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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

Tracking Information
First Submitted Date  ICMJE August 17, 2017
First Posted Date  ICMJE September 15, 2017
Last Update Posted Date May 17, 2018
Actual Study Start Date  ICMJE September 6, 2017
Estimated Primary Completion Date September 25, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 14, 2017)
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).
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03284268 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: September 14, 2017)
  • Tumor response [ Time Frame: 28 days ]
    Tumor response rate (ORR) to VCN-01 at 28 days post-administration.
  • 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.
  • Immune response [ Time Frame: 180 days ]
    The presence of neutralizing antibodies in blood samples in study patients to assess the immune response.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Outcome Measures  ICMJE
 (submitted: September 14, 2017)
Histopathology of enucleated eyes after treatment with VCN-01 [ Time Frame: 180 days ]
Presence of VCN-01 in vitreous humor samples of enucleated eyes
Original Other Outcome Measures  ICMJE Same as current
 
Descriptive Information
Brief Title  ICMJE Evaluate Safety and the Oncolitic Adenovirus VCN-01 Activity in Patients With Refractory Retinoblastoma
Official Title  ICMJE 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase Early Phase 1
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Retinoblastoma, Recurrent
Intervention  ICMJE Genetic: VCN-01
VCN-01 intravitreal injection
Study Arms 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
Intervention: Genetic: VCN-01
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: September 14, 2017)
13
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date November 25, 2019
Estimated Primary Completion Date September 25, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender
Sexes Eligible for Study: All
Ages 1 Year to 12 Years   (Child)
Accepts Healthy Volunteers No
Contacts  ICMJE
Contact: Jaume Catalá Mora, Dr. 932 53 21 00 Jmora@sjdhospitalbarcelona.org
Contact: Guillermo Chantada, Dr 932 53 21 00 gchantada@yahoo.com
Listed Location Countries  ICMJE Spain
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03284268
Other Study ID Numbers  ICMJE FSJD-RTB-2015
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
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
Responsible Party Fundació Sant Joan de Déu
Study Sponsor  ICMJE Fundació Sant Joan de Déu
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Jaume Catalá Mora, DR Hospital Sant Joan de Deu
PRS Account Fundació Sant Joan de Déu
Verification Date May 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP