Advanced Malignant NeoplasmPhase 1/2NCT03069469
Study of Vimseltinib (DCC-3014) in Patients With Advanced Tumors and Tenosynovial Giant Cell Tumor
A Phase 1/2 study testing Vimseltinib in people with Advanced Malignant Neoplasm and Pigmented Villonodular Synovitis, run by Deciphera Pharmaceuticals, LLC at 24 sites in 9 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Advanced Malignant Neoplasm, Pigmented Villonodular Synovitis, Giant Cell Tumor of Tendon Sheath
- What is being tested?
- Vimseltinib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 120 participants
- Where
- 24 sitesin 9 countries
- Study dates
- 16 Feb 2017to Aug 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Show all 19Show fewer
- 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)
- Expansion Cohort B: patients must have prior systemic treatment with anti-CSF1 or anti-CSF1R therapy, with the exception of imatinib or nilotinib
- Adequate organ and bone marrow function
- Must have at least 1 measurable lesion according to RECIST Version 1.1
- 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.
Who cannot join
- 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.
Show all 31Show fewer
- 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.
- Expansion Phase (Cohorts A and B)
- Expansion Cohort A: received systemic therapy targeting CSF1 or CSF1R; previous therapy with imatinib and nilotinib is allowed.
- Expansion Cohort B: discontinued systemic therapy targeting anti-CSF1 or anti-CSF1R due to drug-induced liver injury.
- Treatment with therapy for TGCT, including investigational therapy, within 2 weeks or 28 days for therapies with a t1/2 longer than 3 days prior to the administration of the study drug.
- Known metastatic TGCT or other active cancer that requires concurrent treatment.
- 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) <55%.
- Concurrent treatment with proton-pump inhibitor(s).
- Major surgery within 2 weeks of the first dose of study drug.
- Any clinically significant comorbidities
- Malabsorption syndrome or other illness that could affect oral absorption.
- Known human immunodeficiency virus (HIV), active or chronic hepatitis B, active or chronic 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.
- Contraindication for MRI
- Active liver or biliary disease, including evidence of fatty liver, nonalcoholic steatohepatitis (NASH), or cirrhosis
Trial locations and contacts
24 sites in 9 countries.
24 sites in 9 countries
United States9 sites
Dana Farber
Boston, Massachusetts, 02215
University of Colorado - Denver
Denver, Colorado, 80204
Mayo Clinic
Jacksonville, Florida, 32224
University of Miami
Miami, Florida, 33136
Sarah Cannon Research Institute
Nashville, Tennessee, 37203
MSKCC
New York, New York, 10065
Stanford Cancer Institute
Palo Alto, California, 94304
OHSU
Portland, Oregon, 97239
Oregon Health & Science University
Portland, Oregon, 97239
Italy4 sites
IRCCS Istituto Ortopedico Rizzoli
Bologna
Fondazione IRCCS Istituto Nazionale Dei Tumori
Milan
Istituto Nazionale dei Tumori
Milan
Regina Elena National Cancer Institute
Rome
Spain3 sites
Hospital Universitario Vall d'Hebron
Barcelona
Hospital Clinico San Carlos
Madrid
Hospital Universitario Virgen del Rocío, Sevilla
Seville
Canada2 sites
McGill University Health Centre
Montreal, Quebec
Princess Margaret Cancer Center
Toronto
France2 sites
Centre Leon Berard
Lyon
Gustave Roussy Cancer Campus Grand Paris
Paris
About this study
Official title: A Multicenter Phase 1/2, Open-Label Study of DCC-3014 to Assess the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics in Patients With Advanced Tumors and Tenosynovial Giant Cell Tumor
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
- Being tested
- Vimseltinib
- Keywords
- TGCT, DTGCT, PVNS
- Medical subject headings
- Synovitis, Pigmented Villonodular, Giant Cell Tumor of Tendon Sheath
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VimseltinibDrug
Also called DCC-3014
Colony-stimulating factor 1 receptor (CSF1R) inhibitor
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Vimseltinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose (MTD)
Determine the maximum tolerated dose.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Date from PR or CR to disease progression or death (Estimated up to 24 months)
Secondary outcomes (5)
Response rate (Expansion Phase only)
Assessed by central read using tumor volume score and modified RECIST (mRECIST) Version 1.1
Measured: At Week 25 (Cycle 7, Day 1)
Range of Motion (ROM) (Expansion Phase only)
Measure mean change from baseline in relative ROM
Measured: Baseline to Week 25 (Cycle 7, Day 1)
Brief Pain Inventory (BPI) Worst Pain Numeric Rating Scale (NRS) Score (Expansion Phase only)
Proportion of responders based on Brief Pain Inventory (BPI) worst pain numeric rating scale (NRS) and narcotic analgesic use by Brief Pain Inventory-30 (BPI-30)
Measured: Baseline to Week 25 (Cycle 7, Day 1)
Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Score (Expansion Phase only)
Analysis of patient reported outcomes based upon the patient-reported outcomes measurement information system (PROMIS) physical function questionnaire
Measured: Baseline to Week 25 (Cycle 7, Day 1)
Worst Stiffness Numeric Rating Scale (NRS) Score (Expansion Phase only)
Analysis of patient reported outcomes based upon the Worst Stiffness Numeric Rating Scale (NRS)
Measured: Baseline to Week 25 (Cycle 7, Day 1)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2017
- Study start16 Feb 2017
- Primary completionAug 2028 (estimated)
- Study completionAug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Advanced Malignant NeoplasmStudies of the same condition, open and under way.
- More trials run by Deciphera Pharmaceuticals, LLCThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Deciphera Pharmaceuticals, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Maitreyi Sharma, MD, Study director, Deciphera Pharmaceuticals, LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03069469
- Sponsor's protocol ID
- DCC-3014-01-001
- Other IDs
- 2024-514933-39-00
- Status last verified
- Jun 2026
- First submitted
- 20 Feb 2017
- Last updated
- 25 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Phase 1/2, Open-Label Study of DCC-3014 to Assess the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics in Patients With Advanced Tumors and Tenosynovial Giant Cell Tumor
- ClinicalTrials.gov ID
- NCT03069469
- Acronym / sponsor ID
- DCC-3014-01-001
- 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
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- Deciphera Pharmaceuticals, LLCIndustry (pharma, biotech, devices)
- Study start
- 16 Feb 2017 (actual)
- Primary completion
- Aug 2028 (estimated)
- Study completion
- Aug 2028 (estimated)
- First posted
- 3 Mar 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental Treatment | Other | Drug: VimseltinibDose 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (19 listed)
- Patients ≥18 years of age
- Patients must have:
- 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
Key exclusion criteria (31 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VimseltinibAlso: DCC-3014Colony-stimulating factor 1 receptor (CSF1R) inhibitor | Drug | Experimental Treatment |
Primary outcome
Maximum Tolerated Dose (MTD)
Time frame: Day 1 - Day 28 of Cycle 1 for each dose level tested
Determine the maximum tolerated dose.
Number of Patients with Dose-Limiting Toxicities (DLTs)
Time frame: Day 1- Day 28 of Cycle 1 for each dose level tested
Identify the number of patients with DLTs for each dose level tested.
Time to maximum observed concentration of Vimseltinib
Time frame: 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)
Measure the time to maximum plasma concentration of vimseltinib in patients.
Maximum observed concentration of Vimseltinib
Time frame: 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)
Measure the maximum observed concentration of vimseltinib in patients.
Trough observed concentration of Vimseltinib
Time frame: 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)
Measure the observed trough concentration of vimseltinib in patients.
Area under the concentration-time curve (AUC) of Vimseltinib
Time frame: 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)
Measure the AUC of vimseltinib.
Half life of Vimseltinib
Time frame: 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)
Measure half life of vimseltinib in patients.
Objective response rate (ORR= complete response [CR]+partial response [PR]) (Expansion Phase only)
Time frame: At Week 25 (Cycle 7, Day 1)
Assessed by central read using Response Evaluation Criteria In Solid Tumors (RECIST) Version 1.1.
Duration of response rate (DOR) (Expansion Phase only)
Time frame: Date from PR or CR to disease progression or death (Estimated up to 24 months)
Measure time from partial response (PR) or complete response (CR) to disease progression or death.
5 secondary outcomes
Response rate (Expansion Phase only)
Time frame: At Week 25 (Cycle 7, Day 1)
Range of Motion (ROM) (Expansion Phase only)
Time frame: Baseline to Week 25 (Cycle 7, Day 1)
Brief Pain Inventory (BPI) Worst Pain Numeric Rating Scale (NRS) Score (Expansion Phase only)
Time frame: Baseline to Week 25 (Cycle 7, Day 1)
Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Score (Expansion Phase only)
Time frame: Baseline to Week 25 (Cycle 7, Day 1)
Worst Stiffness Numeric Rating Scale (NRS) Score (Expansion Phase only)
Time frame: Baseline to Week 25 (Cycle 7, Day 1)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jun 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Maitreyi Sharma, MD study director
Deciphera Pharmaceuticals, LLC
24 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Vimseltinib in people with Advanced Malignant Neoplasm and Pigmented Villonodular Synovitis, run by Deciphera Pharmaceuticals, LLC at 24 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 19 inclusion and 31 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 25 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 24 sites in United States, Italy, Spain, Canada, France and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum Tolerated Dose (MTD) (time frame: Day 1 - Day 28 of Cycle 1 for each dose level tested). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Jun 2026.