Inherited Retinal Dystrophy Associated With RPE65 MutationsPhase 1NCT06196827
Safety and Tolerability of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations
A Phase 1 study testing LX101 in people with Inherited Retinal Dystrophy Associated With RPE65 Mutations, run by Innostellar Biotherapeutics Co.,Ltd at 2 sites in China.
Trial summary
The purpose of the study is to evaluate the safety, tolerability and efficacy of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy. 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
- 9 participants
- Where
- 2 sitesChina
- Study dates
- 2 Jul 2022to Dec 2027
- Phase
- 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 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.
Trial locations and contacts
2 sites in 1 country.
China2 sites
Beijing Tongren Hospital, Capital Medical University
Beijing
Shanghai General Hospital
Shanghai
About this study
Official title: A Multi-center Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of rAAV2-RPE65 Gene Therapy (LX101) in Subjects With Biallelic RPE65 Mutation-associated Inherited Retinal Dystrophy
The purpose of the study is to evaluate the safety, tolerability and efficacy of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy.
- 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
| Study group | Role | What they receive |
|---|---|---|
| LX101 Dose 1 | Experimental | Not described Receives: Genetic: LX101 |
| LX101 Dose 2 | Experimental | Not described Receives: Genetic: LX101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events (AEs) and serious adverse events (SAEs)
Incidence of ocular and non-ocular AEs and SAEs following LX101 subretinal injection
Measured: 12 months
Incidence of dose-limiting toxicity (DLT)
Incidence of DLT following LX101 subretinal injection at different doses
Measured: 1 month
Secondary outcomes (2)
Efficacy of LX101 in study eye
Changes in BCVA from baseline
Measured: 12 months
Efficacy of LX101 in study eye
Changes in full-field stimulus threshold (FST) from baseline
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 posted9 Jan 2024
- Study start2 Jul 2022
- Primary completion6 Dec 2023
- Study completionDec 2027 (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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 9 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
- NCT06196827
- Sponsor's protocol ID
- INNOSTELLAR-LX101-1 (stage 1)
- Status last verified
- Mar 2026
- First submitted
- 25 Dec 2023
- Last updated
- 6 Mar 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of rAAV2-RPE65 Gene Therapy (LX101) in Subjects With Biallelic RPE65 Mutation-associated Inherited Retinal Dystrophy
- ClinicalTrials.gov ID
- NCT06196827
- Acronym / sponsor ID
- INNOSTELLAR-LX101-1 (stage 1)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Innostellar Biotherapeutics Co.,LtdIndustry (pharma, biotech, devices)
- Study start
- 2 Jul 2022 (actual)
- Primary completion
- 6 Dec 2023 (actual)
- Study completion
- Dec 2027 (estimated)
- First posted
- 9 Jan 2024 (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 safety, tolerability and efficacy of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| LX101 Dose 1 | Experimental | Genetic: LX101 |
| LX101 Dose 2 | Experimental | Genetic: LX101 |
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 (7 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 | Genetic | LX101 Dose 1, LX101 Dose 2 |
Primary outcome
Incidence of adverse events (AEs) and serious adverse events (SAEs)
Time frame: 12 months
Incidence of ocular and non-ocular AEs and SAEs following LX101 subretinal injection
Incidence of dose-limiting toxicity (DLT)
Time frame: 1 month
Incidence of DLT following LX101 subretinal injection at different doses
2 secondary outcomes
Efficacy of LX101 in study eye
Time frame: 12 months
Efficacy of LX101 in study eye
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 6 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.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing LX101 in people with Inherited Retinal Dystrophy Associated With RPE65 Mutations, run by Innostellar Biotherapeutics Co.,Ltd at 2 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 7 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 6 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 2 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 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.
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?
Incidence of adverse events (AEs) and serious adverse events (SAEs) (time frame: 12 months). The study lists 2 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 6 Mar 2026.