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

NCT06622668 · Status: WITHDRAWN · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL

Last updated 2025-01-17

No results posted yet for this study

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

Conditions

  • Lung Disease
  • Pulmonary Disease
  • AATD
  • Alpha-1 Antitrypsin Deficiency
  • Alpha-1 Antitrypsin Deficiency-associated Lung Disease

Interventions

BIOLOGICAL

Biological NTLA-3001

IV administration of AAV and CRISPR/Cas9 gene editing system

Sponsors & Collaborators

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-11-20
Primary Completion
2025-01-09
Completion
2025-01-09

Countries

  • New Zealand

More Related Trials

Entities

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06622668 on ClinicalTrials.gov