Intra-arterial Melphalan in Treating Younger Patients With Unilateral Retinoblastoma
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|ClinicalTrials.gov Identifier: NCT02097134|
Recruitment Status : Active, not recruiting
First Posted : March 26, 2014
Results First Posted : June 26, 2018
Last Update Posted : September 22, 2020
|Condition or disease||Intervention/treatment||Phase|
|Unilateral Retinoblastoma||Drug: Melphalan||Not Applicable|
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)|
|Actual Enrollment :||14 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|
|Actual Study Start Date :||April 16, 2014|
|Actual Primary Completion Date :||June 30, 2017|
|Estimated Study Completion Date :||September 30, 2021|
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.
- Number of Patients Experiencing 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.
- Incidence of Grade 3 or Higher CTCAE Adverse Events Associated With Multiple Doses of IA Chemotherapy [ Time Frame: Up to 30 days after completion of study treatment ]The percentage of patients with at least 1 occurrence of grade 3 or higher CTCAE adverse experience will be provided. Ineligible patients or patients who do not receive any protocol therapy are excluded from reporting of adverse events.
- Probability of Ocular Salvage [ Time Frame: 2 years ]A patient will be considered an ocular-salvage success if enucleation because of disease progression or toxicity is not required during the 2 years following enrollment.
- Rate of Metastases of Retinoblastoma [ Time Frame: Up to 2 years ]The percentage of patients who experience metastases of retinoblastoma will be estimated. Ineligible patients or patients who do not receive any protocol therapy are excluded from this analysis
- 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.
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): NCT02097134
|United States, California|
|Children's Hospital Los Angeles|
|Los Angeles, California, United States, 90027|
|UCSF Medical Center-Parnassus|
|San Francisco, California, United States, 94143|
|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, Georgia|
|Children's Healthcare of Atlanta - Egleston|
|Atlanta, Georgia, United States, 30322|
|United States, Massachusetts|
|Dana-Farber Cancer Institute|
|Boston, Massachusetts, United States, 02215|
|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/Dan L Duncan Comprehensive Cancer Center|
|Houston, Texas, United States, 77030|
|M D Anderson Cancer Center|
|Houston, Texas, United States, 77030|
|Principal Investigator:||Murali Chintagumpala||Children's Oncology Group|