A Phase 1 interventional study of Biospecimen Collection and Computed Tomography in Acral Lentiginous Melanoma, Central Nervous System Melanoma and Clinical Stage IV Cutaneous Melanoma AJCC v8, sponsored by M.D. Anderson Cancer Center. Active, not recruiting at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-08-19.
Sponsored by M.D. Anderson Cancer Center · Phase 1, Interventional, and Treatment
This phase I/Ib trial studies the side effects and best dose of intrathecal nivolumab, and how well it works in combination with intravenous nivolumab in treating patients with leptomeningeal disease. Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.
PRIMARY OBJECTIVE:
I. To determine the safety and/or recommended dose of intrathecal (IT) nivolumab in combination with systemic nivolumab treatment in melanoma and lung cancer with leptomeningeal disease (LMD).
SECONDARY OBJECTIVE:
I. To assess overall survival with combined intrathecal and systemic administration of nivolumab in this patient population.
EXPLORATORY OBJECTIVES:
I. Compare the immunological effects of this treatment on immune cells in the cerebrospinal fluid (CSF) to those observed in the peripheral blood and in non-LMD tumors.
II. Evaluation of predictors (clinical, molecular, and/or immune) of the efficacy and safety of this regimen.
III. To assess the effect of nivolumab on subsequent treatment. IV. To compare levels of nivolumab in the CSF and peripheral blood.
OUTLINE: This is a phase I, dose-escalation study followed by a phase Ib study.
Patients receive nivolumab IT over 5 minutes on day 1 of every cycle. Beginning in cycle 2, patients also receive nivolumab intravenously (IV) over 30 minutes on day 1 (4 hours after the IT dose). Cycles repeat every 14 days for 18 cycles and then every 28 days (cycles 19 and beyond) in the absence of disease progression or unacceptable toxicity. Patients will have CSF and blood specimen collection on days 1, 2, 8 of each cycle and end of treatment. Patients undergo computed tomography (CT) or positron emission tomography (PET) at baseline, cycle 5 and then every 8 weeks. Patients undergo magnetic resonance imaging (MRI) at baseline, cycles 3, 5, and then every 8 weeks.
After completion of study treatment, patients are followed up within 4 weeks and then every 12 weeks thereafter.
Patients who have been treated with an approved targeted therapy (BRAF inhibitor and/or MEK inhibitor) will be allowed to remain on concurrent approved targeted therapy. No other concomitant intrathecal therapy with another agent will be allowed. For patients that have received other systemic therapies, the minimum wash out period is as follows:
For patients with lung cancer:
Women are eligible to participate if:
Females treated with hormone replacement therapy, (HRT) are likely to have artificially suppressed FSH levels and may require a washout period in order to obtain a physiologic FSH level. The duration of the washout period is a function of the type of HRT used. The duration of the washout period below are suggested guidelines and the investigators should use their judgment in checking serum FSH levels. If the serum FSH level is >40 mIU/ml at any time during the washout period, the woman can be considered postmenopausal:
Exclusion Criteria:
Subjects with a condition requiring systemic treatment with either corticosteroids (> 4 mg daily dexamethasone equivalents) or other immunosuppressive medications within 14 days of study drug administration. Tocilizumab and vedolizumab are permitted, as are inhaled or topical steroids and adrenal replacement doses in the absence of active autoimmune disease
Patients receive nivolumab IT over 5 minutes on day 1 of every cycle. Beginning in cycle 2, patients also receive nivolumab IV over 30 minutes on day 1 (4 hours after the IT dose). Cycles repeat every 14 days for 18 cycles and then every 28 days (cycles 19 and beyond) in the absence of disease progression or unacceptable toxicity. Patients will have CSF and blood specimen collection on days 1, 2, 8 of each cycle and end of treatment. Patients undergo CT or PET at baseline, cycle 5 and then every 8 weeks. Patients undergo MRI at baseline, cycles 3, 5, and then every 8 weeks.
Procedure: Biospecimen Collection · Procedure: Computed Tomography · Procedure: Lumbar Puncture · Procedure: Magnetic Resonance Imaging · Biological: Nivolumab · Procedure: Positron Emission Tomography
Correlative studies
Also known as: Biological Sample Collection, Biospecimen Collected, Specimen Collection
Undergo CT
Also known as: CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography, Computerized Tomography, CT, CT Scan, tomography
Undergo lumbar puncture for cerebrospinal fluid collection
Also known as: LP, Spinal Tap
Undergo MRI of brain and spine
Also known as: Magnetic Resonance Imaging Scan, Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance, MR, MR Imaging, MRI, MRI Scan, NMR Imaging, NMRI, Nuclear Magnetic Resonance Imaging
Given IV or IT
Also known as: BMS-936558, CMAB819, MDX-1106, NIVO, Nivolumab Biosimilar CMAB819, ONO-4538, Opdivo
Undergo PET
Also known as: Medical Imaging, Positron Emission Tomography, PET, PET Scan, Positron Emission Tomography Scan, Positron-Emission Tomography, proton magnetic resonance spectroscopic imaging
Incidence of adverse events
Safety and tolerability of treatment will be assessed by vital signs, laboratory assessments, adverse events, and serious adverse events for the safety population. Adverse events will be graded by the Common Terminology Criteria for Adverse Events version 4.0. Categorical measures will be summarized using frequencies and percentages while continuous variables will be summarized using mean, standard deviation, median, minimum, and maximum.
Time frame: Up to 2 years
Recommended dose of combined intrathecal (IT) and intravenous (IV) nivolumab defined as the highest dose for which the posterior probability of toxicity is closest to 30% (dose escalation part)
The Bayesian modified toxicity probability interval method will be used to find the recommended dose.
Time frame: Up to 28 days
Overall survival (OS) in patients treated with IT and IV nivolumab (dose expansion part)
Time frame: Up to 2 years
OS
The Kaplan-Meier method will be used to estimate the distribution of OS from the start of study treatment, and Cox proportional hazard regression will be used to assess the relationship between OS and various covariates of interest, including but not limited to patient demographics, tumor characteristics, disease characteristics, and the expression of biomarkers.
Time frame: Up to 2 years
Immunological effects of nivolumab
Time frame: Up to 2 years
This study is active, not recruiting, as verified in Aug 2026. You cannot join it, but the record below documents what was studied.
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M.D. Anderson Cancer Center