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Study to Evaluate Discontinuation and Re-Treatment in Participants With Tenosynovial Giant Cell Tumor (TGCT) Previously Treated With Pexidartinib (PLX3397)

Primary Purpose

Tenosynovial Giant Cell Tumor

Status
Completed
Phase
Phase 4
Locations
International
Study Type
Interventional
Intervention
Pexidartinib
Sponsored by
Daiichi Sankyo, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Tenosynovial Giant Cell Tumor focused on measuring Tenosynovial Giant Cell Tumor, Pexidartinib, TURALIO™️, PLX3397

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Honor Health
  • USC Norris Comprehensive Cancer Center
  • Sylvester Comprehensive Cancer Center
  • Dana-Farber Cancer Institute
  • Washington University
  • Memorial Sloan Kettering Cancer Center
  • Oregon Health & Science University
  • Chris O'Brien Lifehouse
  • Peter MacCallum Cancer Centre
  • Magyar Honvedseg Egeszsegugyi Kozpont
  • Rizzoli-Istituto Ortopedico Rizzoli
  • Fondazione IRCC Istituto Nazionale dei Tumori
  • Leiden University Medical Center (LUMC)
  • Hospital Sant Pau
  • Hospital Virgen del Rocio
  • National Taiwan University Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Treatment Continuation Cohort

Treatment-Free/Re-Treatment Cohort

Arm Description

Previously-treated participants with TGCT continuing their current dose of pexidartinib treatment.

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).

Outcomes

Primary Outcome Measures

Proportion of Treatment-Free Participants at 12 Months
This is the proportion of participants who remain treatment-free at Month 12.
Proportion of Treatment-Free Participants at 24 Months
This is the proportion of participants who remain treatment-free at Month 24.

Secondary Outcome Measures

Change From Baseline in PROMIS Physical Function Scale during the Treatment-Free Period
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.
Change From Baseline in EQ-5D-5L during the Treatment-Free Period
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.
Number of Participants Who Reported Treatment-Emergent Adverse Events (TEAEs) during the Treatment-free Period
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.
Percentage of Participants Achieving Tumor Response as assessed by Magnetic Resonance Imaging (MRI)
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.
Number of Participants Who Reported TEAEs during the Re-treatment Period
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.

Full Information

First Posted
August 21, 2020
Last Updated
August 7, 2023
Sponsor
Daiichi Sankyo, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04526704
Brief Title
Study to Evaluate Discontinuation and Re-Treatment in Participants With Tenosynovial Giant Cell Tumor (TGCT) Previously Treated With Pexidartinib
Acronym
PLX3397
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

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Completed
Study Start Date
October 20, 2020 (Actual)
Primary Completion Date
July 7, 2023 (Actual)
Study Completion Date
July 7, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Daiichi Sankyo, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Tenosynovial Giant Cell Tumor
Keywords
Tenosynovial Giant Cell Tumor, Pexidartinib, TURALIO™️, PLX3397

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
32 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Treatment Continuation Cohort
Arm Type
Experimental
Arm Description
Previously-treated participants with TGCT continuing their current dose of pexidartinib treatment.
Arm Title
Treatment-Free/Re-Treatment Cohort
Arm Type
Experimental
Arm Description
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).
Intervention Type
Drug
Intervention Name(s)
Pexidartinib
Other Intervention Name(s)
TURALIO™️, PLX3397
Intervention Description
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)
Primary Outcome Measure Information:
Title
Proportion of Treatment-Free Participants at 12 Months
Description
This is the proportion of participants who remain treatment-free at Month 12.
Time Frame
Baseline up to 12 months after last participant enrolled in Cohort
Title
Proportion of Treatment-Free Participants at 24 Months
Description
This is the proportion of participants who remain treatment-free at Month 24.
Time Frame
Baseline up to 24 months after last participant enrolled in Cohort
Secondary Outcome Measure Information:
Title
Change From Baseline in PROMIS Physical Function Scale during the Treatment-Free Period
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.
Time Frame
Baseline and then assessed every 3 months until end of study, up to approximately 24 months
Title
Change From Baseline in EQ-5D-5L during the Treatment-Free Period
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.
Time Frame
Baseline and then assessed every 3 months until end of study, up to approximately 24 months
Title
Number of Participants Who Reported Treatment-Emergent Adverse Events (TEAEs) during the Treatment-free Period
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.
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
Title
Percentage of Participants Achieving Tumor Response as assessed by Magnetic Resonance Imaging (MRI)
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.
Time Frame
Baseline and assessed every 6 months (treatment continuation) or every 3 months (treatment-free/re-treatment), up to approximately 24 months
Title
Number of Participants Who Reported TEAEs during the Re-treatment Period
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.
Time Frame
Start of re-treatment up to 30 days after end of study, up to approximately 24 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Team Leader
Organizational Affiliation
Daiichi Sankyo, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Honor Health
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85258
Country
United States
Facility Name
USC Norris Comprehensive Cancer Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
Facility Name
Sylvester Comprehensive Cancer Center
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Dana-Farber Cancer Institute
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Facility Name
Washington University
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Memorial Sloan Kettering Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Facility Name
Oregon Health & Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Chris O'Brien Lifehouse
City
Camperdown
State/Province
New South Wales
ZIP/Postal Code
2050
Country
Australia
Facility Name
Peter MacCallum Cancer Centre
City
East Melbourne
State/Province
Victoria
ZIP/Postal Code
3000
Country
Australia
Facility Name
Magyar Honvedseg Egeszsegugyi Kozpont
City
Budapest
ZIP/Postal Code
H-1062
Country
Hungary
Facility Name
Rizzoli-Istituto Ortopedico Rizzoli
City
Bologna
ZIP/Postal Code
40136
Country
Italy
Facility Name
Fondazione IRCC Istituto Nazionale dei Tumori
City
Milano
ZIP/Postal Code
20133
Country
Italy
Facility Name
Leiden University Medical Center (LUMC)
City
Leiden
ZIP/Postal Code
2333 ZA
Country
Netherlands
Facility Name
Hospital Sant Pau
City
Barcelona
ZIP/Postal Code
08041
Country
Spain
Facility Name
Hospital Virgen del Rocio
City
Sevilla
ZIP/Postal Code
41013
Country
Spain
Facility Name
National Taiwan University Hospital
City
Taipei
Country
Taiwan

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
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/
IPD Sharing Time Frame
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.
IPD Sharing Access Criteria
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.
IPD Sharing URL
https://vivli.org/ourmember/daiichi-sankyo/

Learn more about this trial

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

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