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

Tenosynovial Giant Cell Tumor Clinical Trial

— 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04526704
• Other study ID #: PL3397-A-U4003
• Secondary ID: 2020-000192-20
• Status: Completed
• Phase: Phase 4
• Start date: October 20, 2020
• Est. completion date: July 7, 2023

Study information

• Verified date: August 2023
• Source: Daiichi Sankyo, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study is designed to evaluate the discontinuation/re-treatment of pexidartinib therapy in previously treated participants with tenosynovial giant cell tumor (TGCT).

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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 32
• Est. completion date: July 7, 2023
• Est. primary completion date: July 7, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Giant Cell Tumor of Tendon Sheath
• Giant Cell Tumors
• Tenosynovial Giant Cell Tumor

Intervention

Drug:
• 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)

Locations

Country	Name	City	State
Australia	Chris O'Brien Lifehouse	Camperdown	New South Wales
Australia	Peter MacCallum Cancer Centre	East Melbourne	Victoria
Hungary	Magyar Honvedseg Egeszsegugyi Kozpont	Budapest
Italy	Rizzoli-Istituto Ortopedico Rizzoli	Bologna
Italy	Fondazione IRCC Istituto Nazionale dei Tumori	Milano
Netherlands	Leiden University Medical Center (LUMC)	Leiden
Spain	Hospital Sant Pau	Barcelona
Spain	Hospital Virgen del Rocio	Sevilla
Taiwan	National Taiwan University Hospital	Taipei
United States	Dana-Farber Cancer Institute	Boston	Massachusetts
United States	USC Norris Comprehensive Cancer Center	Los Angeles	California
United States	Sylvester Comprehensive Cancer Center	Miami	Florida
United States	Memorial Sloan Kettering Cancer Center	New York	New York
United States	Oregon Health & Science University	Portland	Oregon
United States	Washington University	Saint Louis	Missouri
United States	Honor Health	Scottsdale	Arizona

Sponsors (1)

Lead Sponsor	Collaborator
Daiichi Sankyo, Inc.

Countries where clinical trial is conducted

United States,  Australia,  Hungary,  Italy,  Netherlands,  Spain,  Taiwan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Proportion of Treatment-Free Participants at 12 Months	This is the proportion of participants who remain treatment-free at Month 12.	Baseline up to 12 months after last participant enrolled in Cohort
Primary	Proportion of Treatment-Free Participants at 24 Months	This is the proportion of participants who remain treatment-free at Month 24.	Baseline up to 24 months after last participant enrolled in Cohort
Secondary	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.	Baseline and then assessed every 3 months until end of study, up to approximately 24 months
Secondary	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.	Baseline and then assessed every 3 months until end of study, up to approximately 24 months
Secondary	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.	Baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to approximately 24 months
Secondary	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.	Baseline and assessed every 6 months (treatment continuation) or every 3 months (treatment-free/re-treatment), up to approximately 24 months
Secondary	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.	Start of re-treatment up to 30 days after end of study, up to approximately 24 months