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NCT02097134 · CITED IN SOURCE DOCUMENTS

Intra-arterial Melphalan in Treating Younger Patients With Unilateral Retinoblastoma

An NCI or FDA source cites this study. A citation does not establish that it applies to an individual diagnosis.

Phase
PHASE1
Status at capture
COMPLETED
Registry last update
2023-10-13

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.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

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baseline

Baseline characteristics

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outcome · POSTED

Number of Patients Experiencing Feasibility Failure

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outcome · POSTED

Incidence of Grade 3 or Higher CTCAE Adverse Events Associated With Multiple Doses of IA Chemotherapy

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outcome · POSTED

Probability of Ocular Salvage

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outcome · POSTED

Rate of Metastases of Retinoblastoma

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outcome · POSTED

Vision Acuity, Assessed According to the Amblyopia Treatment Study Visual Acuity Testing Protocol

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adverse events

Adverse events

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more info

Limitations, agreements, and source contact

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Who could take part
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 \< 6 months: 0.4 mg/dL * 6 months to \< 1 year: 0.5 mg/dL * 1 to \< 2 years: 0.6 mg/dL * 2 to \< 6 years: 0.8 mg/dL * 6 to \< 10 years: 1 mg/dL * 10 to \< 13 years: 1.2 mg/dL * 13 to \< 16 years: 1.5 mg/dL (male); 1.4 mg/dL (female) * \>= 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
healthy Volunteers
false
minimum Age
6 Months
sex
ALL
std Ages
  1. CHILD
  2. ADULT
  3. OLDER_ADULT
Treatment arms and interventions
arm Groups
  1. description
    Patients receive melphalan IA on day 1. Treatment repeats every 28 days for up to 3 courses in the absence of disease progression or unacceptable toxicity.
    intervention Names
    1. Drug: Melphalan
    label
    Treatment (melphalan)
    type
    EXPERIMENTAL
interventions
  1. arm Group Labels
    1. Treatment (melphalan)
    description
    Given IA
    name
    Melphalan
    other Names
    1. Alanine Nitrogen Mustard
    2. CB-3025
    3. L-PAM
    4. L-Phenylalanine Mustard
    5. L-Sarcolysin
    6. L-Sarcolysin Phenylalanine mustard
    7. L-Sarcolysine
    8. Melphalanum
    9. Phenylalanine Mustard
    10. Phenylalanine Nitrogen Mustard
    11. Sarcoclorin
    12. Sarkolysin
    type
    DRUG
Study design
allocation
NA
intervention Model
SINGLE_GROUP
masking Info
masking
NONE
primary Purpose
TREATMENT
Enrollment
count
14
type
ACTUAL
Registered outcome plans (not posted results)
primary Outcomes
  1. description
    Feasibility failure is defined as a) interventional radiologist is unable to access the ophthalmic artery for the 1st chemotherapy administration for any reason; b) patient develops central retinal artery occlusion after the 1st or 2nd course that does not reopen by the time the next injection is due; or c) the patient cannot receive all three treatments because of Common Terminology Criteria for Adverse Events (CTCAE) complications grade III or IV that are considered possibly, probably or likely related treatment.
    measure
    Number of Patients Experiencing Feasibility Failure
    time Frame
    Up to 4 months
secondary Outcomes
  1. description
    The percentage of patients with at least 1 occurrence of grade 3 or higher CTCAE adverse experience will be provided. Ineligible patients or patients who do not receive any protocol therapy are excluded from reporting of adverse events.
    measure
    Incidence of Grade 3 or Higher CTCAE Adverse Events Associated With Multiple Doses of IA Chemotherapy
    time Frame
    Up to 30 days after completion of study treatment
  2. description
    A patient will be considered an ocular-salvage success if enucleation because of disease progression or toxicity is not required during the 2 years following enrollment.
    measure
    Probability of Ocular Salvage
    time Frame
    2 years
  3. description
    The percentage of patients who experience metastases of retinoblastoma will be estimated. Ineligible patients or patients who do not receive any protocol therapy are excluded from this analysis
    measure
    Rate of Metastases of Retinoblastoma
    time Frame
    Up to 2 years
  4. description
    Estimated by the average visual acuity amongst patients evaluated with a 95% confidence interval.
    measure
    Vision Acuity, Assessed According to the Amblyopia Treatment Study Visual Acuity Testing Protocol
    time Frame
    1 year after therapy
Full study description
brief 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.
detailed Description
PRIMARY OBJECTIVES: I. To study the feasibility of delivering melphalan directly into the ophthalmic artery in children with newly diagnosed unilateral group D retinoblastoma, who would otherwise be considered for enucleation. SECONDARY OBJECTIVES: I. To estimate the ocular salvage rate after treatment with intra-arterial melphalan in children with newly diagnosed unilateral retinoblastoma with group D disease. II. To evaluate the toxicities and adverse events associated with delivering multiple doses of intra-arterial chemotherapy. III. To evaluate vision outcomes in children treated with intra-arterial chemotherapy. IV. To monitor the rate of the development of metastatic disease while on protocol therapy. TERTIARY OBJECTIVES: I. To evaluate the effects of intra-arterial therapy on the histopathology of eyes enucleated for progression. OUTLINE: Patients receive melphalan intra-arterially (IA) on day 1. Treatment repeats every 28 days for up to 3 courses in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up every 3 months for 1 year, every 4 months for 1 year, every 6 months for 1 year, and then periodically for 2 years.
Source references
Source notices and limitations
    Discovery and provenance
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      occurrences
      1. context exact
        gist skilled in this area and a pediatric anesthesiologist. The Children&#8217;s Oncology Group conducted a multi-institutional study (<a href="/clinicaltrials/NCT02097134">ARET12P1 [NCT02097134]</a>) to evaluate the feasibility of administering intra-arterial therapy to newly diagnosed patients with Group D retinoblastoma. The
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      2. context exact
        ea and a pediatric anesthesiologist. The Children&#8217;s Oncology Group conducted a multi-institutional study (<a href="/clinicaltrials/NCT02097134">ARET12P1 [NCT02097134]</a>) to evaluate the feasibility of administering intra-arterial therapy to newly diagnosed patients with Group D retinoblastoma. The study failed to meet th
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      source retrieved at
      2026-09-09T23:22:14.810107+00:00
      source string basis
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      source string sha256
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      source title
      Retinoblastoma Treatment (PDQ®)–Health Professional Version
      source update dates
      1. context
        Updated: April 3, 2025
        datetime
        2025-04-03T12:00:00Z
        display
        April 3, 2025

    Preserved source evidence · Not medical advice