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Retinal DystrophyEarly Phase 1NCT07063030

A Study of LX107 Gene Therapy in AIPL1-IRD Patients

A Early Phase 1 study testing LX107 Injection in people with Retinal Dystrophy, run by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine at 1 site in China.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

Administering subretinal injection of LX107 injection (a gene therapy drug) to patients with retinal dystrophy caused by AIPL1 gene mutation to evaluate its efficacy and safety. Read more about this study

Who is it for?
Ages 4 years and older
Conditions the study is for
Retinal Dystrophy
What is being tested?
LX107 Injection
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
13 participants
Where
1 siteChina
Study dates
15 Jul 2025to 31 Dec 2030
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 4 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • The subject and/or their guardian signs a written informed consent form and is willing to comply with the long-term follow-up protocol and supporting protocols.
  • Adult or pediatric patients (aged ≥ 4 years) diagnosed with AIPL1-IRD.
  • Definitive molecular diagnosis of biallelic AIPL1 gene mutations confirmed by next-generation sequencing combined with Sanger validation.
  • The study eye has a best-corrected visual acuity of no more than 58 letters (approximately equivalent to decimal visual acuity ≤ 0.3) using the ETDRS visual acuity chart at baseline.
  • Note: Only one eye will be designated as the "study eye" (i.e., the eye to receive treatment) at the investigator's discretion.
  • Exclusion Criteria:For any eye with the following conditions:
Show all 19Show fewer
  • A history of ocular diseases that, in the investigator's judgment, may hinder the planned treatment or interfere with the interpretation of study endpoints (e.g., glaucoma, diabetic retinopathy, retinal vein occlusion, retinal detachment, posterior or panuveitis, etc.).
  • Any eye with a history of gene therapy for IRD or other hereditary neuro-ophthalmic diseases (including but not limited to other viral vector-based gene therapies, mRNA therapies, etc.).
  • A lack of sufficient viable retinal cells as determined by non-invasive methods such as OCT or ophthalmoscopy.
  • Any active intraocular or periocular infection in the study eye (e.g., infectious conjunctivitis, keratitis, scleritis, endophthalmitis, infectious blepharitis, uveitis).
  • A history of intraocular surgery (e.g., vitrectomy, cataract surgery, trabeculectomy, or other filtering surgery) in the 6 months prior to the screening visit.
  • For any systemic conditions:
  • Uncontrolled hypertension, defined as systolic blood pressure ≥ 160 mmHg or diastolic blood pressure ≥ 100 mmHg. If the initial measurement exceeds the above limits, it may be repeated on the same day or another day during the screening period; if the subject is taking oral antihypertensive drugs, they must have been on a stable dose of the same drug for at least 30 days prior to screening.
  • Diabetic patients meeting any of the following criteria: ① known to have macrovascular complications; ② baseline HbA1c > 7.5%; ③ receiving treatment with two or more oral hypoglycemic agents, insulin, or GLP-1 receptor agonists.
  • A history of any other diseases, metabolic disorders, physical examination findings, or clinical laboratory abnormalities that, in the investigator's judgment, may contraindicate the use of the study drug, interfere with the interpretation of study results, or place the subject at high risk of treatment complications, including but not limited to: AIDS, syphilis, acute/chronic active hepatitis B or C, coagulation disorders, a history of treated or untreated malignancy within the past 5 years (except for localized basal cell carcinoma of the skin or in situ cervical cancer), etc.
  • Any of the following laboratory abnormalities: ① platelet count < 100 × 10⁹/L, hemoglobin (Hb) < 10 g/dL (males) or < 9 g/dL (females); ② aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 3 × ULN; ③ serum creatinine or urea > 1.5 × ULN.
  • Administration of any other investigational drug (except vitamins and minerals) within 3 months prior to screening, or an attempt to participate in another clinical trial during the study period.
  • Other circumstances deemed unsuitable for enrollment by the investigator.
  • Unwillingness to use effective contraceptive methods during the study; pregnant or lactating women, or women planning to become pregnant or lactate during the study period.

Who cannot join

None listed.

Trial locations and contacts

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

China1 site, 1 recruiting

  • Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

    Shanghai

    Xiaodong Sun, +86-13651765503, xdsun@sjtu.edu.cn

    Recruiting

Other trials in China

About this study

Official title: An Exploratory Clinical Study to Evaluate LX107 Gene Therapy in Patients With AIPL1 Biallelic Mutation-related Inherited Retinal Dystrophy (AIPL1-IRD)

Administering subretinal injection of LX107 injection (a gene therapy drug) to patients with retinal dystrophy caused by AIPL1 gene mutation to evaluate its efficacy and safety.

Being tested
LX107 Injection
Medical subject headings
Retinal Dystrophies

What participants receive

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

  • LX107 InjectionGenetic

    Subjects will receive subretinal injection of LX107 on Day 0.

Study groups in NCT07063030
Study groupRoleWhat they receive
InterventionExperimentalExperimental: LX107 Injection Arm Description: * Low dose group: subretinal injection of LX107 -3×10⁹ VG/eye * Medium dose group: subretinal injection of LX107- 1×10¹⁰ VG/eye * High dose group: subretinal injection of LX107 -3×10¹⁰ VG/eye
Receives: Genetic: LX107 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 [Safety and Tolerability]

    Incidence of ocular and non-ocular adverse events (AEs) and serious adverse events (SAEs) following LX107 subretinal injection

    Measured: 6 Months

Secondary outcomes (5)

  1. Changes in Functional Magnetic Resonance Imaging (fMRI) Results

    Measured: 6 months、12 months

  2. Mean Change from Baseline in Multi-luminance Mobility Test (MLMT)

    Assess the subject's mobility ability under different light intensities using MLMT, and compare the mean changes from baseline at 180 and 365 days after treatment to reflect improvements in visual function.

    Measured: 6 months、12 months

  3. Mean Change from Baseline in Full-Field Sensitivity Threshold (FST)

    Measure the subject's light perception ability using FST, and compare the mean changes from baseline at 180 and 365 days after treatment to evaluate improvements in visual sensitivity.

    Measured: 6 months、12 months

  4. Mean Change from Baseline in Visual Acuity (VA)

    Measure VA using ETDRS and BRVT charts, with LogMAR score as the indicator, and compare the mean changes from baseline at 180 and 365 days after treatment to assess visual acuity improvement.

    Measured: 6 months、12 months

  5. Mean Change from Baseline in Visual Fields

    Use Goldmann perimeter (dynamic visual field) and Humphrey perimeter (static visual field) to compare the mean changes from baseline at 180 and 365 days after treatment, evaluating improvements in visual field range and sensitivity.

    Measured: 6 months、12 months

Timeline

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

  1. First posted14 Jul 2025
  2. Study start15 Jul 2025
  3. Primary completion31 Dec 2026 (estimated)
  4. Study completion31 Dec 2030 (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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
13 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07063030
Sponsor's protocol ID
SHGH-LX107
Status last verified
Mar 2026
First submitted
3 Jul 2025
Last updated
10 Mar 2026
Results posted
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 LX107 Injection in people with Retinal Dystrophy, 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?

The sponsor lists 19 inclusion and 0 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 10 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?

Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] (time frame: 6 Months). 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 NCT07063030.

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 10 Mar 2026.

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