Trial Outcomes & Findings for Intra-arterial Melphalan in Treating Younger Patients With Unilateral Retinoblastoma (NCT NCT02097134)

NCT ID: NCT02097134

Last Updated: 2023-10-13

Results Overview

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.

• Recruitment status: COMPLETED
• Study phase: PHASE1
• Target enrollment: 14 participants
• Primary outcome timeframe: Up to 4 months
• Results posted on: 2023-10-13

Participant Flow

Participant milestones

Measure	Melphalan Patients receive melphalan intra-arterial (IA) on day 1. Treatment repeats every 28 days for up to 3 courses in the absence of disease progression or unacceptable toxicity. Melphalan: Given IA
Overall Study STARTED	14
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	9

Reasons for withdrawal

Measure	Melphalan Patients receive melphalan intra-arterial (IA) on day 1. Treatment repeats every 28 days for up to 3 courses in the absence of disease progression or unacceptable toxicity. Melphalan: Given IA
Overall Study Physician Decision	4
Overall Study Non-Protocol Therapy	1
Overall Study Progressive Disease	3
Overall Study Refusal of Further Protocol Therapy	1

Baseline Characteristics

Intra-arterial Melphalan in Treating Younger Patients With Unilateral Retinoblastoma

Baseline characteristics by cohort

Measure	Melphalan n=14 Participants Patients receive melphalan intra-arterial (IA) on day 1. Treatment repeats every 28 days for up to 3 courses in the absence of disease progression or unacceptable toxicity. Melphalan: Given IA
Age, Continuous	1.92 years STANDARD_DEVIATION 0.93 • n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	7 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	8 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	4 Participants n=5 Participants
Region of Enrollment United States	14 participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 4 months

Population: All Patients who are eligible and evaluable for the feasibility outcome are included in this outcome

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.

Outcome measures

Measure	Melphalan n=12 Participants Patients receive melphalan intra-arterial (IA) on day 1. Treatment repeats every 28 days for up to 3 courses in the absence of disease progression or unacceptable toxicity. Melphalan: Given IA
Number of Patients Experiencing Feasibility Failure	4 participants

SECONDARY outcome

Timeframe: Up to 30 days after completion of study treatment

Population: One patient who did not receive protocol therapy was excluded.

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.

Outcome measures

Measure	Melphalan n=13 Participants Patients receive melphalan intra-arterial (IA) on day 1. Treatment repeats every 28 days for up to 3 courses in the absence of disease progression or unacceptable toxicity. Melphalan: Given IA
Incidence of Grade 3 or Higher CTCAE Adverse Events Associated With Multiple Doses of IA Chemotherapy	38 Percentage of patients Interval 18.0 to 64.0

SECONDARY outcome

Timeframe: 2 years

Population: One patient who did not receive protocol therapy was excluded. Two patients who were lost to follow-up or follow-up was terminated electively prior to 2 years by patient or parent preference were excluded.

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.

Outcome measures

Measure	Melphalan n=11 Participants Patients receive melphalan intra-arterial (IA) on day 1. Treatment repeats every 28 days for up to 3 courses in the absence of disease progression or unacceptable toxicity. Melphalan: Given IA
Probability of Ocular Salvage	36 Percentage of patients Interval 15.0 to 65.0

SECONDARY outcome

Timeframe: Up to 2 years

Population: One patient who did not receive protocol therapy was excluded.

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

Outcome measures

Measure	Melphalan n=13 Participants Patients receive melphalan intra-arterial (IA) on day 1. Treatment repeats every 28 days for up to 3 courses in the absence of disease progression or unacceptable toxicity. Melphalan: Given IA
Rate of Metastases of Retinoblastoma	0 Percentage of Patients

SECONDARY outcome

Timeframe: 1 year after therapy

Population: The study was terminated at stage I since the therapy was considered not feasible to deliver. No patients had this data available. Patients either had affected eye enucleated, or did not complete protocol therapy.

Estimated by the average visual acuity amongst patients evaluated with a 95% confidence interval.

Outcome measures

Outcome data not reported

Adverse Events

Melphalan

Serious events: 2 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Measure	Melphalan n=14 participants at risk Patients receive melphalan intra-arterial (IA) on day 1. Treatment repeats every 28 days for up to 3 courses in the absence of disease progression or unacceptable toxicity. Melphalan: Given IA
Vascular disorders Hypotension	7.1% 1/14 • Up to 4 months The Other Adverse Events field contains Common Terminology Criteria for Adverse Events (CTCAEs) reported on protocol therapy, excluding those that were reported as Serious Adverse Events. The Serious Adverse Events field contains NCI CTCAEs submitted via expedited reporting (NCI AdEERs / CAeRs).
Respiratory, thoracic and mediastinal disorders Hypoxia	7.1% 1/14 • Up to 4 months The Other Adverse Events field contains Common Terminology Criteria for Adverse Events (CTCAEs) reported on protocol therapy, excluding those that were reported as Serious Adverse Events. The Serious Adverse Events field contains NCI CTCAEs submitted via expedited reporting (NCI AdEERs / CAeRs).

Other adverse events

Measure	Melphalan n=14 participants at risk Patients receive melphalan intra-arterial (IA) on day 1. Treatment repeats every 28 days for up to 3 courses in the absence of disease progression or unacceptable toxicity. Melphalan: Given IA
Immune system disorders Anaphylaxis	7.1% 1/14 • Up to 4 months The Other Adverse Events field contains Common Terminology Criteria for Adverse Events (CTCAEs) reported on protocol therapy, excluding those that were reported as Serious Adverse Events. The Serious Adverse Events field contains NCI CTCAEs submitted via expedited reporting (NCI AdEERs / CAeRs).
Gastrointestinal disorders Gastrointestinal disorders - Other, specify	7.1% 1/14 • Up to 4 months The Other Adverse Events field contains Common Terminology Criteria for Adverse Events (CTCAEs) reported on protocol therapy, excluding those that were reported as Serious Adverse Events. The Serious Adverse Events field contains NCI CTCAEs submitted via expedited reporting (NCI AdEERs / CAeRs).
Investigations Neutrophil count decreased	14.3% 2/14 • Up to 4 months The Other Adverse Events field contains Common Terminology Criteria for Adverse Events (CTCAEs) reported on protocol therapy, excluding those that were reported as Serious Adverse Events. The Serious Adverse Events field contains NCI CTCAEs submitted via expedited reporting (NCI AdEERs / CAeRs).
Investigations Platelet count decreased	7.1% 1/14 • Up to 4 months The Other Adverse Events field contains Common Terminology Criteria for Adverse Events (CTCAEs) reported on protocol therapy, excluding those that were reported as Serious Adverse Events. The Serious Adverse Events field contains NCI CTCAEs submitted via expedited reporting (NCI AdEERs / CAeRs).

Additional Information

Results Reporting Coordinator

Children's Oncology Group

Phone: 626-447-0064

Email: resultsreportingcoordinator@childrensoncologygroup.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place