Study on C1 Esterase Inhibitor for Treating and Preventing Hereditary Angioedema Attacks in Patients with C1-INH Deficiency

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What is this study about?

This clinical trial is focused on studying a condition known as hereditary angioedema, which is a rare genetic disorder that causes sudden swelling in various parts of the body, such as the face, hands, and feet. The study is testing a treatment called C1-esterase inhibitor, which is derived from human plasma and is designed to help prevent and treat these swelling attacks. The treatment is given through an intravenous injection, meaning it is administered directly into a vein.

The purpose of the study is to determine if the C1-esterase inhibitor is more effective than a placebo in relieving the symptoms of an acute attack in people with hereditary angioedema. Participants in the study will receive either the treatment or a placebo, and neither the participants nor the researchers will know which one is being administered, a method known as a double-blind study. The study will monitor how quickly participants experience relief from their symptoms after receiving the injection.

Participants will be involved in the study for a period of time during which they will receive the treatment or placebo and be observed for any changes in their condition. The study aims to provide valuable information on the safety and effectiveness of the C1-esterase inhibitor in managing hereditary angioedema attacks, potentially leading to improved treatment options for those affected by this condition.

1 joining the study

Upon joining the study, eligibility is confirmed based on age, diagnosis of hereditary angioedema (HAE), and history of attacks.

Informed consent is required, and a negative pregnancy test is necessary for participants of childbearing potential.

2 treatment administration

The study involves the administration of a C1 esterase inhibitor through intravenous injection or infusion.

The medication is given to relieve symptoms of acute hereditary angioedema attacks.

3 symptom monitoring

Symptom relief is monitored every 15 minutes for 4 hours after the injection.

Participants rate their symptom relief at the site of swelling or pain.

4 evaluation of treatment response

The primary goal is to measure the time to the beginning of symptom relief at the attack site.

Secondary evaluations include the percentage of participants responding to treatment and changes in symptom severity.

5 follow-up and additional assessments

Follow-up includes monitoring for any adverse events and changes in quality of life.

Additional assessments involve physical examinations, vital signs, and laboratory tests.

6 end of study

The study is estimated to conclude by December 31, 2025.

Final evaluations compare the end-of-study results with baseline measurements.

Who Can Join the Study?

The participant must be at least 18 years old for the first phase of the study or at least 2 years old for the second phase.
The participant must have a confirmed diagnosis of HAE type I or II. HAE stands for Hereditary Angioedema, a condition that causes sudden swelling.
The participant must have experienced at least 3 moderate or severe HAE attacks (excluding attacks on the arms or legs) in the last 3 months before the screening visit. For participants aged 2 to 12 years, they must have had at least 1 moderate or severe attack in the last 6 months before the screening visit.
The participant must have a documented C1-INH functional activity of less than 50%. C1-INH is a protein in the blood, and low levels can lead to swelling. The participant may or may not have a deficiency of this protein, and their C4 antigen level must be below the normal range.
The participant or their legally authorized representative must have signed an informed consent form, which is a document explaining the study and confirming their agreement to participate. If the participant is capable, they must also agree to participate.
The participant must be willing to follow all study procedures and be available for the entire duration of the study.
If the participant is capable of becoming pregnant, they must have a negative pregnancy test and use a highly effective method of birth control. They must continue using birth control until at least 2 weeks after their last dose of the study medication. Participants not capable of becoming pregnant include those who have had certain surgeries (like removal of the uterus or ovaries) or those who are postmenopausal, meaning they have not had a menstrual period for 12 months without another medical reason.

Who Cannot Join the Study?

Participants who are not experiencing an acute hereditary angioedema attack. This is a sudden swelling that can occur in different parts of the body.
Individuals who are younger than 12 years old or older than 18 years old.
Participants who are not willing to receive treatment through an IV injection. This means medicine is given directly into a vein.
Individuals who are part of a vulnerable population, which means they might need special protection or care.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

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Other Sites

Site Name	City	Country	Status
Institutul Regional De Gastroenterologie Hepatologie Prof. Dr. Octavian Fodor Cluj	Cluj Napoca	Romania
Diagnostic-consultative center “Aleksandrovska” EOOD	Sofia	Bulgaria

Trial status

Country	Status	Recruitment Start
Bulgaria	Recruiting	26.01.2024
Romania	Not recruiting	26.01.2024

Trial locations

Investigated drugs:

OCTA-C1-INH is an intravenous medication derived from human plasma. It is used in this clinical trial to treat and prevent acute attacks of hereditary angioedema (HAE) in patients with a deficiency of C1 esterase inhibitor. The medication works by replacing the missing or malfunctioning C1 inhibitor in the blood, helping to control swelling and other symptoms associated with HAE attacks.

Acute Hereditary Angioedema Attacks – This condition is characterized by sudden episodes of swelling in various parts of the body, including the skin, gastrointestinal tract, and airways. It is a genetic disorder caused by a deficiency or dysfunction of the C1 inhibitor protein, which normally helps regulate inflammation. During an attack, patients may experience painful swelling, abdominal pain, nausea, and difficulty breathing if the airways are affected. The attacks can occur spontaneously or be triggered by stress, trauma, or certain medications. The frequency and severity of attacks can vary widely among individuals. Managing the condition involves understanding triggers and monitoring symptoms to prevent complications.

Trial ID:

2023-503507-29-00

Protocol code:

CONE-02

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on C1 Esterase Inhibitor for Treating and Preventing Hereditary Angioedema Attacks in Patients with C1-INH Deficiency

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?