Metabolic Dysfunction-associated Steatohepatitis (MASH)Phase 1/2RESTORENCT07732400
A Study of VV-14303 for the Treatment of Metabolic Dysfunction-associated Steatohepatitis (MASH)
A Phase 1/2 study testing VV-14303 in people with Metabolic Dysfunction-associated Steatohepatitis (MASH), run by Kriya Therapeutics, Inc. at 3 sites in 2 countries.
Trial summary
A Study of VV-14303 for the Treatment of Metabolic dysfunction-associated steatohepatitis (MASH) Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Metabolic Dysfunction-associated Steatohepatitis (MASH)
- What is being tested?
- VV-14303
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 56 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 30 Jul 2026to Jul 2029
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participant is capable of providing signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol
- Must be 18 to 75 years of age (inclusive) at Screening
- Body Mass Index (BMI) of 25 to <40 kg/m2 (inclusive)
- Male participants must agree to use a highly effective contraception during the Treatment Period and at least 12 months after administration of VV-14303. Female participants must not be a woman of child-bearing potential (WOCBP)
- Biopsy-confirmed MASH
- Previous history or presence of ≥2 of the following metabolic risk factors: obesity (BMI ≥25 kg/m²), hypertension (blood pressure [BP] ≥140/90 mmHg or on antihypertensive medication), dyslipidemia (triglycerides ≥150 mg/dL or high-density lipoprotein cholesterol [HDL-C] <40 mg/dL in men/<50 mg/dL in women or on lipid-lowering therapy), type 2 diabetes mellitus
- Must be willing to refrain from the donation of blood, plasma, platelets, eggs, or sperm during the 12-month post-treatment follow-up period
Who cannot join
- Presence of alternate and/or additional liver disease etiologies at Screening, including but not limited to chronic viral hepatitis, autoimmune hepatitis
- Use of treatments for metabolic syndrome management, including oral antidiabetic drugs (OADs) (e.g., metformin), incretin mimetics (GLP-1 receptor agonists or GLP-1/gastric inhibitory polypeptide [GIP] agonists) or other glucose-lowering agents that has not been stable for at least 6 months prior to Screening
- Use of Resmetirom that has not been stable for at least 6 months prior to Screening visit
- Any medical, cognitive, or psychiatric condition that, in the opinion of the Investigator, could contraindicate the use of the investigational drug, make consistent study assessment and follow-up over the 12-month Post-Treatment Follow-up Period unlikely, or would make the participant an unsafe study candidate.
- Type 1 diabetes, or poorly controlled type 2 diabetes (HbA1c > 8.0% at Screening)
- History of major trauma to the muscle(s) intended for IM injection meeting any of the following criteria:
Show all 11Show fewer
- Within 6 months prior to Screening, or
- At any timepoint prior to Screening with continued neurologic or musculoskeletal symptoms
- Prior participation in any systemic experimental treatment or receiving any other systemic investigational treatment including within 6 weeks or 5 half-lives of the active ingredient (whichever is longer) prior to the start of Screening
- Any vaccination or planned vaccination 30 days prior to dosing, or planned vaccination 8 weeks post dosing
- Previously received AAV or adenoviral therapy or participation in any previous gene therapy trial
Trial locations and contacts
3 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
3 sites in 2 countries
United States2 sites, 2 recruiting
- Not yet recruiting
Kriya Clinical Trial Site
Chandler, Arizona, 85224
VP, Medical Affairs, 1.984.884.5058, clinicaltrials@kriyatx.com
- Recruiting
Kriya Clinical Trial Site
Lady Lake, Florida, 32159
New Zealand1 site, 1 recruiting
- Not yet recruiting
Kriya Clinical Trial Site
Auckland
About this study
Official title: A Phase 1/2, First-in-Human, Multi-Arm, Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), 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)
A Study of VV-14303 for the Treatment of Metabolic dysfunction-associated steatohepatitis (MASH)
Read the detailed description
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)
- Being tested
- VV-14303
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VV-14303Genetic
will be administered via ultrasound guided intramuscular injections
| Study group | Role | What they receive |
|---|---|---|
| Part 1 Cohort 1, dose #1 | Experimental | Dose #1 of VV-14303 will be administered Receives: Genetic: VV-14303 |
| Part 1 Cohort 2, dose #2 | Experimental | Dose #2 of VV-14303 will be administered Receives: Genetic: VV-14303 |
| Part 1 Cohort 3, dose #3 | Experimental | Dose #3 of VV-14303 will be administered Receives: Genetic: VV-14303 |
| Part 2 dose | Experimental | VV-14303 will be administered at the dose determined from Part 1 (Cohorts 1, 2, and 3) Receives: Genetic: VV-14303 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence 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
Measured: 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
Measured: 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
Measured: 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
Measured: 26 weeks
Secondary outcomes (7)
Efficacy associated with VV-14303 in participants with MASH
Liver stiffness as measured by change from Baseline transient elastography as assessed by FibroScan®
Measured: 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)
Measured: 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®
Measured: 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®
Measured: 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®
Measured: 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®
Measured: 26 Weeks
Concentration of adeno-associated virus (AAV) vector-mediated transgene product in serum
Pharmacokinetics of VV-14303 transgene product
Measured: 52 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jul 2026
- Study start30 Jul 2026
- Primary completionJul 2028 (estimated)
- Study completionJul 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Metabolic Dysfunction-associated Steatohepatitis (MASH)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 56 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kriya Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07732400
- Sponsor's protocol ID
- KRIYA-497-101
- Acronym
- RESTORE
- Status last verified
- Aug 2026
- First submitted
- 14 Jul 2026
- Last updated
- 1 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, First-in-Human, Multi-Arm, Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), 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)
- ClinicalTrials.gov ID
- NCT07732400
- Acronym / sponsor ID
- RESTORE / KRIYA-497-101
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 56 participants (estimated)
- Lead sponsor
- Kriya Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 30 Jul 2026 (actual)
- Primary completion
- Jul 2028 (estimated)
- Study completion
- Jul 2029 (estimated)
- First posted
- 28 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A Study of VV-14303 for the Treatment of Metabolic dysfunction-associated steatohepatitis (MASH)
Detailed description
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Part 1 Cohort 1, dose #1 | Experimental | Genetic: VV-14303Dose #1 of VV-14303 will be administered |
| Part 1 Cohort 2, dose #2 | Experimental | Genetic: VV-14303Dose #2 of VV-14303 will be administered |
| Part 1 Cohort 3, dose #3 | Experimental | Genetic: VV-14303Dose #3 of VV-14303 will be administered |
| Part 2 dose | Experimental | Genetic: VV-14303VV-14303 will be administered at the dose determined from Part 1 (Cohorts 1, 2, and 3) |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Participant is capable of providing signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol
- Must be 18 to 75 years of age (inclusive) at Screening
- Body Mass Index (BMI) of 25 to <40 kg/m2 (inclusive)
- Male participants must agree to use a highly effective contraception during the Treatment Period and at least 12 months after administration of VV-14303. Female participants must not be a woman of child-bearing potential (WOCBP)
- Biopsy-confirmed MASH
Key exclusion criteria (11 listed)
- Presence of alternate and/or additional liver disease etiologies at Screening, including but not limited to chronic viral hepatitis, autoimmune hepatitis
- Use of treatments for metabolic syndrome management, including oral antidiabetic drugs (OADs) (e.g., metformin), incretin mimetics (GLP-1 receptor agonists or GLP-1/gastric inhibitory polypeptide [GIP] agonists) or other glucose-lowering agents that has not been stable for at least 6 months prior to Screening
- Use of Resmetirom that has not been stable for at least 6 months prior to Screening visit
- Any medical, cognitive, or psychiatric condition that, in the opinion of the Investigator, could contraindicate the use of the investigational drug, make consistent study assessment and follow-up over the 12-month Post-Treatment Follow-up Period unlikely, or would make the participant an unsafe study candidate.
- Type 1 diabetes, or poorly controlled type 2 diabetes (HbA1c > 8.0% at Screening)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VV-14303will be administered via ultrasound guided intramuscular injections | Genetic | Part 1 Cohort 1, dose #1, Part 1 Cohort 2, dose #2, Part 1 Cohort 3, dose #3, Part 2 dose |
Primary outcome
Incidence and severity of adverse events, abnormal clinical laboratory values, abnormal physical exams, abnormal vital signs, abnormal ECGs, and abnormal imaging
Time frame: 52 Weeks
Safety of VV-14303 in participants with MASH
Number of participants with improvement in overall metabolic health, as assessed by changes in serum biomarker levels
Time frame: 6 weeks
Evaluate safety and efficacy of VV-14303 in participants with MASH in Part 1
Changes in liver fat content as assessed by Magnetic Resonance Proton Density Fat Fraction (MRI-PDFF) as assessed by FibroScan®
Time frame: 26 Weeks
Efficacy of VV-14303 in participants with MASH in Part 2
Change in liver fat content as assessed by controlled attenuation parameter (CAP) as assessed by FibroScan®
Time frame: 26 weeks
Efficacy of VV-14303 in participants with MASH in Part 2
7 secondary outcomes
Efficacy associated with VV-14303 in participants with MASH
Time frame: Week 26 and 52
Efficacy associated with VV-14303 in participants with MASH
Time frame: Week 26 and 52
Efficacy associated with VV-14303 in participants with MASH
Time frame: 52 Weeks
Efficacy associated with VV-14303 in participants with MASH
Time frame: 52 Weeks
Part 1: Efficacy associated with VV-14303 in participants with MASH
Time frame: 26 Weeks
Part 1: Efficacy associated with VV-14303 in participants with MASH
Time frame: 26 Weeks
Concentration of adeno-associated virus (AAV) vector-mediated transgene product in serum
Time frame: 52 Weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Sep 2026.
Related trials for Metabolic Dysfunction-associated Steatohepatitis (MASH)
RecruitingPhase 3
A Study Evaluating Efruxifermin in Subjects With Non-Cirrhotic Nonalcoholic Steatohepatitis (NASH)/Metabolic Dysfunction-Associated Steatohepatitis (MASH) and Fibrosis
NASH With Fibrosis, MASH With Fibrosis
RecruitingPhase 1
A Study of INI-822 in Healthy Volunteers and Participants With Metabolic Dysfunction-Associated Steatohepatitis (MASH) or Presumed MASH
Metabolic Dysfunction-Associated Steatohepatitis
RecruitingPhase 3
Trial to Evaluate ALN-CIDEB in Adults With Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD)
Metabolic Dysfunction-Associated Steatotic Liver Disease, MASLD
RecruitingPhase 3
A Pivotal Clinical Study to Investigate the Safety and Efficacy of Efimosfermin Compared With Placebo in Adult Participants With Compensated Cirrhosis Due to Metabolic Dysfunction-associated Steatohepatitis (MASH)
Metabolic Dysfunction-associated Steatohepatitis
RecruitingPhase 3
A Study to Investigate Safety and Efficacy of Efimosfermin Compared With Placebo in Adult Participants With Compensated Cirrhosis Due to Metabolic Dysfunction-associated Steatohepatitis (MASH)
Metabolic Dysfunction-associated Steatohepatitis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing VV-14303 in people with Metabolic Dysfunction-associated Steatohepatitis (MASH), run by Kriya Therapeutics, Inc. at 3 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 1 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, New Zealand. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact VP, Medical Affairs (1.984.884.5058, clinicaltrials@kriyatx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of adverse events, abnormal clinical laboratory values, abnormal physical exams, abnormal vital signs, abnormal ECGs, and abnormal imaging (time frame: 52 Weeks). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Sep 2026.