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Trial record 12 of 27 for:    Recruiting, Not yet recruiting, Available Studies | "Retinoblastoma"

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

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ClinicalTrials.gov Identifier: NCT03284268
Recruitment Status : Recruiting
First Posted : September 15, 2017
Last Update Posted : May 17, 2018
Sponsor:
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 Early Phase 1

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 13 participants
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 : September 25, 2019
Estimated Study Completion Date : November 25, 2019

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



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Ages Eligible for Study:   1 Year to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patients with retinoblastoma with either a somatic mutation of the geneRB1 with active tumor in a single eye, or with germinal mutation of RB1 with active tumor in a single eye and the contralateral eye unaffected or with active tumor in a single eye and the contralateral already enucleated eye relapsed or refractory with the use of systemic, intraarterial or intravitreal chemotherapy or radiotherapy in whom enucleation is the only recommended treatment.
  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 ClinicalTrials.gov identifier (NCT number): NCT03284268


Contacts
Contact: Jaume Catalá Mora, Dr. 932 53 21 00 Jmora@sjdhospitalbarcelona.org
Contact: Guillermo Chantada, Dr 932 53 21 00 gchantada@yahoo.com

Locations
Spain
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
Investigators
Principal Investigator: Jaume Catalá Mora, DR Hospital Sant Joan de Deu

Responsible Party: Fundació Sant Joan de Déu
ClinicalTrials.gov Identifier: NCT03284268     History of Changes
Other Study ID Numbers: FSJD-RTB-2015
First Posted: September 15, 2017    Key Record Dates
Last Update Posted: May 17, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
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

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:
Retinoblastoma
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
Retinal Diseases