Clinical Trials for Sarcoma

A Phase 1/2 Clinical Trial of the Novel Topoisomerase I Inhibitor PEEL-224 as a Single Agent and in Combination With Vincristine and Temozolomide in Children With Refractory, Progressive or Relapsed 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: Children and adolescents with refractory, progressive, or relapsed non-CNS solid tumors (Phase 1; 1–18 years) receive PEEL-224, a PEGylated topoisomerase I inhibitor prodrug delivering SN22, either alone or with vincristine and temozolomide; Phase 2 enrolls neuroblastoma (1–30 years) and rhabdomyosarcoma (1–18 years) cohorts at the RP2D for the combination. CNS primaries/metastases are excluded; prior irinotecan/temozolomide permitted.

ClinicalTrials.gov ID: NCT06721689

Phase 1, Open-Label, Multicenter, First-In-Human Trial of Ds-2243a 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 HLA-A2–positive advanced solid tumors (synovial sarcoma, myxoid/round cell liposarcoma, NSCLC adenocarcinoma/squamous, or urothelial carcinoma) after appropriate prior therapy receive subcutaneous DS-2243a, a bispecific T‑cell engager targeting CD3 and the HLA‑A*02/NY‑ESO‑1 peptide complex, with step-up dosing. Includes dose escalation and tumor-specific expansion; excludes prior NY‑ESO‑1–targeted therapy and requires measurable disease and ECOG 0–1.

ClinicalTrials.gov ID: NCT06644755

A Phase 1, Open-label, Multicenter Study of LTZ-301 in Subjects With Relapsed/Refractory Non-Hodgkin Lymphoma

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 relapsed/refractory B‑cell non‑Hodgkin lymphoma after ≥2 prior regimens (ECOG 0–1) receive LTZ‑301 monotherapy, an investigational bispecific myeloid‑cell engager antibody targeting CD79b to recruit monocytes/macrophages and induce phagocytosis for B‑cell depletion. Excludes CLL/Richter, prior allo/solid organ transplant, recent ASCT/CAR‑T, active CNS disease, HIV, active autoimmune disease, and significant cardiovascular events.

ClinicalTrials.gov ID: NCT07121946

TCR-αβ+ and CD19+ Depleted KIR/KIR Ligand-mismatched Haploidentical Hematopoietic Stem Cell Transplant and Zoledronate for Pediatric Relapsed/Refractory Hematologic Malignancies and 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: Pediatric patients with relapsed/refractory hematologic malignancies (AML with <20% blasts, high-risk ALL, HL/NHL, MDS/MPN) or selected high-risk solid tumors (neuroblastoma, Ewing/PNET, rhabdomyosarcoma, osteosarcoma) lacking a timely matched donor undergo TCR-αβ+ and CD19+ depleted KIR/KIR-ligand–mismatched haploidentical PBSC transplant after risk-adapted conditioning. Post-transplant zoledronate (nitrogen-containing bisphosphonate; farnesyl pyrophosphate synthase inhibitor) is given to activate γδ T cells and potentially enhance antitumor immunity while monitoring GVHD, engraftment, and immune reconstitution.

ClinicalTrials.gov ID: NCT02508038

Phase I Study of Aerosol Gemcitabine in Patients With Solid Tumors and Pulmonary Metastases

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: Adolescents and adults (12–50) with histologically confirmed solid tumors metastatic to the lungs, ECOG ≤2 and adequate organ/pulmonary function, receive on‑site aerosolized gemcitabine via nebulizer twice weekly in 28‑day cycles, including those previously treated with systemic gemcitabine. Gemcitabine is a nucleoside analog prodrug that inhibits ribonucleotide reductase and DNA synthesis; trial excludes active asthma or significant pulmonary symptoms and seeks to define pulmonary MTD and preliminary activity.

ClinicalTrials.gov ID: NCT03093909

A Multi-cohort Study of Retifanlimab With or Without Gemcitabine and Docetaxel in Patients With Advanced Sarcoma

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 advanced, unresectable or metastatic high‑grade soft tissue sarcoma (ECOG 0–1, no prior systemic therapy for advanced disease) receive gemcitabine/docetaxel with the anti–PD‑1 antibody retifanlimab, with histology-specific cohorts; a separate biomarker-selected cohort tests retifanlimab monotherapy in previously treated patients with an immune-enriched tumor microenvironment. Retifanlimab blocks PD‑1 to restore antitumor T‑cell activity.

ClinicalTrials.gov ID: NCT04577014

SAINT: A Phase 1/2 Study of Safe Amounts of IPLIMUMAB, NIVOLUMAB and TRABECTEDIN for Previously Treated Advanced Soft Tissue Sarcoma (STS)

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 previously treated, unresectable or metastatic soft tissue sarcoma (ECOG 0–1) receive trabectedin (24‑hr IV q3wk at MTD) combined with fixed-dose ipilimumab (CTLA‑4 inhibitor) and nivolumab (PD‑1 inhibitor) to assess safety and preliminary efficacy. Excludes active autoimmune disease, untreated CNS metastases, significant immunosuppression, and active hepatitis B/C or HIV.

ClinicalTrials.gov ID: NCT03138161

Evolutionary Inspired Therapy for Newly Diagnosed, Metastatic, Fusion Positive Rhabdomyosarcoma

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: Newly diagnosed metastatic PAX/FOXO1 fusion–positive rhabdomyosarcoma (children to adults) treated with standard cytotoxic regimens using alternative scheduling strategies: VAC-based “first strike” (vinorelbine/actinomycin D/cyclophosphamide), “second strike” with prolonged vinorelbine + oral cyclophosphamide maintenance after VAC response, adaptive therapy with response-guided intermittent VAC, or conventional 42-week VAC±irinotecan/doxorubicin/ifosfamide/etoposide. No novel agents; compares front-loaded, maintenance, and adaptive timing of standard chemotherapy to optimize EFS/OS with acceptable toxicity.

ClinicalTrials.gov ID: NCT04388839

Phase 1/2 Trial to Evaluate the Safety and Efficacy of PEEL-224 in Combination With Vincristine and Temozolomide in Adolescents and Young Adults With Relapsed or Refractory Sarcomas

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: Adolescents and young adults (12–49 years, ≥40 kg) with relapsed/refractory sarcomas—Phase 2 cohorts for Ewing sarcoma (FET-ETS fusion), DSRCT (EWSR1::WT1), and an “other sarcoma” cohort (including RMS, osteosarcoma, and other translocation-associated round cell sarcomas)—receive PEEL-224 plus vincristine and temozolomide on 21-day cycles with growth factor support. PEEL-224 is an investigational PEGylated topoisomerase I inhibitor prodrug (PEG-[SN22]4) designed to release SN22 and overcome common camptothecin resistance mechanisms; CNS metastases are excluded for EWS/DSRCT cohorts.

ClinicalTrials.gov ID: NCT06709495

A Phase I Study of Pomalidomide and Nivolumab in Patients With Virus-Associated Malignancies With or Without HIV

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 relapsed/refractory or non-curable virus-associated malignancies (including EBV-, HPV-, HBV/HCV-, MCPyV-, and KSHV-associated cancers; HIV-positive allowed on suppressive ART) receive nivolumab (PD-1 inhibitor) plus oral pomalidomide (IMiD enhancing T/NK/dendritic-cell function) in 28-day cycles. Designed to establish safety/MTD and explore activity, with expansion cohorts enriched for Kaposi sarcoma and EBV/KSHV-associated lymphomas.

ClinicalTrials.gov ID: NCT04902443