TSRA 196-AAT-201
Research type
Research Study
Full title
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
IRAS ID
1013044
Contact name
Narin Ahmed
Contact email
Sponsor organisation
Tessera Therapeutics, Inc.
Research summary
This is an open-label, 3-part study of TSRA-196 that will enrol adults who have the PiZZ genotype, blood levels of AAT <11 µM, and AATD-associated lung and/or liver disease.
• Part 1 is the single-ascending dose-escalation phase, and Part 2 is the dose-expansion phase. Participants in Part 1 and Part 2 who have AAT <LLN at Month 6 and Month 12 may be eligible to receive a second dose of TSRA-196 in Part 3 of the study. Part 3 will only proceed after submission of a major protocol amendment to regulatory agencies to define the dose(s) to be used based on the results of Parts 1 and 2.
• Two populations of participants will be enrolled:
participants with clinically significant AATD lung disease with no liver fibrosis or minimal liver fibrosis (Parts 1A, 2A, and 3A), and
participants with significant or severe liver fibrosis with or without lung disease (Parts 1B, 2B, and 3B).
The Screening period will be up to 48 days. Eligible participants will receive prophylactic medications on Day -1 and Day 1 to reduce the risk of a clinically significant infusion-related reaction. On Day 1 a single dose of TSRA-196 will be administered by IV infusion.
Participants will be inpatient for observation for a minimum of 24 hours after the completion of TSRA-196 administration unless safety evaluations suggest that a longer observation period is needed. Participants will have study visits at Days 2, 3, 4, 7, 14, 21, 28, 60, 90, 180, 270, and 365 (Month 12, end of study). There is also an optional visit at Day 330 for participants who had AAT <11 µM at Month 3 and then resumed augmentation therapy (see Section 6.9.3).
Following the end-of-study visit, all participants who received TSRA-196 will be asked to enrol in a long-term follow-up study to assess the safety and efficacy of TSRA-196 for 14 years (total follow-up period of 15 years after TSRA-196 dosing).REC name
North East - York Research Ethics Committee
REC reference
25/NE/0221
Date of REC Opinion
28 Jan 2026
REC opinion
Further Information Favourable Opinion