A Study to Evaluate Treatment With Nivolumab Alone and Nivolumab Combined With Ipilimumab in Children With Melanoma

Real-World Evaluation of Patient Characteristics, Treatment Patterns, Safety, and Efficacy of Nivolumab Monotherapy and Nivolumab in Combination With Ipilimumab for Pediatric Melanoma

The purpose of this study is to describe real-world safety outcomes in children with melanoma who are treated with nivolumab alone or nivolumab in combination with ipilimumab for unresectable or metastatic melanoma, or treated with adjuvant nivolumab after resection of stage IIB-IV melanoma. Demographic and clinical characteristics, and treatment patterns, will also be described in this population.

Study Overview

• Status: Completed
• Conditions: Melanoma
• Intervention / Treatment: Drug: Nivolumab +/- ipilimumab; Drug: Nivolumab

• Study Type: Observational
• Enrollment (Actual): 100

Contacts and Locations

Study Locations

• United States
  • Ohio
    • Dublin, Ohio, United States, 43017
      • Cardinal Health

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Pediatric participants (<18 years of age) who were treated with nivolumab monotherapy or nivolumab combined with ipilimumab for unresectable or metastatic melanoma or treated with adjuvant nivolumab after resection of stage IIb-IV melanoma

Description

Inclusion Criteria:

• Participants with confirmed diagnosis of unresectable or metastatic cutaneous melanoma, or stage IIB-IV cutaneous melanoma who have undergone complete resection
• Initiated treatment with nivolumab monotherapy or in combination with ipilimumab for unresectable or metastatic melanoma, or as adjuvant therapy for resected stage IIb-IV melanoma, between January 1, 2015 and 6 months prior to data collection
• For the adjuvant nivolumab cohort, initiated treatment with nivolumab for stage IIb-IV melanoma within 12 weeks following index resection
• Aged <18 years at the time of nivolumab/ipilimumab treatment initiation
• At least 6 months of follow-up from index treatment date (unless deceased prior to end of 6 months)

Exclusion Criteria:

• Prior malignancy active within 3 years prior to nivolumab or ipilimumab treatment index date except for locally curable cancers that have been apparently cured (such as basal or squamous cell skin cancer, superficial bladder cancer, or carcinoma in situ of the prostate, cervix, or breast)
• Receiving nivolumab therapy (monotherapy, combination with ipilimumab, or adjuvant therapy) for melanoma as part of a clinical trial during the study period

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Participants treated with nivolumab +/- ipilimumab for unresectable/metastatic melanoma	Drug: Nivolumab +/- ipilimumab; Participants treated with nivolumab +/- ipilimumab for unresectable/metastatic melanoma
Participants treated with nivolumab as adjuvant therapy for resected stage IIB-IV melanoma	Drug: Nivolumab; Participants treated with nivolumab as adjuvant therapy for resected stage IIB-IV melanoma

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of treatment-related adverse events	Up to 100 days after treatment discontinuation
Incidence of all-cause adverse events	Up to 100 days after treatment discontinuation

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Participant year of birth		Six months prior to index date
Participant gender		Six months prior to index date
Participant race		Six months prior to index date
Participant ethnicity		Six months prior to index date
Participant state/region of residence		Six months prior to index date
Participant primary insurance payer at treatment initiation		Six months prior to index date
Date of initial melanoma diagnosis		Six months prior to index date
Date of index resection for participants treated with adjuvant nivolumab		Six months prior to index date
Participant height		Initiation of index treatment
Participant weight		Initiation of index treatment
Stage of melanoma at diagnosis, assessed by American Joint Committee on Cancer criteria		Six months prior to index date
Margins at index resection for participants treated with adjuvant nivolumab after resection		Six months prior to index date
Lymph node biopsy results		Six months prior to index date
Lymph node involvement - Stage III/IV only		Six months prior to index date
Tumor ulceration status		Six months prior to index date
Tumor ulceration characteristics		Six months prior to index date
Eastern Cooperative Oncology Group Performance Status (ECOG-PS)		Initiation of index treatment
Lactate dehydrogenase level		Initiation of index treatment
Index treatment		Initiation of index treatment
Number of lines of therapy received for unresectable/metastatic melanoma		From index therapy initiation to death or the date of last follow-up/study end date, whichever came first, assessed up to 108 months
Number of lines of therapy received post resection		From index therapy initiation to death or the date of last follow-up/study end date, whichever came first, assessed up to 108 months
Disease response characteristics		From index therapy initiation to death or the date of last follow-up/study end date, whichever came first, assessed up to 108 months
Disease recurrence characteristics		From index therapy initiation to death or the date of last follow-up/study end date, whichever came first, assessed up to 108 months
Duration of therapy (DOT)		From index therapy initiation to death or the date of last follow-up/study end date, whichever came first, assessed up to 108 months
Time to next treatment (TTNT)		From index therapy initiation to next subsequent systemic melanoma treatment post-nivolumab, death, or the date of last follow-up/study end date, whichever came first, assessed up to 108 months
Treatment-free interval (TFI)		From discontinuation of index treatment to date of initiation of subsequent therapy, assessed up to 108 months
Overall survival (OS)		From index therapy initiation to death or the date of last follow-up/study end date, whichever came first, assessed up to 108 months
Healthcare resource utilization assessed by the number of inpatient hospitalizations		From initiation of therapy until end of data collection date or death, whichever came first, assessed up to 108 months
Healthcare resource utilization assessed by the number of emergency department (ED) visits		From initiation of therapy until end of data collection date or death, whichever came first, assessed up to 108 months
Overall response rate (ORR) in participants with unresectable or metastatic disease	Number of participants with complete or partial response divided by the total number of participants	From index therapy initiation to the date of last follow-up/study end date, whichever came first, assessed up to 108 months
Progression-free survival (PFS) in participants with unresectable or metastatic disease		From index therapy initiation to date of first documented disease progression, death, or the date of last follow-up/study end date, whichever came first, assessed up to 108 months
Time to initial response (TTR) in participants with unresectable or metastatic disease		From index therapy initiation to date of the first physician-reported response (complete or partial response), assessed up to 108 months
Duration of response (DOR) in participants with unresectable or metastatic disease		From date of first physician-reported response (complete or partial response) to date of the first physician-reported disease progression, death, or date of last follow-up/study end date, whichever came first, assessed up to 108 months
Time to progression (TTP) in participants with unresectable or metastatic disease		From index therapy initiation to date of physician-reported disease progression, or the date of last follow-up/study end date, whichever came first, assessed up to 108 months
Time to local disease recurrence in participants with resected disease		From index therapy initiation to date of last follow-up/study end date, whichever came first, assessed up to 108 months
Recurrence free survival (RFS) in participants with resected disease		From index therapy initiation to date of first melanoma recurrence, death, or the date of last follow-up/study end date, whichever came first, assessed up to 108 months
Time to distant metastases in participants with resected disease		From index therapy initiation to date of last follow/up or study end date, whichever came first, assessed up to 108 months
Distant-metastasis free survival (DMFS) in participants with resected disease		From index therapy initiation to date of first distant melanoma metastasis, death, or the date of last follow-up/study end date, whichever came first, assessed up to 108 months
Type of disease recurrence/metastasis in participants with resected disease		From index therapy initiation to date of last follow-up visit or study end date, whichever came first, assessed up to 108 months

Collaborators and Investigators

• Sponsor: Bristol-Myers Squibb
• Investigators: Study Director: Bristol-Myers Squibb, Bristol-Myers Squibb

Publications and helpful links

Helpful Links

• BMS Clinical Trial Information
• FDA Safety Alerts and Recalls

Study record dates

Study Major Dates

• Study Start (Actual): August 14, 2023
• Primary Completion (Actual): September 25, 2023
• Study Completion (Actual): November 10, 2023

Study Registration Dates

• First Submitted: October 20, 2023
• First Submitted That Met QC Criteria: November 30, 2023
• First Posted (Actual): December 8, 2023

Study Record Updates

• Last Update Posted (Actual): April 18, 2024
• Last Update Submitted That Met QC Criteria: April 17, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Neoplasms by Histologic Type; Neoplasms; Neoplasms by Site; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Neuroendocrine Tumors; Nevi and Melanomas; Skin Neoplasms; Melanoma; Molecular Mechanisms of Pharmacological Action; Antineoplastic Agents; Antineoplastic Agents, Immunological; Immune Checkpoint Inhibitors; Nivolumab; Ipilimumab
• Other Study ID Numbers: CA209-1068

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No