Not recruitingRare disease

Study on NTLA-2002 for Adults with Hereditary Angioedema Using Ziclumeran and Lonvoguran

Verified siteInvestigationalNo placebo

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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 a condition called Hereditary Angioedema (HAE), 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 new treatment called NTLA-2002, which is designed to help manage and reduce the frequency of these swelling attacks. NTLA-2002 is a special type of treatment that uses advanced technology involving nucleic acids, which are the building blocks of genetic material. This treatment is given through an infusion, which means it is delivered directly into the bloodstream through a vein.

The purpose of this study is to evaluate the safety and effectiveness of NTLA-2002 in adults with Hereditary Angioedema. The study is divided into different phases. In the first phase, the main goal is to determine the safety of NTLA-2002 and find the right dose for further testing. In the second phase, the focus is on understanding how well NTLA-2002 can reduce the number of HAE attacks. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of NTLA-2002. There is also a part of the study where participants who initially received a placebo or a lower dose of NTLA-2002 may receive a higher dose to further assess safety.

Participants in the study will receive the treatment through an intravenous infusion, which means it is administered directly into a vein. The study will monitor participants over a period of time to observe any changes in the frequency of HAE attacks and to ensure the treatment is safe. The study aims to provide valuable information that could lead to better management of Hereditary Angioedema in the future.

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
Enrollment
Upon joining the study, eligibility is confirmed based on specific criteria, including age and medical history related to hereditary angioedema (HAE).
Informed consent is required, ensuring understanding of the study's purpose and procedures.
Step 2
Phase 1: Initial Treatment
The primary goal is to assess the safety of NTLA-2002 and determine appropriate doses for the next phase.
NTLA-2002 is administered intravenously as a dispersion for infusion.
Monitoring for any adverse effects is conducted to ensure safety and tolerability.
Step 3
Phase 2: Efficacy Evaluation
The focus shifts to evaluating the effect of NTLA-2002 on reducing HAE attacks.
Participants receive either NTLA-2002 or a placebo, with the frequency and severity of HAE attacks being closely monitored.
The study aims to determine the number of HAE attacks per month over a 16-week period.
Step 4
Placebo Crossover and Follow-on Dosing Substudy
Participants who initially received a placebo may receive a 50 mg dose of NTLA-2002.
Those who received a 25 mg dose in earlier phases may continue with the same or adjusted dosage.
Safety and the number of HAE attacks are assessed during this period.
Step 5
Monitoring and Follow-up
Throughout the study, regular monitoring of health and any side effects is conducted.
Participants are required to report any HAE attacks and use of acute medications.
The study is expected to conclude by June 2026, with ongoing assessments to ensure participant safety.

Who can join the trial?

11 criteria

Participants must be **18 years or older** at the time of signing the consent form.
Participants must have a **documented diagnosis of Hereditary Angioedema (HAE)**, which is a condition confirmed by specific laboratory tests.
Participants must have had at least **3 HAE attacks** in the past 3 months, confirmed by a doctor.
Participants must be able to **provide signed informed consent**, meaning they understand and agree to the study's terms.
Participants must agree **not to join another study** while participating in this one.
Participants must have access to and be able to use at least **one medication** to treat angioedema attacks.
Participants must meet certain **laboratory test criteria** during the screening process, such as normal liver and kidney function tests.
**Female participants** who can have children must agree to use a highly effective method of **birth control** from the time they join the study until 12 months after the last dose of the study drug. This does not apply to women who are postmenopausal or surgically sterile.
**Male participants** with partners who can have children must agree to use a **condom** from the time they join the study until 4 months after the last dose of the study drug.
Male participants must agree **not to donate sperm** for 4 months after the last dose of the study drug.
Participants must agree to **limit alcohol consumption** to one drink per day for a specified period after receiving the study drug.

Who cannot join the trial?

10 criteria

Individuals who do not have a diagnosis of Hereditary Angioedema, a condition that causes sudden swelling in different parts of the body.
Individuals who are not within the specified age range for the study.
Individuals who are not part of the specified clinical trial groups.
Individuals who are not willing or able to follow the study procedures.
Individuals who have other medical conditions that might interfere with the study.
Individuals who are pregnant or breastfeeding.
Individuals who have participated in another clinical trial recently.
Individuals who have allergies or reactions to the study medication.
Individuals who are taking medications that might interfere with the study.
Individuals who have a history of substance abuse or alcohol dependency.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Amsterdam UMC
Amsterdam, The Netherlands
Verified
CHU Grenoble Alpes
La Tronche, France
Verified
Centre Hospitalier Universitaire De Lille
Lille, France

Trial locations

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Investigated drugs

NTLA-2002 is a medication being studied for its safety and effectiveness in treating adults with Hereditary Angioedema (HAE). The trial aims to determine how well NTLA-2002 can prevent or reduce the frequency of HAE attacks. The study also seeks to find the most appropriate dose for future use.

What is already known about the treatment

NTLA-2002 – This medication is administered through an injection and is currently being studied in clinical trials for its safety and effectiveness. It is primarily being investigated for the treatment of Hereditary Angioedema, a condition characterized by recurrent episodes of severe swelling. NTLA-2002 works by targeting specific genetic components to reduce the frequency and severity of these swelling attacks. It falls under the category of gene-editing therapies, aiming to provide a long-term solution by modifying the underlying genetic causes of the disease.

Investigated diseases

Hereditary Angioedema – Hereditary Angioedema is a rare genetic disorder characterized by recurrent episodes of severe swelling, known as angioedema. This swelling can affect various parts of the body, including the face, extremities, gastrointestinal tract, 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 painful and may last for several days. The frequency and severity of attacks can vary widely among individuals. It is important to manage the condition to prevent complications associated with swelling, especially in the airway.