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NCT Number: NCT05059262

Study of Vimseltinib for Tenosynovial Giant Cell Tumor

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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Tenosynovial Giant Cell Tumor Giant Cell Tumor of Tendon Sheath Giant Cell Tumors Joint Diseases Muscular Diseases Musculoskeletal Diseases Neoplasms Neoplasms by Histologic Type Neoplasms, Connective Tissue Neoplasms, Connective and Soft Tissue Pigmented Villonodular Synovitis Synovitis Synovitis, Pigmented Villonodular Tendinopathy Tenosynovial Giant Cell Tumor, Diffuse Tenosynovial Giant Cell Tumor, Localized

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Chris O'Brien Lifehouse, Camperdown, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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

Treatment and study plan

Vimseltinib

Drug

Administered orally

Other names: DCC-3014

Placebo

Drug

Administered orally

Primary outcomes

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

    Time frame: Baseline to Week 25 (Cycle 7, Day 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.

Secondary outcomes

  1. ORR at Week 25 Per Tumor Volume Score (TVS)

    Time frame: Baseline to Week 25 (Cycle 7, Day 1)

    TVS is a semi-quantitative magnetic resonance imaging (MRI) scoring system that describes tumor mass and is based on 10% increments of the estimated volume of the maximally distended synovial cavity or tendon sheath involved. A tumor that is equal in volume to that of a maximally distended synovial cavity or tendon sheath was scored 10; a score of 0 indicated no evidence of tumor. ORR was the percentage of participants who achieved either CR or PR as assessed by blinded IRR using TVS.

    • CR: Lesion completely gone
    • PR: ≥50% decrease in volume score relative to Baseline.

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

    Time frame: Baseline to Week 25 (Cycle 7, Day 1)

    Presented here is the change from baseline in active ROM to Week 25. Measurement of the affected and contralateral, non-affected joint was assessed by goniometer and measured in degrees. At baseline, the motion with the smallest relative ROM value (worst) was identified, and this motion was used for evaluating the change in relative ROM subsequently. The affected joint measurement was used to derive a relative ROM based on active measurement relative to reference standard value provided by the American Medical Association. Relative ROM is expressed in percent: 100 x (joint ROM measure)/(reference ROM standard).

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

    Time frame: Baseline to Week 25 (Cycle 7, Day 1)

    All participants were asked 15 questions from the PROMIS-PF item bank. The questions used one of two 5-point verbal rating scales: either 1 = "unable to do", 2 = "with much difficulty", 3 = "with some difficulty", 4 = "with a little difficulty", and 5 = "without any difficulty"; or 1 = "cannot do", 2 = "quite a lot", 3 = "somewhat", 4 = "very little", and 5 = "not at all." Total scores were converted to T-scores that ranged from 0 to 100, with higher scores representing less physical function interference and better health outcomes.

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

    Time frame: Baseline to Week 25 (Cycle 7, Day 1)

    The Worst Stiffness NRS is a single question that asks the participant to assess their worst stiffness in the last 24 hours. Participants rate their worst stiffness on a scale of 0 to 10, where 0 is "no stiffness" and 10 is "worst imaginable." Lower scores represented better level of stiffness.

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

    Time frame: Baseline to Week 25 (Cycle 7, Day 1)

    The EQ-VAS is a standardized tool for measuring overall health. EQ-VAS recorded the participant's self-rated health on a vertical VAS scale ranging from a minimum of 0 (worst imaginable health state) to a maximum of 100 (best imaginable health state). Higher scores indicated better health state.

  6. Percentage of Participants With Response at Week 25 Based on Brief Pain Inventory (BPI) Worst Pain NRS Score and Narcotic Analgesic Use

    Time frame: Baseline to Week 25 (Cycle 7, Day 1)

    Participants reported responses to the BPI Worst Pain NRS. The BPI Worst Pain NRS ranged from 0 to 10, where 0 is "no pain" and 10 is "pain as bad as you can imagine." A responder was defined as a participant who:

    (i) experienced a decrease of at least 30% in the mean BPI Worst Pain NRS item and (ii) did not experience a 30% or greater increase in narcotic analgesic use.

Sponsors and collaborators

Lead sponsor

Deciphera Pharmaceuticals, LLC

Industry

Registry information

Official study title

A Phase 3, Randomized, Placebo-controlled, Double-blind Study of Vimseltinib to Assess the Efficacy and Safety in Patients With Tenosynovial Giant Cell Tumor (MOTION)

Acronym: MOTION

Important dates

Study start
2021
Primary completion
2023
Study completion
2028
First posted
Sep 28, 2021
Registry last updated
Mar 20, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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