RecruitingRare disease

Long-Term Safety Study of NTLA-2002 for Patients with Hereditary Angioedema, Using Messenger RNA Encoding Cas9 and HU-G012267

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 the long-term follow-up of individuals who have been treated with a medication called NTLA-2002. The study is specifically looking at people with a condition known as Hereditary Angioedema, which is a rare genetic disorder that causes sudden and severe swelling in different parts of the body. The treatment being studied, NTLA-2002, is a solution for infusion that contains messenger RNA, a type of genetic material, which is designed to target the kallikrein B1 gene (KLKB1). This gene is involved in the process that leads to the symptoms of Hereditary Angioedema.

The purpose of this study is to evaluate the long-term safety of NTLA-2002 in individuals who have previously received this treatment. Participants in the study will be monitored over an extended period to observe any potential side effects or adverse events related to the treatment. The study will also track the frequency of Hereditary Angioedema attacks and any changes in the use of medications needed to manage these attacks. Additionally, the study will assess changes in the overall quality of life for participants, using specific tools designed to measure this aspect.

Throughout the study, participants will attend regular visits to ensure their health and safety while collecting important data about the treatment's effects. The study aims to provide valuable information about the long-term impact of NTLA-2002 on individuals with Hereditary Angioedema, contributing to a better understanding of how this treatment can be used effectively and safely in the future.

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
Joining the study
Participation begins after completing or discontinuing a previous treatment protocol involving NTLA-2002.
Informed consent is required to participate in the long-term follow-up study.
Commitment to attend study visits and comply with follow-up schedules is necessary.
Step 2
Treatment administration
The study involves monitoring after receiving NTLA-2002, a solution for infusion administered through intravenous use.
NTLA-2002 contains messenger RNA encoding cas9, hu-g012267.
Step 3
Monitoring and follow-up
The main objective is to evaluate the long-term safety of NTLA-2002.
Monitoring includes tracking the incidence of treatment-related adverse events (AEs), serious adverse events (SAEs), and adverse events of special interest (AESIs).
Step 4
Assessment of hereditary angioedema (HAE) attacks
The study assesses the rate of HAE attacks and those requiring acute therapy.
Changes in the consumption of on-demand HAE medications and healthcare utilization for HAE attacks are evaluated.
Step 5
Quality of life evaluation
Quality of life (QoL) parameters are measured using specific instruments such as the MOXIE Angioedema QoL instrument, EQ-5D-5L, and WPAI:GH.
Changes from baseline in these QoL parameters are assessed.
Step 6
Study duration
The estimated end date for the study is February 15, 2038.
The recruitment for the study started on February 15, 2023.

Who can join the trial?

5 criteria

The patient must have completed or stopped participating in a treatment study supported by Intellia, where they received a full or partial dose of **NTLA-2002**.
The patient must have given their agreement to participate in the **LTFU study**, which means they understand the study and agree to be part of it.
The patient must be willing to attend study visits, follow the schedule required by the study, and meet all the study requirements.
The study is open to both male and female patients.
The study includes patients from vulnerable populations, which means it considers people who might need extra care or protection.

Who cannot join the trial?

3 criteria

Patients who have not been previously treated for **Hereditary Angioedema** cannot participate. **Hereditary Angioedema** is a condition that causes sudden swelling in different parts of the body.
Patients who are not within the specified age range cannot participate. The age range includes certain age groups, but the exact ages are not specified here.
Patients who belong to a vulnerable population may not be eligible. A vulnerable population includes groups of people who might need special protection or care.

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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 long-term safety in patients who have already been treated with it. This trial aims to monitor any potential side effects or health changes over an extended period after the initial treatment. The focus is on ensuring that the medication remains safe for use in the long term.

What is already known about the treatment

NTLA-2002 – This medication is administered through an intravenous infusion, allowing it to be delivered directly into the bloodstream. Currently, NTLA-2002 is under investigation in clinical trials, with a focus on its long-term safety for individuals who have previously received treatment. It is primarily being studied for its potential to treat hereditary angioedema, a condition characterized by sudden swelling attacks. The medication works by targeting specific genetic components to reduce the frequency and severity of these swelling episodes. NTLA-2002 falls under the category of gene-editing therapies, which aim to modify genetic material to achieve therapeutic effects.

Investigated diseases

Hereditary Angioedema – Hereditary Angioedema is a rare 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.