X-linked RetinoschisisNCT05814952
Safety and Efficacy Study of LX103 Treatment of X-Linked Retinoschisis (XLRS)
A study testing LX103 Injection in people with X-linked Retinoschisis, run by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine at 1 site in China.
Trial summary
The goal of this study is to evaluate the safety and efficacy of LX103 treatment of X-linked retinoschisis. This study will enroll subjects aged ≥ 6 years old to receive a single unilateral intravitreal (IVT) injection of LX103 to evaluate its safety and efficacy. Read more about this study
- Who is it for?
- Ages 6 years and older, men only
- Conditions the study is for
- X-linked Retinoschisis
- What is being tested?
- LX103 Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteChina
- Study dates
- 13 Dec 2022to Aug 2028
- Phase
- Interventional
- 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
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Willing to sign the informed consent, and willing to attend follow-up visits.
- Male individual at least 6 years of age with a diagnosis of XLRS and documented mutations in the RS1 gene.
- The study eye must have a best-corrected E-ETDRS visual acuity letter score of less than or equal to 63.
Who cannot join
- Any eye with disease that would interfere with the fundus examinations.
- The study eye is receiving topical carbonic anhydrase inhibitor, or has received topical carbonic anhydrase inhibitors in the past three months.
- The study eye has undergone intraocular surgery within six months prior to enrollment.
- Participant has uncontrolled hypertension or diabetes.
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
- Recruiting
Shanghai General Hospital, Shanghai Jiao Tong University
Shanghai, 200080
Junran Sun, MD, +86 13661995933, emiliesun@sina.com
Xiaodong Sun, PhD
About this study
Official title: An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Efficacy of LX103 in Patients With X-Linked Retinoschisis (XLRS)
The goal of this study is to evaluate the safety and efficacy of LX103 treatment of X-linked retinoschisis. This study will enroll subjects aged ≥ 6 years old to receive a single unilateral intravitreal (IVT) injection of LX103 to evaluate its safety and efficacy.
- Conditions
- X-linked Retinoschisis
- Being tested
- LX103 Injection
- Medical subject headings
- Retinoschisis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LX103 InjectionGenetic
Qualified subjects will receive a single unilateral intravitreal injection of LX103 at Day 0 in the trial.
| Study group | Role | What they receive |
|---|---|---|
| LX103 Injection | Experimental | Potential doses:
5E10 vg, 0.1 mL/eye/dose (low dose)
1E11 vg, 0.05 mL/eye/dose (high dose) Receives: Genetic: LX103 Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events (AEs)
Incidence of adverse events (AEs) within 24 weeks of LX103 intravitreal injection at different doses
Measured: 24 weeks
Incidence of serious adverse events (SAEs)
Incidence of serious adverse events (SAEs) within 24 weeks of LX103 intravitreal injection at different doses
Measured: 24 weeks
Secondary outcomes (3)
Mean change from baseline in BCVA (LogMAR) in the study eye
ETDRS visual acuity charts will be used to assess BCVA
Measured: 24 weeks, 52 weeks
Mean change from baseline in central subfield thickness (CST) in the study eye
Mean change of CST measured using Optical Coherence Tomography (OCT)
Measured: 24 weeks, 52 weeks
Change from baseline in Electroretinogram (ERG) parameters in the study eye
Change in dark-adapted 3.0 B-wave amplitude (μV)
Measured: 24 weeks, 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Apr 2023
- Study start13 Dec 2022
- Primary completionAug 2024 (estimated)
- Study completionAug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for X-linked RetinoschisisStudies of the same condition, open and under way.
- More trials run by Shanghai General Hospital, Shanghai Jiao Tong University School of MedicineThe sponsor's other registered studies.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Innostellar Biotherapeutics
- Responsible party
- Principal investigator, Xiaodong Sun, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05814952
- Sponsor's protocol ID
- SHGH-LX103
- Status last verified
- Apr 2023
- First submitted
- 4 Apr 2023
- Last updated
- 18 Apr 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Efficacy of LX103 in Patients With X-Linked Retinoschisis (XLRS)
- ClinicalTrials.gov ID
- NCT05814952
- Acronym / sponsor ID
- SHGH-LX103
- Conditions
- X-linked Retinoschisis
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Shanghai General Hospital, Shanghai Jiao Tong University School of MedicineUniversity, hospital or other
- Collaborators
- Innostellar Biotherapeutics
- Study start
- 13 Dec 2022 (actual)
- Primary completion
- Aug 2024 (estimated)
- Study completion
- Aug 2028 (estimated)
- First posted
- 18 Apr 2023 (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 goal of this study is to evaluate the safety and efficacy of LX103 treatment of X-linked retinoschisis. This study will enroll subjects aged ≥ 6 years old to receive a single unilateral intravitreal (IVT) injection of LX103 to evaluate its safety and efficacy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| LX103 Injection | Experimental | Genetic: LX103 InjectionPotential doses: 5E10 vg, 0.1 mL/eye/dose (low dose) 1E11 vg, 0.05 mL/eye/dose (high dose) |
Who may qualify
- Ages
- 6 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Willing to sign the informed consent, and willing to attend follow-up visits.
- Male individual at least 6 years of age with a diagnosis of XLRS and documented mutations in the RS1 gene.
- The study eye must have a best-corrected E-ETDRS visual acuity letter score of less than or equal to 63.
Key exclusion criteria (4 listed)
- Any eye with disease that would interfere with the fundus examinations.
- The study eye is receiving topical carbonic anhydrase inhibitor, or has received topical carbonic anhydrase inhibitors in the past three months.
- The study eye has undergone intraocular surgery within six months prior to enrollment.
- Participant has uncontrolled hypertension or diabetes.
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 |
|---|---|---|
| LX103 InjectionQualified subjects will receive a single unilateral intravitreal injection of LX103 at Day 0 in the trial. | Genetic | LX103 Injection |
Primary outcome
Incidence of adverse events (AEs)
Time frame: 24 weeks
Incidence of adverse events (AEs) within 24 weeks of LX103 intravitreal injection at different doses
Incidence of serious adverse events (SAEs)
Time frame: 24 weeks
Incidence of serious adverse events (SAEs) within 24 weeks of LX103 intravitreal injection at different doses
3 secondary outcomes
Mean change from baseline in BCVA (LogMAR) in the study eye
Time frame: 24 weeks, 52 weeks
Mean change from baseline in central subfield thickness (CST) in the study eye
Time frame: 24 weeks, 52 weeks
Change from baseline in Electroretinogram (ERG) parameters in the study eye
Time frame: 24 weeks, 52 weeks
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 18 Apr 2023.
- Individual participant dataSharing planned: No
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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.
Shanghai General Hospital, Shanghai Jiao Tong University
Shanghai, China
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing LX103 Injection in people with X-linked Retinoschisis, 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 3 inclusion and 4 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 18 Apr 2023. 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.
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 adverse events (AEs) (time frame: 24 weeks). 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 18 Apr 2023.