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

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2014 by University of California, San Francisco
Sponsor:
Information provided by (Responsible Party):
University of California, San Francisco
ClinicalTrials.gov Identifier:
NCT02116959
First received: February 18, 2014
Last updated: April 24, 2014
Last verified: April 2014
  Purpose

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
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: 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 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 ] [ Designated as safety issue: Yes ]
  • Evaluation of patients' commitment to taking the study medication [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]
  • Dose-Limiting Toxicity (DLT) and minimum tolerated dosing interval for combination IAM and CEV. [ Time Frame: Up to 2 years ] [ Designated as safety issue: Yes ]

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 ] [ Designated as safety issue: Yes ]
  • Ocular event free survival with the use of combination IAM therapy and systemic CEV in this patient population. [ Time Frame: Up to 2 years ] [ Designated as safety issue: Yes ]
  • Visual outcomes using a standardized age based assessment [ Time Frame: Up to 2 years ] [ Designated as safety issue: Yes ]

Other Outcome Measures:
  • Collection of melphalan pharmacokinetics after intra-arterial administration to the retina [ Time Frame: Up to 2 years ] [ Designated as safety issue: No ]

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

  Eligibility

Ages Eligible for Study:   4 Months and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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 ClinicalTrials.gov identifier: NCT02116959

Contacts
Contact: Katherine Matthay, MD 415-476-3831 matthayk@peds.ucsf.edu
Contact: Oscar Alcantar 415-476-2218 alcantaro@cc.ucsf.edu

Locations
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
Investigators
Principal Investigator: Katherine Matthay, MD University of California, San Francisco
  More Information

No publications provided

Responsible Party: University of California, San Francisco
ClinicalTrials.gov Identifier: NCT02116959     History of Changes
Other Study ID Numbers: 13087
Study First Received: February 18, 2014
Last Updated: April 24, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by University of California, San Francisco:
Systemic
Chemotherapy
Melphalan

Additional relevant MeSH terms:
Retinoblastoma
Eye Diseases
Eye Neoplasms
Neoplasms
Neoplasms by Histologic Type
Neoplasms by Site
Neoplasms, Germ Cell and Embryonal
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Retinal Diseases
Retinal Neoplasms
Melphalan
Alkylating Agents
Antineoplastic Agents
Antineoplastic Agents, Alkylating
Immunologic Factors
Immunosuppressive Agents
Molecular Mechanisms of Pharmacological Action
Myeloablative Agonists
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses

ClinicalTrials.gov processed this record on October 22, 2014