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Study to Evaluate Discontinuation and Re-Treatment in Participants With Tenosynovial Giant Cell Tumor (TGCT) Previously Treated With Pexidartinib (PLX3397)

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04526704
Recruitment Status : Active, not recruiting
First Posted : August 26, 2020
Last Update Posted : May 3, 2022
Sponsor:
Information provided by (Responsible Party):
Daiichi Sankyo, Inc.

Brief Summary:
This study is designed to evaluate the discontinuation/re-treatment of pexidartinib therapy in previously treated participants with tenosynovial giant cell tumor (TGCT).

Condition or disease Intervention/treatment Phase
Tenosynovial Giant Cell Tumor Drug: Pexidartinib Phase 4

Detailed Description:
This multicenter study in previously pexidartinib-treated participants with TGCT will provide the Investigators and participants the option at Screening to either continue pexidartinib treatment (Treatment Continuation Cohort) or discontinue treatment with the possibility of re-initiating pexidartinib treatment (Treatment-Free/Re-Treatment Cohort).

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 32 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 4, Multicenter Study to Evaluate Discontinuation and Re-Treatment in Subjects With Tenosynovial Giant Cell Tumor (TGCT) Previously Treated With Pexidartinib
Actual Study Start Date : October 20, 2020
Estimated Primary Completion Date : September 28, 2023
Estimated Study Completion Date : April 18, 2024

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Treatment Continuation Cohort
Previously-treated participants with TGCT continuing their current dose of pexidartinib treatment.
Drug: Pexidartinib
200 mg capsules administered orally twice daily on an empty stomach (at least 1 hour before or at least 2 hours after a meal or snack)
Other Names:
  • TURALIO™️
  • PLX3397

Experimental: Treatment-Free/Re-Treatment Cohort
Previously-treated participants with TGCT who discontinue pexidartinib treatment (Treatment-Free Period) and resume pexidartinib treatment at dose at completion of prior study (Re-Treatment Period).
Drug: Pexidartinib
200 mg capsules administered orally twice daily on an empty stomach (at least 1 hour before or at least 2 hours after a meal or snack)
Other Names:
  • TURALIO™️
  • PLX3397




Primary Outcome Measures :
  1. Proportion of Treatment-Free Participants at 12 Months [ Time Frame: Baseline up to 12 months after last participant enrolled in Cohort ]
    This is the proportion of participants who remain treatment-free at Month 12.

  2. Proportion of Treatment-Free Participants at 24 Months [ Time Frame: Baseline up to 24 months after last participant enrolled in Cohort ]
    This is the proportion of participants who remain treatment-free at Month 24.


Secondary Outcome Measures :
  1. Change From Baseline in PROMIS Physical Function Scale during the Treatment-Free Period [ Time Frame: Baseline and then assessed every 3 months until end of study, up to approximately 24 months ]
    The PROMIS Physical Function Scale is based on a 5-point rating scale, where 1 is unable to do and 5 is without any difficulty.

  2. Change From Baseline in EQ-5D-5L during the Treatment-Free Period [ Time Frame: Baseline and then assessed every 3 months until end of study, up to approximately 24 months ]
    The EQ-5D-5L questionnaire will ask the participant to describe their health in the areas of mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. The overall health is rated on a scale from 0 to 100, where 0 is worst health you can imagine and 100 is best health you can imagine.

  3. Number of Participants Who Reported Treatment-Emergent Adverse Events (TEAEs) during the Treatment-free Period [ Time Frame: Baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to approximately 24 months ]
    TEAEs are defined as new Adverse Events or pre-existing conditions that worsen in CTCAE grade after the first dose of study drug and up to 30 days after last dose of study drug.

  4. Percentage of Participants Achieving Tumor Response as assessed by Magnetic Resonance Imaging (MRI) [ Time Frame: Baseline and assessed every 6 months (treatment continuation) or every 3 months (treatment-free/re-treatment), up to approximately 24 months ]
    Individual participant outcomes by tumor volume score (TVS) will be classified according to the following criteria via RECIST: Complete response (CR), Lesion completely gone; Partial response (PR), ≥50% decrease in volume score relative to baseline; Progressive disease (PD), ≥30% increase in volume relative to lowest score during the study whether at baseline or some other visit; Stable disease (SD), Does not meet any of the prior criteria based on score during study.

  5. Number of Participants Who Reported TEAEs during the Re-treatment Period [ Time Frame: Start of re-treatment up to 30 days after end of study, up to approximately 24 months ]
    TEAEs are defined as new Adverse Events or pre-existing conditions that worsen in CTCAE grade after the first dose of study drug and up to 30 days after last dose of study drug.



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:

  • Currently enrolled and on pexidartinib treatment in one of the following studies: Study PLX108-10 (ENLIVEN), Study PLX108-01, Study PL3397-A-A103 or Study PL3397-A-U126.
  • Willing and able to complete the PROMIS Physical Function Scale and EQ-5D-5L throughout the study.
  • Willing and able to provide written informed consent prior to any study-related procedures and to comply with all study requirements.
  • Females of reproductive potential must have a negative urine pregnancy test at Screening/Baseline (to be confirmed by a serum pregnancy test taken on the last treatment visit of their prior study). They are advised to use an effective, non-hormonal method of contraception during treatment with pexidartinib and for 1 month after the last dose. Males with female partners of reproductive potential should be advised to use an effective method of contraception during treatment with pexidartinib and for 1 month after the last dose. Female partners of male patients should concurrently use effective contraceptive methods (hormonal or non-hormonal).

Note: A female is considered of reproductive potential following menarche and until becoming postmenopausal (no menstrual period for a minimum of 12 months) unless permanently sterile (undergone a hysterectomy, bilateral salpingectomy or bilateral oophorectomy) with a confirmed by follicle stimulating hormone (FSH) test level >40 mIU/mL.

  • Male participants must not freeze or donate sperm starting at Screening and throughout the study period, and for at least 5 half-lives or 1 month after the final study drug administration, whichever is longer. Female participants must not donate, or retrieve for their own use, ova from the time of Screening and throughout the study treatment period, and for at least 1 month or 5 half-lives after the final study drug administration, whichever is longer.

Exclusion Criteria:

  • Participant has a clinically significant abnormality identified by the Investigator at Screening on physical examination, laboratory tests, or electrocardiogram (ECG) which, in the judgement of the Investigator, would preclude the participant's safe completion of the study.
  • Exposure to another investigational drug or current participation in other therapeutic investigational procedures, besides pexidartinib studies, within 1 month prior to start of study treatment. Any known contraindication to treatment with, including hypersensitivity to, the study drug(s) or excipients in pexidartinib.

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): NCT04526704


Locations
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United States, Arizona
Honor Health
Scottsdale, Arizona, United States, 85258
United States, California
USC Norris Comprehensive Cancer Center
Los Angeles, California, United States, 90033
United States, Florida
Sylvester Comprehensive Cancer Center
Miami, Florida, United States, 33136
United States, Massachusetts
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02215
United States, Missouri
Washington University
Saint Louis, Missouri, United States, 63110
United States, New York
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10065
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239
Australia, New South Wales
Chris O'Brien Lifehouse
Camperdown, New South Wales, Australia, 2050
Australia, Victoria
Peter MacCallum Cancer Centre
East Melbourne, Victoria, Australia, 3000
Hungary
Magyar Honvedseg Egeszsegugyi Kozpont
Budapest, Hungary, H-1062
Italy
Rizzoli-Istituto Ortopedico Rizzoli
Bologna, Italy, 40136
Fondazione IRCC Istituto Nazionale dei Tumori
Milano, Italy, 20133
Netherlands
Leiden University Medical Center (LUMC)
Leiden, Netherlands, 2333 ZA
Spain
Hospital Sant Pau
Barcelona, Spain, 08041
Hospital Virgen del Rocio
Sevilla, Spain, 41013
Taiwan
National Taiwan University Hospital
Taipei, Taiwan
Sponsors and Collaborators
Daiichi Sankyo, Inc.
Investigators
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Study Director: Clinical Team Leader Daiichi Sankyo, Inc.
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Responsible Party: Daiichi Sankyo, Inc.
ClinicalTrials.gov Identifier: NCT04526704    
Other Study ID Numbers: PL3397-A-U4003
2020-000192-20 ( EudraCT Number )
First Posted: August 26, 2020    Key Record Dates
Last Update Posted: May 3, 2022
Last Verified: April 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: De-identified individual participant data (IPD) and applicable supporting clinical trial documents may be available upon request at https://vivli.org/. In cases where clinical trial data and supporting documents are provided pursuant to our company policies and procedures, Daiichi Sankyo will continue to protect the privacy of our clinical trial participants. Details on data sharing criteria and the procedure for requesting access can be found at this web address: https://vivli.org/ourmember/daiichi-sankyo/
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Clinical Study Report (CSR)
Time Frame: Studies for which the medicine and indication have received European Union (EU) and United States (US), and/or Japan (JP) marketing approval on or after 01 January 2014 or by the US or EU or JP Health Authorities when regulatory submissions in all regions are not planned and after the primary study results have been accepted for publication.
Access Criteria: Formal request from qualified scientific and medical researchers on IPD and clinical study documents from clinical trials supporting products submitted and licensed in the United States, the European Union and/or Japan from 01 January 2014 and beyond for the purpose of conducting legitimate research. This must be consistent with the principle of safeguarding study participants' privacy and consistent with provision of informed consent.
URL: https://vivli.org/ourmember/daiichi-sankyo/

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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 Daiichi Sankyo, Inc.:
Tenosynovial Giant Cell Tumor
Pexidartinib
TURALIO™️
PLX3397
Additional relevant MeSH terms:
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Giant Cell Tumors
Giant Cell Tumor of Tendon Sheath
Neoplasms
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Synovitis
Joint Diseases
Musculoskeletal Diseases
Tendinopathy
Muscular Diseases