Study on Long-Term Safety of Donidalorsen for Patients with Hereditary Angioedema

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What is this study about?

This clinical trial is focused on studying Hereditary Angioedema (HAE), a rare genetic condition that causes sudden swelling in various parts of the body, such as the face, hands, and feet. The study will use a treatment called Donidalorsen, which is an injection designed to help prevent these swelling attacks. Donidalorsen is a type of medication known as an antisense oligonucleotide, which works by targeting specific genetic material to reduce the frequency of HAE attacks.

The purpose of this study is to evaluate the long-term safety of using Donidalorsen in patients with HAE. Participants in the study will receive regular injections of Donidalorsen and will be monitored over an extended period to assess how well the treatment works and to identify any potential side effects. The study will also track the number of HAE attacks participants experience each month and how the treatment affects their quality of life.

Throughout the study, participants will have regular check-ups with healthcare professionals to ensure their safety and to gather information on the effectiveness of the treatment. The study aims to provide valuable insights into the long-term use of Donidalorsen for managing Hereditary Angioedema and to improve the overall understanding of this condition.

1 joining the study

Upon joining the study, participants must provide a written and signed informed consent form. This is a document that confirms understanding and agreement to participate in the study.

Participants must have access to at least one medication to treat angioedema attacks, such as plasma-derived or recombinant C1-INH concentrate or a bradykinin receptor antagonist.

2 eligibility confirmation

Participants must have completed a previous study with the medication ISIS 721744 through Week 25 or have an acceptable safety profile if exiting early.

Participants must be at least 12 years old and have a documented diagnosis of hereditary angioedema (HAE) type 1 or 2.

Participants must be on a stable dose of prophylaxis treatment with lanadelumab, berotralstat, or C1-esterase inhibitor for at least 12 weeks before the screening period.

3 treatment administration

The medication used in this study is called donidalorsen, also known as ISIS 721744. It is administered as a subcutaneous injection, which means it is injected under the skin.

The study aims to evaluate the safety of long-term dosing with donidalorsen in patients with hereditary angioedema.

4 monitoring and evaluation

The primary focus is on the incidence and severity of any adverse events that occur during treatment.

Secondary evaluations include the frequency of hereditary angioedema attacks, the percentage of attack-free patients, and the quality of life over the course of 53 weeks.

5 study duration

The study is expected to continue until November 6, 2026.

Participants will be monitored throughout the study to ensure safety and to assess the effectiveness of the treatment.

Who Can Join the Study?

Participants and, if needed, their legally authorized representatives (like a parent or legal guardian) must provide a written and signed informed consent form. This means they agree to join the study after understanding what it involves.
Participants must have access to and be able to use at least one medication to treat sudden swelling attacks. This is for those who are already part of the Open-Label Extension group.
Participants must have successfully completed a previous study called ISIS 721744-CS5 through Week 25, or be allowed to leave that study with a good safety and tolerability profile. This is for new participants who have not used donidalorsen before.
Participants must be at least 12 years old at the time they give their consent to join the study.
Participants must have a documented diagnosis of Hereditary Angioedema (HAE), specifically types HAE-1 or HAE-2.
Participants must have been on a stable dose of preventive treatment with medications like lanadelumab, berotralstat, or C1-esterase inhibitor for at least 12 weeks before the screening period of the study.

Who Cannot Join the Study?

Patients who do not have Hereditary Angioedema cannot participate. Hereditary Angioedema is a rare genetic condition that causes sudden swelling in different parts of the body.
Patients who are not within the specified age ranges cannot participate. The age ranges include children, teenagers, and adults.
Both male and female patients are considered for participation, so gender is not an exclusion criterion.
Patients who are part of a vulnerable population may not be eligible. A vulnerable population includes groups like children, pregnant women, or those unable to give consent.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Bellvitge University Hospital	L'hospitalet De Llobregat	Spain
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
Azienda Ospedaliera Universitaria Federico II Di Napoli	Naples	Italy
Aix Marseille University	Marseille	France

Other Sites

Site Name	City	Country
Hopital Saint Antoine	Paris	France
Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di Catania	Catania	Italy
Klinikum der Technischen Universitaet Muenchen (TUM Klinikum)	Munich	Germany
Azienda Ospedaliera di Padova	Padua	Italy
Diagnostic-consultative center “Aleksandrovska” EOOD	Sofia	Bulgaria
Virgen del Rocío University Hospital	Sevilla	Spain
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie	Cracow	Poland
Fondazione Salvatore Maugeri Clinica Del Lavoro E Della Riabilitazione	Pavia	Italy
Convex Ltd.	Sofia	Bulgaria
Ueemcnptsxfd Mjyivcr Ctrtlow Gmllzwniu	Groningen	The Netherlands
Auotwdh Oouaeyasokb Ofpxpcwz Rdifpyl Vormt Sfybs Czawbcrt	Palermo	Italy
Ahjzcwavy Ueq	Amsterdam	The Netherlands
Gpgvft Ulfljmduwv Fwjxgwwef	Frankfurt	Germany
Uirourjypd Op Awhdkeb	Edegem	Belgium
Fdapyywgz Pnml Ll Iuvfrfsohxgeq Bndqcbjzx Dzp Hpljiqth Ukuoovjvtnecc Li Pth	Madrid	Spain
Hfusegtz Vxax dgbfysup	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	15.11.2022
Bulgaria	Not recruiting	15.11.2022
France	Not recruiting	15.11.2022
Germany	Not recruiting	15.11.2022
Italy	Not recruiting	15.11.2022
Poland	Not recruiting	15.11.2022
Spain	Not recruiting	15.11.2022
The Netherlands	Not recruiting	15.11.2022

Trial locations

Investigated drugs:

Donidalorsen

Donidalorsen is a medication being studied for its ability to prevent attacks in patients with Hereditary Angioedema (HAE). This trial aims to assess the long-term safety and effectiveness of using donidalorsen regularly to reduce the frequency and severity of HAE episodes.

Investigated diseases:

Hereditary angioedema

Hereditary Angioedema – Hereditary Angioedema is a rare genetic disorder characterized by recurrent episodes of severe swelling. This swelling can affect 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.

Trial ID:

2023-509201-77-00

Protocol code:

ISIS 721744-CS7

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Long-Term Safety of Donidalorsen for Patients with Hereditary Angioedema

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