Clinical Trials for Sarcoma

A Pilot Study of BTX-A51 in Patients With Metastatic and/or Recurrent Liposarcomas Characterized by MDM2 Amplifications, Myxoid Liposarcoma, and CIC-Rearranged Sarcoma

Active drug

Investigational drug(s) in this trial have shown anticancer activity 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 metastatic/recurrent liposarcoma (WD/DD with MDM2 amplification or myxoid subtype) or CIC-rearranged sarcoma receive oral BTX-A51 three times weekly; BTX-A51 is an investigational multikinase inhibitor (targets CK1α and CDK7/CDK9) intended to stabilize p53 by suppressing MDM2 and downregulate oncogenic transcription (e.g., MYC, MCL1). Two fixed dose levels (21 mg or 30 mg) are explored with treatment until progression or unacceptable toxicity.

ClinicalTrials.gov ID: NCT06414434

A Phase 1, Multicenter, Open-label, 2-stage, Single-arm Study to Evaluate the Safety and Tolerability of an Autologous Tumor-infiltrating Lymphocytes (TIL) Regimen and Preliminary Antitumor Activity of TIL in Pediatric, Adolescent, and Young Adult Participants With Relapsed or Refractory Solid Tumors

Active drug

Investigational drug(s) in this trial have shown anticancer activity 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: Single-arm TIL therapy (lifileucel-based autologous tumor-infiltrating lymphocytes expanded ex vivo with IL-2) for children, adolescents, and young adults (≤21 years) with relapsed/refractory solid tumors lacking effective options, including sarcomas, primary CNS tumors, and melanoma. Patients undergo tumor resection for TIL manufacture, nonmyeloablative lymphodepletion, then a single TIL infusion with supportive care (typically IL-2); outcomes include safety and preliminary antitumor activity.

ClinicalTrials.gov ID: NCT06566092

A Phase 1 Study of Peposertib (M3814) and Low-Dose Liposomal Doxorubicin (Doxil®) in Patients With Metastatic Leiomyosarcoma and Other Soft Tissue Sarcomas

Active drug

Investigational drug(s) in this trial have shown anticancer activity 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 metastatic or unresectable soft tissue sarcoma—emphasis on leiomyosarcoma—after prior therapy (including limited prior anthracycline) receive oral peposertib (DNA-PK inhibitor targeting NHEJ repair) continuously with low-dose pegylated liposomal doxorubicin every 28 days. Includes dose escalation across selected STS subtypes and a leiomyosarcoma expansion; requires biopsy-amenable disease, allows treated/stable CNS metastases, and excludes significant cardiac dysfunction and prior DNA-PK inhibitor exposure.

ClinicalTrials.gov ID: NCT05711615

Phase 1/2 Study of Vincristine and Temozolomide in Combination With PEN-866 for Adolescents and Young Adults With Relapsed or Refractory Solid Tumors

Active drug

Investigational drug(s) in this trial have shown anticancer activity 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: Adolescents and young adults (12–39) with relapsed/refractory solid tumors (phase 1) and specifically Ewing sarcoma with EWS translocation or embryonal/alveolar rhabdomyosarcoma with measurable disease (phase 2) receive PEN-866 (locnartecan), an HSP90-targeted miniature drug conjugate delivering SN-38, combined with fixed-dose vincristine and temozolomide in 21-day cycles. Excludes prior irinotecan/temozolomide in phase 2; aims to define MTD/RP2D and assess response.

ClinicalTrials.gov ID: NCT04890093

Feasibility of CSF (Cerebrospinal Fluid) and Plasma ctDNA (Circulating Tumor Deoxyribonucleic) in BRAF (V-raf Murine Sarcoma Viral Oncogene Homolog B1)-Altered Glioma During Treatment With Plixorafenib

Active drug

Investigational drug(s) in this trial have shown anticancer activity 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 recurrent, measurable primary CNS gliomas harboring BRAF V600E mutations after prior BRAF/MEK inhibitor therapy, eligible for resection/biopsy with ventricular reservoir, receive plixorafenib (paradox-breaking BRAF inhibitor that disrupts RAF dimer signaling) plus cobicistat. The study includes serial CSF and plasma sampling to assess ctDNA dynamics alongside MRI response while patients continue oral therapy until progression.

ClinicalTrials.gov ID: NCT06610682

Phase I/II Study of Silmitasertib (CX-4945) in Combination With Chemotherapy in Children and Young Adults With Relapsed Refractory Solid Tumors

Active drug

Investigational drug(s) in this trial have shown anticancer activity 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 with relapsed/refractory solid tumors—phase II focused on neuroblastoma and Ewing sarcoma—receive silmitasertib, an oral CK2 inhibitor (modulates PI3K/AKT and DNA damage response), combined with standard salvage chemotherapy backbones. Regimens include silmitasertib + irinotecan/temozolomide for neuroblastoma and silmitasertib + vincristine/irinotecan/temozolomide for Ewing sarcoma.

ClinicalTrials.gov ID: NCT06541262

A Phase I/II, Open-label, Multicenter Study to Assess the Safety, Tolerability, Pharmacokinetics, and Antitumor Activity of Vactosertib as a Single Agent in Adolescents and Adults With Recurrent, Refractory or Progressive Osteosarcoma

Active drug

Investigational drug(s) in this trial have shown anticancer activity 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: Enrolling adolescents and adults (≥12) with measurable, recurrent/refractory or progressive osteosarcoma after standard therapy and ECOG 0–2 for oral vactosertib monotherapy. Vactosertib is a selective TGF-β type I receptor (ALK5) kinase inhibitor aimed at blocking TGF-β signaling to reduce tumor progression and immune evasion; excludes prior TGF-β inhibitors and significant cardiac/GI risks or strong CYP3A4 interactions.

ClinicalTrials.gov ID: NCT05588648

Phase I Clinical Trial of Autologous B7-H3 Chimeric Antigen Receptor T Cells (B7-H3CART) in Children and Young Adults With Relapsed or Refractory Solid Tumor Expressing B7-H3 Target

Active drug

Investigational drug(s) in this trial have shown anticancer activity 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 (2–30 years) with relapsed/refractory solid tumors (e.g., neuroblastoma, sarcomas, Wilms tumor) after standard therapy receive lymphodepleting fludarabine/cyclophosphamide followed by a single IV infusion of autologous B7-H3–directed CAR T cells (targets CD276). B7-H3 expression testing is required on tissue (positivity not mandatory); key exclusions include uncontrolled infection, active viral hepatitis/HIV, recent significant cardiac disease, untreated brain metastases, and need for systemic immunosuppression.

ClinicalTrials.gov ID: NCT06500819

LIGHTBEAM-U01 Substudy 01A: A Phase 1/2 Substudy to Evaluate the Safety and Efficacy of Zilovertamab Vedotin in Pediatric and Young Adult Participants With Hematologic Malignancies or Solid Tumors

Active drug

Investigational drug(s) in this trial have shown anticancer activity 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: Enrolling pediatric (1 to <18 years) and young adult patients with relapsed/refractory B-ALL, DLBCL/Burkitt lymphoma, neuroblastoma, or Ewing sarcoma to receive single‑agent zilovertamab vedotin IV every 21 days in dose-escalation/expansion cohorts. Zilovertamab vedotin is a ROR1‑targeted antibody–drug conjugate delivering MMAE to tumor cells; primary aims are safety/PK and preliminary efficacy with disease‑specific response criteria.

ClinicalTrials.gov ID: NCT06395103

A First-in-human, Open-label Trial to Evaluate the Combination of ACTengine® IMA203 With mRNA-4203 in Previously Treated, Unresectable or Metastatic Cutaneous Melanoma or Synovial Sarcoma Patients (ACTengine® IMA203-102)

Active drug

Investigational drug(s) in this trial have shown anticancer activity 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 HLA‑A*02:01–positive unresectable or metastatic cutaneous melanoma (post–PD‑1) or synovial sarcoma needing further therapy receive autologous PRAME‑targeted TCR‑T cells (ACTengine IMA203) after lymphodepletion plus low‑dose IL‑2, combined with a PRAME mRNA vaccine (mRNA‑4203) to boost PRAME‑specific T‑cell responses. Key exclusions include active brain metastases, significant autoimmune/cardiac disease, prior allogeneic transplant, active viral infections, and hypersensitivity to study agents.

ClinicalTrials.gov ID: NCT06946225