Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

Phase I Trial of Periocular Topotecan in Retinoblastoma

This study has been completed.
Information provided by:
Hospital JP Garrahan Identifier:
First received: April 13, 2007
Last updated: April 30, 2008
Last verified: April 2008

April 13, 2007
April 30, 2008
March 2007
December 2007   (final data collection date for primary outcome measure)
Dose limiting toxicity [ Designated as safety issue: Yes ]
Dose limiting toxicity
Complete list of historical versions of study NCT00460876 on Archive Site
Response rate, description of toxicity, pharmacokinetic profile
Same as current
Not Provided
Not Provided
Phase I Trial of Periocular Topotecan in Retinoblastoma
Toxicity and Activity of Periocular Topotecan in Children With Retinoblastoma

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

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.

Phase 1
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Drug: Topotecan
Not Provided
Chantada GL, Fandino AC, Carcaboso AM, Lagomarsino E, de Davila MT, Guitter MR, Rose AB, Manzitti J, Bramuglia GF, Abramson DH. A phase I study of periocular topotecan in children with intraocular retinoblastoma. Invest Ophthalmol Vis Sci. 2009 Apr;50(4):1492-6. Epub 2008 Oct 31.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
April 2008
December 2007   (final data collection date for primary outcome measure)

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
up to 18 Years
Contact information is only displayed when the study is recruiting subjects
Hospital JP Garrahan
Hospital JP Garrahan
Not Provided
Principal Investigator: Guillermo L Chantada, MD Hospital JP Garrahan
Hospital JP Garrahan
April 2008

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