Phase I study, single site, open label with dose escalation, for evaluate safety and the oncolitic Adenovirus VCN-01 activity in patients with refractory retinoblastoma.
Phase I clinical trial, single-center, open-label, dose-escalation study, to evaluate the safety of the VCN-01 and, secondarily, its anti-tumoral activity. The trial sample consists of approximately 13 patients aged between 1 and 12 years old.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
13
VCN-01 intravitreal injection
Hospital Sant Joan de Déu
Barcelona, Spain
Incidence of Treatment-Emergent Adverse Events
The safety and tolerability of two intravitreal injections of VCN-01 will be determined with dose escalation until the maximum tolerated dose (MTD) is established based on the evaluation of adverse events and the observation of any dose-limiting toxicity (TLD).
Time frame: 180 days
Tumor response
Tumor response rate (ORR) to VCN-01 at 28 days post-administration.
Time frame: 28 days
VCN-01
Virus presence in blood samples, aqueous humor, ocular surface and nasal swabs, and its difference between the viral particles before and after the treatment to evaluate the excretion profile of the CNV-01.
Time frame: 180 days
Immune response
The presence of neutralizing antibodies in blood samples in study patients to assess the immune response.
Time frame: 180 days
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.