Hereditary AngioedemaNCT06262399
Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002
An observational study of Hereditary Angioedema, run by Intellia Therapeutics at 7 sites in 6 countries.
Trial summary
This is a follow-up study of subjects who received NTLA-2002 in a previous clinical trial as an observational evaluation of the long-term effects of the investigational therapy. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hereditary Angioedema
- How many people
- 100 participants
- Where
- 7 sitesin 6 countries
- Study dates
- 12 Apr 2024to Apr 2040
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Participating sites in an Intellia-sponsored clinical study of NTLA-2002.
Who may be able to join
- A subject has completed or discontinued from an Intellia-sponsored or -supported treatment protocol in which a complete or partial dose of NTLA-2002 was received.
- A subject has provided informed consent for the LTFU study.
- A subject is willing to attend study visits, complete protocol-required follow-up schedule, and comply with the study requirements.
Who cannot join
- None
Trial locations and contacts
7 sites in 6 countries. Contact the study team or a site near you to ask about taking part.
7 sites in 6 countries
France2 sites
Centre National de Reference - Grenoble
Grenoble
Hôpital Claude Huriez
Lille, 59037
Netherlands1 site
University of Amsterdam Academic Medical Center
Amsterdam
United Kingdom1 site
Cambridge University Hospitals NHS Foundation Trust, Addenbrooke's Hospital
Cambridge
About this study
Official title: Long-Term Follow-Up of Subjects Treated With NTLA 2002
This is a follow-up study of subjects who received NTLA-2002 in a previous clinical trial as an observational evaluation of the long-term effects of the investigational therapy.
- Conditions
- Hereditary Angioedema
- Medical subject headings
- Angioedemas, Hereditary
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-related Adverse Events (AEs); incidence of treatment-related Serious Adverse Events (SAEs); incidence of treatment-related Adverse Events of Special Interest (AESIs) defined per protocol
Measured: up to 15 years
Secondary outcomes (6)
To evaluate the long-term efficacy of NTLA-2002 in previously treated subjects
Rate of Hereditary Angioedema (HAE) attacks overall and rate of HAE attacks requiring acute therapy. Total plasma kallikrein protein level.
Measured: up to 15 years
Change from baseline in consumption of on-demand HAE medications for reported HAE attacks
Measured: up to 15 years
Change from baseline in healthcare utilization for HAE attacks
Measured: up to 15 years
Change from baseline in QoL parameters as measured by the MOXIE Angioedema-QoL instrument.
Measured: up to 5 years
Change from baseline in QoL parameters as measured by the EQ-5D-5L instrument.
Measured: up to 5 years
Change from baseline in QoL parameters as measured by the WPAI:GH instrument.
Measured: up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Feb 2024
- Study start12 Apr 2024
- Primary completionApr 2040 (estimated)
- Study completionApr 2040 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary AngioedemaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Intellia Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06262399
- Sponsor's protocol ID
- ITL-2002-CL-999
- Other IDs
- 2022-003778-22
- Status last verified
- Apr 2025
- First submitted
- 12 Jan 2024
- Last updated
- 28 Nov 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-Term Follow-Up of Subjects Treated With NTLA 2002
- ClinicalTrials.gov ID
- NCT06262399
- Acronym / sponsor ID
- ITL-2002-CL-999
- Conditions
- Hereditary Angioedema
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Intellia TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 12 Apr 2024 (actual)
- Primary completion
- Apr 2040 (estimated)
- Study completion
- Apr 2040 (estimated)
- First posted
- 16 Feb 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a follow-up study of subjects who received NTLA-2002 in a previous clinical trial as an observational evaluation of the long-term effects of the investigational therapy.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participating sites in an Intellia-sponsored clinical study of NTLA-2002.
Key inclusion criteria (3 listed)
- A subject has completed or discontinued from an Intellia-sponsored or -supported treatment protocol in which a complete or partial dose of NTLA-2002 was received.
- A subject has provided informed consent for the LTFU study.
- A subject is willing to attend study visits, complete protocol-required follow-up schedule, and comply with the study requirements.
Key exclusion criteria (1 listed)
- None
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Incidence of treatment-related Adverse Events (AEs); incidence of treatment-related Serious Adverse Events (SAEs); incidence of treatment-related Adverse Events of Special Interest (AESIs) defined per protocol
Time frame: up to 15 years
6 secondary outcomes
To evaluate the long-term efficacy of NTLA-2002 in previously treated subjects
Time frame: up to 15 years
Change from baseline in consumption of on-demand HAE medications for reported HAE attacks
Time frame: up to 15 years
Change from baseline in healthcare utilization for HAE attacks
Time frame: up to 15 years
Change from baseline in QoL parameters as measured by the MOXIE Angioedema-QoL instrument.
Time frame: up to 5 years
Change from baseline in QoL parameters as measured by the EQ-5D-5L instrument.
Time frame: up to 5 years
Change from baseline in QoL parameters as measured by the WPAI:GH instrument.
Time frame: up to 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Nov 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
The record lists no contact. Use the official record to reach the sponsor.
7 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hereditary Angioedema, run by Intellia Therapeutics at 7 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 28 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in France, Australia, Germany, Netherlands, New Zealand and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-related Adverse Events (AEs); incidence of treatment-related Serious Adverse Events (SAEs); incidence of treatment-related Adverse Events of Special Interest (AESIs) defined per protocol (time frame: up to 15 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Nov 2025.