NCT04465643

Neoadjuvant Nivolumab Plus Ipilimumab for Newly Diagnosed Malignant Peripheral Nerve Sheath Tumor

Completed Phase 1 Last updated 8 October 2025

What this trial tests

Phase 1 trial testing Nivolumab in Nerve Sheath Tumors in 13 participants. Completed in 30 July 2025.

Timeline

8 June 2021

Primary endpoint
30 July 2025

30 July 2025

Quick facts

Lead sponsor	Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	13
Start date	8 June 2021
Primary completion	30 July 2025
Estimated completion	30 July 2025
Sites	1 location across United States

Drugs / interventions tested

• Nivolumab (nivolumab) — full drug profile →
• Ipilimumab — full drug profile →

Conditions studied

• Nerve Sheath Tumors — all drugs for Nerve Sheath Tumors →

Sponsor

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins — full company profile →

Who can join

Adults 12 to 100, any sex, with Nerve Sheath Tumors. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The purpose of the study is to evaluate safety and feasibility of neoadjuvant nivolumab plus ipilimumab prior to standard therapy (surgery, chemotherapy or radiation therapy) in patients with Neurofibromatosis Type 1 (NF1) and newly diagnosed pre-malignant and malignant peripheral nerve sheath tumors (MPNST) for whom surgery for resection of tumor is indicated.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Biomarkers for immune checkpoint inhibition in sarcomas - are we close to clinical implementation?
   Yiong CS, Lin TP, Lim VY, Toh TB, et al · · 2023 · cited 26× · PMID 37612756 · DOI 10.1186/s40364-023-00513-5
2. The Role of Macrophages in Sarcoma Tumor Microenvironment and Treatment.
   Zając AE, Czarnecka AM, Rutkowski P. · · 2023 · cited 22× · PMID 37958467 · DOI 10.3390/cancers15215294
3. Diagnosis and Treatment of Peripheral and Cranial Nerve Tumors with Expert Recommendations: An EUropean Network for RAre CANcers (EURACAN) Initiative.
   Pellerino A, Verdijk RM, Nichelli L, Andratschke NH, et al · · 2023 · cited 20× · PMID 37046591 · DOI 10.3390/cancers15071930
4. Transcriptomic subtyping of malignant peripheral nerve sheath tumours highlights immune signatures, genomic profiles, patient survival and therapeutic targets.
   Høland M, Berg KCG, Eilertsen IA, Bjerkehagen B, et al · · 2023 · cited 17× · PMID 37837931 · DOI 10.1016/j.ebiom.2023.104829
5. Advances in immunotherapeutic targets for childhood cancers: A focus on glypican-2 and B7-H3.
   Li N, Spetz MR, Li D, Ho M. · · 2021 · cited 15× · PMID 33992682 · DOI 10.1016/j.pharmthera.2021.107892
6. Malignant Peripheral Nerve Sheath Tumor, a Heterogeneous, Aggressive Cancer with Diverse Biomarkers and No Targeted Standard of Care: Review of the Literature and Ongoing Investigational Agents.
   Somaiah N, Paudyal B, Winkler RE, Van Tine BA, et al · · 2024 · cited 14× · PMID 38954182 · DOI 10.1007/s11523-024-01078-5
7. Trends in clinical development of pediatric cancer for PD-1 and PD-L1 inhibitors: an analysis of ClinicalTrials.gov.
   Que Y, Hu Y, Hong D, Zhang Y. · · 2021 · cited 11× · PMID 34583971 · DOI 10.1136/jitc-2021-002920
8. The quest for effective immunotherapies against malignant peripheral nerve sheath tumors: Is there hope?
   Paudel SN, Hutzen B, Cripe TP. · · 2023 · cited 10× · PMID 37680255 · DOI 10.1016/j.omto.2023.07.008