A Study of CTX460 in Adults with Emphysema Associated with Severe Alpha-1 Antitrypsin Deficiency (AATD).
Age: ≥18 and ≤75 years.
Able to provide written informed consent.
Diagnosis of AATD with PiZZ homozygous genotype.
Serum total AAT level <11µM in the absence of augmentation.
Received augmentation therapy within the 6 weeks prior to CTX460 infusion
Lung or liver transplant or on waiting list for lung or liver transplant
Moderate to severe lung disease other than COPD that may confound results as per investigator discretion
Evidence of significant liver fibrosis/cirrhosis
Sequential
CTX460
DrugCTX460 is a lipid nanoparticle (LNP) formulation of synthetic nucleotide template polymerase (SyNTase) editor for in vivo gene editing of the serine protease inhibitor A1 (SERPINA1) gene in adults with emphysema associated with severe alpha-1 antitrypsin deficiency (AATD).
Phase 1: To evaluate the safety and tolerability of CTX460 in adults with emphysema associated with severe AATD and to determine the recommended Phase 2 dose(s)
Incidence of DLTs and frequency of AEs
Phase 2: To assess the efficacy of CTX460 in adults with severe AATD and emphysema
Absolute blood levels of total AAT at Month 6
Phase 1: To assess the preliminary efficacy of CTX460
Absolute blood levels of total AAT at Month 6
Phase 2: To further characterize the safety of CTX460
Frequency and severity of clinically significant laboratory abnormalities, and clinically significant abnormal vital signs
Phase 1/2: To assess the efficacy of CTX460
Rate of change in lung density (g/L per year) as measured by centralized, standardized CT lung densitometry
Phase 1/2: To assess the efficacy of CTX460
Time to first moderate or severe COPD exacerbation
Click on the lead sponsor to view all of their trials.
This trial is not recruiting at the moment. You can still explore other options:
