Clinical Trials for Sarcoma

Single-arm, Open-label, Phase 1b/2 Trial of Nivolumab Therapy Following Partially HLA Mismatched (Haploidentical) Bone Marrow Transplant in Children and Young Adults With High Risk, Recurrent or Refractory Sarcomas

High burden on patient

The trial has a high burden on patients. An example would be a phase 1 trial with extra tumor biopsies and frequent pharmacokinetic blood draws.

Started >3 years ago

The trial was activated more than 3 years ago. This is not always bad but can indicate that the trial has less current treatments, or has had trouble getting enough patients to enroll due to other issues.

HealthScout AI summary: Children, adolescents, and young adults (12 months–50 years) with high-risk, recurrent, or refractory sarcomas (and select poor-prognosis solid tumors) who are ≥day +120 after reduced-intensity haploidentical bone marrow transplant with PTCy receive post-transplant nivolumab. Nivolumab is a PD-1–blocking monoclonal antibody given IV (weight/flat dosing) for up to 24 cycles to enhance donor T‑cell antitumor activity while monitoring for GVHD and immune-related toxicities.

ClinicalTrials.gov ID: NCT03465592

Phase I Study of HER2 Chimeric Antigen Receptor (CAR) T Cells in Combination With Checkpoint Blockade in Patients With Advanced Sarcoma (HEROS 3.0)

High burden on patient

The trial has a high burden on patients. An example would be a phase 1 trial with extra tumor biopsies and frequent pharmacokinetic blood draws.

Started >3 years ago

The trial was activated more than 3 years ago. This is not always bad but can indicate that the trial has less current treatments, or has had trouble getting enough patients to enroll due to other issues.

HealthScout AI summary: Pediatric, adolescent, and young adult patients (1–25 years) with recurrent/progressive HER2‑positive sarcoma (≥1+ by IHC) after prior systemic therapy receive autologous HER2‑targeted second‑generation (CD28ζ) CAR T cells after cyclophosphamide/fludarabine lymphodepletion, followed about a week later by PD‑1 blockade with pembrolizumab (q3w) or nivolumab (q2w), with option for repeat CAR T infusions. Excludes significant autoimmune disease, active infection, bulky disease, prior severe Cy/Flu or checkpoint reactions, and major cardiopulmonary contraindications.

ClinicalTrials.gov ID: NCT04995003

A Phase 1, Two-Part, Multicenter, Open-Label First in Human Study of Anti-GD2 Antibody Drug Conjugate M3554 in Participants With Advanced Solid Tumors

No known activity

No investigational drug(s) in this trial have shown anticancer activity yet in clinical trials

High burden on patient

The trial has a high burden on patients. An example would be a phase 1 trial with extra tumor biopsies and frequent pharmacokinetic blood draws.

HealthScout AI summary: Adults with ECOG 0–1 and either advanced, unresectable/metastatic soft tissue sarcoma after at least one prior metastatic-line (including anthracycline) or IDH‑wildtype glioblastoma after first-line chemoradiation are eligible. Investigational therapy is M3554, an anti‑GD2 antibody–drug conjugate delivering the topoisomerase I inhibitor exatecan via a cleavable linker, given as monotherapy in dose escalation/expansion.

ClinicalTrials.gov ID: NCT06641908

A Phase 1/2a, Open-Label Study of Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SA53-OS, an MDM2 Inhibitor, in Patients With Locally Advanced or Metastatic p53 Wild-Type Solid Tumors

No known activity

No investigational drug(s) in this trial have shown anticancer activity yet in clinical trials

High burden on patient

The trial has a high burden on patients. An example would be a phase 1 trial with extra tumor biopsies and frequent pharmacokinetic blood draws.

HealthScout AI summary: Adults with refractory locally advanced/metastatic TP53 (p53) wild-type solid tumors (Phase 1 also allows relapsed/refractory non-Hodgkin lymphoma) receive SA53-OS, an oral small-molecule MDM2 inhibitor designed to reactivate wild-type p53. Phase 2a expands to dedifferentiated liposarcoma with MDM2 amplification and other TP53 wild-type solid tumors, with SA53-OS given 3 days every 3 weeks at the RP2D.

ClinicalTrials.gov ID: NCT06578624

A Phase II Trial of Reduced Intensity Conditioning and Partially HLA-mismatched (HLA-haploidentical) Related Donor Bone Marrow Transplantation for High-risk Solid Tumors

High burden on patient

The trial has a high burden on patients. An example would be a phase 1 trial with extra tumor biopsies and frequent pharmacokinetic blood draws.

Started >3 years ago

The trial was activated more than 3 years ago. This is not always bad but can indicate that the trial has less current treatments, or has had trouble getting enough patients to enroll due to other issues.

HealthScout AI summary: Children and adults (1–50 years) with very high-risk solid tumors (e.g., refractory neuroblastoma, stage 4 rhabdomyosarcoma, metastatic Ewing sarcoma, osteosarcoma with unresectable/extrapulmonary mets, desmoplastic small round cell tumor) undergo reduced-intensity conditioning (fludarabine/melphalan/200 cGy TBI) followed by T cell–replete haploidentical bone marrow transplant. Post-transplant cyclophosphamide is used for GVHD prophylaxis with a shortened tacrolimus course (to day +90) plus mycophenolate, aiming to enhance graft-versus-tumor while monitoring GVHD and non-relapse mortality.

ClinicalTrials.gov ID: NCT01804634

A Phase I Trial of CRD3874-SI, a STING Agonist, in Patients With Advanced/Metastatic Malignant Solid Tumors

No known activity

No investigational drug(s) in this trial have shown anticancer activity yet in clinical trials

High burden on patient

The trial has a high burden on patients. An example would be a phase 1 trial with extra tumor biopsies and frequent pharmacokinetic blood draws.

HealthScout AI summary: Adults with advanced/metastatic solid tumors after ≥1 prior therapy, with dose expansion focused on anti–PD-1/PD-L1–refractory or –progressed sarcoma subtypes (UPS/MFS, angiosarcoma, bone sarcoma) and Merkel cell carcinoma; ECOG 0–1 required. Patients receive weekly intravenous CRD3874-SI, an investigational small‑molecule allosteric STING agonist designed to enhance cGAMP-mediated type I interferon signaling, with 3+3 dose escalation to RP2D followed by expansion.

ClinicalTrials.gov ID: NCT06021626

A Phase I/Ib Study of Losartan in Combination With Sunitinib in the Treatment of Pediatric and Adult Patients With Relapsed or Refractory Osteosarcoma

High burden on patient

The trial has a high burden on patients. An example would be a phase 1 trial with extra tumor biopsies and frequent pharmacokinetic blood draws.

Started >3 years ago

The trial was activated more than 3 years ago. This is not always bad but can indicate that the trial has less current treatments, or has had trouble getting enough patients to enroll due to other issues.

HealthScout AI summary: Pediatric and adult patients (≥10 years) with relapsed/refractory osteosarcoma after ≥1 prior systemic therapy, without curative options and with adequate organ function, receive oral losartan (AT1R blocker aimed at modulating tumor stroma/immune chemokines) plus sunitinib (multikinase VEGFR/PDGFR/c-KIT inhibitor) on a 4-weeks-on/2-weeks-off schedule. Key exclusions include surface/periosteal osteosarcoma, active CNS metastases, significant cardiac disease/QTc prolongation, uncontrolled hypertension, recent major surgery, and use of strong CYP3A4 modulators or QT‑prolonging drugs.

ClinicalTrials.gov ID: NCT03900793

Phase I Dose Escalation Study of FGFR4 Chimeric Antigen Receptor (CAR) T Cells in Children and Young Adults With Recurrent or Refractory Rhabdomyosarcoma

No known activity

No investigational drug(s) in this trial have shown anticancer activity yet in clinical trials

High burden on patient

The trial has a high burden on patients. An example would be a phase 1 trial with extra tumor biopsies and frequent pharmacokinetic blood draws.

HealthScout AI summary: Children and young adults (3–39 years) with relapsed/refractory rhabdomyosarcoma after ≥2 prior regimens receive fludarabine/cyclophosphamide lymphodepletion followed by a single infusion of autologous FGFR4-directed CAR T cells (3A11; second-generation CAR targeting the FGFR4 receptor tyrosine kinase, with truncated EGFR tag for tracking/depletion). Prior stable CNS disease allowed; trial assesses safety, feasibility, and preliminary activity across dose levels (1×10^5–1×10^7 CAR T cells/kg).

ClinicalTrials.gov ID: NCT06865664

Interleukin-15 Armored Glypican-3-specific Chimeric Antigen Receptor Expressing Autologous T Cells as Immunotherapy for Children With Solid Tumors

High burden on patient

The trial has a high burden on patients. An example would be a phase 1 trial with extra tumor biopsies and frequent pharmacokinetic blood draws.

Started >3 years ago

The trial was activated more than 3 years ago. This is not always bad but can indicate that the trial has less current treatments, or has had trouble getting enough patients to enroll due to other issues.

HealthScout AI summary: Pediatric and young adult patients (1–21 years) with relapsed/refractory GPC3-positive solid tumors, including eligible hepatocellular carcinoma, receive autologous CAR T cells engineered to target glypican-3 and constitutively express IL-15, with an inducible caspase-9 safety switch, after fludarabine/cyclophosphamide lymphodepletion. Retreatment at the same dose is permitted for responders without dose-limiting toxicity; key exclusions include uncontrolled infection, prior transplant, and high-dose steroids.

ClinicalTrials.gov ID: NCT04377932

The TNT Protocol: A Phase 2 Study Using Talimogene Laherparepvec,Nivolumab and Trabectedin as First, Second/Third Line Therapy for Advanced Sarcoma, Including Desmoid Tumor and Chordoma

High burden on patient

The trial has a high burden on patients. An example would be a phase 1 trial with extra tumor biopsies and frequent pharmacokinetic blood draws.

Started >3 years ago

The trial was activated more than 3 years ago. This is not always bad but can indicate that the trial has less current treatments, or has had trouble getting enough patients to enroll due to other issues.

HealthScout AI summary: Adults with unresectable or metastatic sarcoma (including desmoid tumor and chordoma) with measurable disease and at least one injectable lesion receive intratumoral talimogene laherparepvec (oncolytic HSV‑1 expressing GM‑CSF) plus nivolumab (PD‑1 inhibitor) and trabectedin (DNA minor-groove binder) as first–third line therapy. Open-label single-arm regimen treats up to 1 year; excludes active significant immunosuppression/autoimmunity, active CNS mets needing high-dose steroids, HSV-1 complications, and need for systemic antivirals.

ClinicalTrials.gov ID: NCT03886311