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Intra-arterial Chemotherapy for the Treatment of Intraocular Retinoblastoma

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ClinicalTrials.gov Identifier: NCT01293539
Recruitment Status : Terminated (IAC is now recognized as part of standard of care treatment options in the US.)
First Posted : February 10, 2011
Results First Posted : July 21, 2017
Last Update Posted : August 28, 2017
Information provided by (Responsible Party):

Study Description
Brief Summary:
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 or disease Intervention/treatment Phase
Retinoblastoma Drug: Melphalan hydrochloride Phase 2

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.

Study Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Intra-arterial Chemotherapy for the Treatment of Intraocular Retinoblastoma
Study Start Date : March 2011
Primary Completion Date : August 2016
Estimated Study Completion Date : August 2021

Arms and Interventions

Arm Intervention/treatment
Intraocular Retinoblastoma Patients
Single group assignment of patients with intraocular retinoblastoma, unilateral or bilateral.
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

Outcome Measures

Primary Outcome Measures :
  1. 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.

Eligibility Criteria

Information from the National Library of Medicine

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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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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
ClinicalTrials.gov Identifier: NCT01293539     History of Changes
Other Study ID Numbers: J1071
NA_00040637 ( Other Identifier: JHMIRB )
First Posted: February 10, 2011    Key Record Dates
Results First Posted: July 21, 2017
Last Update Posted: August 28, 2017
Last Verified: July 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