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
Brief Summary
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.
Study Type
Interventional
Primary Outcome
Incidence of Treatment-Emergent Adverse Events
Secondary Outcome
Tumor response
Condition
Retinoblastoma, Recurrent
Intervention
VCN-01
Study Arms / Comparison Groups
Dose escalation of VCN-01
Description: Dose lower : 2E+9 viral particules/eye Dose medium: 2E+10 viral particules/eye Dose high: 2E+11 viral particules/eye
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Genetic
Estimated Enrollment
13
Start Date
September 6, 2017
Completion Date
June 30, 2022
Primary Completion Date
June 30, 2022
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.
Gender
All
Ages
1 Year - 12 Years
Accepts Healthy Volunteers
No
Contacts
Jaume Catalá Mora, Dr, 932 53 21 00, [email protected]
Location Countries
Spain
Location Countries
Spain
Administrative Informations
NCT ID
NCT03284268
Organization ID
FSJD-RTB-2015
Responsible Party
Sponsor
Study Sponsor
Fundació Sant Joan de Déu
Study Sponsor
Jaume Catalá Mora, Dr, Principal Investigator, Hospital Sant Joan de Deu
Verification Date
June 2022