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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

Study Description
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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.

Condition or disease Intervention/treatment Phase
Advanced Malignant Neoplasm Pigmented Villonodular Synovitis Giant Cell Tumor of Tendon Sheath Tenosynovial Giant Cell Tumor Tenosynovial Giant Cell Tumor, Diffuse Drug: DCC-3014 Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 120 participants
Allocation: N/A
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : February 16, 2017
Estimated Primary Completion Date : July 2022
Estimated Study Completion Date : June 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
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.

 Drug: DCC-3014
CSF1R inhibitor


Outcome Measures
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Primary Outcome Measures :
  1. 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

  2. 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

  3. 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

  4. 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

  5. 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

  6. 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

  7. 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

  8. 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

  9. 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


Secondary Outcome Measures :
  1. 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

  2. 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

  3. 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)

  4. 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

  5. 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)


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 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
Contacts and Locations
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Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03069469


Contacts
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Contact: Clinical Team 785-830-2100 clinicaltrials@deciphera.com

Locations
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Sponsors and Collaborators
Deciphera Pharmaceuticals LLC
Investigators
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Study Director: Maitreyi Sharma, MD Deciphera Pharmaceuticals LLC
More Information
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Responsible Party: Deciphera Pharmaceuticals LLC
ClinicalTrials.gov Identifier: NCT03069469    
Other Study ID Numbers: DCC-3014-01-001
First Posted: March 3, 2017    Key Record Dates
Last Update Posted: December 22, 2021
Last Verified: December 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Deciphera Pharmaceuticals LLC:
TGCT
DTGCT
PVNS
Additional relevant MeSH terms:
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Neoplasms
Giant Cell Tumors
Giant Cell Tumor of Tendon Sheath
Synovitis, Pigmented Villonodular
Synovitis
Joint Diseases
 Musculoskeletal Diseases
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Tendinopathy
Muscular Diseases