A Phase 1/2 Study of NTLA-3001 in AATD-Associated Lung Disease
Research type
Research Study
Full title
Phase 1/2 Multicenter, Open-label Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-3001 in Participants with Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease
IRAS ID
1009607
Contact name
Peggy Sung
Contact email
Sponsor organisation
Intellia Therapeutics, Inc.
Eudract number
2023-508138-33
Clinicaltrials.gov Identifier
Research summary
Alpha-1 antitrypsin deficiency (AATD) is a rare, inherited disorder which can lead to disability and sometimes death. It is caused by mutations in a gene called SERPINA1, which produces a protein called alpha-1 antitrypsin (AAT; also known as A1AT). AAT blocks the activity of a proteolytic enzyme (a “protein cutter”) called neutrophil elastase (NE). AAT works to reduce the harm to healthy lung tissue that is caused when white blood cells (part of your immune system) release powerful enzymes to fight infection (and other harmful substances). In AATD patients, who do not make enough functional AAT, this enzymatic damage can lead to early onset of a lung disease that closely resembles chronic obstructive pulmonary disease (COPD).
NTLA-3001 is a one-time administered investigational treatment for AATD-associated lung disease that works by providing the patients with a functioning version of the mutated gene, so they begin to produce the correct AAT protein.
This clinical study has two parts (Phase 1 and Phase 2). Participants in the United Kingdom will take part in both Phase 1 and Phase 2 of this study:
In Phase 1:
In this part of the study, up to 18 participants will be recruited to receive an increasing dose of the study drug, with the aim of identifying a safe and effective dose for the Phase 2 part of the study.In Phase 2:
In this phase of the study, up to 12 additional participants will be enrolled to receive the dose level selected from the Phase 1 part. The aim of this part of the study is to further understand the effectiveness of the drug on the participants’ pulmonary function.REC name
North East - York Research Ethics Committee
REC reference
24/NE/0105
Date of REC Opinion
23 Jul 2024
REC opinion
Further Information Favourable Opinion