Clinical Trial

Disease: Alpha-1 Antitrypsin Deficiency, AATD, (NCT06622668)

Disease info:

Alpha-1 antitrypsin deficiency (AATD) is an inherited genetic disorder that is most commonly caused by a G-to-A point mutation in the SERPINA1 gene. This mutation results in the production of insufficient levels of circulating M-AAT protein, which protects the lungs from proteolytic enzymes, and aggregation of misfolded Z-AAT protein in hepatocytes. This leads to lung and liver disease. Individuals with AATD carry Z mutations on both SERPINA1 alleles; this is known as the PiZZ genotype.

Augmentation therapy via delivery of functional AAT protein is currently the only treatment option for AATD-associated lung disease and requires weekly intravenous infusions. There are no treatments for AATD-associated liver disease, besides liver transplantation.

Frequency:
There are approximately 200,000 patients in the United States and Europe who have Z mutations on both alleles.
Official 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
Who:

Contact

Phone: 1-857-285-6200 ext 6

Email: medicalinformation@intelliatx.com

Partners:
Locations:
Study start:
Oct. 1, 2024
Enrollment:
30 participants
Gene editing method:
CRISPR-Cas9
Type of edit:
Gene insertion
Gene:
SERPINA1
Delivery method:
Lipid-based nanoparticle (LNP) - In-vivo
IndicatorIndicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Not yet recruiting

Description

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

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.

Last updated: Nov. 2, 2024
close
Search CRISPR Medicine