Clinical Study in Patients With DSRCT

NCT07328425 | Recruiting Phase 2

• 2 other identifiers: ISG-TULIPS2024-519261-21-00
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

Patients participating in this study have DSRCT that has spread locally or to other parts of the body and can no longer be surgically removed without causing significant harm. Treatment will continue until the tumor progresses further, severe side effects occur, or either patient or investigator decision. In addition, patients may participate in an optional biological study. The study will analyze the tumor's genes and the molecules related to them. By studying genes and their products, the investigators can better understand the behavior of the tumor and how the body responds to therapies.

Conditions

Desmoplastic Small Round Cell Tumor

Outcome Measures

Primary Outcomes (1)

• Overall tumour Response Rate (ORR), according to RECIST v 1.1

  Overall tumour Response Rate (ORR), according to RECIST v 1.1

  Week 6, week 12, week 18, week 30, week 42, week 54, week 66, week 78, week 90, week 102 and until progression

Secondary Outcomes (6)

• Overall Survival (OS)

  From date of study therapy start until the date of death from any cause, assessed up to 60 months

• Progression Free Survival (PFS)

  From date of study therapy start until first documented progression or date of death from any cause, whichever came first, assessed up to 60 months

• Duration of Response (DoR)

  From date of study therapy start until first documented assessment without progression or date of death from any cause, whichever came first, assessed up to 60 months

• Adverse events and Serious adverse event (according to CTC-AE v.5)

  After initiation of study treatments(s), all adverse events will be reported until 30 days after the final dose of study treatment or until initiation of new systemic anti-cancer therapy, whichever occurs first.

• European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30)

  Screening, cycle1 day1 (each cycle is 21 days), cycle2 day1, End Of Treatment visit (performed 30 days after the last study drug administration), at follow-up visits performed at months 6, 12, 18 and 24 after the last study drug administration

Study Arms (1)

Lurbinectedine and Irinotecan

EXPERIMENTAL

lurbinectedin in combination with intravenous irinotecan administered until progression or unacceptable toxicity, in a population of =/\>15 years old patients with histologically and molecularly confirmed (EWSR1-WT1 translocation positive), advanced (locally advanced or metastatic) DSRCT, from 2nd to 4th line, following progression to anthracycline-based chemotherapy.

Drug: Lurbinectedin; Drug: Irinotecan

Interventions

Lurbinectedin DRUG

Lurbinectedin and Irinotecan

Lurbinectedine and Irinotecan

Irinotecan DRUG

Lurbinectedin and Irinotecan

Lurbinectedine and Irinotecan

Eligibility Criteria

• Age: 15 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Histological centrally confirmed diagnosis of DSRCT with the documented presence of EWSR1-WT1 translocation.
• Age ≥ 15 years.
• Locally advanced (i.e. radical surgical resection of local disease unfeasible or surgery declined by the patient or surgery deemed to become less demolitive and / or easier after cytoreduction) and/or metastatic disease.
• Measurable disease by RECIST v1.1.
• Clinical or objective disease progression after the last administration of the last standard therapy, or have stopped standard therapy due to intolerability within 6 months from enrollment.
• At least one prior chemotherapy based on anthracycline (considering chemotherapy administered for primary tumour) and no more than 3 prior chemotherapy lines.
• Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) ≤ 2.
• platelet count ≥ 100 × 109/L, hemoglobin ≥ 9.0 g/dL, white blood cells ≥ 3.0 × 109/L and absolute neutrophil count (ANC) ≥ 2.0 × 109/L,
• aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3.0 × the upper limit of normal (ULN), even in the presence of liver metastases,
• total bilirubin ≤ 1.5 × ULN or direct bilirubin ≤ ULN,
• International Normalized Ratio (INR) \< 1.5 (except if patient is on oral anticoagulation therapy),
• calculated creatinine clearance (CrCL) ≥ 30 mL/minute (using Cockcroft-Gault formula),
• creatine phosphokinase (CPK) ≤ 2.5 × ULN,
• albumin ≥ 3.0 g/dL.
• Cardiac ejection fraction ≥50% as measured by echocardiogram.

You may not qualify if:

• Prior treatment with lurbinectedin or trabectedin, Ecubectedin (PM 14) or PM54.
• Known hypersensitivity to irinotecan or lurbinectedin or any of their components of the drugs products (excipients).
• Other primary malignancy with \<5 years clinically assessed disease free interval, except basal cell skin cancer, cervical carcinoma in situ or other neoplasm judged to entail a low risk of relapse.
• History or presence of unstable angina, myocardial infarction, or clinically significant valvular heart disease within 12 months of the study.
• Grade III/IV cardiac problems as defined by the New York Heart Association Criteria (i.e. congestive heart failure, myocardial infarction within 12 months of study).
• Symptomatic arrhythmia or any uncontrolled arrhythmia requiring ongoing treatment within 12 months of study.
• Myopathy or any clinical situation that causes significant and persistent elevation of CPK (\> 2.5 × ULN in two different determinations performed one week apart).
• Severe and/or uncontrolled medical disease (i.e. uncontrolled diabetes, chronic renal disease, or active uncontrolled infection).
• Known active brain metastasis.
• Known chronic liver disease (i.e. chronic active hepatitis and cirrhosis).
• Diagnosis of human deficiency virus (HIV), hepatitis C virus (HCV) infection or active hepatitis B (to be excluded during the screening period).
• Any past or present chronic inflammatory colon and/or liver disease, past intestinal obstruction, pseudo or sub-occlusion or paralysis.
• Evident symptomatic pulmonary fibrosis or interstitial pneumonitis, pleural or cardiac effusion rapidly increasing and/or necessitating prompt local treatment within seven days.
• Any other major illness that, in the Investigator's judgment, will substantially increase the risk associated with the patient's participation in this study.
• Known active COVID-19 disease (this includes positive test for SARS-CoV-2 in nasopharyngeal/oropharyngeal swabs or nasal swabs by PCR).

Sponsors & Collaborators

• Italian Sarcoma Group: lead

Study Sites (1)

Fondazione IRCCS Istituto Nazionale dei Tumori

Milan, MI, 20133, Italy

RECRUITING

Related Publications (41)

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MeSH Terms

Conditions

Desmoplastic Small Round Cell Tumor

Interventions

PM 01183; Irinotecan

Condition Hierarchy (Ancestors)

Sarcoma; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms

Intervention Hierarchy (Ancestors)

Camptothecin; Alkaloids; Heterocyclic Compounds

Study Officials

• Silvia Syecchiotti, MD

  Findazione IRCCS Istituto Nazionale Tumori Milano

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Gianluca Ignazzi

CONTACT

+393335359192; gianluca.ignazzi@italiansarcomagroup.org

Viviana Appolloni

CONTACT

+393756073789; viviana.appolloni@italiansarcomagroup.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 17, 2025
• First Posted: January 9, 2026
• Study Start: November 21, 2025
• Primary Completion (Estimated): October 1, 2030
• Study Completion (Estimated): October 1, 2030
• Last Updated: January 12, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

IT (1)