Actively Recruiting
Retinoblastoma Phase II Expanded Access Clinical Trial
Led by Targeted Therapy Technologies, LLC · Updated on 2026-04-14
30
Participants Needed
2
Research Sites
192 weeks
Total Duration
On this page
Sponsors
T
Targeted Therapy Technologies, LLC
Lead Sponsor
M
Morgan Stanley Children's Hospital
Collaborating Sponsor
AI-Summary
What this Trial Is About
In this research study investigators want to learn more about treatment of advanced or recurrent retinoblastoma. For children with retinoblastoma that have an advanced stage of presentation in one eye or if they have failed all conventional treatment, eye removal is considered. This study will investigate the utility of a chemoplaque(s) to salvage eyes involved with retinoblastoma. The goal of the study is to further determine/assess the safety and efficacy and optimal chemotherapy dose for retinoblastoma.
CONDITIONS
Official Title
Retinoblastoma Phase II Expanded Access Clinical Trial
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants must be younger than 8 years old.
- Diagnosis of Group D or earlier intraocular retinoblastoma where eye removal is recommended, OR active residual or recurrent intraocular retinoblastoma after first-line therapy.
- If both eyes require treatment, each will be treated separately; non-study eye may receive focal therapy.
- Intraocular calcium must be visible in the tumor eye by ultrasound or imaging.
- The study eye must have vision potential with at least light perception and no signs of high risk for cancer spread outside the eye.
- Performance level must be Lansky ≥ 50 (for children under 16 years) or Karnofsky ≥ 50 (for ages 16 and older).
- Adequate bone marrow function: neutrophils ≥ 1000/mm3, platelets ≥ 100,000/mm3 without recent transfusion, hemoglobin ≥ 8.0 g/dL.
- Adequate kidney function: creatinine clearance or GFR ≥ 70 ml/min/1.73 m2 or age/gender appropriate serum creatinine levels.
- Adequate liver function: bilirubin ≤ 1.5 times upper limit normal, SGPT (ALT) ≤ 110 U/L, serum albumin ≥ 2 g/dL.
- Females of reproductive potential must agree to use effective contraception during the study and 40 days after treatment.
- Ability to understand and sign informed consent; assent obtained when appropriate.
You will not qualify if you...
- Tumor involving the optic nerve rim.
- Evidence of cancer spread outside the eye, including optic nerve invasion or metastatic retinoblastoma.
- Allergy to topotecan, camptothecin, or related drugs.
- Receipt of chemotherapy, other retinoblastoma therapy, or investigational agents within 3 weeks before treatment.
- Uncontrolled illness that increases risk or limits study compliance.
- Recent febrile illness within one week before starting treatment.
- Positive pregnancy test or breastfeeding during the study.
- Any condition that may interfere with study participation or affect study results as judged by the investigator.
AI-Screening
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Trial Site Locations
Total: 2 locations
1
NewYork Presbyterian Morgan Stanley Children's Hospital
New York, New York, United States, 10032
Actively Recruiting
2
Children's Wisconsin
Milwaukee, Wisconsin, United States, 53226
Actively Recruiting
Research Team
B
Brian Marr, MD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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