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Tests treatment safety and results for Oculocutaneous Albinism (OCA)
Official title Safety and Efficacy of a Single Suprachoroidal Injection of JWK010 Gene Therapy in Subjects With Oculocutaneous Albinism Type 1 (OCA1)
ClinicalTrials.gov ID: NCT07313618
What this study is testing
What is JWK010 gene therapy?
JWK010 gene therapy is an investigational medicine, being studied as a potential treatment for oculocutaneous albinism (oca).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Oculocutaneous albinism (OCA) is the most common type of albinism. People with OCA have little or no pigment (melanin) in their eyes, skin, and hair.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 5 to 12
You may be able to join if
- Fully understand the purpose and requirements of this trial, voluntarily participate in the clinical study and sign the informed consent form (for...
- Aged ≥5 years and ≤12years (inclusive of the threshold values, based on the date of signing the informed consent form), regardless of gender.
- Clinically diagnosed with OCA1A type, with ocular and cutaneous manifestations consistent with the clinical presentation of OCA1A.
- Confirmed by genetic testing to carry pathogenic mutations in both TYR alleles, without carrying pathogenic mutations associated with other...
- The visual acuity of the fellow eye is better than that of the study eye, and the visual acuity of the fellow eye is no less than 20/400
You likely can't join if
- Presence of any other condition in the study eye that may cause vision loss (e.g., optic atrophy, advanced glaucoma, uveitis).
- The presence of lens, cornea or other refractive stromal opacity in the study eye affects retinal observation and examination.
- Presence of ocular conditions that may affect suprachoroidal injection or the assessment of study endpoints.
- Have undergone intraocular surgery in the study eye within 6 months.
- Have received any gene therapy or cell therapy in the past.
- people with childbearing potential are unwilling to use contraceptive measures.
See the full eligibility criteria
- Fully understand the purpose and requirements of this trial, voluntarily participate in the clinical study and sign the informed consent form (for minor people, the informed consent form shall be signed by their...
- Aged ≥5 years and ≤12years (inclusive of the threshold values, based on the date of signing the informed consent form), regardless of gender.
- Clinically diagnosed with OCA1A type, with ocular and cutaneous manifestations consistent with the clinical presentation of OCA1A.
- Confirmed by genetic testing to carry pathogenic mutations in both TYR alleles, without carrying pathogenic mutations associated with other ophthalmic genetic diseases.
- The visual acuity of the fellow eye is better than that of the study eye, and the visual acuity of the fellow eye is no less than 20/400
- Presence of any other condition in the study eye that may cause vision loss (e.g., optic atrophy, advanced glaucoma, uveitis).
- The presence of lens, cornea or other refractive stromal opacity in the study eye affects retinal observation and examination.
- Presence of ocular conditions that may affect suprachoroidal injection or the assessment of study endpoints.
- Have undergone intraocular surgery in the study eye within 6 months.
- Have received any gene therapy or cell therapy in the past.
- people with childbearing potential are unwilling to use contraceptive measures.
- Presence of any of the following: active infection requiring systemic treatment which, in the opinion of the investigator, may affect the patient's participation or study results; positive hepatitis B surface antigen...
- Diagnosis of malignancy within 5 years prior to screening (except for adequately treated carcinoma in situ of the cervix, basal cell or squamous cell skin cancer, or ductal carcinoma in situ of the breast after radical...
- Suffering or having suffered from systemic immune system diseases.
- Abnormal laboratory values considered 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...
- There is severe allergy or known allergy to the drugs used for treatment or examination in the research protocol, including allergy to study drugs.
- Pregnant or lactating women; people of childbearing potential who are unable to use effective contraception from 2 weeks prior to screening until 6 months after administration.
- Other circumstances that the researcher believes are not suitable for participating in this study
The study team makes the final eligibility decision.
Where it's taking place
- Chengdu, Sichuan, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 5 years to 12 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Chengdu, Sichuan, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.