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
Tessera Therapeutics, Inc.
Summary
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
- Age range
- 18–70 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * 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 * Nonsmoker for at least 6 months before screening and must remain nonsmoking for the entire study duration * Either AAT treatment-naïve or washed out of all investigational or approved treatments that modify AAT levels for 5 half-lives or at least 4 weeks, whichever is longer, before TSRA-196 administration…
Interventions
- DrugTSRA-196
TSRA-196 is an in-vivo genome editing product formulated in lipid nanoparticles (LNPs) for the treatment of patients with alpha-1 antitrypsin deficiency (AATD), via intravenous (IV) infusion
Locations (3)
- Clinical Study CenterBoston, Massachusetts
- Clinical Study CenterCharleston, South Carolina
- Clinical Study CenterFitzroy, Victoria