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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03069469




Registration number
NCT03069469
Ethics application status
Date submitted
20/02/2017
Date registered
3/03/2017

Titles & IDs
Public title
Study of Vimseltinib (DCC-3014) in Patients With Advanced Tumors and Tenosynovial Giant Cell Tumor
Scientific 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
Secondary ID [1] 0 0
DCC-3014-01-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Advanced Malignant Neoplasm 0 0
Pigmented Villonodular Synovitis 0 0
Giant Cell Tumor of Tendon Sheath 0 0
Tenosynovial Giant Cell Tumor 0 0
Tenosynovial Giant Cell Tumor, Diffuse 0 0
Condition category
Condition code
Cancer 0 0 0 0
Bone
Cancer 0 0 0 0
Other cancer types
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Vimseltinib

Other: Experimental Treatment - 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.


Treatment: Drugs: Vimseltinib
Colony-stimulating factor 1 receptor (CSF1R) inhibitor
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Maximum Tolerated Dose (MTD)
Timepoint [1] 0 0
Day 1 - Day 28 of Cycle 1 for each dose level tested
Primary outcome [2] 0 0
Number of Patients with Dose-Limiting Toxicities (DLTs)
Timepoint [2] 0 0
Day 1- Day 28 of Cycle 1 for each dose level tested
Primary outcome [3] 0 0
Time to maximum observed concentration of Vimseltinib
Timepoint [3] 0 0
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)
Primary outcome [4] 0 0
Maximum observed concentration of Vimseltinib
Timepoint [4] 0 0
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)
Primary outcome [5] 0 0
Trough observed concentration of Vimseltinib
Timepoint [5] 0 0
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)
Primary outcome [6] 0 0
Area under the concentration-time curve (AUC) of Vimseltinib
Timepoint [6] 0 0
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)
Primary outcome [7] 0 0
Half life of Vimseltinib
Timepoint [7] 0 0
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)
Primary outcome [8] 0 0
Objective response rate (ORR= complete response [CR]+partial response [PR]) (Expansion Phase only)
Timepoint [8] 0 0
At Week 25 (Cycle 7, Day 1)
Primary outcome [9] 0 0
Duration of response rate (DOR) (Expansion Phase only)
Timepoint [9] 0 0
Date from PR or CR to disease progression or death (Estimated up to 24 months)
Secondary outcome [1] 0 0
Response rate (Expansion Phase only)
Timepoint [1] 0 0
At Week 25 (Cycle 7, Day 1)
Secondary outcome [2] 0 0
Range of Motion (ROM) (Expansion Phase only)
Timepoint [2] 0 0
Baseline to Week 25 (Cycle 7, Day 1)
Secondary outcome [3] 0 0
Brief Pain Inventory (BPI) Worst Pain Numeric Rating Scale (NRS) Score (Expansion Phase only)
Timepoint [3] 0 0
Baseline to Week 25 (Cycle 7, Day 1)
Secondary outcome [4] 0 0
Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Score (Expansion Phase only)
Timepoint [4] 0 0
Baseline to Week 25 (Cycle 7, Day 1)
Secondary outcome [5] 0 0
Worst Stiffness Numeric Rating Scale (NRS) Score (Expansion Phase only)
Timepoint [5] 0 0
Baseline to Week 25 (Cycle 7, Day 1)

Eligibility
Key inclusion criteria
Inclusion Criteria

Dose Escalation Phase:

1. Patients =18 years of age
2. Patients must have:

1. advanced malignant solid tumors; or
2. symptomatic TGCT for which surgical resection is not an option (tumor biopsy to confirm diagnosis required if no histology/pathology available at screening)
3. Malignant solid tumor patients only: Able to provide a tumor tissue sample
4. Must have 1 measurable lesion according to RECIST Version 1.1
5. Malignant solid tumor patients only: Must have Eastern Cooperative Oncology Group (ECOG) performance status of 0-1
6. Adequate organ and bone marrow function
7. If a female of childbearing potential, must have a negative pregnancy test prior to enrollment and agree to follow the contraception requirements.
8. Must provide signed consent to participate in the study and is willing to comply with study-specific procedures.

Expansion Phase (Cohorts A and B)

1. Patients =18 years of age
2. 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
3. Adequate organ and bone marrow function
4. Must have at least 1 measurable lesion according to RECIST Version 1.1
5. If a female of childbearing potential, must have a negative pregnancy test prior to enrollment and agree to follow the contraception requirements.
6. Must provide signed consent to participate in the study and is willing to comply with study-specific procedures.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion Criteria

Dose Escalation Phase:

1. 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.
2. Unresolved toxicity (Grade >1 or baseline) from previous anticancer therapy or TGCT therapy, excluding alopecia.
3. Known active central nervous system (CNS) metastases.
4. History or presence of clinically relevant cardiovascular abnormalities.
5. Systemic arterial or venous thrombotic or embolic events.
6. QT interval corrected by Fridericia's formula (QTcF) >450 ms in males or >470 ms in females or history of long QT syndrome.
7. Left ventricular ejection fraction (LVEF) <50%.
8. Concurrent treatment with proton-pump inhibitor(s).
9. Major surgery within 2 weeks of the first dose of study drug.
10. Malabsorption syndrome or other illness that could affect oral absorption.
11. Known human immunodeficiency virus, active hepatitis B, active hepatitis C, or active mycobacterium tuberculosis infection.
12. If female, the patient is pregnant or lactating.
13. Known allergy or hypersensitivity to any component of the study drug.
14. Any other clinically significant comorbidities.

Expansion Phase (Cohorts A and B)

1. Expansion Cohort A: received systemic therapy targeting CSF1 or CSF1R; previous therapy with imatinib and nilotinib is allowed.
2. Expansion Cohort B: discontinued systemic therapy targeting anti-CSF1 or anti-CSF1R due to drug-induced liver injury.
3. 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.
4. Known metastatic TGCT or other active cancer that requires concurrent treatment.
5. QT interval corrected by Fridericia's formula (QTcF) >450 ms in males or >470 ms in females or history of long QT syndrome.
6. Left ventricular ejection fraction (LVEF) <55%.
7. Concurrent treatment with proton-pump inhibitor(s).
8. Major surgery within 2 weeks of the first dose of study drug.
9. Any clinically significant comorbidities
10. Malabsorption syndrome or other illness that could affect oral absorption.
11. Known human immunodeficiency virus (HIV), active or chronic hepatitis B, active or chronic hepatitis C, or active mycobacterium tuberculosis infection.
12. If female, the patient is pregnant or lactating.
13. Known allergy or hypersensitivity to any component of the study drug.
14. Contraindication for MRI
15. Active liver or biliary disease, including evidence of fatty liver, nonalcoholic steatohepatitis (NASH), or cirrhosis

Study design
Purpose of the study
Treatment
Allocation to intervention
NA
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
16/02/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/08/2028
Actual
Sample size
Target
120
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Peter MacCallum Cancer Centre - Melbourne
Recruitment postcode(s) [1] 0 0
- Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
Oregon
Country [7] 0 0
United States of America
State/province [7] 0 0
Tennessee
Country [8] 0 0
Canada
State/province [8] 0 0
Quebec
Country [9] 0 0
Canada
State/province [9] 0 0
Toronto
Country [10] 0 0
France
State/province [10] 0 0
Lyon
Country [11] 0 0
France
State/province [11] 0 0
Paris
Country [12] 0 0
Italy
State/province [12] 0 0
Bologna
Country [13] 0 0
Italy
State/province [13] 0 0
Milan
Country [14] 0 0
Italy
State/province [14] 0 0
Rome
Country [15] 0 0
Netherlands
State/province [15] 0 0
Leiden
Country [16] 0 0
Poland
State/province [16] 0 0
Warsaw
Country [17] 0 0
Spain
State/province [17] 0 0
Barcelona
Country [18] 0 0
Spain
State/province [18] 0 0
Madrid
Country [19] 0 0
Spain
State/province [19] 0 0
Sevilla
Country [20] 0 0
United Kingdom
State/province [20] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Deciphera Pharmaceuticals, LLC
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Maitreyi Sharma, MD
Address 0 0
Deciphera Pharmaceuticals, LLC
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT03069469