Study of Treatment for Patients With Cancer of the Eye -Retinoblastoma

This study is ongoing, but not recruiting participants.

Sponsor:

Information provided by (Responsible Party):

St. Jude Children's Research Hospital

ClinicalTrials.gov Identifier:

NCT00186888

First received: September 12, 2005

Last updated: February 25, 2013

Last verified: June 2012

History of Changes

Results First Received: June 29, 2011

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized; Endpoint Classification: Efficacy Study; Intervention Model: Parallel Assignment; Masking: Open Label; Primary Purpose: Treatment
Conditions:	Retinoblastoma Retinal Neoplasm
Interventions:	Procedure: Enucleation Drug: Vincristine, Carboplatin Procedure: Focal Therapies Radiation: External Beam Radiation Drug: Vincristine + Topotecan Drug: Vincristine + Carboplatin + Etoposide Drug: Vincristine + Cyclophosphamide + Doxorubicin Drug: Vincristine+Carboplatin+Etoposide Procedure: Periocular carboplatin Other: G-CSF

Participant Flow

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Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
107 patients were recruited between February, 2005 and June, 2010 (stratum B) and between February, 2005 and November, 2010 (strata A & C).The primary objective was designed only for stratum B patients who had advanced bilateral retinoblastoma and received the investigational window therapy.

Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
107 patients were enrolled on the study. Two patients were excluded as they were deemed to be ineligible after study enrollment. One patient was found to have retinal dysplasia rather than retinoblastoma and the other patient had retinoblastoma but did not fit into any of the defined treatment strata for this study.

Reporting Groups

	Description
Stratum A	Early Unilateral or Bilateral Retinoblastoma. Stratum A includes mainly patients with early stage (Reese-Ellsworth group I, II, or III) bilateral retinoblastoma. Patients with unilateral disease diagnosed at an early stage, and patients with early multifocal unilateral disease are rare, but these patients are also candidates for conservative management and were treated in stratum A.
Stratum B	Advanced Bilateral Retinoblastoma. Stratum B includes patients with at least one Reese-Ellsworth group IV or V eye that after careful evaluation by the treating team is considered not to require upfront enucleation. A proportion of patients treated on this stratum will not have advanced disease in both eyes. Only stratum B patients received window therapy consisting of 2 courses of vincristine and topotecan.
Stratum C	Advanced Unilateral Retinoblastoma. Research participants with unilateral (unifocal or multifocal) advanced (Reese-Ellsworth group IV or V) intraocular disease will undergo upfront enucleation. Adjuvant therapy was also indicated in certain cases.

Participant Flow: Overall Study

	Stratum A	Stratum B	Stratum C
STARTED	23	27	55
Window Therapy	0	27	0
COMPLETED	19	24	55
NOT COMPLETED	4	3	0
Toxicity	0	1	0
Relapse or progression of disease	4	2	0

Baseline Characteristics

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Reporting Groups

	Description
Stratum A	Early Unilateral or Bilateral Retinoblastoma. Stratum A includes mainly patients with early stage (Reese-Ellsworth group I, II, or III) bilateral retinoblastoma. Patients with unilateral disease diagnosed at an early stage, and patients with early multifocal unilateral disease are rare, but these patients are also candidates for conservative management and were treated in stratum A.
Stratum B	Advanced Bilateral Retinoblastoma. Stratum B includes patients with at least one Reese-Ellsworth group IV or V eye that after careful evaluation by the treating team is considered not to require upfront enucleation. A proportion of patients treated on this stratum will not have advanced disease in both eyes. Only stratum B patients received window therapy consisting of 2 courses of vincristine and topotecan.
Stratum C	Advanced Unilateral Retinoblastoma. Research participants with unilateral (unifocal or multifocal) advanced (Reese-Ellsworth group IV or V) intraocular disease will undergo upfront enucleation. Adjuvant therapy was also indicated in certain cases.
Total	Total of all reporting groups

Baseline Measures

	Stratum A	Stratum B	Stratum C	Total
Number of Participants [units: participants]	23	27	55	105
Age [units: months] Mean ± Standard Deviation	5.7 ± 3.6	8.5 ± 4.6	29.6 ± 22.1	18.9 ± 19.7
Gender [units: participants]
Female	11	13	28	52
Male	12	14	27	53

Outcome Measures

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1. Primary:

Stratum B Response to Window Therapy [ Time Frame: Six weeks post window therapy ]

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2. Secondary:

Stratum B Response Rate of Early Stage Eyes to Window Therapy [ Time Frame: Six weeks post window therapy. ]

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3. Secondary:

Relationship Between Topotecan Clearance (CL) and CYP3A4/5 Genotype in Stratum B Participants. [ Time Frame: Courses 1, 2, 5, and 8 ]

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4. Secondary:

Relationship Between Topotecan Clearance (CL) and ABCG2/B1 Genotype in Stratum B Participants. [ Time Frame: Courses 1, 2, 5, and 8 ]

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5. Post-Hoc:

Number of Patients Recommended for and Utilizing Rehabilitation Services [ Time Frame: At diagnosis, and at 3, 6, 9, and 12 months from diagnosis ]

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Serious Adverse Events

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Other Adverse Events

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More Information

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Certain Agreements:

All Principal Investigators ARE employed by the organization sponsoring the study.

Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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Results Point of Contact:

Name/Title: Ibrahim Qaddoumi MD
Organization: St. Jude Children's Research Hospital
phone: 1-866-278-5833
e-mail: info@stjude.org

No publications provided

Responsible Party:	St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier:	NCT00186888 History of Changes
Other Study ID Numbers:	RET5
Study First Received:	September 12, 2005
Results First Received:	June 29, 2011
Last Updated:	February 25, 2013
Health Authority:	United States: Institutional Review Board United States: Food and Drug Administration

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