Trial in Patients With Metastatic or Locally Advanced Leiomyosarcoma

NCT ID: NCT04383119

Last Updated: 2024-12-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

100 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-10-29

Study Completion Date

2025-12-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

Study is aimed at evaluating the activity of Trabectedin (arm A) in advanced leiomyosarcomas, having Gemcitabine (arm B) as the comparator.

In addition to the randomized cohort, the study has also an observational prospective cohort which include patients who will refuse the randomization or for whom the investigator will not judge the randomization as an appropriate option.

In order to allow the participation of sites only to the prospective-observational (non randomized) cohort, it was introduced the possibility to participate to the study and receive the ethical approval only to the Observational Prospective Cohort In parallel an optional translational study will be performed, in both cohorts, to identify factors predictive of the activity of Trabectedin or Gemcitabine in this specific histotype.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

The management of patients with leiomyosarcomas determines many difficulties. Despite patients with metastatic disease at diagnosis or who recur after initial treatment have a dismal prognosis and, except for a subset of selected patients with completely resectable disease, the median survival is less than two years.

At the advanced-disease stage, the main aim of treatment is to improve patient's quality of life, possibly survival, with the best compromise between toxicity and symptoms. Trabectedin (T) is a marine-derived cytotoxic approved by European MEdicine Agency (EMEA) and FDA.

It is indicated for the treatment of patients with advanced soft tissue sarcoma, after failure of anthracyclines-based chemotherapy or who are unsuitable to receive these agents.

Among Soft Tissue Sarcoma (STS), activity has been mainly detected in synovial sarcoma, liposarcoma and leiomyosarcoma. Although the response rate did not exceed 10%, T was demonstrated to provide disease control, with progression arrest rates exceeding 50% and progression-free survival rates exceeding 20% at 6 months. So far no phase II or III studies have been addressed to test the activity of T in leiomyosarcoma specifically (without differentiation between site of primary localization) in comparison with Gemcitabine.

This study is aimed at evaluating the activity of Trabectedin (arm A) in advanced leiomyosarcomas, having Gemcitabine (arm B) as the comparator. In parallel an optional translational study will be performed to identify factors predictive of the activity of Trabectedin or Gemcitabine in this specific histotype.

In addition to the randomized cohort, the study has also an observational prospective cohort which include patients who will refuse the randomization or for whom the investigator will not judge the randomization as an appropriate option.

In order to allow the participation of sites only to the prospective-observational (non randomized) cohort, it was introduced the possibility to participate to the study and receive the ethical approval only to the Observational Prospective Cohort In parallel an optional translational study will be performed, in both cohorts, to identify factors predictive of the activity of Trabectedin or Gemcitabine in this specific histotype.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Leiomyosarcoma of Ovary Soft Tissue Sarcoma

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

advanced leiomyosarcoma

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

multicenter, randomized, open-label, two arms with crossover. Patients will be randomized to receive Trabectedin (arm A) or Gemcitabine (arm B).

In case of progressive disease (PD) or unacceptable toxicity during the assigned treatment, the patient will switched to the other arm
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Not applicable (open label study)

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Arm A

Trabectedin at the dose of 1.5 mg/m2-1.3 mg/m2 with a top-dose of 2.6 total mg per cycle (according the clinical practice in pretreated patients and in all our ISG studies) will be administered via a central venous catheter as a 24-hour infusion on day 1 of 21-days treatment cycles

Group Type EXPERIMENTAL

Trabectedin

Intervention Type DRUG

Trabectedin in monotherapy

Arm B

Gemcitabine 800-1000 mg/m2 will be administered via a central venous catheter on days 1,8 every 21 days

Group Type ACTIVE_COMPARATOR

Gemcitabine

Intervention Type DRUG

Gemcitabine, control arm

Observational Cohort

Treatmen according clinical practice (not defined in advance). The patient who will refuse randomization between Arm A and B can choose to participate to the observational cohort to the study, where they will be treated according clinical practice

Group Type ACTIVE_COMPARATOR

No Intervention: Observational Cohort

Intervention Type DRUG

Treatment according clinical practice

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Trabectedin

Trabectedin in monotherapy

Intervention Type DRUG

Gemcitabine

Gemcitabine, control arm

Intervention Type DRUG

No Intervention: Observational Cohort

Treatment according clinical practice

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Trabectedin arm Gemcitabine arm

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

1. Patients with histologically documented diagnosis of leiomyosarcoma
2. Patients with diagnosis of unresectable or metastatic leiomyosarcoma
3. Patients who received at least on previous systemic treatment with anthracycline-based chemotherapy.
4. Patients suitable to receive gemcitabine or trabectedin therapy.
5. Measurable or evaluable disease with RECIST 1.1 criteria.
6. Evidence of progression according RECIST 1.1 during the 6 months before study entry.
7. Age ≥18 years
8. Eastern Cooperative Oncology Group (ECOG) Performance Status ≤ 2
9. All previous anticancer treatments must have completed ≥ 3 weeks prior to first dose of study drug.
10. The patient has resolution of adverse events, with the exception of alopecia, and of all clinically significant toxic effects of prior loco-regional therapy, surgery, radiotherapy or systemic anticancer therapy to ≤ Grade 1, by National Cancer Institute - Common Terminology Criteria for Adverse Events (NCI-CTCAE) Version 5.0
11. Adequate bone marrow, liver and renal function
12. Left Ventricular Ejection Fraction ≥ 50% and/or above lower institutional limit of normality.
13. Female patients of child-bearing potential must have negative pregnancy test within 7 days before initiation each cycle of chemotherapy.
14. No history of arterial and/or venous thromboembolic event within the previous 12 months.
15. The patient or legal representative must be able to read and understand the informed consent form and must have been willing to give written informed consent prior to any study specific procedure. The subject may also provide an optional consent for the biological/translational sub-study associated.

Exclusion Criteria

1. Prior treatment with Trabectedin and/or Gemcitabine
2. Known hypersensitivity to any of the study drugs, study drug classes, or excipients in the formulation of the study drugs
3. History of other malignancies (except basal cell carcinoma or cervical carcinoma in situ, adequately treated), unless in remission from 5 years or more and judged of negligible potential of relapse.
4. Persistent toxicities with the exception of alopecia, caused by previous anticancer therapies
5. Metastatic brain or meningeal tumors
6. Active viral hepatitis
7. Immunocompromised patients, e.g., patients who are known to be serologically positive for human immunodeficiency virus
8. Patients with any severe and/or uncontrolled medical conditions
9. Medical history of hemorrhage or a bleeding event ≥ Grade 3 (NCI-CTCAE v 5.0) within 4 weeks prior to the initiation of study treatment
10. Active clinically serious infections
11. Immunocompromised patients, e.g., patients who are known to be serologically positive for human immunodeficiency virus
12. Previous treatment with radiation therapy within 14 days of first day of study drug dosing,
13. Major surgery within 4 weeks prior to study entry
14. Concomitant use of known strong CYP3A inhibitors or moderate CYP3A inhibitors
15. Concomitant use of known strong or moderate CYP3A inducers
16. Patients undergoing renal dialysis or with Creatinin Clearance \<30 ml/min or Creatinine \>1,5 mg/dL
17. Pregnant or breast feeding patients
18. Patients with severe and/or uncontrolled concurrent medical disease that in the opinion of the investigator could cause unacceptable safety risks or compromise compliance with the protocol
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

PharmaMar

INDUSTRY

Sponsor Role collaborator

Italian Sarcoma Group

NETWORK

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Bruno Vincenzi, Prof/MD

Role: PRINCIPAL_INVESTIGATOR

Campus Biomedico of Rome

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Azienda Ospedaliera S. Orsola-Malpighi

Bologna, BO, Italy

Site Status RECRUITING

Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori - IRST

Meldola, FC, Italy

Site Status RECRUITING

Nuovo Ospedale di Prato

Prato, Firenze, Italy

Site Status RECRUITING

Istituto Clinico Humanitas

Rozzano, MI, Italy

Site Status RECRUITING

Centro di Riferimento Oncologico di Aviano

Aviano, PD, Italy

Site Status NOT_YET_RECRUITING

Policlinico Universitario Campus Biomedico

Roma, RM, Italy

Site Status RECRUITING

IRCCS Fondazione Piemonte per l'Oncologia

Candiolo, Torino, Italy

Site Status RECRUITING

A.O.U.San Luigi Gonzaga

Orbassano, Torino, Italy

Site Status RECRUITING

Istituto Ortopedico Rizzoli - Unit of Chemotherapy of Muscoloskeletal Tumors

Bologna, , Italy

Site Status RECRUITING

H.San Martino di Genova

Genova, , Italy

Site Status RECRUITING

Fondazione IRCCS INT Milano

Milan, , Italy

Site Status NOT_YET_RECRUITING

Istituto Europeo di Oncologia

Milan, , Italy

Site Status RECRUITING

IRCCS Istituto nazionale Tumori "Fondazione G.Pascale"

Napoli, , Italy

Site Status RECRUITING

Irccs Istituto Oncologico Veneto (Iov)

Padua, , Italy

Site Status RECRUITING

Ospedale Giaccone

Palermo, , Italy

Site Status RECRUITING

Istituto Regina Elena - IFO

Rome, , Italy

Site Status RECRUITING

ASL Città di Torino (Dipartimento di Oncologia)

Torino, , Italy

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

Italy

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Bruno Vincenzi, Prof/MD

Role: CONTACT

Phone: 003906-22541

Email: [email protected]

Emanuela Marchesi, PhD

Role: CONTACT

Phone: 003905101459

Email: [email protected]

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

Margherita Nannini, MD

Role: primary

Valentina Fausti, MD

Role: primary

Giacomo G. Baldi, MD

Role: primary

Alexia Bertuzzi, MD

Role: primary

Angela Buonadonna, MD

Role: primary

Bruno Vincenzi, MD

Role: primary

Sandra Aliberti, MD

Role: primary

Lorenzo D'Ambrosio, MD

Role: primary

Toni Ibrahim, MD

Role: primary

Comandini Danila, MD

Role: primary

Roberta Sanfilippo, MD

Role: primary

Elisabetta Setola, MD

Role: primary

Salvatore Tafuto, Prof

Role: primary

Antonella Brunello, MD

Role: primary

Giuseppe Giuseppe, MD

Role: primary

Virginia Ferraresi, MD

Role: primary

Antonella Boglione, MD

Role: primary

Boglione, MD

Role: backup

References

Explore related publications, articles, or registry entries linked to this study.

Hensley ML, Miller A, O'Malley DM, Mannel RS, Behbakht K, Bakkum-Gamez JN, Michael H. Randomized phase III trial of gemcitabine plus docetaxel plus bevacizumab or placebo as first-line treatment for metastatic uterine leiomyosarcoma: an NRG Oncology/Gynecologic Oncology Group study. J Clin Oncol. 2015 Apr 1;33(10):1180-5. doi: 10.1200/JCO.2014.58.3781. Epub 2015 Feb 23.

Reference Type BACKGROUND
PMID: 25713428 (View on PubMed)

Pautier P, Floquet A, Chevreau C, Penel N, Guillemet C, Delcambre C, Cupissol D, Selle F, Isambert N, Piperno-Neumann S, Thyss A, Bertucci F, Bompas E, Alexandre J, Collard O, Lavau-Denes S, Soulie P, Toulmonde M, Le Cesne A, Lacas B, Duffaud F; French Sarcoma Group. Trabectedin in combination with doxorubicin for first-line treatment of advanced uterine or soft-tissue leiomyosarcoma (LMS-02): a non-randomised, multicentre, phase 2 trial. Lancet Oncol. 2015 Apr;16(4):457-64. doi: 10.1016/S1470-2045(15)70070-7. Epub 2015 Mar 18.

Reference Type BACKGROUND
PMID: 25795402 (View on PubMed)

Demetri GD, Chawla SP, von Mehren M, Ritch P, Baker LH, Blay JY, Hande KR, Keohan ML, Samuels BL, Schuetze S, Lebedinsky C, Elsayed YA, Izquierdo MA, Gomez J, Park YC, Le Cesne A. Efficacy and safety of trabectedin in patients with advanced or metastatic liposarcoma or leiomyosarcoma after failure of prior anthracyclines and ifosfamide: results of a randomized phase II study of two different schedules. J Clin Oncol. 2009 Sep 1;27(25):4188-96. doi: 10.1200/JCO.2008.21.0088. Epub 2009 Aug 3.

Reference Type BACKGROUND
PMID: 19652065 (View on PubMed)

Patel SR, Gandhi V, Jenkins J, Papadopolous N, Burgess MA, Plager C, Plunkett W, Benjamin RS. Phase II clinical investigation of gemcitabine in advanced soft tissue sarcomas and window evaluation of dose rate on gemcitabine triphosphate accumulation. J Clin Oncol. 2001 Aug 1;19(15):3483-9. doi: 10.1200/JCO.2001.19.15.3483.

Reference Type BACKGROUND
PMID: 11481354 (View on PubMed)

Pautier P, Floquet A, Penel N, Piperno-Neumann S, Isambert N, Rey A, Bompas E, Cioffi A, Delcambre C, Cupissol D, Collin F, Blay JY, Jimenez M, Duffaud F. Randomized multicenter and stratified phase II study of gemcitabine alone versus gemcitabine and docetaxel in patients with metastatic or relapsed leiomyosarcomas: a Federation Nationale des Centres de Lutte Contre le Cancer (FNCLCC) French Sarcoma Group Study (TAXOGEM study). Oncologist. 2012;17(9):1213-20. doi: 10.1634/theoncologist.2011-0467. Epub 2012 Aug 20.

Reference Type BACKGROUND
PMID: 22907974 (View on PubMed)

Seddon B, Strauss SJ, Whelan J, Leahy M, Woll PJ, Cowie F, Rothermundt C, Wood Z, Benson C, Ali N, Marples M, Veal GJ, Jamieson D, Kuver K, Tirabosco R, Forsyth S, Nash S, Dehbi HM, Beare S. Gemcitabine and docetaxel versus doxorubicin as first-line treatment in previously untreated advanced unresectable or metastatic soft-tissue sarcomas (GeDDiS): a randomised controlled phase 3 trial. Lancet Oncol. 2017 Oct;18(10):1397-1410. doi: 10.1016/S1470-2045(17)30622-8. Epub 2017 Sep 4.

Reference Type BACKGROUND
PMID: 28882536 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

ISG-ARTICLE

Identifier Type: -

Identifier Source: org_study_id