Inherited Retinal DystrophyPhase 1/2NCT06212297
Fellow-eye Study (FE) of LX101 in Subjects With Inherited Retinal Dystrophy
A Phase 1/2 study testing LX101 in people with Inherited Retinal Dystrophy, run by Innostellar Biotherapeutics Co.,Ltd at 1 site in China.
Trial summary
Up to nine subjects who have participated in the earlier LX101 clinical study, and who meet all study eligibility criteria, will receive LX101 administration in the previously uninjected, contralateral eye to evaluate the safety of bilateral, sequential subretinal administration of LX101. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Inherited Retinal Dystrophy
- What is being tested?
- LX101
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteChina
- Study dates
- 12 Sep 2023to Mar 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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Prior subretinal administration of LX101 (unilateral)
- ≥ 6 years old Signed written informed consent
Who cannot join
- Prior gene therapy except LX101 Active intraocular or periocular infections Lacking of sufficient surviving retinal cells Prior ocular surgery within six months Retinoid like compounds or precursors were taken within three months Complicating systemic diseases Clinically significant abnormal baseline laboratory values Using of any retinal toxic compounds
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Clinical Trial to Evaluate the Safety and Efficacy of Subretinal Re-Administration of LX101 to the Contralateral Eye in Subjects With Biallelic RPE65 Mutation-associated Inherited Retinal Dystrophy
Up to nine subjects who have participated in the earlier LX101 clinical study, and who meet all study eligibility criteria, will receive LX101 administration in the previously uninjected, contralateral eye to evaluate the safety of bilateral, sequential subretinal administration of LX101.
- Conditions
- Inherited Retinal Dystrophy
- Being tested
- LX101
- Keywords
- RPE65 Mutations
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 contralateral, previously uninjected eye
| Study group | Role | What they receive |
|---|---|---|
| LX101 Subretinal Administration | Experimental | Not described Receives: Genetic: LX101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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: 6 months
Secondary outcomes (3)
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、12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jan 2024
- Study start12 Sep 2023
- Primary completion6 Feb 2025
- Study completionMar 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 DystrophyStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
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
- NCT06212297
- Sponsor's protocol ID
- INNOSTELLAR-LX101-2
- Status last verified
- Jun 2025
- First submitted
- 25 Dec 2023
- Last updated
- 29 Jun 2025
- 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 29 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Clinical Trial to Evaluate the Safety and Efficacy of Subretinal Re-Administration of LX101 to the Contralateral Eye in Subjects With Biallelic RPE65 Mutation-associated Inherited Retinal Dystrophy
- ClinicalTrials.gov ID
- NCT06212297
- Acronym / sponsor ID
- INNOSTELLAR-LX101-2
- Conditions
- Inherited Retinal Dystrophy
- Keywords
- RPE65 Mutations
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Innostellar Biotherapeutics Co.,LtdIndustry (pharma, biotech, devices)
- Study start
- 12 Sep 2023 (actual)
- Primary completion
- 6 Feb 2025 (actual)
- Study completion
- Mar 2029 (estimated)
- First posted
- 18 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.
Up to nine subjects who have participated in the earlier LX101 clinical study, and who meet all study eligibility criteria, will receive LX101 administration in the previously uninjected, contralateral eye to evaluate the safety of bilateral, sequential subretinal administration of LX101.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| LX101 Subretinal Administration | 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 (2 listed)
- Prior subretinal administration of LX101 (unilateral)
- ≥ 6 years old Signed written informed consent
Key exclusion criteria (1 listed)
- Prior gene therapy except LX101 Active intraocular or periocular infections Lacking of sufficient surviving retinal cells Prior ocular surgery within six months Retinoid like compounds or precursors were taken within three months Complicating systemic diseases Clinically significant abnormal baseline laboratory values Using of any retinal toxic compounds
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 contralateral, previously uninjected eye | Genetic | LX101 Subretinal Administration |
Primary outcome
Safety: Incidence of adverse events (AEs) and serious adverse events (SAEs)
Time frame: 6 months
Incidence of ocular and non-ocular AEs and SAEs following LX102 subretinal injection
3 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、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 29 Jun 2025.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing LX101 in people with Inherited Retinal Dystrophy, run by Innostellar Biotherapeutics Co.,Ltd 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 2 inclusion and 1 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 29 Jun 2025. 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 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?
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?
Safety: Incidence of adverse events (AEs) and serious adverse events (SAEs) (time frame: 6 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 29 Jun 2025.