NTLA-3001 in Adults with Alpha-1 Antitrypsin Deficiency-Associated Lung Disease
NCT ID: NCT06622668
Last Updated: 2025-01-17
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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WITHDRAWN
PHASE1/PHASE2
INTERVENTIONAL
2024-11-20
2025-01-09
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Arm 1
Biological NTLA-3001
IV administration of AAV and CRISPR/Cas9 gene editing system
Interventions
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Biological NTLA-3001
IV administration of AAV and CRISPR/Cas9 gene editing system
Eligibility Criteria
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Inclusion Criteria
2. Diagnosis of AATD ZZ/ZNull genotypes
3. FEV1 ≥35% and ≤65%
4. No evidence of liver cirrhosis
5. Adequate chemistry and hematology measures at screening
6. Participants must agree not to participate in another interventional study for the duration of this trial.
7. Participants must be capable of providing signed informed consent
Exclusion Criteria
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.
18 Years
75 Years
ALL
No
Sponsors
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Intellia Therapeutics
INDUSTRY
Responsible Party
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Locations
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New Zealand Clinical Research
Aukland, , New Zealand
Countries
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Other Identifiers
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ITL-3001-CL-101
Identifier Type: -
Identifier Source: org_study_id
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