Healthy Participants, MASH - Metabolic Dysfunction-Associated Steatohepatitis
Conditions
Brief summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamic (PD) effects of single ascending doses of TGM-312-SC01 in healthy adults and multiple ascending doses in patients with metabolic dysfunction-associated steatohepatitis (MASH).
Detailed description
This is a Phase 1/2, randomised, masked, placebo-controlled study evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of TGM-312-SC01 administered by subcutaneous injection. The study includes a single ascending dose component in healthy participants and a multiple ascending dose component in adults with metabolic dysfunction-associated steatohepatitis, with an optional expansion phase.
Interventions
DRUGTGM-312-SC01
TGM-312-SC01 is an investigational medicinal product administered by subcutaneous injection according to a protocol-defined regimen.
DRUGPlacebo
Placebo administered by subcutaneous injection according to a protocol-defined regimen.
Sponsors
Tangram Therapeutics Plc
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
Yes
Inclusion criteria
* Adults aged 18 to 70 years who are able to provide written informed consent. * Medically suitable for study participation based on protocol-defined assessments. * For the disease cohort, participants with clinical features consistent with metabolic dysfunction-associated steatohepatitis, as defined in the protocol.
Exclusion criteria
* Clinically significant medical conditions, laboratory abnormalities, or other findings that, in the opinion of the investigator, could increase risk, interfere with study participation, or confound interpretation of study results. * Recent participation in another investigational study. * Use of medications that are prohibited by the protocol. * Any other condition that would make the individual unsuitable for study participation as determined by the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence and severity of treatment-emergent adverse events [Safety and tolerability] | From start of study drug administration through 16 weeks after the last study drug administration. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Maximum observed plasma concentration (Cmax) of TGM-312-SC01 (ng/mL) | From start of study drug administration through 48 hours after the last study drug administration. | Maximum observed plasma concentration derived from plasma concentration-time data. |
| Area under the plasma concentration-time curve (AUC) of TGM-312-SC01 (ng·h/mL) | From start of study drug administration through 48 hours after the last study drug administration. | Area under the plasma concentration-time curve derived from plasma concentration-time data. |
| Change from baseline in target gene mRNA expression levels in tissue homogenate | From start of study drug administration through 16 weeks after the last study drug administration. | Change from baseline in mRNA expression levels of the target gene in homogenised tissue samples following administration of TGM-312-SC01, expressed as relative change from baseline. |
| Change from baseline in target protein levels in tissue homogenate | From start of study drug administration through 16 weeks after the last study drug administration. | Change from baseline in protein levels of the target gene product in homogenised tissue samples following administration of TGM-312-SC01, expressed as concentration or relative change from baseline. |
Countries
United Kingdom
Contacts
CONTACTSponsor Clinical Trials Office
STUDY_DIRECTORSponsor Medical Representative
Tangram Therapeutics
Outcome results
None listed