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TSRA-196

Phase 1

Alpha-1 Antitrypsin Deficiency (AATD) | Small molecule | Other |cbdMD, Inc.|Last Updated: Aug 21, 2026

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment72

FDA Designations

No designations recorded

Clinical trial landscape

TSRA-196 · 1 trial · 1 indication

Phase 1 1
NCT07227207A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)Alpha-1 Antitrypsin Deficiency (AATD)
RECRUITING72 Analytics
PHASE1RECRUITING
A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)
Alpha-1 Antitrypsin Deficiency (AATD)
NCT0722720772
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Study Endpoints

Primary Endpoints

Part 1 (Dose Escalation): Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
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
1 Year
Part 2 (Dose Expansion): Change in functional AAT concentrations (determined using an elastase inhibition assay) from baseline to end of study
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
1 Year

Secondary Endpoints

Part 1 (Dose Escalation): Proportion of participants who have serum levels of total AAT greater than or equal to LLN after TSRA-196 treatment
1 Year
Part 2 (Dose Expansion): Incidence of TEAEs and SAEs
1 Year
Part 1 (Dose Escalation) and Part 2 (Dose Expansion): Proportion of participants who have serum levels of total AAT greater than or equal to 11 μM after TSRA-196 treatment
1 Year
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
TSRA-196 Drug ProductEXPERIMENTAL -

Interventions

NameTypeDescription
TSRA-196DRUGTSRA-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
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Eligibility Criteria

Age Range18 Years to 70 Years
SexALL
Healthy VolunteersNo
Study Sites6

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 * Nonsmoke...

Countries:United StatesAustraliaUnited Kingdom
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