Study of Emactuzumab for Tenosynovial Giant Cell Tumor (TGCT)

NCT ID: NCT05417789

Last Updated: 2025-12-23

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

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

128 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-10-09

Study Completion Date

2027-09-30

Brief Summary

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This is a multicenter, Phase 3, randomised, double-blind, placebo-controlled study, which aims to evaluate the efficacy and safety of the investigational drug emactuzumab for the treatment of patients with localized or diffuse TGCT where surgical removal of the tumor is not viewed as an option.

The study consists of two parts. In Part 1, eligible subjects will be assigned in a 2:1 ratio to receive either emactuzumab or matching placebo in a double-blind fashion, that will be administered in total 5 times as an intravenous (i.v.) infusion once every 2 weeks. This will be followed by an observation period of 3 months leading to a total duration of 24 weeks in Part 1. A number of assessments will be carried out during the course of the study, including physical examinations, blood tests, imaging studies, electrocardiograms, and questionnaires. Part 2 is a long-term double-blind follow-up phase of the subjects on emactuzumab or placebo. Subjects assigned to placebo in Part 1 have the option, subject to eligibility, to crossover to receive open-label emactuzumab in Part 2. Subjects assigned to active drug in Part 1 have the option to receive open-label retreatment under certain circumstances.

Detailed Description

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Conditions

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TGCT

Keywords

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TGCT, Tenosynovial Giant Cell Tumour PVNS, Pigmented Villonodular Synovitis Synovitis Emactuzumab Local TGCT Diffuse TGCT

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Group 1 in Part 1/Part 2: Emactuzumab

Group 1: Subjects receiving emactuzumab administered intravenously (i.v) on Day(D)1 and repeated once every two weeks (Q2W) for a total of 5 times, followed by an observation period of 3 months leading to a total period of 24 weeks in Part 1 and continued with a follow-up phase in Part 2. Eligible Subjects assigned to active drug in Part 1 have the option to receive open-label retreatment in Part 2.

Group Type EXPERIMENTAL

Emactuzumab

Intervention Type DRUG

Emactuzumab administered once every 2 weeks (q2w)

Group 2 in Part 1 and Part 2: Placebo

Group 2: Subjects receiving placebo administered intravenously (i.v) on D1 and repeated once every two weeks (Q2W) for 5 times followed by an observation period of 3 months to a total period of 24 weeks in Part 1. In Part 2, Eligible Subjects will have the option to receive open-label emactuzumab, administered by i.v once every 2 weeks (Q2W) for a total of 5 times.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Matching placebo administered once every 2 weeks (q2w)

Interventions

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Emactuzumab

Emactuzumab administered once every 2 weeks (q2w)

Intervention Type DRUG

Placebo

Matching placebo administered once every 2 weeks (q2w)

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Age \>12 years
* Biopsy-confirmed (standard of care diagnosis history) local or diffuse TGCT where surgical resection would be associated with predicted worsening functional limitations through surgical joint damage, and/or subject has an anticipated high risk of early recurrence as determined by a multidisciplinary tumour board or equivalent, or any other morbidity associated with the surgery, and/or surgical treatment is not expected to improve the clinical outcomes of the subject.
* Measurable disease: longest diameter ≥20 mm.
* Adequate organ and bone marrow function
* If a woman of childbearing potential (WOCBP), must have a negative pregnancy test prior to starting treatment and agree to use a highly effective method of contraception
* Participants must have given written consent

Exclusion Criteria

* If a female, the subject is pregnant or breast feeding.
* Medical conditions, including auto-immune, requiring systemic immunosuppression. Any systemic treatment for these conditions (eg, glucocorticoids) is not allowed within 4 weeks of Screening and during the study.
* Known metastatic TGCT or other active cancer that requires concurrent or planned treatment
* Received systemic therapy for TGCT (investigational or approved) targeting CSF-1 or CSF-1R or any multi-tyrosine kinase inhibitor (eg nilotinib and imatinib) within 3 months prior to screening
* Any surgery, chemotherapy or radiotherapy within 3 months of screening
* Unresolved clinically significant toxicity from a previous treatment or any history of serious liver toxicity.
* Current or chronic history of liver disease.
* Inadequate renal and liver function
* Systemic antiretroviral therapy within 3 months of baseline
* Within 6 months of baseline has experienced: clinically significant myocardial infarction, severe/unstable angina pectoris, congestive heart failure New York Heart Association (NYHA) Class III or IV, or pulmonary disease (NYHA Criteria 1994) including severe thromboembolic event; incompletely healed clinically significant wounds, including bone fractures; pathological fracture or significant hypercalcaemia.
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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SynOx Therapeutics Limited

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Jean Y Blay, Prof, MD

Role: PRINCIPAL_INVESTIGATOR

Comprehensive Cancer Centre of Lyon

Locations

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NextGen Oncology

Beverly Hills, California, United States

Site Status

Sarcoma Oncology Research Center, LLC

Los Angeles, California, United States

Site Status

USC Norris Comprehensive Cancer Center

Los Angeles, California, United States

Site Status

University of Kansas Cancer Center (Overland Park) - USOR

Overland Park, Kansas, United States

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Duke Cancer Center

Durham, North Carolina, United States

Site Status

The Ohio State University

Columbus, Ohio, United States

Site Status

OHSU Knight Cancer Institute Hematology Oncology

Portland, Oregon, United States

Site Status

MD Anderson Cancer Center

Houston, Texas, United States

Site Status

MedStar Washington Hospital Center

Georgetown, Washington, United States

Site Status

LKH-Universitätsklinikum Graz

Graz, , Austria

Site Status

Cliniques Universitaires Saint-Luc

Woluwe-Saint-Lambert, Brussels Capital, Belgium

Site Status

UZ Gent - Department of Medical Oncology

Ghent, , Belgium

Site Status

Arthur J.E. Child Comprehensive Cancer Centre

Calgary, Alberta, Canada

Site Status

Princess Margaret Cancer Centre

Toronto, Ontario, Canada

Site Status

McGill University Health Centre (MUHC)

Montreal, Quebec, Canada

Site Status

Centre Antoine Lacassagne

Nice, Alpes-Maritimes, France

Site Status

Institut Bergonie - PPDS

Bordeaux, Gironde, France

Site Status

Institut de Cancerologie de Ouest - Saint Herblain

Saint-Herblain, Loire-Atlantique, France

Site Status

Centre Léon Bérard Centre Régional de Lutte Contre Le Cancer Rhône Alpes

Lyon, Rhône, France

Site Status

UNICANCER - Centre Oscar Lambret

Lille, , France

Site Status

AP-HP - Hôpital Cochin - Port-Royal, site Cochin

Paris, , France

Site Status

Institut Curie - Hôpital de Paris

Paris, , France

Site Status

Oncopole Claudius Regaud Oncologie Médicale

Toulouse, , France

Site Status

Fondazione Policlinico Universitario Campus Bio-Medico

Rome, Lazio, Italy

Site Status

Fondazione del Piemonte per l'Oncologia (IRCCS)

Candiolo, Piedmont, Italy

Site Status

Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone

Palermo, Sicily, Italy

Site Status

Azienda Ospedaliero Universitaria Pisana - Stabilimento di Cisanello

Pisa, Tuscany, Italy

Site Status

Nuovo Ospedale di Prato

Prato, Tuscany, Italy

Site Status

IRCCS lstituto Ortopedico Rizzoli

Bologna, , Italy

Site Status

Fondazione IRCCS Istituto Nazionale dei Tumori

Milan, , Italy

Site Status

Azienda Ospedaliero Universitaria Pisana - Stabilimento di Cisanello

Pisa, , Italy

Site Status

Leids Universitair Medisch Centrum

Leiden, , Netherlands

Site Status

Seoul National University Hospital

Seoul, , South Korea

Site Status

Hospital Clinico San Carlos

Madrid, Madrin, Spain

Site Status

Hospital de La Santa Creu i Sant Pau

Barcelona, , Spain

Site Status

Hospital Universitario Virgen del Rocio - PPDS

Seville, , Spain

Site Status

Hospital Universitari i Politecnic La Fe de Valencia

Valencia, , Spain

Site Status

Hospital Universitario Fundacion Jimenez Diaz

Zaragoza, , Spain

Site Status

Hospital Universitario Miguel Servet

Zaragoza, , Spain

Site Status

Skanes Universitetssjukhus Lund

Lund, Skåne County, Sweden

Site Status

Universitäts Kinderspital Beider Basel (UKBB)

Basel, , Switzerland

Site Status

Inselspital - Universitätsspital Bern

Bern, , Switzerland

Site Status

National Taiwan University Hospital

Taipei, , Taiwan

Site Status

Taipei Veterans General Hospital

Taipei, , Taiwan

Site Status

St James's University Hospital

Leeds, , United Kingdom

Site Status

University College Hospital

London, , United Kingdom

Site Status

Churchill Hospital, Cancer Haematology Center

Oxford, , United Kingdom

Site Status

Countries

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United States Austria Belgium Canada France Italy Netherlands South Korea Spain Sweden Switzerland Taiwan United Kingdom

Other Identifiers

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SNX-301-020

Identifier Type: -

Identifier Source: org_study_id