Not recruitingRare disease

Study on the Safety of Donidalorsen for Patients 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 clinical trial is focused on studying Hereditary Angioedema, a rare genetic condition that causes sudden swelling in various parts of the body, such as the face, hands, and feet. The treatment being tested in this study is called ISIS 721744, also known by its active substance name, Donidalorsen. This medication is administered as an injection and is a type of treatment known as an antisense oligonucleotide, which is designed to target specific genetic material in the body.

The purpose of this study is to evaluate the safety of using ISIS 721744 over an extended period and to explore different dosing schedules. Participants in this study will receive the medication through injections and will be monitored for any side effects or adverse reactions. The study will also look at how often participants experience attacks of swelling and how the treatment affects their quality of life. Additionally, the study will measure certain substances in the blood to understand how the medication works in the body.

This study is an open-label extension, meaning that all participants will receive the active medication rather than a placebo. The trial will last for approximately 64 weeks, during which participants will have regular check-ups and assessments to ensure their safety and to gather information about the treatment's effects. The goal is to provide valuable insights into the long-term use of ISIS 721744 for managing Hereditary Angioedema.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Participation begins after satisfactory completion of the previous study, ISIS 721744-CS2, through Week 17 with an acceptable safety and tolerability profile.
Participants must be able and willing to engage in a 64-week study period.
Step 2
Eligibility requirements
Females must be non-pregnant, non-lactating, and either surgically sterile or post-menopausal.
Males must be surgically sterile or abstinent, or if engaged in sexual relations with a female of child-bearing potential, must use an acceptable contraceptive method.
Participants must have access to and the ability to use at least one acute medication to treat angioedema attacks.
Step 3
Treatment administration
The medication used in this study is ISIS 721744, administered as a solution for injection.
The study aims to evaluate the safety of extended dosing, and alternative dosing and/or dose frequency with ISIS 721744.
Step 4
Monitoring and evaluation
The primary endpoint is the percentage of participants with at least one treatment-emergent adverse event, graded by severity.
Secondary endpoints include the time-normalized hereditary angioedema (HAE) attacks (monthly) by treatment, plasma PKK levels, plasma proenzyme activation and cHK levels, consumption of on-demand medications, and the Angioedema Quality of Life (AE-QoL) questionnaire score.
Step 5
Study duration
The study is designed to last for 64 weeks.
The estimated end date for the study is March 3, 2025.

Who can join the trial?

5 criteria

Must have completed the previous study called ISIS 721744-CS2 through Week 17 with a safety profile that is acceptable according to the study team.
Must be able and willing to participate in a study that lasts 64 weeks.
Females must not be pregnant or breastfeeding and should be either surgically sterile (unable to have children due to surgery) or post-menopausal (no longer having menstrual periods).
Males must be surgically sterile, abstinent (not engaging in sexual activity), or if sexually active with a female who can have children, must use an acceptable method of birth control.
Must have access to and be able to use at least one medication to treat sudden swelling attacks. These medications could include plasma-derived or lab-made C1-inhibitor concentrates or a type of medication called a bradykinin-2 antagonist.

Who cannot join the trial?

3 criteria

Patients who are not diagnosed with Hereditary Angioedema cannot participate. Hereditary Angioedema is a condition that causes sudden swelling in different parts of the body.
Patients who are under 18 years old cannot participate.
Patients who are part of a vulnerable population, such as those who cannot make decisions for themselves, cannot participate.

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Amsterdam UMC
Amsterdam, The Netherlands

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Investigated drugs

ISIS 721744 is a medication being studied for its safety and effectiveness in patients with Hereditary Angioedema (HAE). This trial aims to understand how well patients tolerate the medication when taken over a longer period and to explore different ways of administering it, such as changing how often it is taken.

Donidalorsen

What is already known about the treatment

ISIS 721744 – This medication is administered through injection and is currently being studied in clinical trials for its safety and effectiveness in treating hereditary angioedema (HAE). It is not yet widely available in medical practice, as it is still under investigation. The main therapeutic indication for ISIS 721744 is to manage and prevent the symptoms of HAE, a condition characterized by recurrent episodes of severe swelling. At the molecular level, ISIS 721744 works by targeting specific genetic pathways to reduce the frequency and severity of these swelling episodes. It is classified pharmacologically as an antisense oligonucleotide, which is a type of medication designed to interfere with the production of proteins that contribute to disease.

Investigated diseases

Hereditary Angioedema – Hereditary Angioedema is a genetic disorder characterized by recurrent episodes of severe swelling. This swelling can occur in various parts of the body, including the hands, feet, face, and airway. The condition is caused by a deficiency or dysfunction of a protein called C1 inhibitor, which leads to an overproduction of bradykinin, a peptide that increases blood vessel permeability. Swelling episodes can be triggered by stress, trauma, or hormonal changes, but they often occur without a clear cause. The frequency and severity of attacks can vary widely among individuals. Swelling in the airway can be particularly dangerous, requiring immediate medical attention.