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Intra-arterial Chemotherapy for the Treatment of Intraocular Retinoblastoma

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2014 by Sidney Kimmel Comprehensive Cancer Center
Sponsor:
Information provided by (Responsible Party):
Sidney Kimmel Comprehensive Cancer Center
ClinicalTrials.gov Identifier:
NCT01293539
First received: January 21, 2011
Last updated: August 6, 2014
Last verified: August 2014
  Purpose

The purpose of this study is to show that chemotherapy delivered directly through the artery supplying the eye (ophthalmic artery) to patients with retinoblastoma is a safe and effective treatment alternative to conventional systemic chemotherapy, external beam radiation, and surgical removal of the eye.


Condition Intervention Phase
Retinoblastoma
Drug: Melphalan hydrochloride
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Intra-arterial Chemotherapy for the Treatment of Intraocular Retinoblastoma

Resource links provided by NLM:


Further study details as provided by Sidney Kimmel Comprehensive Cancer Center:

Primary Outcome Measures:
  • Number of patients who complete therapy without the need for additional treatment including systemic chemotherapy, external beam radiation, or enucleation. [ Time Frame: Within the first six months after the initial treatment. ] [ Designated as safety issue: No ]
    The primary objective of this study is to show that intra-arterial delivery of the chemotherapeutic agent is successful in treating intraocular retinoblastoma, defined as avoiding systemic chemotherapy, external beam radiation, and enucleation.


Estimated Enrollment: 20
Study Start Date: March 2011
Estimated Study Completion Date: August 2021
Estimated Primary Completion Date: August 2015 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Melphalan hydrochloride

    Drug administered intra-arterially (injection in the artery).

    Standard dose:

    2.5mg (3-6 month old) 3.0 mg (6-12 month old) 4.0 mg (1-3 year old) 5.0 mg (>3 years old)

    Dose modification: decrease standard dose by 25% if there are signs of toxicity. Increase the dose by 25% if there is inadequate tumor response.

    Frequency: 2 treatment cycles at 3-4 week intervals, with a third treatment cycle administered if the tumor requires it.

    Dose not to exceed 0.5mg/kg, per treatment cycle.

    Other Name: Alkeran
Detailed Description:

Delivering the chemotherapeutic agent in the arterial system through the ophthalmic artery transforms the treatment of retinoblastoma from systemic chemotherapy to local chemotherapy. Administration of the drug directly to the targeted site thus avoids the complications and adverse events associated with toxicity from systemic, rather than local, chemotherapy.

  Eligibility

Ages Eligible for Study:   up to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients newborn to 18 years old.
  • Patients with intraocular retinoblastoma, unilateral or bilateral, who would be treated either by systemic chemotherapy, EBR, or enucleation would be considered for this study.

Exclusion Criteria:

  • Patients over the age of 18.
  • Patients with small, localized intraocular Rb amenable to focal therapy (laser or cryotherapy).
  • Patients with extraocular disease evident on MRI (extension into the optic nerve), massive choroidal/uveal invasion (grade IIC or IID per ARET0332) or disease outside the globe evident on MRI or physical examination.
  • Documented hypercoagulable disorders or vasculopathies.
  • Laboratory exclusion criteria: GFR < 60 mL/min/1.73 m2
  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: NCT01293539

Contacts
Contact: Monica Pearl, M.D. 410-955-8525 msmit135@jhmi.edu
Contact: Amber Jones, B.A., CCRP 410-502-0736 ajones78@jhmi.edu

Locations
United States, Maryland
The Johns Hopkins Hospital Recruiting
Baltimore, Maryland, United States, 21287
Sub-Investigator: James Handa, M.D.         
Sub-Investigator: Alan Friedman, M.D.         
Sub-Investigator: Philippe Gailloud, M.D.         
Sponsors and Collaborators
Sidney Kimmel Comprehensive Cancer Center
Investigators
Principal Investigator: Monica Pearl, M.D. The Johns Hopkins Hospital
  More Information

Publications:
Responsible Party: Sidney Kimmel Comprehensive Cancer Center
ClinicalTrials.gov Identifier: NCT01293539     History of Changes
Other Study ID Numbers: J1071, NA_00040637
Study First Received: January 21, 2011
Last Updated: August 6, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Sidney Kimmel Comprehensive Cancer Center:
Retinoblastoma
intra-arterial chemotherapy
angiography

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 November 25, 2014