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

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What is this study about?

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.

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.

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.

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).

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.

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.

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 Study?

  • 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 Study?

  • 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.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Centre Hospitalier Universitaire De Lille Lille France
CHU Grenoble Alpes La Tronche France

Other Sites

Site Name City Country Status
Hopitaux Universitaires Pitie Salpetriere Paris France
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Aqopmwqjn Uur Amsterdam The Netherlands

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
France France
Recruiting
15.02.2023
Germany Germany
Not yet recruiting
15.02.2023
The Netherlands The Netherlands
Recruiting
15.02.2023

Trial locations

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.

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.

Trial ID:
2023-507956-56-00
Protocol code:
ITL-2002-CL-999
Trial Phase:
Human Pharmacology (Phase I) – Other

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