Neoplasms

Eye Diseases

All Conditions

Diseases and Abnormalities at or Before Birth

Rare Diseases

Retinoblastoma

Neoplasms, Neuroepithelial

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Retinal Neoplasms

Eye Neoplasms

Eye Diseases, Hereditary

Retinal Diseases

Neuroepithelioma

The enrolled patients underwent blood sample collection and clinical evaluation before the first injection of the targeted drug. Intravitreal injection of the targeted drug at a dosage of 100-1000 μg was performed on day 1 of week 1 and week 3, respectively. Collection of aqueous humor was performed on weeks 1, 3, and 7, respectively. Collection of blood samples and sputum specimens was performed on weeks 1, 3, 5, and 7. And clinical assessment was performed weekly with continuous follow-up until 12 weeks.

It is performed for intravitreal chemotherapy.

Targeting NUDT21 siRNA drugs

Ruiqi Ma, MD

Jiang Qian, MD

Kang Xue, MD

Kang Zhang, MD

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.

Targeting NUDT21 siRNA Drugs for Patients With Refractory Retinoblastoma

Targeting NUDT21 siRNA Drugs for Patients With Refractory Retinoblastoma (A Two-center Prospective Single Arm Trial)

Tumor response rate (ORR) to targeting NUDT21 siRNA at 28 days post-administration.

To detect the presence of drugs in blood samples and aqueous humor. To find out the difference in drug concentrations before and after treatment to evaluate the excretion profile of the drug.

Inflammatory factor testing was performed on the patients' aqueous humor samples.

The Eye Hospital of Wenzhou Medical University

Eye & ENT Hospital of Fudan University

Professor

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Targeting NUDT21 siRNA Drugs for Patients With Refractory Retinoblastoma	NCT06424301	Retinoblastoma Refractory	Targeting NUDT2...	1 Year - 12 Years	Eye & ENT Hospital of Fudan University
Targeting NUDT21 siRNA Drugs for Patients With Refractory Retinoblastoma	NCT06424301	Retinoblastoma Refractory	Targeting NUDT2...	1 Year - 12 Years	Eye & ENT Hospital of Fudan University
Targeting NUDT21 siRNA Drugs for Patients With Refractory Retinoblastoma	NCT06424301	Retinoblastoma Refractory	Targeting NUDT2...	1 Year - 12 Years	Eye & ENT Hospital of Fudan University

Spots Global Cancer Trial Database for targeting nudt21 sirna drugs