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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03069469
Recruitment Status : Recruiting
First Posted : March 3, 2017
Last Update Posted : December 22, 2021
Sponsor:
Information provided by (Responsible Party):
Deciphera Pharmaceuticals LLC

Tracking Information
First Submitted Date  ICMJE February 20, 2017
First Posted Date  ICMJE March 3, 2017
Last Update Posted Date December 22, 2021
Actual Study Start Date  ICMJE February 16, 2017
Estimated Primary Completion Date July 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 1, 2020)
  • Maximum tolerated dose [ Time Frame: Day 1 - Day 28 of Cycle 1 for each dose level tested ]
    Identify the dose limiting toxicities for each dose level tested and determine the maximum tolerated dose and recommended Phase 2 dose
  • Incidence of Adverse Events [ Time Frame: Cycle 1 through study completion (~ 24 months) ]
    Identify the observed adverse events, serious adverse events associated with DCC-3014
  • Time to maximum observed concentration of DCC-3014 [ 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 DCC-3014 in patients
  • Maximum observed concentration of DCC-3014 [ 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 DCC-3014 in patients
  • Trough observed concentration of DCC-3014 [ 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 DCC-3014 in patients
  • Area under the concentration-time curve of DCC-3014 [ 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 DCC-3014
  • Half life of DCC-3014 [ 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 DCC-3014 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: Baseline through 24 months ]
    Measure time from PR or CR to disease progression or death
Original Primary Outcome Measures  ICMJE
 (submitted: February 27, 2017)
  • Maximum tolerated dose [ Time Frame: Day 1 - Day 28 of Cycle 1 for each dose level tested ]
    Identify the dose limiting toxicities for each dose level tested and determine the maximum tolerated dose and recommended phase 2 dose
  • Incidence of Adverse Events [ Time Frame: Day -7 pre-Cycle 1 through study completion (~ 18 months) ]
    Identify the observed adverse events, serious adverse events associated with DCC-3014
  • Time to observed concentration of DCC-3014 [ Time Frame: Cycle 1 Day 1 and Cycle 2 Day 2 through study completion (~18 months) at the following time points: 0.5, 1, 2, 4, 6, 8, 10, 24 hours post-dose ]
    Measure the concentration of DCC-3014 in patients
  • Maximum observed concentration of DCC-3014 [ Time Frame: Cycle 1 Day 1 and Cycle 2 Day 2 through study completion (~ 18 months) at the following time points: 0.5, 1, 2, 4, 6, 8, 10, 24 hours post-dose ]
    Measure the observed concentration of DCC-3014 in patients
  • Trough observed concentration of DCC-3014 [ Time Frame: Cycle 1 Day 1 and Cycle 2 Day 2 through study completion (~18 months) at the following time points: 0.5, 1, 2, 4, 6, 8, 10, 24 hours post-dose ]
    Measure the concentration of DCC-3014 in patients
  • Area under the concentration-time curve of DCC-3014 [ Time Frame: Cycle 1 Day 1 and Cycle 2 Day 2 through study completion (~18 months) at the following time points: 0.5, 1, 2, 4, 6, 8, 10, 24 hours post-dose ]
    Measure the AUC of DCC-3014
  • Half life of DCC-3014 [ Time Frame: Cycle 1 Day 1 and Cycle 2 Day 2 through study completion (~18 months) at the following time points: 0.5, 1, 2, 4, 6, 8, 10, 24 hours post-dose ]
    Measure the half life of DCC-3014 in patients
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 1, 2020)
  • Response rate (Expansion Phase only) [ Time Frame: At Week 25 (Cycle 7, Day 1) ]
    Assessed by central read using tumor volume score and modified RECIST (mRECIST) Version 1.1
  • Range of Motion (ROM) (Expansion Phase only) [ Time Frame: Baseline to Week 25 (Cycle 7, Day 1) ]
    Measure mean change from baseline in relative ROM
  • Brief Pain Inventory (BPI) Worst Pain Numeric Rating Scale (NRS) Score (Expansion Phase only) [ Time Frame: Baseline to Week 25 (Cycle 7, Day 1) ]
    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)
  • Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Score (Expansion Phase only) [ Time Frame: Baseline to Week 25 (Cycle 7, Day 1) ]
    Analysis of patient reported outcomes based upon the patient-reported outcomes measurement information system (PROMIS) physical function questionnaire
  • Worst Stiffness Numeric Rating Scale (NRS) Score (Expansion Phase only) [ Time Frame: Baseline to Week 25 (Cycle 7, Day 1) ]
    Analysis of patient reported outcomes based upon the Worst Stiffness Numeric Rating Scale (NRS)
Original Secondary Outcome Measures  ICMJE
 (submitted: February 27, 2017)
Levels of CSF1R-dependent myeloid cells [ Time Frame: Day 0 Cycle 1 through study completion (~18 months) ]
Measure the amount of CSF1R-dependent myeloid cells in patients
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of DCC-3014 in Patients With Advanced Tumors and Tenosynovial Giant Cell Tumor
Official Title  ICMJE 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
Brief Summary This is a multicenter, open-label Phase 1/2 study of DCC-3014 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Advanced Malignant Neoplasm
  • Pigmented Villonodular Synovitis
  • Giant Cell Tumor of Tendon Sheath
  • Tenosynovial Giant Cell Tumor
  • Tenosynovial Giant Cell Tumor, Diffuse
Intervention  ICMJE Drug: DCC-3014
CSF1R inhibitor
Study Arms  ICMJE Experimental Treatment

Dose Escalation Phase: Increasing doses of DCC-3014 beginning at 10 mg 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.

Intervention: Drug: DCC-3014
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: May 1, 2020)
120
Original Estimated Enrollment  ICMJE
 (submitted: February 27, 2017)
55
Estimated Study Completion Date  ICMJE June 2024
Estimated Primary Completion Date July 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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 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.

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 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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Clinical Team 785-830-2100 clinicaltrials@deciphera.com
Listed Location Countries  ICMJE Australia,   Canada,   France,   Italy,   Netherlands,   Poland,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03069469
Other Study ID Numbers  ICMJE DCC-3014-01-001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party Deciphera Pharmaceuticals LLC
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Deciphera Pharmaceuticals LLC
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Maitreyi Sharma, MD Deciphera Pharmaceuticals LLC
PRS Account Deciphera Pharmaceuticals LLC
Verification Date December 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP