Description:
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.
Title
- Brief Title: Evaluate Safety and the Oncolitic Adenovirus VCN-01 Activity in Patients With Refractory Retinoblastoma
- Official Title: 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
Clinical Trial IDs
- ORG STUDY ID:
FSJD-RTB-2015
- NCT ID:
NCT03284268
Conditions
- Retinoblastoma, Recurrent
Purpose
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.
Trial Arms
Name | Type | Description | Interventions |
---|
Dose escalation of VCN-01 | Experimental | Dose lower : 2E+9 viral particules/eye Dose medium: 2E+10 viral particules/eye Dose high: 2E+11 viral particules/eye | |
Eligibility Criteria
Inclusion Criteria:
1. Patients with retinoblastoma with a somatic mutation of the geneRB1 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.
2. Normal renal function: serum creatinine: <45μmol/L (0-2 years); <57μmol/L (3-6 years);
<60μmol/L (7-10 years); <80μmol/L (11-13 years).
3. Normal Hepatic function: serum ALT: <0,52μkat/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 one year and less than 12 years at the time of inclusion in the
study.
6. Informed consent form signed.
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.
11. Pregnancy. Female patients with procreative potential should be agree to undergo a
blood or urine pregnancy test and the result should be negative to enter the study.
Maximum Eligible Age: | 12 Years |
Minimum Eligible Age: | 1 Year |
Eligible Gender: | All |
Healthy Volunteers: | No |
Primary Outcome Measures
Measure: | Incidence of Treatment-Emergent Adverse Events |
Time Frame: | 180 days |
Safety Issue: | |
Description: | 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). |
Secondary Outcome Measures
Measure: | Tumor response |
Time Frame: | 28 days |
Safety Issue: | |
Description: | Tumor response rate (ORR) to VCN-01 at 28 days post-administration. |
Measure: | VCN-01 |
Time Frame: | 180 days |
Safety Issue: | |
Description: | 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. |
Measure: | Immune response |
Time Frame: | 180 days |
Safety Issue: | |
Description: | The presence of neutralizing antibodies in blood samples in study patients to assess the immune response. |
Details
Phase: | N/A |
Primary Purpose: | Interventional |
Overall Status: | Recruiting |
Lead Sponsor: | Fundació Sant Joan de Déu |
Trial Keywords
Last Updated
February 25, 2020