NCT03628209

A Phase Ib/II Study to Evaluate the Safety, Feasibility and Efficacy of Nivolumab or Nivolumab in Combination With Azacitidine in Patients With Recurrent, Resectable Osteosarcoma

Active, enrolled Phase 1 Last updated 3 February 2026

What this trial tests

Phase 1 trial testing Nivolumab in Osteosarcoma in 21 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

3 October 2019

Primary endpoint
29 December 2024

1 April 2026

Quick facts

Lead sponsor	H. Lee Moffitt Cancer Center and Research Institute
Phase	Phase 1
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	21
Start date	3 October 2019
Primary completion	29 December 2024
Estimated completion	1 April 2026
Sites	22 locations across United States

Drugs / interventions tested

• Nivolumab (nivolumab) — full drug profile →
• Azacitidine (azacitidine) — full drug profile →
• Post Treatment Surgery

Conditions studied

• Osteosarcoma — all drugs for Osteosarcoma →
• Osteosarcoma in Children — all drugs for Osteosarcoma in Children →
• Osteosarcoma Recurrent — all drugs for Osteosarcoma Recurrent →
• Sarcoma — all drugs for Sarcoma →

Sponsor

H. Lee Moffitt Cancer Center and Research Institute

Who can join

Under 39, any sex, with Osteosarcoma or Osteosarcoma in Children. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Phase I: Recommended Phase II Dose (RP2D)
  Time frame: 60 days
  If one or fewer dose limiting toxicities (DLT's) occur in 6 participants the study will advance to the next dose level. If 2 or more DLT's occur at a dose level, the prior dose level will be identified as the RP2D.
• Phase II: Rate of Continued Complete Remission (CR)
  Time frame: 1 year post surgery
  Continued complete remission by computed tomography (CT) scan 1 year after surgery.

Sponsor's own description

The purpose of this study is to evaluate the safety and efficacy of nivolumab, or nivolumab in combination with azacitidine in participants with recurrent, resectable osteosarcoma

Publications & conference data

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

1. Targeting epigenetic regulators for cancer therapy: mechanisms and advances in clinical trials.
   Cheng Y, He C, Wang M, Ma X, et al · · 2019 · cited 760× · PMID 31871779 · DOI 10.1038/s41392-019-0095-0
2. Epigenetic regulation in the tumor microenvironment: molecular mechanisms and therapeutic targets.
   Yang J, Xu J, Wang W, Zhang B, et al · · 2023 · cited 251× · PMID 37217462 · DOI 10.1038/s41392-023-01480-x
3. Advances on immunotherapy for osteosarcoma.
   Yu S, Yao X. · · 2024 · cited 146× · PMID 39245737 · DOI 10.1186/s12943-024-02105-9
4. Pathogenesis and Current Treatment of Osteosarcoma: Perspectives for Future Therapies.
   Rathore R, Van Tine BA. · · 2021 · cited 81× · PMID 33809018 · DOI 10.3390/jcm10061182
5. Effects of microenvironment in osteosarcoma on chemoresistance and the promise of immunotherapy as an osteosarcoma therapeutic modality.
   Yu L, Zhang J, Li Y. · · 2022 · cited 47× · PMID 36311748 · DOI 10.3389/fimmu.2022.871076
6. Systematic Review of Recurrent Osteosarcoma Systemic Therapy.
   Gazouli I, Kyriazoglou A, Kotsantis I, Anastasiou M, et al · · 2021 · cited 43× · PMID 33917001 · DOI 10.3390/cancers13081757
7. The Crossroads of Cancer Epigenetics and Immune Checkpoint Therapy.
   Micevic G, Bosenberg MW, Yan Q. · · 2023 · cited 41× · PMID 36449280 · DOI 10.1158/1078-0432.ccr-22-0784
8. Targeting epigenetic regulators as a promising avenue to overcome cancer therapy resistance.
   Song J, Yang P, Chen C, Ding W, et al · · 2025 · cited 40× · PMID 40675967 · DOI 10.1038/s41392-025-02266-z