Alternating Systemic Chemotherapy and Intra-Arterial Melphalan Chemotherapy in Children With Intra-Ocular Retinoblastoma
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02116959|
Recruitment Status : Active, not recruiting
First Posted : April 17, 2014
Last Update Posted : June 28, 2018
|Condition or disease||Intervention/treatment||Phase|
|Advanced Intra-Ocular Retinoblastoma Retinoblastoma||Drug: Melphalan Drug: Carboplatin Drug: Etoposide Drug: Vincristine||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||6 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Pilot Study Evaluating the Safety of Alternating Systemic Chemotherapy and Intra-Arterial Melphalan Chemotherapy in Children With Newly Diagnosed Advanced Intra-Ocular Retinoblastoma|
|Study Start Date :||April 2014|
|Actual Primary Completion Date :||May 25, 2017|
|Estimated Study Completion Date :||May 2019|
Experimental: Cohort 1
Patients will receive alternating treatments beginning with systemic chemotherapy then followed by IA (intra-arterial) therapy.
Bilateral retinoblastoma patients will be in Cohort 1.
For bilateral Bilateral retinoblastoma patients where one eye is stage A or B and the other eye is C, D, or E, only the higher stage eye (C, D, E) will be treated with IA chemotherapy unless the stage A or B eye is not amenable or has failed local therapy.
Experimental: Cohort 2
Patients will receive only IA (intra-arterial) therapy for more limited disease.
- Adverse Event evaluation for newly diagnosed advanced retinoblastoma treated with melphalan therapy and systemic chemotherapy [ Time Frame: Up to 2 years ]
- Dose-Limiting Toxicity (DLT) and minimum tolerated dosing interval for combination IAM and CEV. [ Time Frame: Up to 2 years ]
- Response rates after IAM combined with systemic CEV in patients with newly diagnosed advanced intraocular retinoblastoma [ Time Frame: Up to 2 years ]
- Ocular event free survival with the use of combination IAM therapy and systemic CEV in this patient population. [ Time Frame: Up to 2 years ]
- Visual outcomes using a standardized age based assessment [ Time Frame: Up to 2 years ]
- Salvage rate with triple IA therapy after failure of IA melphalan [ Time Frame: Up to 2 years ]
- Ocular event free survival for unilateral retinoblastoma with IA chemotherapy only for Group C or Group A/B that failed local therapy. [ Time Frame: Up to 2 years ]
- Visual outcomes using a standardized age based assessment. [ Time Frame: Up to 2 years ]
- Collection of melphalan pharmacokinetics after intra-arterial administration to the retina [ Time Frame: Up to 2 years ]
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): NCT02116959
|United States, California|
|University of California, San Francisco|
|San Francisco, California, United States, 94143|
|Principal Investigator:||Anuradha Banerjee, MD||University of California, San Francisco|