NCT01293539

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.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Mar 2011

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 21, 2011

Completed
20 days until next milestone

First Posted

Study publicly available on registry

February 10, 2011

Completed
19 days until next milestone

Study Start

First participant enrolled

March 1, 2011

Completed
5.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2016

Completed
12 months until next milestone

Results Posted

Study results publicly available

July 21, 2017

Completed
Last Updated

November 27, 2018

Status Verified

November 1, 2018

Enrollment Period

5.4 years

First QC Date

January 21, 2011

Results QC Date

June 22, 2017

Last Update Submit

November 1, 2018

Conditions

Retinoblastoma

Keywords

Retinoblastomaintra-arterial chemotherapyangiography

Outcome Measures

Primary Outcomes (1)

  • Number of Patients Who Complete Therapy Without the Need for Additional Treatment Including Systemic Chemotherapy, External Beam Radiation, or Enucleation.

    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.

    Within the first six months after the initial treatment.

Study Arms (1)

Intraocular Retinoblastoma Patients

OTHER

Single group assignment of patients with intraocular retinoblastoma, unilateral or bilateral.

Drug: Melphalan hydrochloride

Interventions

Melphalan hydrochlorideDRUG

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.

Also known as: Alkeran
Intraocular Retinoblastoma Patients

Eligibility Criteria

AgeUp to 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

The Johns Hopkins Hospital

Baltimore, Maryland, 21287, United States

Location

Related Publications (2)

  • Abramson DH, Dunkel IJ, Brodie SE, Kim JW, Gobin YP. A phase I/II study of direct intraarterial (ophthalmic artery) chemotherapy with melphalan for intraocular retinoblastoma initial results. Ophthalmology. 2008 Aug;115(8):1398-404, 1404.e1. doi: 10.1016/j.ophtha.2007.12.014. Epub 2008 Mar 14.

    PMID: 18342944BACKGROUND

  • Abramson DH. Chemosurgery for retinoblastoma: what we know after 5 years. Arch Ophthalmol. 2011 Nov;129(11):1492-4. doi: 10.1001/archophthalmol.2011.354. No abstract available.

    PMID: 22084221BACKGROUND

MeSH Terms

Conditions

Retinoblastoma

Interventions

Melphalan

Condition Hierarchy (Ancestors)

Neoplasms, NeuroepithelialNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms by Histologic TypeNeoplasmsNeoplasms, Glandular and EpithelialNeoplasms, Nerve TissueRetinal NeoplasmsEye NeoplasmsNeoplasms by SiteEye Diseases, HereditaryEye DiseasesRetinal Diseases

Intervention Hierarchy (Ancestors)

Nitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhenylalanineAmino Acids, AromaticAmino Acids, CyclicAmino AcidsAmino Acids, Peptides, and Proteins

Results Point of Contact

Title
Monica Pearl, M.D.
Organization
Johns Hopkins
msmit135@jhmi.edu
410-955-8525

Study Officials

  • Monica Pearl, M.D.

    Johns Hopkins University

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 21, 2011

First Posted

February 10, 2011

Study Start

March 1, 2011

Primary Completion

August 1, 2016

Study Completion

August 1, 2016

Last Updated

November 27, 2018

Results First Posted

July 21, 2017

Record last verified: 2018-11

Locations

US(1)