to treat hereditary angioedema. All participants will receive the drug.
How Is the Trial Designed?
3
Treatment groups
Experimental Treatment
Participant weighing ≥41kg, will receive donidalorsen over the period of one year.
Participant weighing greater than or equal to (≥)26 kg to \<41 kg, will be administered donidalorsen over the period of one year.
Participant weighing 9 kilograms (kg) to less than (\<)26 kg, will be administered donidalorsen over the period of one year.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Ionis Pharmaceuticals, Inc.
Lead Sponsor
Dr. Brett P. Monia
Ionis Pharmaceuticals, Inc.
Chief Executive Officer since 2020
PhD in Pharmacology from the University of Pennsylvania, BSc in Molecular Biology and Analytical Chemistry from Stockton State College
Dr. Eric Bastings
Ionis Pharmaceuticals, Inc.
Chief Medical Officer
MD
Citations
Donidalorsen Treatment of Hereditary Angioedema in ...
Conclusions: Donidalorsen was well tolerated, decreased HAE attack rate, and improved QoL and disease control. Most patients preferred ...
2.
ir.ionis.com
ir.ionis.com/news-releases/news-release-details/ionis-presents-new-data-demonstrating-long-term-disease-controlIonis presents new data demonstrating long-term disease ...
In the Phase 2 OLE study, patients treated with DAWNZERA Q4W showed a 97% mean HAE attack rate reduction over four years. The median attack-free ...
Efficacy and Safety of Donidalorsen for Hereditary ...
Donidalorsen treatment reduced the hereditary angioedema attack rate, a finding that supports potential prophylactic use for hereditary angioedema.
Donidalorsen Treatment of Hereditary Angioedema in ...
Clinical efficacy outcomes. Overall, patients switching from any prior LTP had a mean 62% reduction in HAE attacks from baseline (with the prior LTP) after ...
Safety and Efficacy of Donidalorsen in Adolescent Patients ...
In the phase 3 OASIS-HAE study (NCT05139810), donidalorsen reduced hereditary angioedema (HAE) attack rates overall and in adolescent patients.
Efficacy and Safety of Donidalorsen for Hereditary ...
Donidalorsen treatment reduced the hereditary angioedema attack rate, a finding that supports potential prophylactic use for hereditary ...
219407Orig1s000 - accessdata.fda.gov
09/19/2023: Donidalorsen orphan drug designation granted for IND 142564 for the treatment of hereditary angioedema. • 08/21/2024: NDA 219407 ...
Efficacy and Safety of Donidalorsen for Hereditary ...
Donidalorsen treatment reduced the hereditary angioedema attack rate, a finding that supports potential prophylactic use for hereditary angioedema.
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