Badanie bezpieczeństwa i skuteczności TSRA-196 (rnaivt9315, rnacs24757) u dorosłych z ciężkim niedoborem alfa‑1‑antytrypsyny i chorobą płuc lub wątroby

Baseline Assessment

After enrollment, a comprehensive baseline Assessment is performed. This includes medical history, physical Examination, blood sampling to determine current levels of alpha‑1 antitrypsin (AAT), and lung function testing. The purpose is to establish reference values before any study medication is given.

First intravenous infusion of <b>tsra-196</b>

A single dose of tsra-196 is administered by intravenous infusion. the medication is supplied as a solution for infusion; the exact amount is defined by the study protocol and is given only once in this part of the trial.

The infusion is performed in a clinical setting under medical supervision. Vital signs and safety parameters are monitored during and for several hours after the infusion to detect any immediate adverse reactions.

Early safety monitoring

Following the infusion, the participant remains in the clinic for observation. Routine checks of blood pressure, heart rate, and laboratory tests are repeated to identify any early safety concerns.

First follow‑up visit – month 3

Approximately three months after the infusion, a follow‑up visit is scheduled. Blood is drawn to measure total AAT levels and functional AAT activity (elastase inhibition assay). lung function (post‑bronchodilator ppfev1) may also be assessed. The visit includes evaluation of any adverse events that occurred since the infusion.

Second follow‑up visit – month 6

At six months, another visit is conducted with the same assessments as at month 3. the participant’s serum AAT concentration is checked to determine whether it remains at or above the lower limit of normal (lln) and whether it reaches the target of 11 µM. safety monitoring continues throughout this period.

Optional second intravenous infusion of <b>tsra-196</b>

Participants who meet the study Criteria may receive a second dose of tsra-196 after the month‑6 assessment. This dose is also given as a single intravenous infusion following the same procedure as the first infusion.

Post‑second‑dose monitoring

After the second infusion, immediate safety monitoring identical to the first infusion is performed. The participant stays in the clinic for observation and early laboratory checks.

Long‑term follow‑up – month 12

The final scheduled visit occurs at twelve months from the first infusion (or from the second infusion, if applicable). comprehensive blood tests evaluate total and functional AAT levels over the entire study period. Lung function, clinical laboratory parameters, vital signs, and electrocardiogram (ecg) results are reviewed. Any adverse events that occurred at any time are recorded.