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Intra-arterial Chemotherapy for the Treatment of Intraocular Retinoblastoma

This study has been terminated.
(IAC was initially not an available retinoblastoma treatment at Johns Hopkins. It is now recognized as part of the standard of care treatment options in the US.)
Information provided by (Responsible Party):
Sidney Kimmel Comprehensive Cancer Center Identifier:
First received: January 21, 2011
Last updated: June 22, 2017
Last verified: June 2017
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: Intervention Model: Single Group Assignment
Masking: None (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. ]
    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.

Enrollment: 10
Study Start Date: March 2011
Estimated Study Completion Date: August 2021
Primary Completion Date: August 2016 (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.

Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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
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Please refer to this study by its identifier: NCT01293539

United States, Maryland
The Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
Sponsors and Collaborators
Sidney Kimmel Comprehensive Cancer Center
Principal Investigator: Monica Pearl, M.D. Johns Hopkins University
  More Information

Responsible Party: Sidney Kimmel Comprehensive Cancer Center Identifier: NCT01293539     History of Changes
Other Study ID Numbers: J1071
NA_00040637 ( Other Identifier: JHMIRB )
Study First Received: January 21, 2011
Results First Received: June 22, 2017
Last Updated: June 22, 2017

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

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, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs processed this record on August 22, 2017