Neoplasms

Eye Diseases

All Conditions

Diseases and Abnormalities at or Before Birth

Rare Diseases

Retinoblastoma

Eye Neoplasms

Neoplasms, Neuroepithelial

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Retinal Neoplasms

Eye Diseases, Hereditary

Retinal Diseases

Neuroepithelioma

Central Nervous System Depressants

All Drugs and Chemicals

Anesthetics

Radiation given daily for 5 days in a row each week (except for Saturdays, Sundays, and holidays). The whole treatment will take about 4-6 weeks. Ophthalmic examination under anesthesia including dilated eye exam, ocular fundus photography (Ret-Cam), ocular echography and neuro-radiologic assessment (as deemed necessary)

Radiation given daily for 5 days in a row each week (except for Saturdays, Sundays, and holidays). The whole treatment will take about 4-6 weeks.

Proton Beam Radiation Therapy

Ophthalmic examination under anesthesia including dilated eye exam, ocular fundus photography (Ret-Cam), ocular echography and neuro-radiologic assessment (as deemed necessary)

Ophthalmic EUA

Dan Gombos, MD

The purpose of this trial is to evaluate proton beam radiation therapy as an alternative to external photon beam irradiation in children with retinoblastoma as a means of local tumor control and ocular retention.

External photon beam radiation therapy (which is current standard of care) has been used in the treatment of retinoblastoma. It is a form of radiation therapy in which the radiation is delivered by a machine pointed at the area to be radiated.

Proton beam radiation therapy is a form of external photon beam radiation therapy, but it may be more effective because its adjusted dosing delivers less radiation to surrounding areas of the tumor, which helps preserve other tissues and cause fewer side effects.

Before you can start treatment on this study, you will have what are called "screening tests." These tests will help the doctor decide if you are eligible to take part in this study. Your eye doctor will perform an assessment of your eye, including an exam while you are under anesthesia. This exam will include a dilated eye exam, photography of your affected eye (by a Ret-Cam), ultrasound of your affected eye, and imaging scans, such as computed tomography (CT) or magnetic resonance imaging (MRI), if the doctor thinks it is necessary. The assessments will be done to check and confirm the status of the disease.

After diagnosis of retinoblastoma, the study doctor will first see if external photon beam radiation therapy is a reasonable treatment option for you. If external photon beam radiotherapy is deemed appropriate for you, you will be eligible to receive proton beam radiation therapy in this study.

If you are found to be eligible to take part in this study, you will be treated with proton beam radiation therapy. Treatment will begin within 1 week of referral to the M. D. Anderson Proton Center. It will be given daily for 5 days in a row each week (except for Saturdays, Sundays, and holidays). The whole treatment will take about 4-6 weeks.

Within 2-4 weeks after completion of therapy, you will have an eye exam performed under anesthesia (like the one during screening). You will have repeated eye exams under anesthesia, depending on the appearance and response of the tumor to therapy. This will occur about every 1-4 months.

You will continue to have these eye exams until the tumors are considered to be stable or unless your disease gets worse.

If the disease gets worse or you experience any intolerable side effects, you may be taken off treatment.

You will be required to have lifelong eye and medical assessments to continue to monitor you for disease. If no disease is found 5 years after completion of proton beam radiation therapy, you will be considered free of disease. You will then have annual (yearly) eye and pediatric assessment through adulthood.

This is an investigational study. Up to 20 patients will take part in this study. All will be enrolled at M.D. Anderson.

Proton Beam Radiation Therapy for Intraocular and Periocular Retinoblastoma

A Phase II Trial of Proton Beam Radiation Therapy for Intra- and Periocular Retinoblastoma

Where proton beam radiation therapy used as an alternative to external photon beam irradiation in children with retinoblastoma as a means of local tumor control and ocular retention, local tumor control measured for participants with a globe as tumor regression with ocular retention, and for post enucleation measured as lack of orbital tumor recurrence.

M.D. Anderson Cancer Center

Proton Beam Radiation Therapy given daily for 5 days in a row each week where whole treatment takes about 4-6 weeks. Ophthalmic examination under anesthesia including dilated eye exam, ocular fundus photography (Ret-Cam), ocular echography and neuro-radiologic assessment (as deemed necessary).

Age, Categorical

Sex: Female, Male

United States

Region of Enrollment

No analysis performed as a result of small study population.

Dan Gombos, MD/Professor, Ophthalmology

No analysis performed. Study did not meet anticipated enrollment.

Rate of Local Control in the Globe at 12 Months

Overall Study

Antineoplastic Agents

Melphalan

Antineoplastic Agents, Alkylating

Alkylating Agents

Immunosuppressive Agents

Immunologic Factors

Intra-arterial chemotherapy with melphalan, via direct administration by catheterization of the ophthalmic artery. Dosage range from 3 to 5 mg, depending of patient's weight and estimated tumour volume: a)3 mg for patients under 10 kg and tumour volume size under 1,5 cm3; b)5 mg for patients over 10 kg and tumour volume over 1,5 cm3; c)4 mg in all other situations(tumour volume over 1,5 cm3 in patients under 10 kg or tumour volume under 1,5 cm3 in patients over 10 kg).

The usual number of chemotherapy cycles will be 3. First treatment with Melphalan will be initiated as soon as possible, once the patients are selected for the study. Once the first treatment has been completed, day 0, successive treatments will be considered, second and third (days 21 and 42). In those cases in which some patients could be considered for an additional treatments after the third cycle, these will be administrated in intervals at least of 21 days.Dosage range from 3 to 5 mg, depending of patient's weight and estimated tumour volume.

Andreu Parareda, MD

For selected cases with advanced Retinoblastoma (RTB) intraocular involvement(stage V of the Reese-Ellsworth classification) in which enucleation would usually be the standard therapeutic approach, in this project the investigators propose an alternative conservative treatment using intra-arterial chemotherapy with melphalan, via direct administration by catheterization of the ophthalmic artery.

Retinoblastoma (RTB) is the most frequent tumour of the eye in early childhood and the commonest cancer in the first year of life. Approximately 60% of cases are sporadic and unilateral. Unilateral tumours are usually diagnosed in the advanced intraocular stage and the most frequent treatment prescribed is enucleation. This prevents disease progression but has an important visual risk, and also constitutes a mutilation, with potentially devastating psychological effects on patients and their relatives.

At diagnosis, patients affected with RTB and their relatives are faced with the important effects of this disease, such as a threat to life, although rare in developed countries, and the risk of losing their sight, which depends on the uni- or bilateral nature of the tumour, the topography of the tumour or tumours, and the still prevalent need for enucleation as a treatment. In fact, almost all advanced stage unilateral RTBs are treated with enucleation. In addition to the risk to life and the patient's sight associated with this treatment, it is also important to take into account the risk to the eye itself. For selected cases with advanced intraocular involvement (stage V of the Reese-Ellsworth classification) in which enucleation would usually be the standard therapeutic approach, in this project we propose an alternative conservative treatment using intra-arterial chemotherapy with melphalan, via direct administration by catheterization of the ophthalmic artery.

The treatment aims to preserve the eye ball and visual acuity as much as possible in these patients, and has been demonstrated to be extremely effective at achieving volumetric reduction of tumours, which permits, if necessary, the subsequent conservative treatment, mainly with brachytherapy for anterior tumours or thermotherapy with laser diode for posterior tumours. In cases of retinal detachment, significant volume reduction, such as that achieved after injection with melphalan would, in most cases, permit retinal reapplication that would favour visual prognosis.

This technique was first described by David H. Abramson in the Sloan-Kettering Cancer Center Memorial Hospital of New York (A Phase I/II Study of direct Intra-arterial (Ophthalmic Artery) Chemotherapy with Melphalan for Intraocular Retinoblastoma). Here, in this study we propose using this technique for the first time outside New York city, in our own clinical setting (Retinoblastoma Unit, affiliated to the Oncology Development Department and the Ophthalmology Department of the Sant Joan de Déu Hospital in Barcelona).

The attainment of positive results, in addition to those previously obtained by the New York project, could consolidate this treatment as an alternative to enucleation in most cases of advanced intraocular RTB, and open the way for the future indication of this technique in other stages of RTB.

Intra-arterial Chemotherapy With Melphalan for the Treatment of Retinoblastoma (RTB) in Advanced Intraocular Stage

An Open, Single-centre Non-randomized Phase II Clinical Trial on Intra-arterial Chemotherapy With Melphalan for the Treatment of Retinoblastoma (RTB) in Advanced Intraocular Stage

The primary endpoint will be the objective response to treatment determined by funduscopy and RetCam explorations, recorded as a percentage of partial response (PR) or complete response (CR) to the treatment administered.

The response will be evaluated as a function of tumoral volumetric size by comparing images obtained in successive funduscopy and RetCam explorations.

The secondary endpoint studied will correspond to changes in visual acuity of the affected eye after the third cycle of treatment, assessed by a visual acuity study.

The secondary endpoint studied will correspond to changes in electroretinographic studies and visual evoked potentials after the third cycle of treatment

The secondary safety endpoints studied will correspond to analytical changes in the CBC (essentially in the number of absolute neutrophils) and biochemistry analysis after 10 days of each treatment, and potential ophthalmologic side effects.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Proton Beam Radiation Therapy for Intraocular and Periocular Retinoblastoma	NCT00432445	Retinoblastoma	Proton Beam Rad... Ophthalmic EUA	-	M.D. Anderson Cancer Center
Intra-arterial Chemotherapy With Melphalan for the Treatment of Retinoblastoma (RTB) in Advanced Intraocular Stage	NCT01393769	Retinoblastoma	Melphalan	6 Months - 6 Years	Hospital Sant Joan de Deu

Spots Global Cancer Trial Database for tumor of the eye