Long-Term Follow-Up of Subjects Treated With NTLA-2001

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

6 May 2024 → ongoing

Decision date (initial)

2024-10-18

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Intellia Therapeutics, Inc.

External identifiers

EU CT number

2023-507385-11-00

EudraCT number

2022-003405-30

WHO UTN

U1111-1296-5487

ClinicalTrials.gov

NCT05697861

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy, Pharmacodynamic

To evaluate the long-term safety of NTLA-2001 in previously treated participants

Secondary objectives 1

1. To evaluate long-term pharmacodynamic effects of NTLA-2001 in previously treated participants

Conditions and MedDRA coding

-Hereditary Transthyretin Amyloidosis with Polyneuropathy (ATTRv-PN) -Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. 1. A participant has completed or discontinued from an Intellia-sponsored clinical study in which a complete or partial dose of NTLA-2001 was received.
2. 2. A participant has provided informed consent for the LTFU study.
3. 3. A participant is willing to attend study visits, complete protocol-required follow-up schedule, and comply with the study requirements.
4. 4. Male participants must agree to the following until 4 months after the last administration of study intervention in the parent study: a. Refrain from donating sperm. The time frame may be extended beyond 4 months if sperm donation is contraindicated based on country-specific guidelines. AND b. Use a male condom, in addition to the female partner using a highly effective contraceptive method with a failure rate of < 1% per year, when having sexual intercourse with a WOCBP (Section 10.3.1).
5. 5. Female participants must agree to the following until 7 months after the last administration of study intervention in the parent study: a. Use a protocol-specified highly effective method of contraception, if a WOCBP. This is not required for female participants who are postmenopausal or surgically sterile, as defined in Section 10.3.1. AND b. Not undergo oocyte retrieval for in vitro fertilization for at least 7 months after the last administration of study intervention. The time frame may be extended beyond 7 months if oocyte retrieval is contraindicated based on country-specific guidelines.

Exclusion criteria 1

1. None.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. For ATTR-CM and ATTRv-PN participants, incidence of: • Treatment-related SAEs • Protocol-specified AESIs

Secondary endpoints 1

1. PD biomarkers for ATTR over time: • Serum TTR • Serum prealbumin

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

02139-4254

Country

United States

Scientific contact point

Organisation

Intellia Therapeutics Inc.

Contact name

Trial Manager at Intellia

Public contact point

Organisation

Intellia Therapeutics Inc.

Contact name

Trial Manager at Intellia

Third parties 2

Locations

2 EU/EEA countries · 2 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 24 files

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

Application history

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