Study of Vimseltinib for Tenosynovial Giant Cell Tumor

NCT05059262 | Active Not Recruiting Phase 3 Results DMC FDA Drug

• 2 other identifiers: DCC-3014-03-0012024-513624-42-00
• Study Type: interventional
• Target: 123
• Locations: 13 countries
• Sites: 35

Brief Summary

This is a multicenter Phase 3 clinical study, which aims to evaluate the effectiveness of an investigational drug called vimseltinib for the treatment of tenosynovial giant cell tumor (TGCT) in cases where surgical removal of the tumor is not an option. The study consists of two parts. In Part 1, eligible study participants will be assigned to receive either vimseltinib or matching placebo for 24 weeks. A number of assessments will be carried out during the course of the study, including physical examinations, blood tests, imaging studies, electrocardiograms, and questionnaires. MRI scans will be used to evaluate the response of the tumors to the treatment. Participants assigned to placebo in Part 1 will have the option to receive vimseltinib for Part 2. Part 2 is a long-term treatment phase in which all participants receive open-label vimseltinib.

Conditions

Pigmented Villonodular Synovitis; Giant Cell Tumor of Tendon Sheath; Tenosynovial Giant Cell Tumor, Localized; Tenosynovial Giant Cell Tumor; Tenosynovial Giant Cell Tumor, Diffuse

Keywords

TGCT; PVNS

Outcome Measures

Primary Outcomes (1)

• Objective Response Rate (ORR) at Week 25 Per Response Evaluation Criteria In Solid Tumors (RECIST) Version 1.1

  ORR was assessed by blinded independent radiologic review (IRR) using RECIST Version 1.1. ORR was defined as the percentage of participants who achieved either complete response (CR) or partial response (PR). * CR: Disappearance of all target lesions. Any pathological lymph nodes must be \<10 millimeter (mm) in short axis. Non-nodal targets must be absent. * PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters.

  Baseline to Week 25 (Cycle 7, Day 1)

Secondary Outcomes (6)

• ORR at Week 25 Per Tumor Volume Score (TVS)

  Baseline to Week 25 (Cycle 7, Day 1)

• Change From Baseline in Active Range of Motion (ROM) at Week 25

  Baseline to Week 25 (Cycle 7, Day 1)

• Change From Baseline in the Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function (PF) Score at Week 25

  Baseline to Week 25 (Cycle 7, Day 1)

• Change From Baseline in the Worst Stiffness Numeric Rating Scale (NRS) Score at Week 25

  Baseline to Week 25 (Cycle 7, Day 1)

• Change From Baseline in EuroQoL Visual Analogue Scale (EQ-VAS) at Week 25

  Baseline to Week 25 (Cycle 7, Day 1)

Study Arms (2)

Part 1/Part 2 - Vimseltinib/Vimseltinib

EXPERIMENTAL

Participants received blinded treatment of 30 mg twice a week (BIW) vimseltinib for 24 weeks in Part 1 and open-label 30 mg BIW vimseltinib in Part 2.

Drug: Vimseltinib

Part 1/Part 2: Placebo/Vimseltinib

PLACEBO COMPARATOR

Participants received blinded treatment of BIW matching placebo for 24 weeks in Part 1 and open-label 30 mg BIW vimseltinib in Part 2.

Drug: Vimseltinib; Drug: Placebo

Interventions

Vimseltinib DRUG

Administered orally

Also known as: DCC-3014

Part 1/Part 2 - Vimseltinib/Vimseltinib; Part 1/Part 2: Placebo/Vimseltinib

Placebo DRUG

Administered orally

Part 1/Part 2: Placebo/Vimseltinib

Eligibility Criteria

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

You may qualify if:

• Patients ≥18 years of age
• TGCT for which surgical resection is not an option (tumor biopsy to confirm diagnosis required if no histology/pathology available at screening)
• Symptomatic disease as defined as at least moderate pain or at least moderate stiffness (defined as a score of 4 or more, with 10 describing the worst condition) within the screening period and documented in the medical record
• Participants should complete 14 consecutive days of questionnaires during the screening period and must meet minimum requirements as outlined in study protocol
• Must have stable analgesic regimen, as judged by the investigator, for at least 2 weeks prior to first dose of study drug
• Must have measurable disease, as per RECIST Version 1.1, with at least one lesion having a minimum size of 2cm
• Adequate organ and bone marrow function
• If a female of childbearing potential, must have a negative pregnancy test prior to enrollment and agree to follow the contraception requirements
• Must provide signed consent to participate in the study and is willing to comply with study-specific procedures
• Willing and able to complete the patient-reported outcome (PRO) assessments on an electronic device

You may not qualify if:

• Previous use of systemic therapy (investigational or approved) targeting colony-stimulating factor 1 (CSF1) or colony-stimulating factor 1 receptor (CSF1R); previous therapy with imatinib and nilotinib is allowed
• Received therapy for TGCT, including investigational therapy during the screening period. Participated in a non-TGCT investigational drug study within 30 days of screening.
• Known metastatic TGCT or other active cancer that requires concurrent treatment (exceptions will be considered on a case-by-case basis)
• QT interval corrected by Fridericia's formula (QTcF) \>450 ms in males or \>470 ms in females or history of long QT syndrome
• Concurrent treatment with any study-prohibited medications
• Major surgery within 14 days of the first dose of study drug
• Any clinically significant comorbidities
• Active liver or biliary disease including nonalcoholic steatohepatitis (NASH) or cirrhosis
• Malabsorption syndrome or other illness that could affect oral absorption
• Known active human immunodeficiency virus (HIV), acute or chronic hepatitis B, acute or chronic hepatitis C, or known active mycobacterium tuberculosis infection
• If female, the participant is pregnant or breastfeeding
• Known allergy or hypersensitivity to any component of the study drug
• Contraindication to MRI

Sponsors & Collaborators

• Deciphera Pharmaceuticals, LLC: lead

Study Sites (35)

City of Hope

Duarte, California, 91010, United States

Location

UC Davis Comprehensive Cancer Center

Sacramento, California, 95817, United States

Location

University of Colorado

Aurora, Colorado, 80045, United States

Location

University of Kansas

Kansas City, Kansas, 66160, United States

Location

Dana Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

Mayo Clinic Rochester

Rochester, Minnesota, 55902, United States

Location

Memorial Sloan-Kettering Cancer Center

New York, New York, 10016, United States

Location

Duke Sarcoma Research

Durham, North Carolina, 27710, United States

Location

Ohio State University

Columbus, Ohio, 43210, United States

Location

University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Seattle Cancer Care Alliance

Seattle, Washington, 98109, United States

Location

Chris O'Brien Lifehouse

Camperdown, Australia

Location

McGill University

Montreal, Canada

Location

Princess Margaret Hospital

Toronto, Canada

Location

Institut Bergonié

Bordeaux, France

Location

Centre Léon Bérard

Lyon, France

Location

Institut Gustave Roussy

Villejuif, France

Location

Helios Klinikum Berlin-Buch

Berlin, Germany

Location

University Hospital Essen (Universitätsklinikum Essen)

Essen, Germany

Location

Prince of Wales Hospital

Hong Kong, Hong Kong

Location

Istituto Ortopedico Rizzoli

Bologna, Italy

Location

Fondazione IRCCS Istituto Nazionale dei Tumori

Milan, Italy

Location

Istituto Nazionale Tumori IRCCS "Fondazione G. Pascale"

Naples, Italy

Location

Istituto Oncologico Veneto

Padua, Italy

Location

Istituto Nazionale Tumori Regina Elena

Rome, Italy

Location

Leiden University Medical Center

Leiden, Netherlands

Location

Oslo University Hospital

Oslo, Norway

Location

Klinika Nowotworów Tkanek Miękkich, Kości i Czerniaków Narodowy Instytut Onkologii im. Marii Skłodowskiej-Curie - Państwowy Instytut Badawczy

Warsaw, Poland

Location

Hospital de la Santa Creu i Sant Pau

Barcelona, Spain

Location

Hospital Universitario Vall d'Hebron

Barcelona, Spain

Location

Fundacion Jimenez Diaz

Madrid, Spain

Location

Hospital Clinico San Carlos

Madrid, Spain

Location

Universitäts-Kinderspital beider Basel (UKBB)

Basel, Switzerland

Location

Cancer & Haematology Centre, The Churchill Hospital - Oxford University Hospitals NHS Foundation Trust

London, United Kingdom

Location

University College London Hospitals

London, United Kingdom

Location

Related Publications (2)

• Gelderblom H, Bhadri V, Stacchiotti S, Bauer S, Wagner AJ, van de Sande M, Bernthal NM, Lopez Pousa A, Razak AA, Italiano A, Ahmed M, Le Cesne A, Tinoco G, Boye K, Martin-Broto J, Palmerini E, Tafuto S, Pratap S, Powers BC, Reichardt P, Casado Herraez A, Rutkowski P, Tait C, Zarins F, Harrow B, Sharma MG, Ruiz-Soto R, Sherman ML, Blay JY, Tap WD; MOTION investigators. Vimseltinib versus placebo for tenosynovial giant cell tumour (MOTION): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2024 Jun 22;403(10445):2709-2719. doi: 10.1016/S0140-6736(24)00885-7. Epub 2024 Jun 3.

  PMID: 38843860 DERIVED

• Tap WD, Sharma MG, Vallee M, Smith BD, Sherman ML, Ruiz-Soto R, de Sande MV, Randall RL, Bernthal NM, Gelderblom H. The MOTION study: a randomized, phase III study of vimseltinib for the treatment of tenosynovial giant cell tumor. Future Oncol. 2024 Mar;20(10):593-601. doi: 10.2217/fon-2023-0238. Epub 2023 Aug 18.

  PMID: 37593881 DERIVED

MeSH Terms

Conditions

Giant Cell Tumor of Tendon Sheath; Synovitis, Pigmented Villonodular

Condition Hierarchy (Ancestors)

Giant Cell Tumors; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Synovitis; Joint Diseases; Musculoskeletal Diseases; Tendinopathy; Muscular Diseases

Results Point of Contact

• Title: Clinical Trials
• Organization: Deciphera Pharmaceuticals, LLC

clinicaltrials@deciphera.com

785-830-2100

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 17, 2021
• First Posted: September 28, 2021
• Study Start: October 14, 2021
• Primary Completion: August 10, 2023
• Study Completion (Estimated): July 1, 2028
• Last Updated: March 20, 2026
• Results First Posted: February 24, 2025

Record last verified: 2026-03

Locations

US (11); CA (2); AU (1); DE (2); NL (1); GB (2); IT (5); ES (4); NO (1); FR (3); PL (1); CH (1); HK (1)