Description:
This study is designed to evaluate the discontinuation/re-treatment of pexidartinib therapy
in previously treated participants with tenosynovial giant cell tumor (TGCT).
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
Drug | Synonyms | Arms |
---|
Pexidartinib | TURALIO™️, PLX3397 | Treatment 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
Name | Type | Description | Interventions |
---|
Treatment Continuation Cohort | Experimental | Previously-treated participants with TGCT continuing their current dose of pexidartinib treatment. | |
Treatment-Free/Re-Treatment Cohort | Experimental | 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). | |
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