A study of donidalorsen for children with hereditary angioedema

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What is this study about?

This study focuses on Hereditary Angioedema, a rare condition that causes unexpected swelling in different parts of the body. This swelling can occur under the skin or in deeper tissues, such as the throat or digestive tract. The study aims to evaluate the safety and how the body processes donidalorsen, an experimental medication provided as a subcutaneous injection, which is an injection given just under the skin.

During the study, participants will receive the medication ISIS 721744. Researchers will monitor how the body handles the drug and look for any side effects. The study will also observe changes in plasma, which is the liquid part of the blood, and levels of plasma kallikrein, a specific protein involved in the swelling process. This investigation will take place over a period of 12 months to see how the treatment affects the frequency and severity of swelling episodes.

Who Can Join the Study?

  • You must be between 2 and 11 years old at the time you agree to join the study.
  • You must weigh at least 9 kg (about 20 pounds).
  • You must have a confirmed diagnosis of Hereditary Angioedema (HAE), which is a rare condition that causes sudden swelling in different parts of the body.
  • The diagnosis must be specifically for HAE-1 or HAE-2, which are types of the condition caused by missing or malfunctioning proteins in the blood.
  • Your diagnosis must be supported by a medical history (a record of your past health issues) that shows signs of the condition and diagnostic testing (medical tests like blood work) that proves you have it.

Who Cannot Join the Study?

  • You cannot participate if your blood tests or other medical exams show unusual results that would make it unsafe for you to join the study.
  • You cannot join if you have been diagnosed with any other type of recurrent angioedema, which is a condition that causes repeated, sudden swelling under the skin. This includes idiopathic angioedema (swelling with no known cause) or a specific form of this disease called HAE with normal C1-INH (a type of swelling where your protein levels appear normal).
  • You cannot participate if you have used another investigational drug (a medicine being tested in a study), a biological agent (medicine made from living organisms), or a medical device within the last month, or for a period of time equal to five half-lives of that medicine. A half-life is the amount of time it takes for the amount of a drug in your body to reduce by half.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Other Sites

Site Name City Country Status
ASST Fatebenefratelli Sacco Milan Italy
Azienda Ospedaliera di Padova Padua Italy
IRCCS Policlinico San Donato San Donato Milanese Italy
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie Cracow Poland
Fvrweuwab Pkfj Lb Iloorsjclpgcf Bnjluwsjh Dsg Hbcgeqfv Ugjwexpxxkfch Ln Ppp Madrid Spain
Hfdjolcr Vlce dpgxsley Barcelona Spain
Wzt Wkijll Inp Pyuol Pvjouhtu Kkjknfl Warsaw Poland

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Italy Italy
Recruiting
01.04.2026
Poland Poland
Recruiting
01.04.2026
Spain Spain
Recruiting
01.04.2026

Trial locations

Investigated drugs:

Donidalorsen is an injection given under the skin that is being tested to see if it is safe and well-tolerated in children with hereditary angioedema. It is being studied to understand how the body processes the medicine and how it might help manage this condition.

Hereditary Angioedema – This is a rare genetic condition that causes episodes of severe swelling in various parts of the body. These swelling episodes occur due to a deficiency or dysfunction of specific proteins in the blood. The swelling typically affects the skin, face, limbs, or gastrointestinal tract. As the condition progresses, these episodes can occur more frequently or with varying levels of intensity. The swelling is caused by increased blood vessel permeability, allowing fluid to leak into surrounding tissues.

Trial ID:
2025-523499-22-00
Protocol code:
ISIS 721744-CS8
Trial Phase:
Therapeutic confirmatory (Phase III)

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