X Linked RetinoschisisEarly Phase 1NCT06345898
Safety and Efficacy of a Single Subretinal Injection of JWK002 Gene Therapy in Subjects With X-linked Retinoschisis(XLRS)
A Early Phase 1 study testing JWK002 in people with X Linked Retinoschisis, run by West China Hospital at 1 site in China.
Trial summary
This trial is to evaluate the safety and efficacy of JWK002 treatment of X-linked retinoschisis(XLRS). This study will enroll subjects aged 5-18 years old to receive a sub-retinal injection of JWK002. Read more about this study
- Who is it for?
- Ages 5 to 18 years, men only
- Conditions the study is for
- X Linked Retinoschisis
- What is being tested?
- JWK002
- 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
- 17 Nov 2023to 30 Nov 2033
- 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 5 to 18 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Fully understand the purpose and requirements of the trial, voluntarily participate in the clinical trial and sign the informed consent, and be able to cooperate with the testing required in various studies according to the requirements of the program;
- Male, ≥5 years old and < 18 years old (subject to the date of signing the informed consent);
- Clinically diagnosed XLRS, identified by genetic testing as RS1 gene mutation, and do not carry other mutations causing ophthalmic genetic diseases;
- Best corrected visual acuity (BCVA) of the study eye ≤63 letters (equivalent to Snellen 20/63); Best corrected visual acuity (BCVA) ≥34 letters (equivalent to Snellen 20/200).
Who cannot join
- Carbonic anhydrase inhibitor treatment within 3 months;
- The presence of other conditions in the study eye that may cause vision loss (e.g., atrophy of the optic nerve, advanced glaucoma, uveitis, the presence of vesicular splitting chambers or vesicular retinal detachment that may affect the subretinal injection operator);
- Those who study the retinal observation and examination caused by lens, cornea or other refractive interstitial turbidity;
- There are eye conditions affecting subretinal injection or eye conditions affecting the determination of the study endpoint;
- Patients who had undergone internal eye surgery within 6 months;
- Patients with AAV8 neutralizing antibody titer ≥1:1000;
Show all 13Show fewer
- Have previously received any gene therapy or cell therapy;
- fertile subjects are unwilling to use contraception;
- The presence of any of the following: an active infection that the investigator believes may affect the patient's participation in the study or affect the study results that requires systematic treatment; Hepatitis B surface antigen positive, hepatitis B virus deoxyribonucleic acid (HBV DNA) copy number > ULN; Hepatitis C virus (HCV) antibody positive and HCV-RNA copy number >ULN; Treponema pallidum antibody positive; Positive for human immunodeficiency virus (HIV) antibodies;
- Malignant tumors (basal cell or squamous cell skin cancer) were diagnosed within 5 years prior to screening;
- has or has had a systemic immune system disease;
- Laboratory values considered to be clinically significant were abnormal: alanine and/or aspartate transaminase >2.5×ULN, total bilirubin >1.5×ULN, serum creatinine >1.5×ULN, prothrombin time ≥1.5×ULN, activated partial thromboplastin time ≥1.5×ULN; (13) have a severe allergy or a known allergy to the drugs used in the treatment or examination in the study protocol, including the investigational drug allergy;
- Other situations that the investigator considers inappropriate for participation in this study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Licong Liang contact
Kaiqin She contact
China1 site, 1 recruiting
- Recruiting
West China Hospital
Chengdu, Sichuan, 610023
Licong Liang, 932968425@qq.com
Fang Lu, lufang@wchscu.cn
About this study
Official title: Clinical Study on the Safety and Efficacy of JWK002 Injection Administered as a Single Subretinal Injection in Subjects With X-linked Retinoschisis
This trial is to evaluate the safety and efficacy of JWK002 treatment of X-linked retinoschisis(XLRS). This study will enroll subjects aged 5-18 years old to receive a sub-retinal injection of JWK002.
- Conditions
- X Linked Retinoschisis
- Being tested
- JWK002
- Keywords
- X Linked Retinoschisis
- Medical subject headings
- Retinoschisis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
JWK002Drug
Subretinal administration of gene therapy vector JWK002 to one eye.
| Study group | Role | What they receive |
|---|---|---|
| JWK002 at Low Dose Group | Experimental | Subretinal injection low dose of JWK002 in one eye Receives: Drug: JWK002 |
| JWK002 at Medium Dose Group | Experimental | Subretinal injection medium dose of JWK002 in one eye Receives: Drug: JWK002 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety(Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events)
The primary outcome measures are safety, determined by the number of ocular and non-ocular Study Drug-related adverse events (SDAE), treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs).
Measured: baseline to day 7, day 14, month 1, 3, 6, 12
Secondary outcomes (4)
Best corrected visual acuity (BCVA)
Visual acuity of the study eye was assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS)
Measured: baseline to day 7, day 14, month 1, 2, 3, 6, 12
Full-field electroretinogram(ff-ERG)
The ERG measurement will be performed based on the standards of international society for clinical electrophysiology of vision (ISCEV).
Measured: baseline to month 1, 3, 6, 12
Macular structure as assessed by swept source optical coherence tomography
Change in swept source optical coherence tomography(SS-OCT)
Measured: baseline to month 1, 3, 6, 12
Visual field
Change in visual field assessed by Humphrey perimetry
Measured: baseline to month 1, 3, 6, 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2024
- Study start17 Nov 2023
- Primary completion30 Nov 2033 (estimated)
- Study completion30 Nov 2033 (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 West China HospitalThe 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- West China Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Fang Lu, West China Hospital
- Investigators
- Fang Lu, Principal investigator, lufang@wchscu.cn
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06345898
- Sponsor's protocol ID
- 2023-1479
- Status last verified
- Feb 2025
- First submitted
- 28 Mar 2024
- Last updated
- 7 Feb 2025
- 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 7 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study on the Safety and Efficacy of JWK002 Injection Administered as a Single Subretinal Injection in Subjects With X-linked Retinoschisis
- ClinicalTrials.gov ID
- NCT06345898
- Acronym / sponsor ID
- 2023-1479
- Conditions
- X Linked Retinoschisis
- Keywords
- X Linked Retinoschisis
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- West China HospitalUniversity, hospital or other
- Study start
- 17 Nov 2023 (actual)
- Primary completion
- 30 Nov 2033 (estimated)
- Study completion
- 30 Nov 2033 (estimated)
- First posted
- 3 Apr 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.
This trial is to evaluate the safety and efficacy of JWK002 treatment of X-linked retinoschisis(XLRS). This study will enroll subjects aged 5-18 years old to receive a sub-retinal injection of JWK002.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| JWK002 at Low Dose Group | Experimental | Drug: JWK002Subretinal injection low dose of JWK002 in one eye |
| JWK002 at Medium Dose Group | Experimental | Drug: JWK002Subretinal injection medium dose of JWK002 in one eye |
Who may qualify
- Ages
- 5 to 18 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Fully understand the purpose and requirements of the trial, voluntarily participate in the clinical trial and sign the informed consent, and be able to cooperate with the testing required in various studies according to the requirements of the program;
- Male, ≥5 years old and < 18 years old (subject to the date of signing the informed consent);
- Clinically diagnosed XLRS, identified by genetic testing as RS1 gene mutation, and do not carry other mutations causing ophthalmic genetic diseases;
- Best corrected visual acuity (BCVA) of the study eye ≤63 letters (equivalent to Snellen 20/63); Best corrected visual acuity (BCVA) ≥34 letters (equivalent to Snellen 20/200).
Key exclusion criteria (13 listed)
- Carbonic anhydrase inhibitor treatment within 3 months;
- The presence of other conditions in the study eye that may cause vision loss (e.g., atrophy of the optic nerve, advanced glaucoma, uveitis, the presence of vesicular splitting chambers or vesicular retinal detachment that may affect the subretinal injection operator);
- Those who study the retinal observation and examination caused by lens, cornea or other refractive interstitial turbidity;
- There are eye conditions affecting subretinal injection or eye conditions affecting the determination of the study endpoint;
- Patients who had undergone internal eye surgery within 6 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 |
|---|---|---|
| JWK002Subretinal administration of gene therapy vector JWK002 to one eye. | Drug | JWK002 at Low Dose Group, JWK002 at Medium Dose Group |
Primary outcome
Safety(Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events)
Time frame: baseline to day 7, day 14, month 1, 3, 6, 12
The primary outcome measures are safety, determined by the number of ocular and non-ocular Study Drug-related adverse events (SDAE), treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs).
4 secondary outcomes
Best corrected visual acuity (BCVA)
Time frame: baseline to day 7, day 14, month 1, 2, 3, 6, 12
Full-field electroretinogram(ff-ERG)
Time frame: baseline to month 1, 3, 6, 12
Macular structure as assessed by swept source optical coherence tomography
Time frame: baseline to month 1, 3, 6, 12
Visual field
Time frame: baseline to month 1, 3, 6, 12
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 7 Feb 2025.
Related trials for X Linked Retinoschisis
Active, not recruitingEarly Phase 1
Safety and Efficacy Study of IVB102 Injection in Subjects With X-linked Retinoschisis
Retinoschisis, Retinal Disease
RecruitingPhase 3
ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
X-linked Retinoschisis
RecruitingObservational study
Inherited Retinal Degenerative Disease Registry
Eye Diseases Hereditary, Retinal Disease
RecruitingObservational study
Development and Evaluation of Functional Visual Field and Navigation Endpoints in Moderate to Profound Inherited Retinal Disease (DEFINE-IRD)
Retinitis Pigmentosa, Stargardt Macular Dystrophy
RecruitingObservational study
Observational Study to Assess Endpoint Operational Feasibility & Measurement Properties in Patients with Retinal Degeneration
Retinitis Pigmentosa, Choroideremia
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.
Licong Liang contact
Kaiqin She contact
Fang Lu principal investigator
lufang@wchscu.cn
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing JWK002 in people with X Linked Retinoschisis, run by West China Hospital 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 4 inclusion and 13 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 7 Feb 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 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?
Contact Licong Liang (932968425@qq.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety(Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events) (time frame: baseline to day 7, day 14, month 1, 3, 6, 12). 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 7 Feb 2025.