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| ID | Type | Description | Link |
|---|---|---|---|
| NA_00040637 | Other Identifier | JHMIRB |
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IAC is now recognized as part of standard of care treatment options in the US.
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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.
Delivering the chemotherapeutic agent in the arterial system through the ophthalmic artery transforms the treatment of retinoblastoma from systemic chemotherapy to local chemotherapy. Administration of the drug directly to the targeted site thus avoids the complications and adverse events associated with toxicity from systemic, rather than local, chemotherapy.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Intraocular Retinoblastoma Patients | Other | Single group assignment of patients with intraocular retinoblastoma, unilateral or bilateral. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Melphalan hydrochloride | Drug | Drug administered intra-arterially (injection in the artery). Standard dose: 2.5mg (3-6 month old) 3.0 mg (6-12 month old) 4.0 mg (1-3 year old) 5.0 mg (>3 years old) Dose modification: decrease standard dose by 25% if there are signs of toxicity. Increase the dose by 25% if there is inadequate tumor response. Frequency: 2 treatment cycles at 3-4 week intervals, with a third treatment cycle administered if the tumor requires it. Dose not to exceed 0.5mg/kg, per treatment cycle. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Patients Who Complete Therapy Without the Need for Additional Treatment Including Systemic Chemotherapy, External Beam Radiation, or Enucleation. | 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. | Within the first six months after the initial treatment. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Monica Pearl, M.D. | Johns Hopkins University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Johns Hopkins Hospital | Baltimore | Maryland | 21287 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 18342944 | Background | Abramson 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. | |
| 22084221 | Background |
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| ID | Title | Description |
|---|---|---|
| FG000 | Intra-arterial Chemotherapy | IAC is offered to all patients with intraocular Rb who are deemed eligible after evaluation under anesthesia (EUA),with the exception of those few patients for whom focal modalities (i.e., laser or cryotherapy) would be effective.In cases of bilateral disease, both eyes would be treated sequentially during the same procedure utilizing the same femoral access site. Two cycles of IAC will be performed at 3 to 4 week intervals. Melphalan dosing: 3-6 mth-old, 2.5mg; 3-12 mth-old, 3mg; 1-3 yr-old, 4mg; >3 yr-old, 5mg. For those tumors that have shown appropriate response and are amenable to local therapies, then no additional IAC will be performed. If the tumors do not regress after 2 cycles, then a third cycle of IAC will be offered. Local therapies (laser, cryotherapy) will not be given during the EUAs conducted prior to each treatment session, but will be allowed after toxicity and response has been assessed 3-4 weeks after the final cycle of intra-arterial therapy. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Intra-arterial Chemotherapy | IAC is offered to all patients with intraocular Rb who are deemed eligible after evaluation under anesthesia (EUA),with the exception of those few patients for whom focal modalities (i.e., laser or cryotherapy) would be effective.In cases of bilateral disease, both eyes would be treated sequentially during the same procedure utilizing the same femoral access site. Two cycles of IAC will be performed at 3 to 4 week intervals. Melphalan dosing: 3-6 mth-old, 2.5mg; 3-12 mth-old, 3mg; 1-3 yr-old, 4mg; >3 yr-old, 5mg. For those tumors that have shown appropriate response and are amenable to local therapies, then no additional IAC will be performed. If the tumors do not regress after 2 cycles, then a third cycle of IAC will be offered. Local therapies (laser, cryotherapy) will not be given during the EUAs conducted prior to each treatment session, but will be allowed after toxicity and response has been assessed 3-4 weeks after the final cycle of intra-arterial therapy. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Patients Who Complete Therapy Without the Need for Additional Treatment Including Systemic Chemotherapy, External Beam Radiation, or Enucleation. | 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. | Posted | Count of Participants | Participants | Within the first six months after the initial treatment. |
|
4.5 years
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Intra-arterial Chemotherapy | IAC is offered to all patients with intraocular Rb who are deemed eligible after evaluation under anesthesia (EUA),with the exception of those few patients for whom focal modalities (i.e., laser or cryotherapy) would be effective.In cases of bilateral disease, both eyes would be treated sequentially during the same procedure utilizing the same femoral access site. Two cycles of IAC will be performed at 3 to 4 week intervals. Melphalan dosing: 3-6 mth-old, 2.5mg; 3-12 mth-old, 3mg; 1-3 yr-old, 4mg; >3 yr-old, 5mg. For those tumors that have shown appropriate response and are amenable to local therapies, then no additional IAC will be performed. If the tumors do not regress after 2 cycles, then a third cycle of IAC will be offered. Local therapies (laser, cryotherapy) will not be given during the EUAs conducted prior to each treatment session, but will be allowed after toxicity and response has been assessed 3-4 weeks after the final cycle of intra-arterial therapy. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Glaucoma | Eye disorders | CTCAE (4.0) | Non-systematic Assessment | Eye-disorder, Grade 1 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Eyelid edema & erythema | Eye disorders | CTCAE (4.0) | Non-systematic Assessment | Eyelid disorder, Grade 1 |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Monica Pearl, M.D. | Johns Hopkins | 410-955-8525 | msmit135@jhmi.edu |
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| ID | Term |
|---|---|
| D012175 | Retinoblastoma |
| ID | Term |
|---|---|
| D018302 | Neoplasms, Neuroepithelial |
| D017599 | Neuroectodermal Tumors |
| D009373 | Neoplasms, Germ Cell and Embryonal |
| D009370 | Neoplasms by Histologic Type |
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| ID | Term |
|---|---|
| D008558 | Melphalan |
| ID | Term |
|---|---|
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
| D006838 | Hydrocarbons |
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|
|
| Abramson 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. |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
|
|
| 3 |
| 10 |
| 6 |
| 10 |
| Retinal vascular disorder | Eye disorders | CTCAE (4.0) | Non-systematic Assessment | Eye-disorder, Grade 2 |
|
| Retinal vascular disorder | Eye disorders | CTCAE (4.0) | Non-systematic Assessment | Eye-disorder, Grade 3 |
|
|
| Eyelid function disorder | Eye disorders | CTCAE (4.0) | Non-systematic Assessment | Eye disorder, Grade 1 |
|
| Injury - cut to lip during play | Injury, poisoning and procedural complications | CTCAE (4.0) | Non-systematic Assessment | Injury, Grade 2 |
|
| Eyelid function disorder | Eye disorders | CTCAE (4.0) | Non-systematic Assessment | Eye disorder, Grade 2 |
|
| Eye pain | Eye disorders | CTCAE (4.0) | Non-systematic Assessment | Eye disorder, Grade 1 |
|
| Mild swelling | Eye disorders | CTCAE (4.0) | Non-systematic Assessment | Eye disorders, other, Grade 1 |
|
| Cough | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Non-systematic Assessment | Cough, Grade 1 |
|
| Conjunctivitis | Eye disorders | CTCAE (4.0) | Non-systematic Assessment | Conjunctivitis, Grade 2 |
|
| Flu-like symptoms | General disorders | CTCAE (4.0) | Non-systematic Assessment | Flu-like symptoms, Grade 2 |
|
| Cough | Respiratory, thoracic and mediastinal disorders | CTCAE (4.0) | Non-systematic Assessment | Cough, Grade 2 |
|
| Vomiting | Gastrointestinal disorders | CTCAE (4.0) | Non-systematic Assessment | Vomiting, Grade 1 |
|
| Fever | General disorders | CTCAE (4.0) | Non-systematic Assessment | Fever, Grade 2 |
|
| Fever | General disorders | CTCAE (4.0) | Non-systematic Assessment | Fever, Grade 1 |
|
| Ear infection | Infections and infestations | CTCAE (4.0) | Non-systematic Assessment | Otitis media, Grade 2 |
|
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| D009369 | Neoplasms |
| D009375 | Neoplasms, Glandular and Epithelial |
| D009380 | Neoplasms, Nerve Tissue |
| D019572 | Retinal Neoplasms |
| D005134 | Eye Neoplasms |
| D009371 | Neoplasms by Site |
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D012164 | Retinal Diseases |
| D009930 |
| Organic Chemicals |
| D010649 | Phenylalanine |
| D024322 | Amino Acids, Aromatic |
| D000598 | Amino Acids, Cyclic |
| D000596 | Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |