Clinical trial NCT04526704: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Tenosynovial Giant Cell Tumor
Keywords: Tenosynovial Giant Cell Tumor
Pexidartinib
TURALIO™️
PLX3397
Lead sponsor: Daiichi Sankyo
Sponsor class: INDUSTRY
First posted: 2020-08-26
Enrollment: 32
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).

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).

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.

Primary outcomes:
measure: Number of Treatment-Free Participants at 12 Months In The Treatment-free/Re-treatment Cohort
timeFrame: Baseline up to 12 months after last participant enrolled in Cohort
measure: Number of Treatment-Free Participants at 24 Months In The Treatment-free/Re-treatment Cohort
timeFrame: Baseline up to 24 months after last participant enrolled in Cohort
Secondary outcomes:
measure: Change From Baseline in PROMIS Physical Function Total Overall Score In The Treatment Continuation and Treatment-free/Re-Treatment Cohorts
timeFrame: Baseline up to Month 24
measure: Change From Baseline in EQ-5D-5L Scale Score In The Treatment Continuation and Treatment-free/Re-Treatment Cohorts
timeFrame: Baseline up to Month 24
measure: Number of Participants With and Without Progressive Disease
timeFrame: Month 18 (Re-treatment Period of the Treatment-free/Re-treatment Cohort), Month 24 (Treatment Continuation Cohort)
measure: Number of Participants Who Reported Treatment-Emergent Adverse Events (TEAEs) In The Treatment Continuation Cohort
timeFrame: Baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to 24 months
measure: Number of Participants Who Reported Treatment-Emergent Adverse Events (TEAEs) In The Re-Treatment Period of the Treatment-free/Re-Treatment Cohort
timeFrame: Baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to 24 months
measure: Number of Patients Reporting AEs (Treatment-free)
timeFrame: Baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to 24 months
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Honor Health
state: Arizona
city: Los Angeles
country: United States
facility: USC Norris Comprehensive Cancer Center
state: California
city: Miami
country: United States
facility: Sylvester Comprehensive Cancer Center
state: Florida
city: Boston
country: United States
facility: Dana-Farber Cancer Institute
state: Massachusetts
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
city: New York
country: United States
facility: Memorial Sloan Kettering Cancer Center
state: New York
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Camperdown
country: Australia
facility: Chris O'Brien Lifehouse
state: New South Wales
city: East Melbourne
country: Australia
facility: Peter MacCallum Cancer Centre
state: Victoria
city: Budapest
country: Hungary
facility: Magyar Honvedseg Egeszsegugyi Kozpont
city: Bologna
country: Italy
facility: Rizzoli-Istituto Ortopedico Rizzoli
city: Milano
country: Italy
facility: Fondazione IRCC Istituto Nazionale dei Tumori
city: Leiden
country: Netherlands
facility: Leiden University Medical Center (LUMC)
city: Barcelona
country: Spain
facility: Hospital Sant Pau
city: Sevilla
country: Spain
facility: Hospital Virgen del Rocio
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
Study officials:
affiliation: Daiichi Sankyo
name: Clinical Team Leader
role: STUDY_DIRECTOR
References: