NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease

Phase 1

Recruiting

• Conditions: Lung Disease; Pulmonary Disease; AATD; Alpha-1 Antitrypsin Deficiency; Alpha-1 Antitrypsin Deficiency-associated Lung Disease
• Interventions: Biological: Biological NTLA-3001

• Registration Number: NCT06622668
• Lead Sponsor: Intellia Therapeutics

Brief Summary

This study will be conducted to evaluate the safety, tolerability, clinical activity, pharmacokinetics, and pharmacodynamics of NTLA-3001 in adults with alpha-1 antitrypsin deficiency (AATD) -associated lung disease

Detailed Description

This study consists of 2 parts: Phase 1 is an open-label, single-arm ascending dose study to characterize the safety and activity of NTLA3001 and identify the dose for evaluation in Phase 2. Phase 2 will follow as an open-label, dose expansion study to further characterize the safety and clinical activity of NTLA-3001 and provide an initial assessment of the effect of NTLA-3001 on clinical measures of pulmonary function.

Study Timeline

• Study First Submitted: 2024-09-30
• Study First Submitted QC: 2024-09-30
• Status Verified: 2024-10
• Study Start: 2024-10
• Study First Posted: 2024-10-02
• Last Update Submitted: 2024-10-28
• Last Update Posted: 2024-10-30
• Primary Completion: 2030-03
• Study Completion: 2030-05

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

Not provided

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Exclusion Criteria

1. AATD genotypes outside of ZZ or ZNull
2. Participants with total antibodies to adeno-associated virus (AAV) serotype above laboratory assay cut off
3. Participants who have known negative reaction or hypersensitivity to any lipid nanoparticles (LNP) component.
4. Any condition that, in the Investigator's opinion, could adversely affect the safety of the participant.
5. Unwilling to comply with study procedures.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Arm 1	Biological NTLA-3001	-

Primary Outcome Measures

Name	Time	Method
Safety and Tolerability	From NTLA-3001 infusion up to week156 post infusion	To evaluate the safety and tolerability of NTLA-3001 as determined by adverse events (AEs) and dose limiting toxicities (DLTs)

Secondary Outcome Measures

Name	Time	Method
Pharmacodynamics	From NTLA-3001 infusion up to week 156 post infusion	To evaluate the PD effect of NTLA-3001
Immune Response	From NTLA-3001 infusion up to week 156 post infusion	To evaluate the immune response to NTLA-3001
Vector Shedding	From NTLA-3001 infusion up to week 156 post infusion, only during phase 2.	To evaluate vector shedding following administration of NTLA-3001

Trial Locations

Locations (1)

New Zealand Clinical Research; 🇳🇿 Aukland, New Zealand