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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03284268
Recruitment Status : Recruiting
First Posted : September 15, 2017
Last Update Posted : June 8, 2022
Information provided by (Responsible Party):
Fundació Sant Joan de Déu

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.

Condition or disease Intervention/treatment Phase
Retinoblastoma, Recurrent Genetic: VCN-01 Not Applicable

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 13 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : September 6, 2017
Estimated Primary Completion Date : June 30, 2022
Estimated Study Completion Date : June 30, 2022

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: Dose escalation of VCN-01
Dose lower : 2E+9 viral particules/eye Dose medium: 2E+10 viral particules/eye Dose high: 2E+11 viral particules/eye
Genetic: VCN-01
VCN-01 intravitreal injection

Primary Outcome Measures :
  1. 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 Outcome Measures :
  1. Tumor response [ Time Frame: 28 days ]
    Tumor response rate (ORR) to VCN-01 at 28 days post-administration.

  2. VCN-01 [ Time Frame: 180 days ]
    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.

  3. Immune response [ Time Frame: 180 days ]
    The presence of neutralizing antibodies in blood samples in study patients to assess the immune response.

Other Outcome Measures:
  1. Histopathology of enucleated eyes after treatment with VCN-01 [ Time Frame: 180 days ]
    Presence of VCN-01 in vitreous humor samples of enucleated eyes

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   1 Year to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03284268

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Contact: Jaume Catalá Mora, Dr. 932 53 21 00
Contact: Guillermo Chantada, Dr 932 53 21 00

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Hospital Sant Joan de Déu Recruiting
Barcelona, Spain, 08950
Contact: Jaume Catalá Mora, Dr    932 53 21 00      
Sponsors and Collaborators
Fundació Sant Joan de Déu
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Principal Investigator: Jaume Catalá Mora, Dr Hospital Sant Joan de Deu
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Responsible Party: Fundació Sant Joan de Déu Identifier: NCT03284268    
Other Study ID Numbers: FSJD-RTB-2015
First Posted: September 15, 2017    Key Record Dates
Last Update Posted: June 8, 2022
Last Verified: June 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Anonymized individual data of participants will be shared with Authorities at the end of the Clinical development plan by the CTD (Common Technical Document). Results will be published in a scientific publication

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Fundació Sant Joan de Déu:
Gene therapy
Additional relevant MeSH terms:
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Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Retinal Neoplasms
Eye Neoplasms
Neoplasms by Site
Eye Diseases, Hereditary
Eye Diseases
Retinal Diseases