Clinical Trials for Sarcoma

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

A Pilot Trial of Autologous Tumor Infiltrating Lymphocytes (LN-144 or LN-145) for Patients With Advanced Uveal Melanoma, Undifferentiated Pleomorphic Sarcoma, or Dedifferentiated Liposarcoma

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 uveal melanoma (any prior therapy) or unresectable/metastatic UPS or DDLPS refractory to ≥1 systemic regimen receive autologous tumor-infiltrating lymphocyte therapy lifileucel (LN-144/LN-145) after nonmyeloablative lymphodepleting chemotherapy and followed by IL-2. Lifileucel consists of ex vivo–expanded, tumor-specific T cells (adoptive cell therapy) and is being studied here for safety/feasibility in these populations.

ClinicalTrials.gov ID: NCT05607095

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 Phase 1 Study of the Safety and Efficacy of CRX100 as Monotherapy and in Combination With Pembrolizumab in Advanced Solid Malignancies

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.

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: This trial targets adult patients with advanced solid malignancies and evaluates the safety and efficacy of CRX100, an investigational immunotherapy combining autologous T cells and oncolytic vaccinia virus, as monotherapy and in combination with Pembrolizumab.

ClinicalTrials.gov ID: NCT04282044

A Phase 1/2, Open-label, Dose-escalation, and Expansion Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Therapeutic Activity of GI-101 As a Single Agent and in Combination with Pembrolizumab, Lenvatinib or Local Radiotherapy in Patients with Advanced or Metastatic Solid Tumors (Keynote B59)

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.

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: This trial involves adult patients with advanced or metastatic solid tumors and evaluates the safety and therapeutic activity of GI-101, a bispecific fusion protein targeting CTLA-4 and the IL-2 pathway, as a single agent and in combination with pembrolizumab, lenvatinib, or radiotherapy.

ClinicalTrials.gov ID: NCT04977453

A Phase 1a/1b, Open-Label, Multi-Center, Dose Escalation and Expansion Study of HFB200301 (TNFR2 Agonist Antibody) as a Single Agent and in Combination With Tislelizumab (Anti-PD-1 Antibody) in Adult Patients With Advanced 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.

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: This trial enrolls adults with advanced or metastatic solid tumors (including gastric, renal, melanoma, sarcoma, testicular germ cell, cervical, mesothelioma, NSCLC, or head and neck cancers) who have progressed after standard therapies, and investigates the investigational TNFR2 agonist antibody HFB200301 (stimulating anti-tumor immunity) as monotherapy or in combination with the anti-PD-1 antibody tislelizumab. Eligible patients must have measurable disease, ECOG 0-1, and no prior TNFR2-targeted therapy or active autoimmune disease.

ClinicalTrials.gov ID: NCT05238883