Clinical Trials /

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

NCT04526704

Description:

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

Related Conditions:
  • Tenosynovial Giant Cell Tumor
Recruiting Status:

Recruiting

Phase:

Phase 4

Trial Eligibility

Document

Title

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

Clinical Trial IDs

  • ORG STUDY ID: PL3397-A-U4003
  • SECONDARY ID: 2020-000192-20
  • NCT ID: NCT04526704

Conditions

  • Tenosynovial Giant Cell Tumor

Interventions

DrugSynonymsArms
PexidartinibTURALIO™️, PLX3397Treatment Continuation Cohort

Purpose

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

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).
    

Trial Arms

NameTypeDescriptionInterventions
Treatment Continuation CohortExperimentalPreviously-treated participants with TGCT continuing their current dose of pexidartinib treatment.
  • Pexidartinib
Treatment-Free/Re-Treatment CohortExperimentalPreviously-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).
  • Pexidartinib

Eligibility 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.
      
Maximum Eligible Age:N/A
Minimum Eligible Age:18 Years
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Proportion of Treatment-Free Participants at 12 Months
Time Frame:Baseline up to 12 months after last participant enrolled in Cohort
Safety Issue:
Description:This is the proportion of participants who remain treatment-free at Month 12.

Secondary Outcome Measures

Measure: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
Safety Issue:
Description: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.
Measure: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
Safety Issue:
Description: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.
Measure: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
Safety Issue:
Description: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.
Measure: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
Safety Issue:
Description: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.
Measure: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
Safety Issue:
Description: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.

Details

Phase:Phase 4
Primary Purpose:Interventional
Overall Status:Recruiting
Lead Sponsor:Daiichi Sankyo, Inc.

Trial Keywords

  • Tenosynovial Giant Cell Tumor
  • Pexidartinib
  • TURALIO™️
  • PLX3397

Last Updated

January 13, 2021