Intra-arterial Melphalan in Treating Younger Patients With Unilateral Retinoblastoma
|ClinicalTrials.gov Identifier: NCT02097134|
Recruitment Status : Active, not recruiting
First Posted : March 26, 2014
Last Update Posted : January 25, 2018
|Condition or disease||Intervention/treatment|
|Unilateral Retinoblastoma||Drug: Melphalan|
I. To study the feasibility of delivering melphalan directly into the ophthalmic artery in children with newly diagnosed unilateral group D retinoblastoma, who would otherwise be considered for enucleation.
I. To estimate the ocular salvage rate after treatment with intra-arterial melphalan in children with newly diagnosed unilateral retinoblastoma with group D disease.
II. To evaluate the toxicities and adverse events associated with delivering multiple doses of intra-arterial chemotherapy.
III. To evaluate vision outcomes in children treated with intra-arterial chemotherapy.
IV. To monitor the rate of the development of metastatic disease while on protocol therapy.
I. To evaluate the effects of intra-arterial therapy on the histopathology of eyes enucleated for progression.
Patients receive melphalan intra-arterially (IA) on day 1. Treatment repeats every 28 days for up to 3 courses in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up every 3 months for 1 year, every 4 months for 1 year, every 6 months for 1 year, and then periodically for 2 years.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||50 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Multi-institutional Feasibility Study of Intra-arterial Chemotherapy Given in the Ophthalmic Artery of Children With Retinoblastoma|
|Study Start Date :||April 2014|
|Estimated Primary Completion Date :||June 30, 2018|
|Estimated Study Completion Date :||June 30, 2018|
Experimental: Treatment (melphalan)
Patients receive melphalan IA on day 1. Treatment repeats every 28 days for up to 3 courses in the absence of disease progression or unacceptable toxicity.
- Incidence of IA feasibility failure [ Time Frame: Up to 4 months ]Feasibility failure is defined as a) interventional radiologist is unable to access the ophthalmic artery for the 1st chemotherapy administration for any reason; b) patient develops central retinal artery occlusion after the 1st or 2nd course that does not reopen by the time the next injection is due; or c) the patient cannot receive all three treatments because of Common Terminology Criteria for Adverse Events (CTCAE) complications grade III or IV that are considered possibly, probably or likely related treatment.
- Catheter insertion complication rate [ Time Frame: Up to 48 hours after catheter insertion procedures ]Defined as (1) thrombosis of the femoral artery; (2) dissection of any artery; (3) hematoma at the site of insertion of 3 centimeters or more in diameter; (4) emboli cerebral; or (5) any embolus in the lower extremity that results in vascular insufficiency.
- Histopathology of eyes enucleated for progression [ Time Frame: Up to 5 years ]The proportion of enucleated eyes with various characteristics, such as viable vitreous seeds, invasion into the optic nerve, ischemic atrophy involving the outer retina and choroid, and extensive choroidal and outer retinal atrophy will be calculated, as well as the 95% confidence intervals.
- Incidence of grade 3 or higher CTCAE adverse events associated with multiple doses of IA chemotherapy [ Time Frame: Up to 5 years ]The occurrence of each grade 3 or higher CTCAE adverse experience will be recorded in each patient-cycle. The percentage of patients with each toxicity will be tabulated per cycle according to the methodology in place for the study progress report at the time.
- Probability of ocular salvage [ Time Frame: 2 years ]
- Rate of metastases of retinoblastoma [ Time Frame: Up to 2 years ]
- Vision acuity, assessed according to the Amblyopia Treatment Study Visual Acuity Testing Protocol [ Time Frame: 1 year after therapy ]Estimated by the average visual acuity amongst patients evaluated with a 95% confidence interval.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02097134
|United States, California|
|Children's Hospital Los Angeles|
|Los Angeles, California, United States, 90027|
|UCSF Medical Center-Mission Bay|
|San Francisco, California, United States, 94158|
|United States, Connecticut|
|New Haven, Connecticut, United States, 06520|
|United States, Florida|
|University of Miami Miller School of Medicine-Sylvester Cancer Center|
|Miami, Florida, United States, 33136|
|United States, Massachusetts|
|Dana-Farber Cancer Institute|
|Boston, Massachusetts, United States, 02115|
|United States, North Carolina|
|Duke University Medical Center|
|Durham, North Carolina, United States, 27710|
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center|
|Cincinnati, Ohio, United States, 45229|
|United States, Pennsylvania|
|Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104|
|United States, Texas|
|Baylor College of Medicine|
|Houston, Texas, United States, 77030|
|Principal Investigator:||Murali Chintagumpala, MD||Children's Oncology Group|