MDACC Study Summary COGARET0321

Study Summary
No. COGARET0321:.......Blood And Marrow Transplantation; Eye; Pediatrics; Solid Tumors......Cynthia E. Herzog......Pediatrics

Study Summary Title



Study Summary Number:	COGARET0321

Study Title:	A Trial of Intensive Multi-Modality Therapy for Extra-Ocular Retinoblastoma

Physician

New Patient Referral



Name:	Cynthia E. Herzog	Patients Call:	800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)

Dept:	Pediatrics	Referring MD Call:	800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)

Phone:	713-792-6620 Contact us about clinical trials

General Information



Disease Group:	Blood And Marrow Transplantation Eye Pediatrics Solid Tumors	Supported By:	NCI through COG

Phase of Study:	Phase III	Return Visit:	Patients must return for chemotherapy cycles every 7 days and periodic tests.

Treatment Agents:	Carboplatin Cisplatin Cyclophosphamide Etoposide Filgrastim Mesna Radiation Radiation & Chemo Thiotepa Vincristine Sulfate	Home Care:	Supportive care may be given at home.

Treatment Loc:	Both at MDACC & outside MDACC at one or more Collaborating Sites or Institutions

Estimated Length of Stay in Houston:	Stages 2, 3, 4a, and 4b: Induction chemotherapy (Cycles 1-4, Days 0, 1, 2, 7, 14). Stages 4a and 4b: Consolidation chemotherapy & stem cell transplantation (4-6 weeks).

Description/
Intervention:

The goal of this clinical research study is to learn if the combination of
chemotherapy, stem cell transplant, and/or radiation will get rid of the cancer
for as long as possible and is better than treatments used in the past.
Researchers also what to find out how well the first phase of the study
treatment (called induction) makes the tumor shrink or disappear and find out
what effects good and/or bad that the therapy has on people with your kind of
retinoblastoma.

Study Objectives / Outcomes

1) To estimate the proportion of 3 groups of patients with extra-ocular retinoblastoma:

• Stage 2 and 3: Regional extra-ocular disease

• Stage 4a: Disseminated metastatic disease not involving the CNS

• Stage 4b: Patients with CNS disease

who achieve long-term event-free survival after aggressive multimodality therapy as prescribed in this protocol.

2) To estimate the response rate to the induction phase of the regimen.

3) To evaluate the toxicities associated with this regimen.

Study Status Information



Study Activation / Registration Date:	10/30/2009

IRB Review and Approval Date:	10/30/2009

Study Type:	Phase Iii

Recruitment Status:	Closed

Projected Accrual:	40

Enrollment Eligibility

If you do not meet the enrollment eligibility, there may be other treatment options for you. Please Contact the Referral Office for more information.

Inclusion Criteria:

1) Patients must be no greater than 10 years of age at study enrollment.

2) Patients must be enrolled before treatment begins. Treatment must start within 5 calendar days of study enrollment.

3) Patients must have histologic or cytologic verification of extra-ocular retinoblastoma. Extra-ocular disease includes orbital disease, optic nerve involvement at the surgical margin, regional nodal disease, and/or overt distant metastatic disease (at sites such as bone, bone marrow, liver and/or the central nervous system). Patients with trilateral retinoblastoma will also be included in this protocol.

4) Patients with a CNS lesion consistent with trilateral or Stage 4b disease may be enrolled without tissue confirmation if (1) unequivocal leptomeningeal disease is present on brain or spine MRI scan and/or (2) the primary tumor is at least 2 cm in diameter, predominantly solid, and demonstrates enhancement on the post-Gadolinium images. However, even in such cases surgery should be given serious consideration.

5) Performance Level: Patients must have a performance status corresponding to ECOG scores of 0, 1, or 2. Use Karnofsky for patients >16 years of age and Lansky for patients < / = 16 years of age.

6) No prior chemotherapy or radiotherapy for the extra-ocular retinoblastoma may have been administered prior to entering this study. Prior treatment (chemotherapy and/or radiation therapy) for intra-ocular retinoblastoma is permissible.

7) Adequate Bone Marrow Function Defined As: Peripheral absolute neutrophil count (ANC) > / = 750/microL; Platelet count > / = 75,000/microL (transfusion independent); If the ANC and/or platelet count are not adequate, but due to bone marrow metastatic disease, these criteria will be waived.

8) Adequate renal function: Creatinine clearance or radioisotope GFR >/= 70 ml/min/1.73 m^2; OR, 2) maximum serum creatinine (md/dL) based on age/gender as follows: 1 month to < 6 months: 0.4 md/dL; 6 months to < 1 yr: 0.5 md/dL; 1 to < 2 yrs: 0.6 md/dL; 2 to < 6 yrs: 0.8 md/dL; 6 to < 10 yrs: 1 md/dL; 10 to < 13 yrs: 1.2 md/dL; 13 to < 16 yrs: Male: 1.5 md/dL; Female: 1.4 md/dL; >/= 16 yrs: Male: 1.7 md/dL; Female: 1.4 mg/dL.

9) Adequate Liver Function Defined As: Total bilirubin < / = 1.5 x upper limit of normal (ULN) for age, and AST (SGOT) or ALT (SGPT) < 2.5 x upper limit of normal (ULN) for age.

10) All patients and/or their parents or legal guardians must sign a written informed consent.

11) All institutional, FDA, and NCI requirements for human studies must be met.

Exclusion Criteria:

N/A

Links

Registration Number: NCT00554788
Study Information on Clinical Trials Registry (clinicaltrials.gov)