A Study of VV-14303 for the Treatment of Metabolic dysfunction-associated steatohepatitis (MASH)
A Phase 1/2, First-in-Human, Multi-Arm, Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of VV-14303, an Adeno-associated Virus Vector-mediated Fibroblast Growth Factor 21 (FGF21) Gene Therapy, in Adults with Metabolic dysfunction associated steatohepatitis (MASH) (the RESTORE Study)
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
56
will be administered via ultrasound guided intramuscular injections
Kriya Clinical Trial Site
Chandler, Arizona, United States
NOT_YET_RECRUITINGKriya Clinical Trial Site
Lady Lake, Florida, United States
RECRUITINGKriya Clinical Trial Site
Auckland, New Zealand
NOT_YET_RECRUITINGIncidence and severity of adverse events, abnormal clinical laboratory values, abnormal physical exams, abnormal vital signs, abnormal ECGs, and abnormal imaging
Safety of VV-14303 in participants with MASH
Time frame: 52 Weeks
Number of participants with improvement in overall metabolic health, as assessed by changes in serum biomarker levels
Evaluate safety and efficacy of VV-14303 in participants with MASH in Part 1
Time frame: 6 weeks
Changes in liver fat content as assessed by Magnetic Resonance Proton Density Fat Fraction (MRI-PDFF) as assessed by FibroScan®
Efficacy of VV-14303 in participants with MASH in Part 2
Time frame: 26 Weeks
Change in liver fat content as assessed by controlled attenuation parameter (CAP) as assessed by FibroScan®
Efficacy of VV-14303 in participants with MASH in Part 2
Time frame: 26 weeks
Efficacy associated with VV-14303 in participants with MASH
Liver stiffness as measured by change from Baseline transient elastography as assessed by FibroScan®
Time frame: Week 26 and 52
Efficacy associated with VV-14303 in participants with MASH
Liver stiffness as measured by change from Baseline in Magnetic Resonance Elastography (MRE)
Time frame: Week 26 and 52
Efficacy associated with VV-14303 in participants with MASH
Mean changes in liver fat content as assessed by MRI-PDFF as assessed by FibroScan®
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Time frame: 52 Weeks
Efficacy associated with VV-14303 in participants with MASH
Mean changes in liver fat content as assessed by CAP as assessed by FibroScan®
Time frame: 52 Weeks
Part 1: Efficacy associated with VV-14303 in participants with MASH
Mean changes in liver fat content as assessed by MRI-PDFF as assessed by FibroScan®
Time frame: 26 Weeks
Part 1: Efficacy associated with VV-14303 in participants with MASH
Mean changes in liver fat content as assessed by CAP as assessed by FibroScan®
Time frame: 26 Weeks
Concentration of adeno-associated virus (AAV) vector-mediated transgene product in serum
Pharmacokinetics of VV-14303 transgene product
Time frame: 52 Weeks