ClinicalTrials.gov Identifier: NCT05059262

A new option being studied for your TGCT / PVNS patients not amenable to surgery

Protocol at a Glance

Sponsor: Deciphera Pharmaceuticals, Inc.

Indication: Tenosynovial Giant Cell Tumor (TGCT), also known as Pigmented Villonodular Synovitis (PVNS)

Protocol 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)

Patient Population: Adult patients living with symptomatic TGCT, not amenable to surgery

Number of Patients: Approximately 120 participants

Number of Study Centers: Approximately 45 centers globally

Investigational Drug: Vimseltinib (DCC-3014): a selective inhibitor of colony-stimulating factor 1 receptor (CSF1R) developed by Deciphera

Administration: Vimseltinib 30 mg or matching placebo administered orally, twice weekly

ClinicalTrials.gov

OBJECTIVES AND ENDPOINTS

Primary Objective:

To evaluate anti-tumor activity of vimseltinib using RECIST v1.1 by blinded independent radiological review

Secondary Objectives:

  • To assess anti-tumor activity of vimseltinib using tumor volume score (TVS) and modified RECIST (mRECIST) by blinded independent radiological review
  • To assess the effects of vimseltinib on range of motion (ROM)
  • To assess the effects of vimseltinib on physical function, worst stiffness, worst pain, and quality of life (QoL) using patient-reported outcome (PRO) measures
  • To assess safety and tolerability of vimseltinib

Primary Endpoint:

Objective response rate (ORR, including complete response [CR] and partial response [PR]) per RECIST v1.1 at Week 25

Secondary Endpoints:

  • ORR per tumor volume score (TVS) at Week 25
  • Change from baseline in active range of motion (ROM) of the affected joint, relative to a reference standard, at Week 25
  • Change from baseline in the Patient-Reported Outcomes Measurement Information System (PROMIS) physical function score at Week 25
  • Change from baseline in the Worst Stiffness numeric rating scale (NRS) score at Week 25
  • Change from baseline in EuroQol Visual Analogue Scale (EQ-VAS) at Week 25
  • Response of at least a 30% improvement in the mean Brief Pain Inventory (BPI) Worst Pain NRS score without a 30% or greater increase in narcotic analgesic use at Week 25
  • ORR assessed by mRECIST at Week 25
  • Duration of response (DOR; time from first PR or CR to disease progression or death) assessed using RECIST v1.1, TVS, and mRECIST
  • Incidence of treatment-emergent serious events, related TEAEs, dose reductions, dose interruptions, and discontinuation of study drug due to adverse event
  • Changes from baseline in laboratory parameters, electrocardiograms (ECGs), and vital signs

Select Eligibility Requirements

Inclusion Criteria

  • Male or female patients ≥18 years of age
  • Histologically confirmed diagnosis of TGCT (also known as Pigmented Villonodular Synovitis [PVNS]) or giant cell tumor of the tendon sheath (GCT-TS). Tumor biopsy to confirm TGCT / PVNS diagnosis will be required if no histology / pathology is available. Note: Participants should have TGCT in a single joint and must have TGCT in joints where range of motion assessments can be assessed
  • Disease for which surgical resection will potentially cause worsening functional limitation or severe morbidity as judged by surgical consultation or a multidisciplinary tumor board
  • Symptomatic disease with at least moderate pain (defined as a score of 4 or more, with 10 describing the worst condition)
  • Participant should complete 14 consecutive days of questionnaires during the screening period and must meet minimum requirements
  • Willing and able to complete the PRO assessments on an electronic device
  • Agrees to follow all contraceptive guidelines

Exclusion Criteria

  • Previous use of systemic therapy targeting CSF1 or CSF1R; previous therapy with imatinib and nilotinib is allowed
  • Treatment for TGCT / PVNS, including investigational treatment, within 2 weeks prior to screening. For immediate prior treatments with a half‑life longer than three days, or if the half-life is not available, the interval must be ≥28 days prior to the first administration of study drug.
  • Known metastatic TGCT / PVNS or other active cancer that requires concurrent treatment (exceptions considered on case-by-case basis)
  • Receive concurrent treatment with any prohibited medications
  • Any clinically significant comorbidities, such as significant concomitant arthropathy related to TGCT in the affected joint, known alcohol abuse, or any other serious medical or psychiatric condition(s), which in the judgment of the Investigator, could compromise compliance with the protocol, interfere with the interpretation of study results, or predispose the patient to safety risks
  • Active liver or biliary disease, nonalcoholic steatohepatitis (NASH), or cirrhosis

We Look Forward to Speaking with You

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