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Protocol for the Study and Treatment of Patients With Intraocular Retinoblastoma


Phase 3
N/A
N/A
Open (Enrolling)
Both
Retinoblastoma, Retinal Neoplasm

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Trial Information

Protocol for the Study and Treatment of Patients With Intraocular Retinoblastoma


This study will determine the following:

1. To estimate the ocular survival and event-free survival of bilateral disease patients
with advanced intraocular retinoblastoma in either eye (R-E IV-V) responding to the
vincristine/topotecan window, with alternating cycles of vincristine and carboplatin
with vincristine, topotecan, and periocular carboplatin, with intensive focal
treatments.

2. To estimate the ocular survival of eye and event-free survival of eye of bilateral
disease patients with advanced intraocular retinoblastoma in either eye (R-E IV-V)
responding to the vincristine/topotecan window, with alternating cycles of vincristine
and carboplatin with vincristine, topotecan, and periocular carboplatin, with intensive
focal treatments.

3. To estimate the ocular survival and event free survival of patients with advanced
intraocular retinoblastoma (R-E IV-V) not responding to the vincristine/topotecan
window, with a combination of vincristine, carboplatin, etoposide, and periocular
carboplatin, with intensive focal treatments.

4. To estimate the ocular survival and event free survival of eye of patients with
advanced intraocular retinoblastoma (R-E IV-V) not responding to the
vincristine/topotecan window, with a combination of vincristine, carboplatin,
etoposide, and periocular carboplatin, with intensive focal treatments.

5. To estimate the ocular survival and event-free survival of patients with early stage
intraocular retinoblastoma (R-E I-III) with vincristine and carboplatin with intensive
focal treatments.

6. To estimate the ocular survival of eye and event-free survival of eye of patients with
early stage intraocular retinoblastoma (R-E I-III) with vincristine and carboplatin
with intensive focal treatments.

7. To estimate the response rate of early stage eyes (R-E I-III) in patients with
contralateral advanced disease treated with vincristine and topotecan.

8. To estimate the ocular survival and event-free survival of early stage eyes (R-E I-III)
of patients with contralateral advanced disease treated with vincristine and topotecan.

9. To describe the outcome of intraocular retinoblastoma with respect to the new
International Classification for Intraocular Retinoblastoma and the AJCC.

10. To describe primary visual cortex function in patients with unilateral and bilateral
retinoblastoma.

11. To describe the cognitive, adaptive, and social/emotional development of children with
retinoblastoma.

12. To describe changes in the pineal gland during treatment in patients with bilateral
retinoblastoma.

13. To provide insight into molecular pathogenesis of retinoblastoma.

14. To describe the incidence and type of germline mutations of the RB gene in patients
with retinoblastoma.

15. To assess the relation between CYP3A4/5 genotype and the pharmacokinetics and
pharmacodynamics of topotecan.

16. To assess the relation between ABCG2 genotype and the pharmacokinetics and
pharmacodynamics of topotecan.

17. To determine if carboplatin can produce changes in cochlear function that are
detectable with measurement of otoacoustic emissions.

18. To evaluate the need for and feasibility of starting early intervention support during
the first year after the diagnosis of retinoblastoma.


Inclusion Criteria:



- Must have newly diagnosed intraocular retinoblastoma, previously untreated. Patients
previously diagnosed with unilateral retinoblastoma treated surgically (or with focal
therapies), who develop asynchronous involvement of the contralateral eye, will be
eligible for study.

- Must have a life expectancy of at least 8 weeks.

- Must have Performance Status (ECOG) of 0-2.

- Patients must have an adequate liver function, as defined by bilirubin less than or
equal to 3 x normal, and SGOT and SGPT less than or equal to 3x normal.

- Patients must have adequate renal function as defined by serum creatinine less than
or equal to 3x normal for age.

- Legal guardians must sign an informed consent indicating that they are aware of this
study, its possible benefits, and toxic side effects. Legal guardians will be given a
copy of the consent form.

Exclusion Criteria:

- Previously treated patients

- Presence of metastatic disease or orbital involvement

- Patients must not have an invasive infection at time of protocol entry.

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Stratum B Response to Window Therapy

Outcome Description:

The primary outcome is to estimate the proportion of stratum B patients responding to 2 courses of window therapy consisting of vincristine and topotecan. Complete Response is the complete regression of all apparent tumor masses in the funduscopic examination and by MRI and ultrasound (US). Partial Response is defined as greater than 50% (but less than 100%) reduction of the tumor masses in the funduscopic examination and by US and MRI, without the appearance of any new lesions. The response must persist for at least 4 weeks. Stratum A and C did not receive window therapy.

Outcome Time Frame:

Six weeks post window therapy

Safety Issue:

No

Principal Investigator

Ibrahim Qaddoumi, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

St. Jude Children's Research Hospital

Authority:

United States: Institutional Review Board

Study ID:

RET5

NCT ID:

NCT00186888

Start Date:

February 2005

Completion Date:

November 2016

Related Keywords:

  • Retinoblastoma
  • Retinal Neoplasm
  • Cancer of the Eye
  • Eye Enucleation
  • Neoplasms
  • Eye Neoplasms
  • Retinoblastoma
  • Retinal Neoplasms

Name

Location

St. Jude Children's Research HospitalMemphis, Tennessee  38105-2794