TSRA196-AAT-201: A Phase 1/2, Open-Label, Multi-Center, Dose Escalation, Dose Expansion, and Single Repeat Dose Study of TSRA-196 in Adults With the PiZZ Genotype Who Have Lung and/or Liver Disease Associated with Severe Alpha-1 Antitrypsin Deficiency

Lung and/or Liver Disease Associated with Alpha-1 Antitrypsin Deficiency (AATD)

Part 1: Incidence of TEAEs and SAEs Part 2: Proportion of participants who have serum levels of total AAT ≥LLN at Month 3 and Month 6 after TSRA-196 treatment Part 3: Proportion of participants who have serum levels of total AAT ≥LLN at Month 3 and Month 6 after a second dose of TSRA-196 Part 2 and 3: Change from baseline in functional AAT concentrations (determined using an elastase inhibition assay) to Month 12

Part 1: Proportion of participants who have serum levels of total AAT ≥LLN: • at Month 3 and Month 6 after TSRA-196 treatment • from Month 6 through Month 12 after TSRA-196 treatment • from Month 3 through Month 12 after TSRA-196 treatment, Part 1: Proportion of participants who have serum levels of total AAT ≥11 μM: • at Month 3 and Month 6 after TSRA-196 treatment • from Month 6 through Month 12 after TSRA-196 treatment • from Month 3 through Month 12 after TSRA-196 treatment, Part 1 and 2 and 3: Change in serum levels of total AAT from baseline over time, Part 1 and 2 and 3: Incidence of AESI from day of dosing through EOS/ET, Part 1 and 2 and 3: Change in clinical laboratory parameters, vital signs, and ECG parameters over time, Part 1 and 2 and 3: PK parameters for • ionizable lipid in blood • DMG-PEG2000 in blood • mRNA in blood • tgRNA in blood, Part 2 and 3: Incidence of TEAEs and SAEs, Part2: Proportion of participants who have serum levels of total AAT ≥LLN • from Month 6 through Month 12 after TSRA-196 treatment • from Month 3 through Month 12 after TSRA-196 treatment, Part 2: Proportion of participants who have serum levels of total AAT ≥11 μM: • at Month 3 and Month 6 after TSRA-196 treatment • from Month 6 through Month 12 after TSRA-196 treatment • from Month 3 through Month 12 after TSRA-196 treatment, Part 3: Proportion of participants who have serum levels of total AAT ≥LLN: • from Month 6 through Month 12 after a second dose of TSRA-196 • from Month 3 through Month 12 after a second dose of TSRA-196, Part 3: Proportion of participants who have serum levels of total AAT ≥11 μM: • at Month 3 and Month 6 after a second dose of TSRA-196 • from Month 6 through Month 12 after a second dose of TSRA-196 • from Month 3 through Month 12 after a second dose of TSRA-196, Part 2 and 3: Change from baseline in functional AAT concentrations (determined using an elastase inhibition assay) to Month 3 and to Month 6, Part 2 and 3: Change from baseline in post-bronchodilator ppFEV1 through Month 12, Part 2 and 3: Incidence of COPD exacerbations from baseline over time

No related papers found yet.