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Stargardt Disease 1Phase 1/2NCT06300476

Safety and Efficacy of a Single Subretinal Injection of JWK006 Gene Therapy in Subjects With Stargardt Disease(STGD1)

A Phase 1/2 study testing JWK006 in people with Stargardt Disease 1, run by West China Hospital at 1 site in China.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

The purpose of the study is to assess the safety and efficacy of JWK006 in Stargardt Disease(STGD1). JWK006 is packed by adeno-associated virus vector that expressing ABCA4 gene. Read more about this study

Who is it for?
Ages 10 to 18 years
Conditions the study is for
Stargardt Disease 1
What is being tested?
JWK006
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
9 participants
Where
1 siteChina
Study dates
20 Nov 2023to 30 Dec 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 10 to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Fully understand the purpose and requirements of this trial, voluntarily participate in the clinical trial and sign the informed consent form (underage subjects must be signed by their guardians), and be able to cooperate with the tests required in various studies according to the protocol requirements;
  • Aged 10 to 18 years old (including the critical value, subject to the time of signing the informed consent form), regardless of gender;
  • Clinical diagnosis of Stargardt disease(STGD1), macular changes consistent with the clinical manifestations of Stargardt disease(STGD1);
  • The biallelic ABCA4 pathogenic mutation has been confirmed by genetic testing and does not carry other pathogenic mutations for ophthalmic genetic diseases;
  • The best corrected visual acuity of the study eye is ≤ETDRS 68 letters (20/50); the best corrected visual acuity of the fellow eye is ≥20/400.

Who cannot join

  • 1) The study eye has other conditions that may cause vision loss (such as optic atrophy, advanced glaucoma, and uveitis); (2) The presence of lens, cornea or other refractive stromal opacity in the study eye affects retinal observation and examination; (3) There are eye conditions that affect subretinal injection, or eye conditions that affect the judgment of the study endpoint; (4) Those who have undergone intraocular surgery in the study eye within 6 months; (5) Patients with AAV8 neutralizing antibody titer ≥1:1000; (6) Have received any gene therapy or cell therapy in the past; (7) Subjects of childbearing age are unwilling to use contraceptive measures; (8) One of the following situations exists: the researcher believes that there is an active infection that may affect the patient's participation in the study or that affects the study results and requires systemic treatment; hepatitis B surface antigen is positive and hepatitis B virus deoxyribonucleic acid (HBV DNA) copies number > ULN; hepatitis C virus (HCV) antibody positive, and HCV-RNA copy number > ULN; Treponema pallidum antibody positive; human immunodeficiency virus (HIV) antibody positive; (9) Malignant tumors diagnosed within 5 years before screening (except for adequately treated cervical carcinoma in situ, basal cell or squamous epithelial cell skin cancer, and breast ductal carcinoma in situ after radical mastectomy); (10) Suffering or having suffered from systemic immune system diseases; (11) Abnormal laboratory values considered to be clinically significant: alanine aminotransferase and/or aspartate aminotransferase >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; (12) There is severe allergy or known allergy to the drugs used for treatment or examination in the research protocol, including allergy to study drugs; (13) Pregnant and lactating women; subjects with childbearing potential who are unable to take effective contraceptive measures within 2 weeks before screening to 6 months after administration; (14) Other circumstances that the researcher believes are not suitable for participating in this study.

Trial locations and contacts

1 site in 1 country.

China1 site

  • West China Hospital

    Chengdu, Sichuan, 610044

Other trials in China

About this study

The purpose of the study is to assess the safety and efficacy of JWK006 in Stargardt Disease(STGD1).

JWK006 is packed by adeno-associated virus vector that expressing ABCA4 gene.

Read the detailed description

This clinical study is a single-center, non-randomized, non-blinded, self-controlled, dose-escalation clinical study, aiming to evaluate the safety, tolerability, and preliminary effectiveness of JWK006 injection in the treatment of approximately 12 patients(aged 10 to 18 years) with Stargardt Disease(STGD1),genotyped with ABCA4 gene mutations.

In this study, a "3+3" dose escalation trial was adopted, with 3 doses (low dose, medium dose, and high dose) set up, and 9 subjects were planned to be enrolled, 3 in each dose group. Following the principle of dose escalation, starting from the low-dose treatment group, one subject was enrolled in each dose group. In order to fully protect the safety of subjects, it is required to adopt an orderly enrollment method within each dose group, with one subject enrolled at a time. No obvious toxic or side effects are observed 28 days after the completion of the administration. The next subject in the group. At the same time, 28 days after the last subject in each dose group is administered, the researchers and others will jointly evaluate the safety results of the dose group before deciding whether to enter the next dose group.

Being tested
JWK006
Medical subject headings
Stargardt Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • JWK006Genetic

    Subretinal administration of gene therapy vector JWK006 to one eye.

Study groups in NCT06300476
Study groupRoleWhat they receive
Low dose groupExperimentalSubretinal injection low dose of JWK006 in one eye
Receives: Genetic: JWK006
Medium dose groupExperimentalSubretinal injection medium dose of JWK006 in one eye
Receives: Genetic: JWK006
High dose groupExperimentalSubretinal injection high dose of JWK006 in one eye
Receives: Genetic: JWK006

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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: 5 years (Screening to 5 years post JWK006 administration)

Secondary outcomes (4)

  1. Visual acuity

    Visual acuity of the study eye was assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS)

    Measured: 5 years (Screening to 5 years post JWK006 administration)

  2. Ophthalmoscope Measurement

    Usese Slit-lamp Biomicroscopy to visualize the anatomy of ocular structures before and after sub-retinal injections and follow-up visits.

    Measured: 5 years (Screening to 5 years post JWK006 administration)

  3. Qualitative and quantitative assessments of autofluorescence pattern (FAF)

    Changes in FAF intensity will be assessed from baseline measurements to assess damage to photoreceptors and retinal pigment epithelium cells.

    Measured: 5 years (Screening to 5 years post JWK006 administration)

  4. multifocal electroretinogram (mfERG)

    mfERG can be used to assess macular function of retina.

    Measured: 5 years (Screening to 5 years post JWK006 administration)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Mar 2024
  2. Study start20 Nov 2023
  3. Primary completion30 Dec 2026 (estimated)
  4. Study completion30 Dec 2029 (estimated)

Resources

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
Non-randomised
Intervention model
Sequential
Masking
Single blind
Who is masked
Participant
Enrolment
9 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06300476
Sponsor's protocol ID
2023-1511
Status last verified
Mar 2024
First submitted
3 Mar 2024
Last updated
8 Mar 2024
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing JWK006 in people with Stargardt Disease 1, 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 5 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 8 Mar 2024. 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(Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events) (time frame: 5 years (Screening to 5 years post JWK006 administration)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06300476.

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 8 Mar 2024.

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