Evaluate Safety and the Oncolitic Adenovirus VCN-01 Activity in Patients With Refractory Retinoblastoma

NCT03284268 | Completed

• 1 other identifier: FSJD-RTB-2015
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 1

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.

Conditions

Retinoblastoma, Recurrent

Keywords

Gene therapy

Outcome Measures

Primary Outcomes (1)

• 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).

  180 days

Secondary Outcomes (3)

• Tumor response

  28 days

• VCN-01

  180 days

• Immune response

  180 days

Other Outcomes (1)

• Histopathology of enucleated eyes after treatment with VCN-01

  180 days

Study Arms (1)

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

Genetic: VCN-01

Interventions

VCN-01 GENETIC

VCN-01 intravitreal injection

Dose escalation of VCN-01

Eligibility Criteria

• Age: 1 Year - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.
• 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).
• 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).
• 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.
• Informed consent form signed.

You may not qualify if:

• Presence of factors that require immediate enucleation of the affected eye such as glaucoma, rubeosis iridis, anterior chamber involvement.
• Comorbidities: Uncontrolled epilepsy with anticonvulsant treatment, cardiac disease not compensated by treatment.
• Active Infections.
• Any cause of Immunosuppression.
• Trilateral Retinoblastoma.
• Extraocular spread.
• Patients who can not complete the study procedures for reasons psychological or social.
• 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.

Sponsors & Collaborators

• Fundació Sant Joan de Déu: lead

Study Sites (1)

Hospital Sant Joan de Déu

Barcelona, 08950, Spain

Location

MeSH Terms

Conditions

Retinoblastoma; Recurrence

Condition Hierarchy (Ancestors)

Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Retinal Neoplasms; Eye Neoplasms; Neoplasms by Site; Eye Diseases, Hereditary; Eye Diseases; Retinal Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Jaume Catalá Mora, Dr

  Hospital Sant Joan de Deu

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 17, 2017
• First Posted: September 15, 2017
• Study Start: September 6, 2017
• Primary Completion: June 14, 2024
• Study Completion: June 14, 2024
• Last Updated: August 27, 2024

Record last verified: 2024-08

Data Sharing

• IPD Sharing: Will not share

Locations

ES (1)