New treatment for children with hard-to-treat retinoblastoma

Targeting NUDT21 siRNA Drugs for Patients With Refractory Retinoblastoma (A Prospective Pilot Study)

This study is testing a new drug for children with hard-to-treat eye cancer to see if it works better and has fewer side effects than standard chemotherapy.

Quick facts

• Phase: Early Phase 1
• Study type: Interventional
• Enrollment: 2 (estimated)
• Ages: 6 Months and up
• Sex: All
• Sponsor: Eye & ENT Hospital of Fudan University (Academic / other)
• Drugs / interventions: chemotherapy, radiation
• Locations: 1 site (Shanghai)
• Trial ID: NCT06424301 on ClinicalTrials.gov

What this trial studies

This clinical trial focuses on patients with refractory retinoblastoma, a serious eye cancer in children. It aims to evaluate the effectiveness of targeting NUDT21 siRNA drugs, which may enhance anti-tumor activity while minimizing side effects compared to traditional chemotherapy. The study includes patients with specific genetic mutations and active tumors who wish to preserve their eyes despite previous treatments failing. The trial is conducted at the Eye & ENT Hospital of Fudan University in Shanghai.

Who should consider this trial

Good fit: Ideal candidates are children with retinoblastoma who have specific genetic mutations and active tumors that have not responded to previous treatments.

Not a fit: Patients with retinoblastoma who do not have the required genetic mutations or those whose tumors are not active may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new option for preserving vision in children with refractory retinoblastoma.

How similar studies have performed: While there have been advancements in retinoblastoma treatment, this specific approach using NUDT21 siRNA drugs is novel and has not been extensively tested in prior studies.

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.

Where this trial is running

Shanghai

• Fudan Eye & ENT Hospital — Shanghai, China (Recruiting)

Study contacts

• Principal investigator: Chen Zhao, MD — Fudan Eye & ENT Hospital
• Study coordinator: Chen Zhao, MD

Conditions Retinoblastoma; Refractory; retinoblastoma; intravitreal Chemotherapy