Targeting NUDT21 siRNA Drugs for Patients With Refractory Retinoblastoma

Status Not yet recruiting

Clinical Trial Summary

Retinoblastoma (RB) is the most common intraocular malignancy in children and accounts for 11% of all cancers in children under the age of 1 year. Although the incidence of RB is low, approximately 1/15,000 ~ 1/20,000, it tends to metastasize to the intracranial area through the optic nerve pathway leading to poor prognosis for patients with RB. Currently, with the emergence of new administration routes, such as intravitreal and intrarterial chemotherapy, the rate of eye preservation has been effectively improved. However, the use of high doses of chemotherapeutic agents may lead to visual impairments due to long-term retinal toxicity and some tumors recur or become resistant to chemotherapeutic agents after treatment. In such cases, ocular resection is the only option to prevent extraocular metastasis and death. Therefore, studies on retinoblastoma are currently focused on finding new targeted therapies to increase anti-tumor activity and reduce side effects. In this study, a novel targeting NUDT21 siRNA drug will be used to treat patients with refractory retinoblastoma. This drug promotes tumor apoptosis by regulating the 3'UTR plus tail of SMC1A, which makes the proliferative activity of tumor cells weaken and achieves tumor control. At the same time, since the targeted drug only focuses on tumor cells, it has reduced side effects compared with existing local chemotherapy regimens. Based on the above background, this study will explore the feasibility and effectiveness of intravitreal injection of NUDT21 siRNA in patients with refractory retinoblastoma through a two-center prospective study.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT06424301
Study type	Interventional
Source	Eye & ENT Hospital of Fudan University
Contact	Ruiqi Ma, MD
Phone	86-021-64377134
Email	ruiqi_ma@fudan.edu.cn
Status	Not yet recruiting
Phase	Early Phase 1
Start date	May 21, 2024
Completion date	May 21, 2027

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