Recruiting
Phase 1
Phase 2

TSRA-196

Sponsor:

Tessera Therapeutics, Inc.

Code:

NCT07227207

Conditions

Alpha-1 Antitrypsin Deficiency (AATD)

Eligibility Criteria

Sex: All

Age: 18 - 70

Healthy Volunteers: Not accepted

Interventions

TSRA-196

Study Details

Brief 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)

Conditions

Alpha-1 Antitrypsin Deficiency (AATD)

Study ID

NCT07227207

Start date

Apr 21, 2026

Status verified date

Aug, 2026

Completion date

Mar, 2029

Anticipated

Primary completion date

Mar, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 70

Healthy Volunteers: Not accepted

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

Parts 1A and 2A (AATD lung disease with no or minimal liver fibrosis)

  • Clinically significant lung disease, defined as 1) evidence of emphysema or bronchiectasis by computed tomography or 2) DLCO <70% of the predicted value or 3) ppFEV1 <80%
  • ppFEV1 ≥35%
  • METAVIR fibrosis score F0 or F1 confirmed by liver biopsy at screening, or a liver stiffness measure by FibroScan ≤7 kPa at screening
  • FIB-4 index score ≤3.25 at screening
  • ALT and/or AST <ULN at screening

Parts 1B and 2B (AATD liver disease with significant or severe liver fibrosis, with or without AATD lung disease)

  • METAVIR fibrosis score F2 or F3 confirmed by liver biopsy at screening. A liver biopsy conducted within 12 months before screening is acceptable as a substitute.
  • Liver stiffness measure by FibroScan >7 and ≤15 kPa at screening
  • ALT and/or AST <2 x ULN at screening

Exclusion Criteria:

  • 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
  • Unstable AATD-related COPD, as determined by the investigator, or severe bronchiectasis
  • Lung volume reduction surgery within 1 year before screening or plan to receive lung volume reduction surgery during the study period
  • Documented chronic need for positive airway pressure therapy beyond nocturnal use
  • Seropositive for human immunodeficiency virus (HIV) (HIV-1 or HIV-2)
  • Seropositive for hepatitis B (hepatitis B surface antigen \[HBsAg\] or hepatitis B core antibody \[HBcAb\] positive) with detectable HBV DNA
  • Hepatitis C virus (HCV) RNA positive at screening (Parts 1A and 2A), or HCV RNA positive and/or HCV antibody positive at screening (Parts 1B and 2B)
  • Has received an organ transplant or is on a waiting list for an organ transplant
  • Prior treatment with gene therapy using viral vectors or intended to permanently change the patient's DNA
  • Any investigational products within 30 days before dosing or plan to take an investigational product before the end of study

Study Design

Enrollment

72 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: TSRA-196 Drug Product

Interventions

TSRA-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

Primary outcome measure

  • Part 1 (Dose Escalation): Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) [ Time Frame: 1 Year ]
  • Part 2 (Dose Expansion): Proportion of participants who have serum levels of total alpha-1 antitrypsin (AAT) greater than or equal to Lower Limit of Normal (LLN) after TSRA-196 treatment [ Time Frame: 1 Year ]
  • Part 2 (Dose Expansion): Change in functional AAT concentrations (determined using an elastase inhibition assay) from baseline to end of study [ Time Frame: 1 Year ]
  • Part 3 (Single Repeated Dose): Proportion of participants who have serum levels of total alpha-1 antitrypsin (AAT) greater than or equal to Lower Limit of Normal (LLN) after a second dose of TSRA-196 [ Time Frame: 1 Year ]

Central Contacts and Locations

Central contacts

Tessera Clinical Trials Information

857-271-4800clinicalinfo@tesseratx.com

Locations

Clinical Study Center

Recruiting

Boston, Massachusetts, United States, 02118

Clinical Study Center

Recruiting

Rochester, Minnesota, United States, 55905

Clinical Study Center

Recruiting

Charleston, South Carolina, United States, 29425

Clinical Study Center

Recruiting

Seattle, Washington, United States, 98195

More Information

Sponsor

Tessera Therapeutics, Inc.

Last update posted

Sep 3, 2026

Last verified

Aug, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Tessera Therapeutics, Inc. on 2026-09-03.