Phase 1
Completed N=14
Intra-arterial Melphalan in Treating Younger Patients With Unilateral Retinoblastoma
Unilateral Retinoblastoma
Source: ClinicalTrials.gov NCT02097134 ↗
Enrolled (actual)
14
Serious AEs
14.3%
Results posted
Jun 2018
Primary outcomePrimary: Number of Patients Experiencing Feasibility Failure — 4 participants
Summary
This pilot clinical trial studies whether unilateral group D retinoblastoma, or retinoblastoma affecting one eye that has spread to the inner jelly like part of the eye, can be treated with a new technique for delivering chemotherapy directly into the blood vessel that supplies the affected eye. This new technique is called intra-arterial injection. Giving melphalan via intra-arterial injection may make it less likely that children will need surgery to remove the eye and may reduce the amount of treatment side effects.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Patients Experiencing Feasibility Failure |
4 | — |
| SECONDARY Incidence of Grade 3 or Higher CTCAE Adverse Events Associated With Multiple Doses of IA Chemotherapy |
38 | — |
| SECONDARY Probability of Ocular Salvage |
36 | — |
| SECONDARY Rate of Metastases of Retinoblastoma |
— | — |
| SECONDARY Vision Acuity, Assessed According to the Amblyopia Treatment Study Visual Acuity Testing Protocol |
— | — |
Eligibility Criteria
Inclusion Criteria
- Newly diagnosed patients with unilateral group D retinoblastoma
- Magnetic resonance imaging (MRI) (or computed tomography [CT] if MRI is not available) of the brain must be performed within 14 days prior to study entry
- Diagnostic examination under anesthesia (EUA) must be performed within 14 days prior to study entry
- Rapid central review confirmation of group D disease based on RetCam images from diagnostic EUA must be obtained before starting treatment
- Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2
- Patients must have a life expectancy of >= 8 weeks
- Patients must have adequate renal function, defined as:
- Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or
- A serum creatinine based on age/gender as follows:
- 1 month to = 16 years: 1.7 mg/dL (male); 1.4 mg/dL (female)
- Total bilirubin =< 1.5 x upper limit of normal (ULN) for age
- Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 2.5 x upper limit of normal (ULN) for age
Exclusion Criteria
- Patients with bilateral disease
- Unilateral retinoblastoma with group A, B, C, or E eyes
- Prior chemotherapy or radiation therapy for this disease (laser and cryotherapy are allowed and are not considered exclusion criteria)
- Clinical or neuroimaging evidence of extraocular disease or orbital optic nerve involvement
Data sourced from ClinicalTrials.gov (NCT02097134). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.