Targeting NUDT21 SiRNA Drugs for Patients with Refractory Retinoblastoma

Launched by EYE & ENT HOSPITAL OF FUDAN UNIVERSITY · May 16, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment for children with refractory retinoblastoma, a type of eye cancer that can be very challenging to treat when it doesn't respond to standard therapies. The researchers are testing a drug called NUDT21 siRNA, which aims to help kill tumor cells while causing fewer side effects than traditional chemotherapy. The goal is to find a way to control the cancer without needing to remove the eye, which is often the last resort.

To participate in this trial, children aged 1 to 12 who have a specific genetic mutation related to retinoblastoma and an active tumor in one eye may be eligible. They should have already tried other treatments like chemotherapy or radiation but still wish to keep their eye. Participants will receive an injection of the new drug directly into the eye and will be monitored closely for its effects. This study is an important step in finding more effective and less harmful treatments for young patients facing this difficult disease.

Gender

ALL

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. Adequate marrow reserve manifested in an absolute neutrophil count \> 1000 / mm3, platelets \> 100,000 / mm3 and hemoglobin\> 8 g / dl, without transfusional or cytokine support at least one month prior to study entry.
• • 5. Age greater than 1 year and less than 12 years at the time of inclusion in the study.
• • 6. 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 for reasons psychological or Social.

Trial Officials