Clinical trial NCT04342572: Intra-arterial Chemotherapy for Retinoblastoma
Official title: Intra-arterial Chemotherapy for Retinoblastoma (IAC)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Retinoblastoma
Lead sponsor: Washington University School of Medicine
Sponsor class: OTHER
First posted: 2020-04-13
Enrollment: 5
Sex: ALL
Minimum age (years): 4
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: Inclusion Criteria:

* 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
    * 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.

Exclusion Criteria:

* 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.
* 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.

Primary outcomes:
measure: Feasibility of intra-arterial chemotherapy as measured by the number of participants who receive 3 intra-arterial injections of melphalan
timeFrame: Completion of enrollment and treatment of all patients (estimated to be 32 months)
Secondary outcomes:
measure: Rate of procedure-related complications
timeFrame: Through 30 days following completion of treatment (estimated to be 4 months)
measure: Rate of ocular salvage
timeFrame: 2 years post-treatment
measure: Visual acuity as measured by Cardiff testing
timeFrame: 6 months post-treatment
measure: Rate of metastatic disease
timeFrame: Completion of treatment (estimated to be 3 months)
Other outcomes:
Locations:
city: Saint Louis
country: United States
facility: Washington University School of Medicine (Saint Louis Children's Hospital)
state: Missouri
Study officials:
affiliation: Washington University School of Medicine
name: Margaret Reynolds, M.D.
role: PRINCIPAL_INVESTIGATOR
References: