NCT06424301 Targeting NUDT21 siRNA Drugs for Patients With Refractory Retinoblastoma
| NCT ID | NCT06424301 |
| Status | Recruiting |
| Phase | EARLY_Phase 1 |
| Sponsor | Eye & ENT Hospital of Fudan University |
| Condition | Retinoblastoma |
| Study Type | INTERVENTIONAL |
| Enrollment | 2 participants |
| Start Date | 2024-12-20 |
| Primary Completion | 2025-09-30 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 2 participants in total. It began in 2024-12-20 with a primary completion date of 2025-09-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
Retinoblastoma (RB) is the most common intraocular malignancy in children, accounting for approximately 11% of all cancers diagnosed in children under the age of one. Although its incidence is relatively low-about 1 in 15,000 to 20,000 live births-RB has a high risk of intracranial metastasis via the optic nerve, often leading to poor prognosis in advanced cases. Recent advances in administration routes, such as intravitreal and intra-arterial chemotherapy, have significantly improved eye preservation rates. However, these strategies are limited by cumulative retinal toxicity and drug resistance. In refractory cases, enucleation remains the only definitive treatment to prevent extraocular spread and death. In light of these challenges, current research efforts are focused on developing novel targeted therapies that enhance anti-tumor efficacy while minimizing local toxicity. In this context, we introduce a first-in-class siRNA-based drug targeting NUDT21, which promotes tumor regression by modulating the 3'UTR tail of SMC1A, thereby suppressing tumor cell proliferation. Importantly, the siRNA drug selectively targets tumor cells, offering a favorable safety profile compared to conventional chemotherapeutic regimens. Given that both the target (NUDT21) and the mode of administration (intraocular siRNA injection) are novel in retinoblastoma treatment, there is an urgent need for early-phase investigator-initiated clinical research. This study is therefore designed to assess the short-term safety and preliminary efficacy of NUDT21 siRNA in patients with refractory retinoblastoma, and to provide an evidence base for future large-scale clinical trials.
Eligibility Criteria
Inclusion Criteria: 1. Patients with retinoblastoma with a somatic mutation of the gene RB1 and active tumor in a single eye, or germinal mutation of RB1 with active tumor/s in an eye and the contralateral eye unaffected, enucleated or without tumor activity. In both cases, relapsed or refractory with the use of systemic, intraarterial or intravitreal chemotherapy or radiotherapy, in accordance with the availability at his/her referral site, in whom enucleation is the only recommended treatment under opinion of the medical team treating the case at the originating referral site. But the patient has a strong desire to preserve the eye. 2. Normal renal function: serum creatinine: \< 45 μmol/L (0-2 years); \< 57 μmol/L (3-6 years); \< 60 μmol/L (7-10 years); \< 80 μmol/L (11-13 years). 3. Normal Hepatic function: serum ALT: \< 0,52 μkat/L (de 9 months -12 years); serum AST: 61-80 g/L (8 months - 5 years); 63-83 g/L (5-9 years); 63-82 g/L (9-12 years). 4. Age greater than 6 months at the time of inclusion in the study. 5. Sign the informed consent form and be willing to follow up at the specified time. Exclusion Criteria: 1. Presence of factors that require immediate enucleation of the affected eye such as glaucoma, rubeosis iridis, anterior chamber involvement. 2. Comorbidities: Uncontrolled epilepsy with anticonvulsant treatment, cardiac disease not compensated by treatment. 3. Active Infections. 4. Other chronic or active acute diseases that under the criterion of the researcher were an exclusion criterion. 5. History of having received attenuated or live vaccines in the 30 days prior to inclusion in the study. 6. Any cause of Immunosuppression. 7. Trilateral Retinoblastoma. 8. Extraocular spread. 9. History of having received treatment for retinoblastoma with chemotherapy or radiation therapy by any means within 30 days prior to inclusion in the study. 10. Patients who can not complete the study procedures.
Contact & Investigator
Chen Zhao, MD
PRINCIPAL INVESTIGATOR
Fudan Eye & ENT Hospital
Frequently Asked Questions
Who can join the NCT06424301 clinical trial?
This trial is open to participants of all sexes, aged 6 Months or older, studying Retinoblastoma. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06424301 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT06424301 currently recruiting?
Yes, NCT06424301 is actively recruiting participants. Contact the research team at dr_zhaochen@fudan.edu.cn for enrollment information.
Where is the NCT06424301 trial being conducted?
This trial is being conducted at Shanghai, China.
Who is sponsoring the NCT06424301 clinical trial?
NCT06424301 is sponsored by Eye & ENT Hospital of Fudan University. The principal investigator is Chen Zhao, MD at Fudan Eye & ENT Hospital. The trial plans to enroll 2 participants.