Clinical trial · Interventional
Intra-arterial Chemotherapy for the Treatment of Intraocular Retinoblastoma
NCT01293539CI-TRIAL-00035942terminatedPhase 2Results postedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Why stopped (as posted): IAC is now recognized as part of standard of care treatment options in the US.
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Retinoblastoma | Retinoblastoma | ONTOLOGY_EXACT | 0.98 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Melphalan hydrochloride | Drug | Melphalan | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- OTHER
- label
- Intraocular Retinoblastoma Patients
- description
- Single group assignment of patients with intraocular retinoblastoma, unilateral or bilateral.
- interventionNames
- Drug: Melphalan hydrochloride
Primary outcomes (1)
- measure
- Number of Patients Who Complete Therapy Without the Need for Additional Treatment Including Systemic Chemotherapy, External Beam Radiation, or Enucleation.
- timeFrame
- Within the first six months after the initial treatment.
- description
- 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
Eligibility (as posted)
- Sex
- All
- Maximum age
- 18 Years
Show eligibility criteria text
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
References
Publications (2)
- BACKGROUNDAbramson 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 18342944
- BACKGROUNDAbramson 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 22084221