Tenosynovial giant cell tumor (TGCT) is a group of neoplasms including pigmented villonodular synovitis (PVNS) and giant cell tumor of the tendon sheath (GCT-TS) MedDRA version: 20.0 Level: LLT Classification code 10062856 Term: Giant cell tumour of tendon sheath benign System Organ Class: 100000004864
Conditions
Interventions
Product Name: Pexidartinib
Product Code: PLX3397
Pharmaceutical Form: Capsule
INN or Proposed INN: PEXIDARTINIB
CAS Number: 1029044-16-3
Current Sponsor code: PLX3397
Other descriptive name: PLX3397 H
Sponsors
DAIICHI SANKYO, INC.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Subjects must meet all of the following criteria to be eligible for enrollment into the study: 1. Currently enrolled and have not been discontinued from pexidartinib treatment in one of the following studies: Study PLX108-10 (ENLIVEN), Study PLX108-01, Study PL3397-A-A103, or Study PL3397-A-U126. 2. Willing and able to complete the PROMIS (Physical Function Scale) and EQ-5D-5L (European Quality of Life) throughout the study. 3. Willing and able to provide written informed consent form (ICF) prior to any study-related procedures and to comply with all study requirements. 4. 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 End-of-Treatment visit of their prior study) and should be 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. 5. Male subjects 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 subjects 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. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 32 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
Exclusion criteria: Subjects who meet any of the following criteria are NOT eligible for enrollment into the study 1. Subject has a clinically significant abnormality identified by the Investigator at Screening on physical examination, laboratory tests, or electrocardiogram which, in the judgement of the Investigator, would preclude the subject’s safe completion of the study. 2. 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Proportion of subjects who remain treatment-free.;Secondary Objective: - Change from Baseline in Patient Reported Outcomes (PROs) (PROMIS PF, EQ-5D-5L) - Safety: Total number of subjects in the safety analysis set with any AE collected between Screening and start of re-treatment or final database lock (whichever occurs first) - Tumor Assessment: Investigator evaluation of tumor;Primary end point(s): Proportion of subjects who remain treatment-free at Month 12 and Month 24;Timepoint(s) of evaluation of this end point: 24 months after last subject enrolled in the Cohort | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Change from Baseline in Patient Reported Outcomes (PROs): Mean change from Baseline* for PROMIS PF and EQ-5D-5L quarterly for the treatment-free and Re-Treatment periods. *Note: Baseline is Screening values for Treatment Free Period Treatment Continuation Cohort. Baseline is reinitiated with subject entering Re-Treatment period. Safety: Incidence of AEs/TEAEs and SAEs, ECGs, and laboratory assessments Tumor Assessment: Qualitative assessment of the tumor (not applicable to Treatment-Free period);Timepoint(s) of evaluation of this end point: Change from Baseline in Patient Reported Outcomes (PROs): every 3 months Safety: - Incidence of AEs/TEAEs and SAEs: after subject signs the ICF and up to 30 days after the last dose of study medication - ECGs: at screening/baseline and at EOS/EOT/FU (and whenever deemed medically necessary) - laboratory assessments - from baseline, every study visit until EOS/EOT/FU; only for Treatment Free Period: at baseline and then every 6 months until EOS/EOT/FU | — |
Countries
Australia, Hungary, Italy, Netherlands, Spain, Taiwan, United States
Contacts
Public ContactClinical Trial Information Contact
Daiichi Sankyo , Inc.
Outcome results
None listed