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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

Status
Completed
Phases
Phase 1Phase 2
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2023-508138-33-00
Acronym
ITL-3001-CL-101
Enrollment
4
Registered
2024-11-18
Start date
Unknown
Completion date
2025-01-09
Last updated
2024-11-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pulmonary emphysema

Brief summary

1. Treatment-emergent adverse events (TEAE).

Detailed description

1. PD. Circulating alpha-1 antitrypsin (AAT) protein., 2. PK. Plasma concentration-time profiles and PK parameters of the components of the study intervention., 3. Immunogenicity. Total antibodies (TAb) and neutralizing antibodies (NAb) to AAV., 4. Immunogenicity. Antibodies to AAT protein., 5. Immunogenicity. Anti-drug antibodies to LNP., 6. Immunogenicity. Anti-Cas9 protein antibodies., 7. Shedding (Phase 2 only). AAV VGCN per cell in blood, urine, saliva, and semen samples., 8. Exploratory. St George’s Respiratory Questionnaire & 36-item short form health survey questionnaire (SF-36).

Interventions

DRUGSERPINA1(human)-AAV8-1 DP
DRUGLNP1265 DP

Sponsors

Intellia Therapeutics Inc.
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
All
Age
18 Years to No maximum

Design outcomes

Primary

MeasureTime frame
1. Treatment-emergent adverse events (TEAE).—

Secondary

MeasureTime frame
1. PD. Circulating alpha-1 antitrypsin (AAT) protein., 2. PK. Plasma concentration-time profiles and PK parameters of the components of the study intervention., 3. Immunogenicity. Total antibodies (TAb) and neutralizing antibodies (NAb) to AAV., 4. Immunogenicity. Antibodies to AAT protein., 5. Immunogenicity. Anti-drug antibodies to LNP., 6. Immunogenicity. Anti-Cas9 protein antibodies., 7. Shedding (Phase 2 only). AAV VGCN per cell in blood, urine, saliva, and semen samples., 8. Exploratory. St George’s Respiratory Questionnaire & 36-item short form health survey questionnaire (SF-36).—

Countries

Ireland

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026