RecruitingRare disease

A study of donidalorsen for children with hereditary angioedema

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What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 2 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Administration of medication
The treatment involves receiving an injection of donidalorsen (brand name isis 721744).
This medication is administered through a subcutaneous injection, which means it is injected into the fatty tissue just under the skin.
Step 2
Monitoring and observation
During the trial, safety and tolerability, which refers to how well the body handles the medicine, are monitored.
Blood samples are used to check pharmacokinetics, which is the study of how the body absorbs, distributes, and removes the medication.
The frequency and number of hereditary angioedema (hae) attacks, which are episodes of swelling, are recorded over a 12-month period.
The effectiveness of the medicine is measured by tracking the reduction in the rate of attacks and the levels of a substance called pkk in the blood.
Changes in quality of life are assessed using specific scales designed for children.

Who can join the trial?

5 criteria

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 trial?

3 criteria

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.

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Verified sites

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Verified
ASST Fatebenefratelli Sacco
Milan, Italy
Verified
Azienda Ospedaliera di Padova
Padua, Italy
Responsive
Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Paz
Madrid, Spain

Trial locations

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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.

Donidalorsen

What is already known about the treatment

Donidalorsen - This medication is an injection given through a small needle just under the skin to treat hereditary angioedema, a rare condition that causes severe swelling. Currently being studied in clinical trials, it belongs to a class of drugs called antisense oligonucleotides. At a molecular level, it works by attaching to specific genetic instructions to prevent the body from producing too much of a protein that triggers swelling.

Investigated diseases

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.