Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
A database of clinical trials and their results from Australia, New Zealand, and other countries.
account_circle
Log in
to register or update your trial
search
Search for trials
Trial Review
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 endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT04526704
Registration number
NCT04526704
Ethics application status
Date submitted
21/08/2020
Date registered
26/08/2020
Date last updated
25/03/2025
Titles & IDs
Public title
Study to Evaluate Discontinuation and Re-Treatment in Participants With Tenosynovial Giant Cell Tumor (TGCT) Previously Treated With Pexidartinib
Query!
Scientific title
A Phase 4, Multicenter Study to Evaluate Discontinuation and Re-Treatment in Subjects With Tenosynovial Giant Cell Tumor (TGCT) Previously Treated With Pexidartinib
Query!
Secondary ID [1]
0
0
2020-000192-20
Query!
Secondary ID [2]
0
0
PL3397-A-U4003
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
PLX3397
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Tenosynovial Giant Cell Tumor
0
0
Query!
Condition category
Condition code
Cancer
0
0
0
0
Query!
Bone
Query!
Cancer
0
0
0
0
Query!
Other cancer types
Query!
Musculoskeletal
0
0
0
0
Query!
Other muscular and skeletal disorders
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Drugs - Pexidartinib
Experimental: Treatment Continuation Cohort - Previously-treated participants with TGCT continuing their current dose of pexidartinib treatment.
Experimental: Treatment-Free/Re-Treatment Cohort - Previously-treated participants with TGCT who discontinue pexidartinib treatment (Treatment-Free Period) and had the option to resume pexidartinib treatment at dose at completion of prior study (Re-Treatment Period).
Treatment: Drugs: 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)
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Number of Treatment-Free Participants at 12 Months In The Treatment-free/Re-treatment Cohort
Query!
Assessment method [1]
0
0
Participants who were not remaining treatment-free were defined as either participants who resumed pexidartinib treatment, death (any cause) or who were receiving systemic therapy or undergoing surgery for the treatment of TGCT, whichever occurs first. The number of participants who remained treatment-free at Month 12 is reported.
Query!
Timepoint [1]
0
0
Baseline up to 12 months after last participant enrolled in Cohort
Query!
Primary outcome [2]
0
0
Number of Treatment-Free Participants at 24 Months In The Treatment-free/Re-treatment Cohort
Query!
Assessment method [2]
0
0
Participants who were not remaining treatment-free were defined as either participants who resumed pexidartinib treatment, death (any cause) or who were receiving systemic therapy or undergoing surgery for the treatment of TGCT, whichever occurs first. The number of participants who remained treatment-free at Month 24 is reported.
Query!
Timepoint [2]
0
0
Baseline up to 24 months after last participant enrolled in Cohort
Query!
Secondary outcome [1]
0
0
Change From Baseline in PROMIS Physical Function Total Overall Score In The Treatment Continuation and Treatment-free/Re-Treatment Cohorts
Query!
Assessment method [1]
0
0
The PROMIS Physical Function Total Overall Score (includes 11 upper extremity questions and 13 lower extremity questions) ranges from 24 to 120, with each individual question being rated on a 5-point rating scale (where 1 is unable to do and 5 is without any difficulty). Higher PROMIS Physical Function Total Overall Scores indicate a better health state. The change from baseline in PROMIS Physical Function Total Overall Scores is being reported.
Query!
Timepoint [1]
0
0
Baseline up to Month 24
Query!
Secondary outcome [2]
0
0
Change From Baseline in EQ-5D-5L Scale Score In The Treatment Continuation and Treatment-free/Re-Treatment Cohorts
Query!
Assessment method [2]
0
0
The EQ-5D-5L questionnaire assessed a participant's 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. The change from baseline in EQ-5D-5L Scale Score is being reported.
Query!
Timepoint [2]
0
0
Baseline up to Month 24
Query!
Secondary outcome [3]
0
0
Number of Participants With and Without Progressive Disease
Query!
Assessment method [3]
0
0
Tumors were assessed based on the RECIST criteria. CR was defined as a disappearance of all target lesions, PR was defined as at least a 30% decrease in the sum of diameters of target lesions, and stable disease (SD) was defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease (PD; =30% increase in volume relative to lowest score during the study whether at baseline or some other visit.
Query!
Timepoint [3]
0
0
Month 18 (Re-treatment Period of the Treatment-free/Re-treatment Cohort), Month 24 (Treatment Continuation Cohort)
Query!
Secondary outcome [4]
0
0
Number of Participants Who Reported Treatment-Emergent Adverse Events (TEAEs) In The Treatment Continuation Cohort
Query!
Assessment method [4]
0
0
Treatment-emergent adverse events (TEAEs) were defined as new adverse events (AEs) 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.
Query!
Timepoint [4]
0
0
Baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to 24 months
Query!
Secondary outcome [5]
0
0
Number of Participants Who Reported Treatment-Emergent Adverse Events (TEAEs) In The Re-Treatment Period of the Treatment-free/Re-Treatment Cohort
Query!
Assessment method [5]
0
0
Treatment-emergent adverse events (TEAEs) were defined as new adverse events (AEs) 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.
Query!
Timepoint [5]
0
0
Baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to 24 months
Query!
Secondary outcome [6]
0
0
Number of Patients Reporting AEs (Treatment-free)
Query!
Assessment method [6]
0
0
Adverse events (AEs) were defined as any untoward medical occurrence in a participant who was administered a pharmaceutical product and that does not necessarily have to have a causal relationship with this treatment. Participants who were not remaining treatment-free were defined as either participants who resumed pexidartinib treatment, death (any cause) or who were receiving systemic therapy or undergoing surgery for the treatment of TGCT, whichever occurs first.
Query!
Timepoint [6]
0
0
Baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to 24 months
Query!
Eligibility
Key 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.
Query!
Minimum age
18
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key 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.
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Non-randomised trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Single group
Query!
Other design features
Query!
Phase
Phase 4
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
20/10/2020
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
7/07/2023
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
32
Query!
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Query!
Recruitment hospital [1]
0
0
Chris O'Brien Lifehouse - Camperdown
Query!
Recruitment hospital [2]
0
0
Peter MacCallum Cancer Centre - East Melbourne
Query!
Recruitment postcode(s) [1]
0
0
2050 - Camperdown
Query!
Recruitment postcode(s) [2]
0
0
3000 - East Melbourne
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
Arizona
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
California
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Florida
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Massachusetts
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Missouri
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
New York
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
Oregon
Query!
Country [8]
0
0
Hungary
Query!
State/province [8]
0
0
Budapest
Query!
Country [9]
0
0
Italy
Query!
State/province [9]
0
0
Bologna
Query!
Country [10]
0
0
Italy
Query!
State/province [10]
0
0
Milano
Query!
Country [11]
0
0
Netherlands
Query!
State/province [11]
0
0
Leiden
Query!
Country [12]
0
0
Spain
Query!
State/province [12]
0
0
Barcelona
Query!
Country [13]
0
0
Spain
Query!
State/province [13]
0
0
Sevilla
Query!
Country [14]
0
0
Taiwan
Query!
State/province [14]
0
0
Taipei
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Daiichi Sankyo
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
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).
Query!
Trial website
https://clinicaltrials.gov/study/NCT04526704
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Clinical Team Leader
Query!
Address
0
0
Daiichi Sankyo
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
Query!
What data in particular will be shared?
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 document/s available: Study protocol, Statistical analysis plan (SAP), Clinical study report (CSR)
Query!
When will data be available (start and end dates)?
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.
Query!
Available to whom?
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.
Query!
Available for what types of analyses?
Query!
How or where can data be obtained?
IPD available at link: https://vivli.org/ourmember/daiichi-sankyo/
Query!
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
Study Protocol and Statistical Analysis Plan
https://cdn.clinicaltrials.gov/large-docs/04/NCT04526704/Prot_SAP_000.pdf
Statistical analysis plan
Study Protocol and Statistical Analysis Plan
https://cdn.clinicaltrials.gov/large-docs/04/NCT04526704/Prot_SAP_000.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT04526704
Download to PDF