A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-70
SponsorTessera Therapeutics, Inc.
About this trial
This is a Phase 1/2, open-label, multi-center, dose escalation (Part 1), dose expansion (Part 2), and single repeat dose (Part 3) study to evaluate the safety, tolerability, efficacy, and PK/PD parameters of TSRA-196 in adults with the PiZZ genotype who have lung and/or liver disease associated with severe alpha-1 antitrypsin deficiency (AATD)
Eligibility criteria
Qualifiers
Males or females who are 18 to 70 years of age, inclusive, at the time of signing the informed consent
Body mass index of 18 to 37 kg/m2, inclusive
Confirmed diagnosis of AATD and PiZZ genotype
At least one previous measure of blood total AAT level <11 µmol/L
Disqualifiers
Presence of genetic variation in SERPINA1 gene that may disrupt the function of TSRA-196, determined by screening genotyping
History of liver disease unrelated to AATD, or history of or clinical signs of cirrhosis
Significant lung disease not attributable to manifestations of AATD, as determined by the investigator
History of one or more hospitalizations due to severe exacerbation of underlying lung disease during the year before screening or received IV antibiotics for treatment of a pulmonary infection within 6 months before screening
Trial design
Treatments tested in this trial
- TSRA-196
Treatment groups
72 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov