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Heterozygous Familial HypercholesterolemiaEarly Phase 1NCT07353398

Early-phase Study of ART002g1 Injection in HeFH: Safety, Tolerability and Preliminary Efficacy

A Early Phase 1 study testing ART002g1 Injection in people with Heterozygous Familial Hypercholesterolemia, run by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine at 1 site in China.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
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Trial summary

This study is an open-label, single ascending dose (SAD) study designed to evaluate the safety and tolerability of ART002g1 in patients with heterozygous familial hypercholesterolemia (HeFH) who require further reduction in low-density lipoprotein cholesterol (LDL-C). Read more about this study

Who is it for?
Ages 18 to 70 years
What is being tested?
ART002g1 Injection
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
24 participants
Where
1 siteChina
Study dates
11 Mar 2026to Jul 2027
Phase
Early Phase 1
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 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Subjects must meet all the following criteria to be eligible for enrollment:

  1. Male or female, aged 18 to 70 years (inclusive) at the time of signing the Informed Consent Form (ICF);
  2. Body weight between 45 and 90 kg (inclusive) at screening;
  3. Definite diagnosis of heterozygous familial hypercholesterolemia (HeFH), meeting either of the following two criteria (1) or (2):

（1) HeFH diagnosed to be caused by mutations in the LDLR, APOB, or PCSK9 gene;

(2) Meeting 2 out of the 3 following criteria for adults per the Dutch Lipid Clinical Network (DLCN) criteria:

  1. Serum LDL-C ≥ 4.7 mmol/L without prior lipid-lowering treatment;
  2. Cutaneous or tendinous xanthomas, or arcus cornealis (in subjects < 45 years old);
  3. Presence of FH or early-onset atherosclerotic cardiovascular disease (ASCVD) in first-degree relatives.

Subjects must not be enrolled if they meet any one or more of the following exclusion criteria:

  1. Diagnosis of compound heterozygous FH, double heterozygous FH, or homozygous FH (HoFH);
  2. Positive for hepatitis B surface antigen (HBsAg), or positive for hepatitis B core antibody (HBcAb) with peripheral blood HBV DNA titer ≥ 1 × 10² copies/L; positive for hepatitis C virus (HCV) antibody with positive peripheral blood HCV RNA; positive for human immunodeficiency virus (HIV) antibody;
  3. Any unstable systemic disease, including but not limited to: unstable angina; cerebrovascular accident or transient ischemic attack (within 6 months prior to screening); myocardial infarction (within 6 months prior to screening); history of heart failure (NYHA Class II-IV); severe arrhythmia requiring pharmacotherapy; liver, kidney, or metabolic diseases; or other unstable systemic diseases as determined by the investigator;
  4. History of percutaneous transluminal coronary angioplasty (PTCA), percutaneous coronary intervention (PCI), or coronary artery bypass grafting (CABG) within 6 months prior to the first dose; or documented severe coronary artery stenosis as confirmed by coronary CT or coronary angiography within 90 days prior to randomization.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Xueying Ding, MD contact

+86-02136126102

dingxueying@126.com

Rong Jiang, MD contact

+86 13795493921

listening39@163.com

China1 site

  • Shanghai General Hospital

    Shanghai

    Xueying Ding, MD, +86-021-, dingxueying@126.com

    Rong Jiang, MD, listening39@163.com

Other trials in China

About this study

Official title: Early-phase Clinical Study on Safety, Tolerability and Preliminary Efficacy of ART002g1 Injection in the Treatment of Heterozygous Familial Hypercholesterolemia

This study is an open-label, single ascending dose (SAD) study designed to evaluate the safety and tolerability of ART002g1 in patients with heterozygous familial hypercholesterolemia (HeFH) who require further reduction in low-density lipoprotein cholesterol (LDL-C). ART002g1 uses base editing technology, which is designed to interfere with the expression of the PCSK9 gene in the liver, thereby reducing the circulating levels of PCSK9 and LDL-C. The primary objectives of this study are to determine the safety and pharmacodynamic (PD) profiles of ART002g1 in this patient population.

Being tested
ART002g1 Injection
Keywords
ART002g1, LNP, HeFH

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ART002g1 InjectionDrug

    Intravenous (IV) infusion

Study groups in NCT07353398
Study groupRoleWhat they receive
Experimental: ART002g1 Injection - Dose Group 1 ( Single IV Infusion)ExperimentalParticipants will receive a single dose of ART002g1.
Receives: Drug: ART002g1 Injection
Experimental: ART002g1 Injection - Dose Group 2 (Single IV Infusion)ExperimentalParticipants will receive a single dose of ART002g1.
Receives: Drug: ART002g1 Injection
Experimental: ART002g1 Injection - Extended Dose Group ( Single IV Infusion)ExperimentalParticipants will receive a single Optimal Biological Dose (OBD) of ART002g1, which is based on the results of the ascending dose escalation.
Receives: Drug: ART002g1 Injection

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)

    Measured: As of Week 48 (W48) post-administration of ART002g1 for Injection

Secondary outcomes (8)

  1. Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: Tmax

    Measured: As of Week 2 (W2) post-administration of ART002g1 for Injection

  2. Pharmacodynamic (PD) Assessments: Serum PCSK9 protein

    Measured: As of Week 48 (W48) post-administration of ART002g1 for Injection

  3. Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: Cmax

    Measured: As of Week 2 (W2) post-administration of ART002g1 for Injection

  4. Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: AUC

    Measured: As of Week 2 (W2) post-administration of ART002g1 for Injection

  5. Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: t½

    Measured: As of Week 2 (W2) post-administration of ART002g1 for Injection

  6. Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1: CL

    Measured: As of Week 2 (W2) post-administration of ART002g1 for Injection

  7. Pharmacokinetic (PK) Assessments: PK Parameters of ART002g1:Vss

    Measured: As of Week 2 (W2) post-administration of ART002g1 for Injection

  8. Pharmacodynamic (PD) Assessments: Serum LDL-C

    Measured: As of Week 48 (W48) post-administration of ART002g1 for Injection

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Jan 2026
  2. Study start11 Mar 2026 (estimated)
  3. Primary completionJul 2027 (estimated)
  4. Study completionJul 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
24 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07353398
Sponsor's protocol ID
IIT-ATMP-2026001
Status last verified
Mar 2026
First submitted
12 Jan 2026
Last updated
17 Mar 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing ART002g1 Injection in people with Heterozygous Familial Hypercholesterolemia, run by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

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: opening soon, as of the sponsor's last update on 17 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. 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 phase mean?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

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 Xueying Ding, MD (+86-02136126102, dingxueying@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) (time frame: As of Week 48 (W48) post-administration of ART002g1 for Injection). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07353398.

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 17 Mar 2026; AzTrials last refreshed its copy on 8 Nov 2026.

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