Inherited Retinal Dystrophy Associated With RPE65 MutationsPhase 3NCT07054632
Efficacy and Safety of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations
A Phase 3 study testing LX101 in people with Inherited Retinal Dystrophy Associated With RPE65 Mutations, run by Innostellar Biotherapeutics Co.,Ltd at 4 sites in China.
Trial summary
The purpose of the study is to evaluate the efficacy and safety of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy. This is an open-label, multicenter, randomized controlled Phase III clinical trial. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Inherited Retinal Dystrophy Associated With RPE65 Mutations
- What is being tested?
- LX101
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 4 sitesChina
- Study dates
- 13 Sep 2023to Aug 2029
- Phase
- Phase 3
- 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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject and/or their guardian signing a written informed consent.
- Diagnosed with biallelic RPE65 mutation-associated inherited retinal dystrophy.
- Subjects are 6 years of age or older.
- Visual acuity of ≤ 20/63 or visual field less than 20 degrees in the eye to be injected.
Who cannot join
- Prior gene therapy for IRD and other hereditary eye diseases.
- Pre-existing eye conditions that would interfere with interpretation of study endpoints.
- Active intraocular or periocular infections in the study eye.
- Lacking of sufficient surviving retinal cells.
- Prior ocular surgery within six months.
- Complicating systemic diseases or clinically significant abnormal baseline laboratory values.
- Pre-existing systemic diseases that should not discontinue the use of any retinal toxic compounds.
- Complicating systemic diseases or clinically significant abnormal baseline laboratory values.
Trial locations and contacts
4 sites in 1 country.
China4 sites
Southwest Hospital of AMU
Chongqing
Zhongshan Ophthalmic Center, Sun Yat sen University
Guangzhou, Guangdong
Shanghai General Hospital
Shanghai
Tianjin Medical University Ophthalmology Hospital
Tianjin
About this study
Official title: Efficacy and Safety of Gene Therapy rAAV-RPE65 (LX101) in Biallelic RPE65 Mutation-associated Inherited Retinal Dystrophy: a Phase III, Multicenter, Randomized Controlled Trial (STAR)
The purpose of the study is to evaluate the efficacy and safety of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy. This is an open-label, multicenter, randomized controlled Phase III clinical trial. Subjects were randomly assigned in a 1:1 ratio to either the intervention group or the control group. Subjects in the intervention group received subretinal injection of LX101, while those in the control group received no treatment.
- Being tested
- LX101
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LX101Genetic
Subretinal administration of LX101 to the study eye
| Study group | Role | What they receive |
|---|---|---|
| Intervention group | Experimental | Not described Receives: Genetic: LX101 |
| Control group | No intervention | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mobility Test
Changes in functional vision from baseline, determined by mobility test score
Measured: 12 months
Secondary outcomes (4)
Full-field Light Sensitivity Threshold (FST) Test
Changes in light sensitivity from baseline, assessed by FST in log cd.s/m2
Measured: 6 months、12 months
Visual Acuity
Changes in visual acuity from baseline, based on the ability to read letters using the Early Treatment Diabetic Retionpathy Study (ETDRS) chart
Measured: 6 months、12 months
Mobility Test
Changes in functional vision from baseline, determined by mobility test score
Measured: 6 months
Safety: Incidence of adverse events (AEs) and serious adverse events (SAEs)
Incidence of ocular and non-ocular AEs and SAEs following LX102 subretinal injection
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jul 2025
- Study start13 Sep 2023
- Primary completion13 Aug 2025
- Study completionAug 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Inherited Retinal Dystrophy Associated With RPE65 MutationsStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Innostellar Biotherapeutics Co.,Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07054632
- Sponsor's protocol ID
- INNOSTELLAR-LX101-1 (stage 2)
- Status last verified
- Mar 2026
- First submitted
- 27 Jun 2025
- Last updated
- 5 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Gene Therapy rAAV-RPE65 (LX101) in Biallelic RPE65 Mutation-associated Inherited Retinal Dystrophy: a Phase III, Multicenter, Randomized Controlled Trial (STAR)
- ClinicalTrials.gov ID
- NCT07054632
- Acronym / sponsor ID
- INNOSTELLAR-LX101-1 (stage 2)
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Innostellar Biotherapeutics Co.,LtdIndustry (pharma, biotech, devices)
- Study start
- 13 Sep 2023 (actual)
- Primary completion
- 13 Aug 2025 (actual)
- Study completion
- Aug 2029 (estimated)
- First posted
- 8 Jul 2025 (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.
The purpose of the study is to evaluate the efficacy and safety of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy. This is an open-label, multicenter, randomized controlled Phase III clinical trial. Subjects were randomly assigned in a 1:1 ratio to either the intervention group or the control group. Subjects in the intervention group received subretinal injection of LX101, while those in the control group received no treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Intervention group | Experimental | Genetic: LX101 |
| Control group | No intervention | – |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Subject and/or their guardian signing a written informed consent.
- Diagnosed with biallelic RPE65 mutation-associated inherited retinal dystrophy.
- Subjects are 6 years of age or older.
- Visual acuity of ≤ 20/63 or visual field less than 20 degrees in the eye to be injected.
Key exclusion criteria (8 listed)
- Prior gene therapy for IRD and other hereditary eye diseases.
- Pre-existing eye conditions that would interfere with interpretation of study endpoints.
- Active intraocular or periocular infections in the study eye.
- Lacking of sufficient surviving retinal cells.
- Prior ocular surgery within six months.
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 |
|---|---|---|
| LX101Subretinal administration of LX101 to the study eye | Genetic | Intervention group |
Primary outcome
Mobility Test
Time frame: 12 months
Changes in functional vision from baseline, determined by mobility test score
4 secondary outcomes
Full-field Light Sensitivity Threshold (FST) Test
Time frame: 6 months、12 months
Visual Acuity
Time frame: 6 months、12 months
Mobility Test
Time frame: 6 months
Safety: Incidence of adverse events (AEs) and serious adverse events (SAEs)
Time frame: 12 months
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 5 Mar 2026.
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing LX101 in people with Inherited Retinal Dystrophy Associated With RPE65 Mutations, run by Innostellar Biotherapeutics Co.,Ltd at 4 sites 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 4 inclusion and 8 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: under way, not looking for participants, as of the sponsor's last update on 5 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 4 sites 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?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mobility Test (time frame: 12 months). 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 5 Mar 2026.