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

NCT04526704 | Completed Phase 4 Results FDA Drug

• 2 other identifiers: PL3397-A-U40032020-000192-20
• Study Type: interventional
• Target: 32
• Locations: 7 countries
• Sites: 16

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).

Conditions

Tenosynovial Giant Cell Tumor

Keywords

Tenosynovial Giant Cell Tumor; Pexidartinib; TURALIO™️; PLX3397

Outcome Measures

Primary Outcomes (2)

• Number of Treatment-Free Participants at 12 Months In The Treatment-free/Re-treatment Cohort

  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.

  Baseline up to 12 months after last participant enrolled in Cohort

• Number of Treatment-Free Participants at 24 Months In The Treatment-free/Re-treatment Cohort

  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.

  Baseline up to 24 months after last participant enrolled in Cohort

Secondary Outcomes (6)

• Change From Baseline in PROMIS Physical Function Total Overall Score In The Treatment Continuation and Treatment-free/Re-Treatment Cohorts

  Baseline up to Month 24

• Change From Baseline in EQ-5D-5L Scale Score In The Treatment Continuation and Treatment-free/Re-Treatment Cohorts

  Baseline up to Month 24

• Number of Participants With and Without Progressive Disease

  Month 18 (Re-treatment Period of the Treatment-free/Re-treatment Cohort), Month 24 (Treatment Continuation Cohort)

• Number of Participants Who Reported Treatment-Emergent Adverse Events (TEAEs) In The Treatment Continuation Cohort

  Baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to 24 months

• Number of Participants Who Reported Treatment-Emergent Adverse Events (TEAEs) In The Re-Treatment Period of the Treatment-free/Re-Treatment Cohort

  Baseline up to 30 days after the start of re-treatment or end of study (whichever occurs first), up to 24 months

Study Arms (2)

Treatment Continuation Cohort

EXPERIMENTAL

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

Drug: Pexidartinib

Treatment-Free/Re-Treatment Cohort

EXPERIMENTAL

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).

Drug: Pexidartinib

Interventions

Pexidartinib DRUG

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)

Also known as: TURALIO™️, PLX3397

Treatment Continuation Cohort; Treatment-Free/Re-Treatment Cohort

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Daiichi Sankyo: lead

Study Sites (16)

Honor Health

Scottsdale, Arizona, 85258, United States

Location

USC Norris Comprehensive Cancer Center

Los Angeles, California, 90033, United States

Location

Sylvester Comprehensive Cancer Center

Miami, Florida, 33136, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

Washington University

St Louis, Missouri, 63110, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Chris O'Brien Lifehouse

Camperdown, New South Wales, 2050, Australia

Location

Peter MacCallum Cancer Centre

East Melbourne, Victoria, 3000, Australia

Location

Magyar Honvedseg Egeszsegugyi Kozpont

Budapest, H-1062, Hungary

Location

Rizzoli-Istituto Ortopedico Rizzoli

Bologna, 40136, Italy

Location

Fondazione IRCC Istituto Nazionale dei Tumori

Milan, 20133, Italy

Location

Leiden University Medical Center (LUMC)

Leiden, 2333 ZA, Netherlands

Location

Hospital Sant Pau

Barcelona, 08041, Spain

Location

Hospital Virgen del Rocio

Seville, 41013, Spain

Location

National Taiwan University Hospital

Taipei, Taiwan

Location

MeSH Terms

Conditions

Giant Cell Tumor of Tendon Sheath

Interventions

pexidartinib

Condition Hierarchy (Ancestors)

Giant Cell Tumors; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Synovitis; Joint Diseases; Musculoskeletal Diseases; Tendinopathy; Muscular Diseases

Results Point of Contact

• Title: Contact for Clinical Trial Information
• Organization: Daiichi Sankyo

CTRinfo@dsi.com

908-992-6400

Study Officials

• Clinical Team Leader

  Daiichi Sankyo

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 21, 2020
• First Posted: August 26, 2020
• Study Start: October 20, 2020
• Primary Completion: July 7, 2023
• Study Completion: July 7, 2023
• Last Updated: December 30, 2024
• Results First Posted: December 30, 2024

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• 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.
• 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.

Locations

TW (1); AU (2); HU (1); ES (2); NL (1); US (7); IT (2)