Study of Vimseltinib (DCC-3014) in Patients With Advanced Tumors and Tenosynovial Giant Cell Tumor

NCT03069469 | Active Not Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: DCC-3014-01-0012024-514933-39-00
• Study Type: interventional
• Target: 120
• Locations: 9 countries
• Sites: 24

Brief Summary

This is a multicenter, open-label Phase 1/2 study of vimseltinib in patients with malignant solid tumors and tenosynovial giant cell tumor (TGCT). There will be 2 distinct parts in this study: Dose Escalation (Phase 1) and Expansion (Phase 2). Phase 1 will enroll both malignant solid tumor and TGCT patients. Phase 2 will comprise two cohorts (Cohort A and Cohort B) and will only enroll TGCT patients.

Conditions

Advanced Malignant Neoplasm; Pigmented Villonodular Synovitis; Giant Cell Tumor of Tendon Sheath; Tenosynovial Giant Cell Tumor; Tenosynovial Giant Cell Tumor, Diffuse

Keywords

TGCT; DTGCT; PVNS

Outcome Measures

Primary Outcomes (9)

• Maximum Tolerated Dose (MTD)

  Determine the maximum tolerated dose.

  Day 1 - Day 28 of Cycle 1 for each dose level tested

• Number of Patients with Dose-Limiting Toxicities (DLTs)

  Identify the number of patients with DLTs for each dose level tested.

  Day 1- Day 28 of Cycle 1 for each dose level tested

• Time to maximum observed concentration of Vimseltinib

  Measure the time to maximum plasma concentration of vimseltinib in patients.

  Cycle 1 Day 1 and Day 8, and Cycle 2 Day 1 (pre-dose and at multiple time points (up to 8 hours) post-dose)

• Maximum observed concentration of Vimseltinib

  Measure the maximum observed concentration of vimseltinib in patients.

  Cycle 1 Day 1 and Day 8, and Cycle 2 Day 1 (pre-dose and at multiple time points (up to 8 hours) post-dose)

• Trough observed concentration of Vimseltinib

  Measure the observed trough concentration of vimseltinib in patients.

  Cycle 1 Day 1 and Day 8, and Cycle 2 Day 1 (pre-dose and at multiple time points (up to 8 hours) post-dose)

• Area under the concentration-time curve (AUC) of Vimseltinib

  Measure the AUC of vimseltinib.

  Cycle 1 Day 1 and Day 8, and Cycle 2 Day 1 (pre-dose and at multiple time points (up to 8 hours) post-dose)

• Half life of Vimseltinib

  Measure half life of vimseltinib in patients.

  Cycle 1 Day 1 and Day 8, and Cycle 2 Day 1 (pre-dose and at multiple time points (up to 8 hours) post-dose)

• Objective response rate (ORR= complete response [CR]+partial response [PR]) (Expansion Phase only)

  Assessed by central read using Response Evaluation Criteria In Solid Tumors (RECIST) Version 1.1.

  At Week 25 (Cycle 7, Day 1)

• Duration of response rate (DOR) (Expansion Phase only)

  Measure time from partial response (PR) or complete response (CR) to disease progression or death.

  Date from PR or CR to disease progression or death (Estimated up to 24 months)

Secondary Outcomes (5)

• Response rate (Expansion Phase only)

  At Week 25 (Cycle 7, Day 1)

• Range of Motion (ROM) (Expansion Phase only)

  Baseline to Week 25 (Cycle 7, Day 1)

• Brief Pain Inventory (BPI) Worst Pain Numeric Rating Scale (NRS) Score (Expansion Phase only)

  Baseline to Week 25 (Cycle 7, Day 1)

• Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Score (Expansion Phase only)

  Baseline to Week 25 (Cycle 7, Day 1)

• Worst Stiffness Numeric Rating Scale (NRS) Score (Expansion Phase only)

  Baseline to Week 25 (Cycle 7, Day 1)

Study Arms (1)

Experimental Treatment

OTHER

Dose Escalation Phase: Increasing doses of vimseltinib beginning at 10 milligram (mg) once daily (QD) for 28 day cycles until disease progression or unacceptable toxicity. Expansion Phase: Dosing of different patient cohorts at the dose level determined from the Dose Escalation Phase of the study.

Drug: Vimseltinib

Interventions

Vimseltinib DRUG

Colony-stimulating factor 1 receptor (CSF1R) inhibitor

Also known as: DCC-3014

Experimental Treatment

Eligibility Criteria

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

You may qualify if:

• Dose Escalation Phase:
• Patients ≥18 years of age
• Patients must have:
• advanced malignant solid tumors; or
• symptomatic TGCT for which surgical resection is not an option (tumor biopsy to confirm diagnosis required if no histology/pathology available at screening)
• Malignant solid tumor patients only: Able to provide a tumor tissue sample
• Must have 1 measurable lesion according to RECIST Version 1.1
• Malignant solid tumor patients only: Must have Eastern Cooperative Oncology Group (ECOG) performance status of 0-1
• 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.
• Expansion Phase (Cohorts A and B)
• Patients ≥18 years of age
• Patients must have symptomatic TGCT for which surgical resection is not an option (tumor biopsy to confirm diagnosis required if no histology/pathology available at screening)
• a) Expansion Cohort B: patients must have prior systemic treatment with anti-CSF1 or anti-CSF1R therapy, with the exception of imatinib or nilotinib

You may not qualify if:

• Dose Escalation Phase:
• Received anticancer therapy or therapy for TGCT, including investigational therapy, within 2 weeks or 28 days for therapies with half-life (t1/2) longer than 3 days prior to the administration of study drug.
• Unresolved toxicity (Grade \>1 or baseline) from previous anticancer therapy or TGCT therapy, excluding alopecia.
• Known active central nervous system (CNS) metastases.
• History or presence of clinically relevant cardiovascular abnormalities.
• Systemic arterial or venous thrombotic or embolic events.
• QT interval corrected by Fridericia's formula (QTcF) \>450 ms in males or \>470 ms in females or history of long QT syndrome.
• Left ventricular ejection fraction (LVEF) \<50%.
• Concurrent treatment with proton-pump inhibitor(s).
• Major surgery within 2 weeks of the first dose of study drug.
• Malabsorption syndrome or other illness that could affect oral absorption.
• Known human immunodeficiency virus, active hepatitis B, active hepatitis C, or active mycobacterium tuberculosis infection.
• If female, the patient is pregnant or lactating.
• Known allergy or hypersensitivity to any component of the study drug.
• Any other clinically significant comorbidities.

Sponsors & Collaborators

• Deciphera Pharmaceuticals, LLC: lead

Study Sites (24)

Stanford Cancer Institute

Palo Alto, California, 94304, United States

Location

University of Colorado - Denver

Denver, Colorado, 80204, United States

Location

Mayo Clinic

Jacksonville, Florida, 32224, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

Dana Farber

Boston, Massachusetts, 02215, United States

Location

MSKCC

New York, New York, 10065, United States

Location

OHSU

Portland, Oregon, 97239, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Sarah Cannon Research Institute

Nashville, Tennessee, 37203, United States

Location

Peter MacCallum Cancer Centre

Melbourne, Australia

Location

McGill University Health Centre

Montreal, Quebec, Canada

Location

Princess Margaret Cancer Center

Toronto, Canada

Location

Centre Leon Berard

Lyon, France

Location

Gustave Roussy Cancer Campus Grand Paris

Paris, France

Location

IRCCS Istituto Ortopedico Rizzoli

Bologna, Italy

Location

Fondazione IRCCS Istituto Nazionale Dei Tumori

Milan, Italy

Location

Istituto Nazionale dei Tumori

Milan, Italy

Location

Regina Elena National Cancer Institute

Rome, Italy

Location

Leiden University Medical Center

Leiden, Netherlands

Location

M. Sklodowska-Curie Memorial Cancer Center

Warsaw, Poland

Location

Hospital Universitario Vall d'Hebron

Barcelona, Spain

Location

Hospital Clinico San Carlos

Madrid, Spain

Location

Hospital Universitario Virgen del Rocío, Sevilla

Seville, Spain

Location

University College Hospital

London, United Kingdom

Location

MeSH Terms

Conditions

Synovitis, Pigmented Villonodular; Giant Cell Tumor of Tendon Sheath

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

Study Officials

• Maitreyi Sharma, MD

  Deciphera Pharmaceuticals, LLC

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 20, 2017
• First Posted: March 3, 2017
• Study Start: February 16, 2017
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): August 1, 2028
• Last Updated: December 18, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

PL (1); FR (2); AU (1); IT (4); NL (1); US (9); GB (1); CA (2); ES (3)