Not recruitingRare disease

Study on NTLA-2002 for Treating Hereditary Angioedema in Patients: Evaluating the Effects of Ziclumeran and Lonvoguran

Verified siteRegistered drugNo 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 Hereditary Angioedema (HAE), a rare genetic condition that causes sudden swelling in various parts of the body. The study will evaluate a new treatment called NTLA-2002, which is a special type of medicine known as an advanced therapy medical product. This treatment involves a dispersion for infusion, which means it is given through a drip directly into the bloodstream. The active substances in NTLA-2002 are ziclumeran and lonvoguran, which are designed to target specific genetic components involved in HAE.

The purpose of the study is to assess how effective NTLA-2002 is in reducing the number of HAE attacks compared to a placebo. Participants in the study will receive either NTLA-2002 or a placebo through an intravenous infusion. The study will monitor participants over several weeks to see how the treatment affects the frequency and severity of their HAE attacks. The trial will also look at the overall quality of life for participants during the study period.

Throughout the study, participants will be closely observed to ensure their safety and to gather information on how well the treatment works. The study aims to provide valuable insights into the potential benefits of NTLA-2002 for people living with hereditary angioedema, offering hope for a new treatment option for this challenging condition.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Run-in period
The run-in period lasts for 8 weeks. During this time, at least 2 hereditary angioedema (HAE) attacks must be documented by an investigator.
If 2 attacks occur within the first 28 days, randomization may occur after Day 28, provided all eligibility criteria are met.
Step 2
Randomization
Participants are randomly assigned to receive either the study medication NTLA-2002 or a placebo.
The study is double-blind, meaning neither the participant nor the investigator knows which treatment is being administered.
Step 3
Treatment administration
The study medication NTLA-2002 is administered via intravenous infusion.
The infusion is a dispersion, which means the medication is mixed in a liquid for administration.
Step 4
Primary observation period
This period lasts from Week 5 through Week 28.
The main objective is to evaluate the effectiveness of NTLA-2002 by measuring the number of HAE attacks compared to the placebo.
Step 5
Secondary observation period
During this time, the number of HAE attacks requiring on-demand treatment is recorded.
The severity of attacks and any changes in quality of life are also assessed.
Step 6
End of study
The study is estimated to end on September 22, 2027.
Participants will have completed all required assessments and observations by this time.

Who can join the trial?

12 criteria

Participants must be at least 18 years old when they sign the consent form.
Participants must have a clinical history of **HAE** (Hereditary Angioedema), which involves repeated episodes of swelling under the skin or in the mucous membranes, without hives. They must also meet specific laboratory criteria related to **C1-INH** (C1 esterase inhibitor) levels.
Participants must have experienced at least 2 confirmed **HAE attacks** during an 8-week period before the study starts.
Participants must agree not to use long-term preventive treatments for HAE during the study period, unless it is confirmed safe by the study doctor.
Participants must have access to and be able to use medication to treat angioedema attacks when they occur.
Participants must meet certain laboratory test criteria, including liver function tests and kidney function tests.
Male participants with partners who can become pregnant must agree to use a condom during the study and for 4 months after the last study treatment.
Male participants must agree not to donate sperm during the study and for 4 months after the last study treatment.
Female participants who can become pregnant must use a highly effective method of birth control during the study and for 7 months after the last study treatment. This does not apply to women who are postmenopausal or have had certain surgeries that prevent pregnancy.
Female participants must agree not to undergo egg retrieval for in vitro fertilization during the study and for 7 months after the last study treatment.
Participants must be able to provide signed consent to participate in the study.
Participants must agree not to join another study that involves treatment during this study.

Who cannot join the trial?

5 criteria

Patients who do not have **hereditary angioedema** due to **C1 esterase inhibitor deficiency** (Type 1 or 2) cannot participate. **Hereditary angioedema** is a condition that causes sudden swelling, and **C1 esterase inhibitor deficiency** is a specific cause of this condition.
Patients who are not within the specified age range for the study cannot participate. The age range is not specified here, but it is important for eligibility.
Patients who are not part of the specified clinical trial group cannot participate. The specific group is not detailed here, but it is a requirement for the study.
Patients who are not male or female cannot participate, as the study includes both genders.
Patients who are not considered part of a vulnerable population cannot participate. A **vulnerable population** includes groups that may need special protection or consideration in research.

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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
Assistance Publique Hopitaux De Marseille
Marseille, France
Verified
CHU Grenoble Alpes
La Tronche, France

Trial locations

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Investigated drugs

NTLA-2002 is a medication being studied for its effectiveness in treating hereditary angioedema (HAE). This condition causes sudden and severe swelling in different parts of the body. The trial aims to see how well NTLA-2002 can reduce the number of HAE attacks over a specific period.

What is already known about the treatment

NTLA-2002 – This medication is administered through an intravenous infusion. It is currently being studied in clinical trials for its effectiveness in treating hereditary angioedema, a condition caused by a deficiency in the C1 esterase inhibitor. The main therapeutic indication is to reduce the frequency of HAE attacks. NTLA-2002 works at the molecular level by using CRISPR technology to edit genes, aiming to correct the underlying genetic defect. It is classified as a gene-editing therapy.

Investigated diseases

Hereditary Angioedema due to C1 Esterase Inhibitor Deficiency (Type 1 or 2) – This is a genetic disorder characterized by recurrent episodes of severe swelling, known as angioedema. The swelling can affect various parts of the body, including the limbs, face, intestinal tract, and airway. It is caused by a deficiency or dysfunction of the C1 esterase inhibitor protein, which plays a role in regulating inflammation and fluid balance. During an attack, the swelling can be painful and may last for several days. The frequency and severity of attacks can vary widely among individuals. This condition is inherited in an autosomal dominant pattern, meaning a single copy of the altered gene in each cell is sufficient to cause the disorder.