A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)

Conditions

• Alpha-1 Antitrypsin Deficiency (AATD)

Eligibility Criteria

Sex

ALL

Age

18 Years - 70 Years

Healthy Volunteers

Not accepted

Interventions

• TSRA-196 — DRUG
  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

Study Details

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)

Key Dates

Start date

Apr 21, 2026

Status verified

May 2026

Primary completion

Mar 31, 2029

Completion

Mar 31, 2029

Study Design

Enrollment

72 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: TSRA-196 Drug Product

Primary Outcome Measure

Part 1 (Dose Escalation): Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) [ Time Frame: 1 Year ]

Central Contacts

• Tessera Clinical Trials Information
  857-271-4800
  [email protected]

Locations (2)

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