Intra-arterial Chemotherapy for the Treatment of Intraocular Retinoblastoma

Phase 2

Terminated

• Conditions: Retinoblastoma
• Interventions: Drug: Melphalan hydrochloride

• Registration Number: NCT01293539
• Lead Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

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.

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 Timeline

• Study First Submitted: 2011-01-21
• Study First Submitted QC: 2011-02-09
• Study First Posted: 2011-02-10
• Study Start: 2011-03
• Primary Completion: 2016-08
• Study Completion: 2016-08
• Results First Submitted: 2017-06-22
• Results First Submitted QC: 2017-06-22
• Results First Posted: 2017-07-21
• Status Verified: 2018-11
• Last Update Submitted: 2018-11-01
• Last Update Posted: 2018-11-27

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 10

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Intraocular Retinoblastoma Patients	Melphalan hydrochloride	Single group assignment of patients with intraocular retinoblastoma, unilateral or bilateral.

Primary Outcome Measures

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

Trial Locations

Locations (1)

The Johns Hopkins Hospital; 🇺🇸 Baltimore, Maryland, United States