RetinoblastomaPhase 1IACNCT04342572
Intra-arterial Chemotherapy for Retinoblastoma
A Phase 1 study testing Melphalan in people with Retinoblastoma, run by Washington University School of Medicine at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 4 months and older
- Conditions the study is for
- Retinoblastoma
- What is being tested?
- Melphalan
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- 11 Aug 2020to 21 Mar 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Washington University School of Medicine (Saint Louis Children's Hospital)
St Louis, Missouri, 63110
About this study
Official title: Intra-arterial Chemotherapy for Retinoblastoma (IAC)
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.
- Conditions
- Retinoblastoma
- Being tested
- Melphalan
- Medical subject headings
- Retinoblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MelphalanDrug
Also called Evomela
-The drug is commerically available
| Study group | Role | What they receive |
|---|---|---|
| Intra-arterial injections of melphalan | Experimental | -Participants will receive intra-arterial injections of melphalan Q4W for 3 cycles. Receives: Drug: Melphalan |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Completion of enrollment and treatment of all patients (estimated to be 32 months)
Secondary outcomes (4)
Rate of procedure-related complications
Measured: Through 30 days following completion of treatment (estimated to be 4 months)
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.
Measured: 2 years post-treatment
Visual acuity as measured by Cardiff testing
Measured: 6 months post-treatment
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.
Measured: Completion of treatment (estimated to be 3 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Apr 2020
- Study start11 Aug 2020
- Primary completion4 Jan 2023
- Study completion21 Mar 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for RetinoblastomaStudies of the same condition, open and under way.
- More trials run by Washington University School of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Washington University School of Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Margaret Reynolds, M.D., Principal investigator, Washington University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04342572
- Sponsor's protocol ID
- 202006160
- Acronym
- IAC
- Status last verified
- May 2025
- First submitted
- 8 Apr 2020
- Last updated
- 11 May 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intra-arterial Chemotherapy for Retinoblastoma (IAC)
- ClinicalTrials.gov ID
- NCT04342572
- Acronym / sponsor ID
- IAC / 202006160
- Conditions
- Retinoblastoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Washington University School of MedicineUniversity, hospital or other
- Study start
- 11 Aug 2020 (actual)
- Primary completion
- 4 Jan 2023 (actual)
- Study completion
- 21 Mar 2025 (actual)
- First posted
- 13 Apr 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Intra-arterial injections of melphalan | Experimental | Drug: Melphalan-Participants will receive intra-arterial injections of melphalan Q4W for 3 cycles. |
Who may qualify
- Ages
- 4 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- 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:
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MelphalanAlso: Evomela-The drug is commerically available | Drug | Intra-arterial injections of melphalan |
Primary outcome
Feasibility of intra-arterial chemotherapy as measured by the number of participants who receive 3 intra-arterial injections of melphalan
Time frame: Completion of enrollment and treatment of all patients (estimated to be 32 months)
-Feasibility is defined as the ability to receive 3 intra-arterial injections of melphalan per patient.
4 secondary outcomes
Rate of procedure-related complications
Time frame: Through 30 days following completion of treatment (estimated to be 4 months)
Rate of ocular salvage
Time frame: 2 years post-treatment
Visual acuity as measured by Cardiff testing
Time frame: 6 months post-treatment
Rate of metastatic disease
Time frame: Completion of treatment (estimated to be 3 months)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 May 2025.
- Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of MedicineLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Margaret Reynolds, M.D. principal investigator
Washington University School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Melphalan in people with Retinoblastoma, run by Washington University School of Medicine at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Feasibility of intra-arterial chemotherapy as measured by the number of participants who receive 3 intra-arterial injections of melphalan (time frame: Completion of enrollment and treatment of all patients (estimated to be 32 months)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 May 2025.