---
title: Evaluate Safety and the Oncolitic Adenovirus VCN-01 Activity in Patients With Refractory Retinoblastoma
nct_id: NCT03284268
overall_status: COMPLETED
phase: NA
sponsor: Fundació Sant Joan de Déu
study_type: INTERVENTIONAL
primary_condition: Retinoblastoma, Recurrent
countries: Spain
canonical_url: "https://parkinsonspathways.com/agent/trials/NCT03284268.md"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT03284268"
ct_last_update_post_date: 2024-08-27
last_seen_at: "2026-05-12T06:03:33.023Z"
source: ClinicalTrials.gov (mirrored, no enrichment)
---
# 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

**NCT ID:** [NCT03284268](https://clinicaltrials.gov/study/NCT03284268)

## Key Facts

- **Status:** COMPLETED
- **Phase:** NA
- **Study Type:** INTERVENTIONAL
- **Target Enrollment:** 13
- **Lead Sponsor:** Fundació Sant Joan de Déu
- **Conditions:** Retinoblastoma, Recurrent
- **Start Date:** 2017-09-06
- **Completion Date:** 2024-06-14
- **CT.gov Last Update:** 2024-08-27

## 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.

## Detailed Description

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.

## Eligibility

- **Minimum age:** 1 Year
- **Maximum age:** 12 Years
- **Sex:** ALL
- **Healthy Volunteers:** No

```
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.
```

## Arms

- **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

## Interventions

- **VCN-01** (GENETIC) — VCN-01 intravitreal injection

## Primary Outcomes

- **Incidence of Treatment-Emergent Adverse Events** _(time frame: 180 days)_ — 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 Outcomes

- **Tumor response** _(time frame: 28 days)_
- **VCN-01** _(time frame: 180 days)_
- **Immune response** _(time frame: 180 days)_

## Locations (1)

- Hospital Sant Joan de Déu, Barcelona, Spain

## Recent Field Changes (last 30 days)

- `status.overallStatus` — added _(2026-05-12)_
- `status.primaryCompletionDate` — added _(2026-05-12)_
- `status.completionDate` — added _(2026-05-12)_
- `status.lastUpdatePostDate` — added _(2026-05-12)_
- `design.phases` — added _(2026-05-12)_
- `design.enrollmentCount` — added _(2026-05-12)_
- `eligibility.criteria` — added _(2026-05-12)_
- `eligibility.minAge` — added _(2026-05-12)_
- `eligibility.maxAge` — added _(2026-05-12)_
- `eligibility.sex` — added _(2026-05-12)_
- `outcomes.primary` — added _(2026-05-12)_
- `outcomes.secondary` — added _(2026-05-12)_
- `armsInterventions.arms` — added _(2026-05-12)_
- `armsInterventions.interventions` — added _(2026-05-12)_
- `sponsor.lead` — added _(2026-05-12)_
- `results.hasResults` — added _(2026-05-12)_
- `locations.hospital sant joan de déu|barcelona||spain` — added _(2026-05-12)_

---

*Canonical: https://parkinsonspathways.com/agent/trials/NCT03284268.md*  
*Source data (authoritative): https://clinicaltrials.gov/study/NCT03284268*  
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