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Alternating Systemic Chemotherapy and Intra-Arterial Melphalan Chemotherapy in Children With Intra-Ocular Retinoblastoma

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified January 2015 by Katherine Matthay, University of California, San Francisco.
Recruitment status was:  Recruiting
Information provided by (Responsible Party):
Katherine Matthay, University of California, San Francisco Identifier:
First received: February 18, 2014
Last updated: January 9, 2015
Last verified: January 2015
The purpose of this study is to test the safety of the treatment combination of alternating standard chemotherapy and another (melphalan) chemotherapy at different interval schedules. Researchers want to find out what effects, good and/or bad, the treatment combination has on the patients and their retinoblastoma.

Condition Intervention
Advanced Intra-Ocular Retinoblastoma Retinoblastoma Drug: Melphalan Drug: Carboplatin Drug: Etoposide Drug: Vincristine

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pilot Study Evaluating the Safety of Alternating Systemic Chemotherapy and Intra-Arterial Melphalan Chemotherapy in Children With Newly Diagnosed Advanced Intra-Ocular Retinoblastoma

Resource links provided by NLM:

Further study details as provided by Katherine Matthay, University of California, San Francisco:

Primary Outcome Measures:
  • Adverse Event evaluation for newly diagnosed advanced retinoblastoma treated with melphalan therapy and systemic chemotherapy [ Time Frame: Up to 2 years ]
  • Evaluation of patients' commitment to taking the study medication [ Time Frame: Up to 2 years ]
  • Dose-Limiting Toxicity (DLT) and minimum tolerated dosing interval for combination IAM and CEV. [ Time Frame: Up to 2 years ]

Secondary Outcome Measures:
  • Response rates after IAM combined with systemic CEV in patients with newly diagnosed advanced intraocular retinoblastoma [ Time Frame: Up to 2 years ]
  • Ocular event free survival with the use of combination IAM therapy and systemic CEV in this patient population. [ Time Frame: Up to 2 years ]
  • Visual outcomes using a standardized age based assessment [ Time Frame: Up to 2 years ]

Other Outcome Measures:
  • Collection of melphalan pharmacokinetics after intra-arterial administration to the retina [ Time Frame: Up to 2 years ]

Estimated Enrollment: 42
Study Start Date: April 2014
Estimated Study Completion Date: July 2017
Estimated Primary Completion Date: January 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Infants
Patients ≥ 6 months of age at time of study enrollment
Drug: Melphalan Drug: Carboplatin Drug: Etoposide Drug: Vincristine
Experimental: Babies
Patients ≥ 4 months of age but < 6 months of age
Drug: Melphalan Drug: Carboplatin Drug: Etoposide Drug: Vincristine


Ages Eligible for Study:   4 Months and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Age greater than or equal to 4 months.

    1. Age greater than or equal to 4 months but less than 6 months of age can receive up to 2 cycles of systemic chemotherapy to bridge until 6 months (cohort 2).
    2. Age greater than or equal to 6 months at study entry (cohort 1) to receive alternating therapy.
  • Intraocular retinoblastoma not previously treated with systemic chemotherapy, radiation therapy, or IA therapy. Local retinal therapy such as laser photocoagulation and cryotherapy will be permitted.
  • Unilateral or bilateral RB patients are eligible
  • Patients will be registered on study based on the local exam under anesthesia (EUA) done for diagnostic purposes prior to study entry. The EUA done at study entry should be done within 14 days prior to study entry
  • Patients may have had enucleation of one eye, as long as the remaining eye meets the eligibility criteria
  • Involved eye(s) must meet the definition for International Classification of Retinoblastoma Group D or E

    1. Group D eye can be unilateral or bilateral
    2. Group E eye will be eligible if bilateral involvement or unilateral involvement in a child less than 1 year of age
    3. Patients with bilateral disease in which one of the eyes has Group C disease are not eligible until interval level 2 in cohort 1 and are not eligible for cohort 2
  • Adequate Renal Function defined as: creatinine clearance or radioisotope GFR ³ 70mL/min/1.73 m2 OR a serum creatinine based on age and gender as follows:

The threshold creatinine values in this Table were derived from the Schwartz formula for estimating GFR (Schwartz et al. J. Peds, 106:522, 1985) utilizing child length and stature data published by the CDC

  • Adequate hematological function defined as:

    1. Absolute Neutrophil Count > 750/microliter
    2. Platelet Count > 100,000/microliter
  • Adequate liver function defined as total bilirubin should be less than or equal to 1.5 x upper limit of normal (ULN) for age and SGOT (AST) and SGPT (ALT) < 5 x upper limit of normal (ULN) for age
  • Adequate coagulation system as defined as an international normalized ratio (INR) of less than 1.4 and a partial thromboplastin time (PTT) of less than 34
  • The effects of melphalan, carboplatin, etoposide and vincristine are harmful on the developing human fetus. For this reason, women of child-bearing potential and men must agree to use adequate contraception: hormonal or barrier method of birth control; abstinence, prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately

Exclusion Criteria:

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02116959

Contact: Katherine Matthay, MD 415-476-3831
Contact: Oscar Alcantar 415-476-2218

United States, California
University of California, San Francisco Recruiting
San Francisco, California, United States, 94143
Sub-Investigator: Steven Dubois, MD         
Sub-Investigator: Sheila Thampi, MD         
Sub-Investigator: Steven Hetts, MD         
Sub-Investigator: Van Halbach, MD         
Sub-Investigator: Bertil Damato, MD         
Sub-Investigator: Alejandra de Alba Campomanes, MD         
Sub-Investigator: Elizabeth Robbins, MD         
Sub-Investigator: Anuradha Banerjee, MD         
Sub-Investigator: Janel Long-Boyle, PhD, PharmD         
Sub-Investigator: Ilana Withop, RN, PNP         
Sponsors and Collaborators
University of California, San Francisco
Principal Investigator: Katherine Matthay, MD University of California, San Francisco
  More Information

Responsible Party: Katherine Matthay, chief of Pediatric Hematology-Oncology at UCSF Benioff Children's Hospital, University of California, San Francisco Identifier: NCT02116959     History of Changes
Other Study ID Numbers: 13087
Study First Received: February 18, 2014
Last Updated: January 9, 2015

Keywords provided by Katherine Matthay, University of California, San Francisco:

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
Antineoplastic Agents
Antineoplastic Agents, Phytogenic
Topoisomerase II Inhibitors
Topoisomerase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Tubulin Modulators
Antimitotic Agents
Mitosis Modulators
Antineoplastic Agents, Alkylating
Alkylating Agents
Myeloablative Agonists
Immunosuppressive Agents processed this record on August 23, 2017