Back to resultsIntra-arterial Chemotherapy for Retinoblastoma (IAC)
Primary Purpose
Retinoblastoma
Status
Active
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Melphalan
Sponsored by
About this trial
This is an interventional treatment trial for Retinoblastoma
Eligibility Criteria
Inclusion Criteria:
- Patients diagnosed with retinoblastoma >4 months of age16
- Patients whose other treatment options would require systemic chemotherapy, radiotherapy, or enucleation
- Patient or parent/legal guardian must sign a written informed consent
One of a, b, or c:
Patients who have bilateral COG stage B, C, D, or E retinoblastoma (refer to Appendix A) who have undergone systemic chemotherapy without resolution (meaning either has not had CR or has progressed despite systemic chemotherapy) and would have the following treatment options remaining:
- IAC
- enucleation of one eye
- local radiation
- Patients with non-germline retinoblastoma with unilateral disease who have COG A, B, C, or D tumors (refer to Appendix A).
- Other patients may be considered on a case by case basis after discussion with pediatric ophthalmology, hematology/oncology, and interventional neuroradiology.
Exclusion Criteria:
- Opaque or hazy media which precluded visualization of the fundus.
- New or recurrent retinoblastoma that can be controlled with other conservative measures such as cryotherapy, thermotherapy, or plaque radiotherapy.
- Unilateral COG group E retinoblastoma (refer to Appendix A) with very poor visual prognosis as defined by pediatric ophthalmologist.
- Patients who would benefit from systemic chemotherapy.
- Patients with clinical or radiological evidence suggestive of retinoblastoma invasion of the optic nerve, choroid, sclera, orbit or metastatic sites.
- Currently receiving any other investigational agents.
- A history of allergic reactions attributed to compounds of similar chemical or biologic composition to melphalan or other agents used in the study.
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia.
- Pregnant and/or breastfeeding. Participants of childbearing potential must have a negative pregnancy test within 14 days of study entry.
- Patients with HIV are eligible unless their CD4+ T-cell counts are < 350 cells/mcL or they have a history of AIDS-defining opportunistic infection within the 12 months prior to registration. Concurrent treatment with effective ART according to DHHS treatment guidelines is recommended.
Sites / Locations
- Washington University School of Medicine (Saint Louis Children's Hospital)
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Intra-arterial injections of melphalan
Arm Description
-Participants will receive intra-arterial injections of melphalan Q4W for 3 cycles.
Outcomes
Primary Outcome Measures
Feasibility of intra-arterial chemotherapy as measured by the number of participants who receive 3 intra-arterial injections of melphalan
-Feasibility is defined as the ability to receive 3 intra-arterial injections of melphalan per patient.
Secondary Outcome Measures
Rate of procedure-related complications
Rate of ocular salvage
A patient will be considered a successful ocular salvage if the child does not experience enucleation because of disease progression or toxicity during 2 years of follow-up.
Visual acuity as measured by Cardiff testing
Rate of metastatic disease
-Patients will be determined to have metastatic disease if they have received 1 or more administrations of IAC and they are found to have progression of disease or appearance of tumor outside of the eye after protocol therapy. Pineal tumors will not be considered metastatic disease.
Full Information
NCT ID
NCT04342572
First Posted
April 8, 2020
Last Updated
May 11, 2023
Sponsor
Washington University School of Medicine
1. Study Identification
Unique Protocol Identification Number
NCT04342572
Brief Title
Intra-arterial Chemotherapy for Retinoblastoma
Acronym
IAC
Official Title
Intra-arterial Chemotherapy for Retinoblastoma (IAC)
Study Type
Interventional
2. Study Status
Record Verification Date
May 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
August 11, 2020 (Actual)
Primary Completion Date
January 4, 2023 (Actual)
Study Completion Date
January 4, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Washington University School of Medicine
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
Children with retinoblastoma who may benefit from intra-arterial chemotherapy will receive up to 3 doses of melphalan and will be assessed for feasibility, toxicity, and response.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Retinoblastoma
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
5 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Intra-arterial injections of melphalan
Arm Type
Experimental
Arm Description
-Participants will receive intra-arterial injections of melphalan Q4W for 3 cycles.
Intervention Type
Drug
Intervention Name(s)
Melphalan
Other Intervention Name(s)
Evomela
Intervention Description
-The drug is commerically available
Primary Outcome Measure Information:
Title
Feasibility of intra-arterial chemotherapy as measured by the number of participants who receive 3 intra-arterial injections of melphalan
Description
-Feasibility is defined as the ability to receive 3 intra-arterial injections of melphalan per patient.
Time Frame
Completion of enrollment and treatment of all patients (estimated to be 32 months)
Secondary Outcome Measure Information:
Title
Rate of procedure-related complications
Time Frame
Through 30 days following completion of treatment (estimated to be 4 months)
Title
Rate of ocular salvage
Description
A patient will be considered a successful ocular salvage if the child does not experience enucleation because of disease progression or toxicity during 2 years of follow-up.
Time Frame
2 years post-treatment
Title
Visual acuity as measured by Cardiff testing
Time Frame
6 months post-treatment
Title
Rate of metastatic disease
Description
-Patients will be determined to have metastatic disease if they have received 1 or more administrations of IAC and they are found to have progression of disease or appearance of tumor outside of the eye after protocol therapy. Pineal tumors will not be considered metastatic disease.
Time Frame
Completion of treatment (estimated to be 3 months)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
4 Months
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Patients diagnosed with retinoblastoma >4 months of age16
Patients whose other treatment options would require systemic chemotherapy, radiotherapy, or enucleation
Patient or parent/legal guardian must sign a written informed consent
One of a, b, or c:
Patients who have bilateral COG stage B, C, D, or E retinoblastoma (refer to Appendix A) who have undergone systemic chemotherapy without resolution (meaning either has not had CR or has progressed despite systemic chemotherapy) and would have the following treatment options remaining:
IAC
enucleation of one eye
local radiation
Patients with non-germline retinoblastoma with unilateral disease who have COG A, B, C, or D tumors (refer to Appendix A).
Other patients may be considered on a case by case basis after discussion with pediatric ophthalmology, hematology/oncology, and interventional neuroradiology.
Exclusion Criteria:
Opaque or hazy media which precluded visualization of the fundus.
New or recurrent retinoblastoma that can be controlled with other conservative measures such as cryotherapy, thermotherapy, or plaque radiotherapy.
Unilateral COG group E retinoblastoma (refer to Appendix A) with very poor visual prognosis as defined by pediatric ophthalmologist.
Patients who would benefit from systemic chemotherapy.
Patients with clinical or radiological evidence suggestive of retinoblastoma invasion of the optic nerve, choroid, sclera, orbit or metastatic sites.
Currently receiving any other investigational agents.
A history of allergic reactions attributed to compounds of similar chemical or biologic composition to melphalan or other agents used in the study.
Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia.
Pregnant and/or breastfeeding. Participants of childbearing potential must have a negative pregnancy test within 14 days of study entry.
Patients with HIV are eligible unless their CD4+ T-cell counts are < 350 cells/mcL or they have a history of AIDS-defining opportunistic infection within the 12 months prior to registration. Concurrent treatment with effective ART according to DHHS treatment guidelines is recommended.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Margaret Reynolds, M.D.
Organizational Affiliation
Washington University School of Medicine
Official's Role
Principal Investigator
Facility Information:
Facility Name
Washington University School of Medicine (Saint Louis Children's Hospital)
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Links:
URL
http://www.siteman.wustl.edu
Description
Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine
Learn more about this trial
Intra-arterial Chemotherapy for Retinoblastoma
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