eligibility_summary
Inclusion: ≤21 at dx, >12 mo at enroll (safety run-in ≥6 y), staged ≤4 wks, R/R high-risk NB with measurable/evaluable disease, washouts: chemo 2 wks (nitrosourea 6), GF 5 d, SMI ≥7 d, IO 4 wks, XRT 30 d, SCT ≥2 mo (no GVHD), MIBG 6 wks, PS ≥50, adequate marrow/liver/renal, QTcF ≤470 ms, neg pregnancy/contraception, CNS steroids stable. Exclusion: <1 y, BSA <0.25, on investigational/anticancer therapy, uncontrolled infection, noncompliance, prior Gr4 naxitamab allergy.
trial_source
clinical_trials.gov from Dec 2, 2025
annotation_status
manual_review_required
ai_summary
Phase II (recruiting) study testing two agents in relapsed/refractory high-risk neuroblastoma: 1) Tipifarnib (R115777) — an oral small-molecule farnesyltransferase inhibitor that blocks farnesylation of proteins such as HRAS, disrupting RAS/MAPK and related survival/proliferation signaling in tumor cells. 2) Naxitamab (Danyelza) — an IV anti-GD2 IgG1 monoclonal antibody that binds GD2 on neuroblastoma cells and induces antibody-dependent cellular cytotoxicity and complement-dependent cytotoxicity via NK cells, macrophages, and complement. Targets/pathways: GD2-expressing neuroblastoma cells (including bone/bone marrow disease), farnesylated RAS-pathway proteins within tumor cells (RAS/MAPK signaling). Goals: evaluate safety, tolerability, and antitumor activity (responses, progression-free survival).