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Alkylating agents

Intra-arterial Chemotherapy for Retinoblastoma (IAC Trial)

Phase 1
Waitlist Available
Led By Margaret Reynolds, M.D.
Research Sponsored by Washington University School of Medicine
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Patients whose other treatment options would require systemic chemotherapy, radiotherapy, or enucleation
Patients who have bilateral COG stage B, C, D, or E retinoblastoma who have undergone systemic chemotherapy without resolution and would have specific treatment options remaining
Must not have
Uncontrolled intercurrent illness including ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia
Unilateral COG group E retinoblastoma with very poor visual prognosis as defined by pediatric ophthalmologist
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 2 years post-treatment
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new way to give chemotherapy to children with retinoblastoma, a cancer of the eye. Up to 3 doses of melphalan will be given through a small tube in the artery. Doctors will check if it is safe and effective.

Who is the study for?
This trial is for children over 4 months old with retinoblastoma, a type of eye cancer. It's specifically for those who haven't responded to systemic chemotherapy and are facing limited treatment options like eye removal or radiation. Patients need consent from a parent or guardian. Those with allergies to melphalan, pregnant/breastfeeding individuals, HIV patients with low CD4+ counts or recent opportunistic infections, and cases where the cancer has spread significantly are excluded.
What is being tested?
The trial tests intra-arterial chemotherapy using melphalan in children with retinoblastoma. Participants will receive up to three doses of this drug directly into the artery supplying the eye tumor. The study aims to evaluate how feasible and toxic this approach is and how well it works in shrinking the tumors.
What are the potential side effects?
Melphalan may cause side effects such as damage to blood cells leading to increased infection risk, bleeding problems, allergic reactions similar to other drugs in its class, nausea, vomiting, mouth sores, and potential impact on fertility later in life.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
My next treatment options include chemotherapy, radiotherapy, or eye removal.
Select...
I have advanced retinoblastoma in both eyes and chemotherapy hasn't worked.
Select...
I have a type of eye cancer in one eye that is not inherited.
Select...
I was diagnosed with retinoblastoma when I was older than 4 months.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I do not have any uncontrolled illnesses or heart problems.
Select...
My child has advanced eye cancer in one eye with a poor outlook for vision.
Select...
My retinoblastoma can be managed with less invasive treatments.
Select...
I have HIV but my CD4+ T-cell count is above 350 and I haven't had an AIDS-related infection in the last year.
Select...
My tests suggest my eye cancer may have spread to critical areas.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~2 years post-treatment
This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 years post-treatment for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Feasibility of intra-arterial chemotherapy as measured by the number of participants who receive 3 intra-arterial injections of melphalan
Secondary study objectives
Rate of metastatic disease
Rate of ocular salvage

Side effects data

From 2021 Phase 3 trial • 30 Patients • NCT01877837
36%
Infection (grade 3 and above)
24%
Graft versus host disease
8%
Renal insufficiency
4%
SupraVentricular Tachycardia
4%
Alerted mental status
4%
Posterior Reversible Encephalopathy Syndrome
4%
Gastrointestinal bleed
4%
Respiratory failure
4%
Hypokalemia
100%
80%
60%
40%
20%
0%
Study treatment Arm
Patients With Sickle Cell Anemia

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Intra-arterial injections of melphalanExperimental Treatment1 Intervention
-Participants will receive intra-arterial injections of melphalan Q4W for 3 cycles.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Melphalan
2008
Completed Phase 3
~1500

Find a Location

Who is running the clinical trial?

Washington University School of MedicineLead Sponsor
1,991 Previous Clinical Trials
2,295,879 Total Patients Enrolled
1 Trials studying Retinoblastoma
14 Patients Enrolled for Retinoblastoma
Margaret Reynolds, M.D.Principal InvestigatorWashington University School of Medicine

Media Library

Melphalan (Alkylating agents) Clinical Trial Eligibility Overview. Trial Name: NCT04342572 — Phase 1
Retinoblastoma Research Study Groups: Intra-arterial injections of melphalan
Retinoblastoma Clinical Trial 2023: Melphalan Highlights & Side Effects. Trial Name: NCT04342572 — Phase 1
Melphalan (Alkylating agents) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04342572 — Phase 1
~1 spots leftby Nov 2025
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