Long-Term Follow-Up of Subjects Treated With NTLA-2002

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Intellia Therapeutics Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

17 May 2024 → ongoing

Decision date (initial)

2023-11-03

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Intellia Therapeutics, Inc.

External identifiers

EU CT number

2023-507956-56-00

EudraCT number

2022-003778-22

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety

To evaluate the long-term safety of NTLA-2002 in previously treated participants

Secondary objectives 3

1. To evaluate the long-term efficacy of NTLA-2002 in previously treated participants
2. To evaluate disease-related outcomes of NTLA-2002 in previously treated participants
3. To evaluate patient-reported outcomes of NTLA-2002 in previously treated participants

Conditions and MedDRA coding

Hereditary Angioedema

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. A participant has completed or discontinued from an Intellia-sponsored or -supported treatment protocol in which a complete or partial dose of NTLA-2002 was received.
2. Participants ≥ 18 years of age, emancipated minors, or legal guardians of participants < 18 years of age must provide informed consent. In addition, participants ≥ 7 to < 18 years of age (or as determined by local regulations), whose legal guardian provides informed consent, must be willing and able to read, understand, and sign an assent form.
3. A participant is willing to attend study visits, complete protocol-required follow-up schedule, and comply with the study requirements.

Exclusion criteria 1

1. Not applicable

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. Incidence of treatment-related AEs;
2. incidence of treatment-related SAEs
3. incidence of treatment-related AESIs defined per protocol

Secondary endpoints 5

1. - Rate of HAE attacks overall and rate of HAE attacks requiring acute therapy
2. - Total plasma kallikrein protein level
3. - Change from baseline in consumption of on demand HAE medications for attacks
4. - Change from baseline in healthcare utilization for HAE attacks
5. - Change from baseline in QoL parameters as measured by the MOXIE Angioedema QoL instrument, EQ-5D-5L, WPAI:GH, and WPAI:HAE

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Intellia Therapeutics Inc.

Sponsor organisation

Intellia Therapeutics Inc.

Address

40 Erie Street

City

Cambridge

Postcode

02139

Country

United States

Scientific contact point

Organisation

Intellia Therapeutics Inc.

Contact name

Clinical Program Management

Public contact point

Organisation

Intellia Therapeutics Inc.

Contact name

Clinical Program Management

Third parties 3

Locations

3 EU/EEA countries · 5 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 70 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

8 submissions — initial application plus substantial / non-substantial modifications