Phase I Trial of Periocular Topotecan in Retinoblastoma

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. Read our disclaimer for details. Identifier: NCT00460876
Recruitment Status : Completed
First Posted : April 17, 2007
Last Update Posted : May 2, 2008
Information provided by:
Hospital JP Garrahan

Brief Summary:
This is a dose-escalation study aimed to assess the toxicity (and marginally the activity) of periocular topotecan in patients with relapsed-resistant retinoblastoma.

Condition or disease Intervention/treatment Phase
Retinoblastoma Drug: Topotecan Phase 1

Detailed Description:
Patients with bilateral retinoblastoma who have relapsed after attempts of conservative therapy with standard regimens such as carboplatin, etoposide, vincristine and external beam radiotherapy who face immediate enucleation of their single remaining eye are eligible for this protocol. Starting dose of topotecan will be 0.5 mg and dose escalation will be done by the accelerated titration method. Any Grade 3 ocular toxicity or grade 4 non ocular toxicity will be designed as the dose limiting toxicity. Grade 2 scleral toxicity will be considered for DLT. In case of grade 2 ocular toxicity or grade 3 systemic toxicity, the escalation dose will be 0.25 mg. Maximal dose will be 2 mg.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 5 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Toxicity and Activity of Periocular Topotecan in Children With Retinoblastoma
Study Start Date : March 2007
Actual Primary Completion Date : December 2007
Actual Study Completion Date : April 2008

Primary Outcome Measures :
  1. Dose limiting toxicity

Secondary Outcome Measures :
  1. Response rate, description of toxicity, pharmacokinetic profile

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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Group Vb (Reese Ellsworth)
  • Relapsed or progressed after carboplatin-based regimens and external beam radiotherapy
  • Enucleation of the contralateral eye
  • Normal renal and liver function

Exclusion Criteria:

  • Presence of glaucoma, rubeosis iridis, anterior chamber extension
  • Extraocular disease
  • Adequate follow up impossible for social reasons

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): NCT00460876

Hospital JP Garrahan
Buenos Aires, CF, Argentina, C1245AAL
Sponsors and Collaborators
Hospital JP Garrahan
Principal Investigator: Guillermo L Chantada, MD Hospital JP Garrahan

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Hospital JP Garrahan Identifier: NCT00460876     History of Changes
Other Study ID Numbers: 14711603062
First Posted: April 17, 2007    Key Record Dates
Last Update Posted: May 2, 2008
Last Verified: April 2008

Keywords provided by Hospital JP Garrahan:

Additional relevant MeSH terms:
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
Retinal Diseases
Topoisomerase I Inhibitors
Topoisomerase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents