Phase 3 Study of Pexidartinib for Pigmented Villonodular Synovitis (PVNS) or Giant Cell Tumor of the Tendon Sheath (GCT-TS)

Completed · Phase 3 · Has a placebo group

Conditions studied: Pigmented Villonodular Synovitis, Giant Cell Tumors of the Tendon Sheath, Tenosynovial Giant Cell Tumor

In brief

This is a Phase 3 clinical study, which aims to evaluate the effectiveness of an investigational drug called pexidartinib for the treatment of certain tumors for which surgical removal could cause more harm than good. The main purpose of this study is to gather information about the investigational drug pexidartinib, which may help to treat tumors of pigmented villonodular synovitis (PVNS) or giant cell tumor of the tendon sheath (GCT-TS). The study consists of two parts with a follow-up period. In Part 1, eligible study participants will be assigned to receive either pexidartinib or matching placebo for 24 weeks. A number of assessments will be carried out during the course of the study, including physical examinations, blood tests, imaging studies, electrocardiograms, and questionnaires. MRI scans will be used to evaluate the response of the tumors to the treatment. Some subjects, assigned to placebo in Part 1 transitioned to pexidartinib for Part 2. Then a protocol amendment was written to allow only pexidartinib patients to continue into Part 2. Part 2 is a long-term treatment phase in which all participants receive open-label pexidartinib. There was also a follow-up period added to Part 2.

Key facts

Study ID: NCT02371369
Run by: Daiichi Sankyo
People needed: 120
Starts: 2015-05-11
Expected to finish: 2021-04-30
Last updated by the study team: 2022-05-11

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Age ≥ 18 years.
A diagnosis of PVNS or GCT-TS (i) that has been histologically confirmed either by a pathologist at the treating institution or a central pathologist, and (ii) where surgical resection would be associated with potentially worsening functional limitation or severe morbidity (locally advanced disease), with morbidity determined consensually by qualified personnel (eg, two surgeons or a multi-disciplinary tumor board).
Measurable disease of at least 2 cm and otherwise based on RECIST 1.1, assessed from MRI scans by a central radiologist.
Symptomatic disease because of active PVNS or GCT-TS, defined as one or more of the following:
a worst pain of at least 4 at any time during the week preceding the Screening Visit (based on scale of 0 to 10, with 10 representing "pain as bad as you can imagine").
a worst stiffness of at least 4 at any time during the week preceding the Screening Visit (based on a scale of 0 to 10, with 10 representing "stiffness as bad as you can imagine").
Stable prescription of analgesic regimen during the 2 weeks prior to randomization.
During the 2 weeks prior to randomization, at least 4 of 7 consecutive days of Brief Pain Inventory (BPI) Worst Pain Numeric Rating Scale (NRS) items and Worst Stiffness NRS items completed correctly.
Women of childbearing potential must have a negative serum pregnancy test within the 14-day period prior to randomization. (Where demanded by local regulations, this test may be required within 72 hours of randomization.)
Males and females of childbearing potential are permitted in the study so long as they consent to avoid getting their partner pregnant or becoming pregnant, respectively, by using a highly effective contraception method, as described below, throughout the study and for up to 90 days after completion. Highly effective methods of contraception include: intra-uterine device (non-hormonal or hormonal), bilateral tubal occlusion, vasectomy, sexual abstinence, or barrier methods (eg, condom, diaphragm) used in combination with hormonal methods associated with inhibition of ovulation. Women of non-childbearing potential may be included if they are either surgically sterile or have been postmenopausal for ≥ 1 year. Women who have documentation of at least 12 months of spontaneous amenorrhea and have a follicle stimulating hormone (FSH) level > 40 milli-International units (mIU/mL) will be considered postmenopausal.
Adequate hematologic, hepatic, and renal function, defined by:
Absolute neutrophil count ≥ 1.5 × 10\^9/L
aspartate aminotransferase/alanine (AST/ALT) ≤ 1.5 × upper limit of normal (ULN)
Hemoglobin > 10 g/dL
Total bilirubin ≤ 1.5 × ULN
Platelet count ≥ 100 × 10\^9/L
Serum creatinine ≤ 1.5 × ULN
Willingness and ability to complete the Worst Pain NRS item, Worst Stiffness NRS item, Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Scale, and other self-assessment instruments throughout the study.
Willingness and ability to use an electronic diary.
Willingness and ability to provide written informed consent prior to any study-related procedures and to comply with all study requirements.

You may not qualify if…

Investigational drug use within 28 days of randomization.
Previous use of pexidartinib or any biologic treatment targeting CSF-1 or the CSF-1R; previous use of oral tyrosine kinase inhibitors, eg, imatinib or nilotinib, are allowed.
Active cancer (either concurrent or within the last year of starting study treatment) that requires therapy (eg, surgical, chemotherapy, or radiation therapy), with the exception of adequately treated basal or squamous cell carcinoma of the skin, melanoma in-situ, carcinoma in-situ of the cervix or breast, or prostate carcinoma with a prostate-specific antigen value <0.2 ng/mL.
Known metastatic PVNS/GCT-TS.
Active or chronic infection with hepatitis C virus (HCV) or hepatitis B virus or known active or chronic infection with human immunodeficiency virus.
Known active tuberculosis.
Significant concomitant arthropathy in the affected joint, serious illness, uncontrolled infection, or a medical or psychiatric history that, in the Investigator's opinion, would likely interfere with the person's study participation or the interpretation of his or her results.
Women who are breastfeeding.
A screening Fridericia corrected QT interval (QTcF) ≥ 450 ms (men) or ≥ 470 ms (women).
MRI contraindications.
History of hypersensitivity to any excipients in the investigational product.
Inability to swallow capsules.

Where it is running

Mayo Clinic — Scottsdale, Arizona, United States
University of Southern California — Los Angeles, California, United States
Stanford Cancer Center — Palo Alto, California, United States
UCLA Medical Center — Santa Monica, California, United States
Mayo Clinic Cancer Center — Jacksonville, Florida, United States
Sylvester Comprehensive Cancer Center — Miami, Florida, United States
Moffitt Cancer Center — Tampa, Florida, United States
Massachusetts General Hospital — Boston, Massachusetts, United States
: Dana Farber Cancer Institute — Boston, Massachusetts, United States
Michigan Comprehensive Cancer Center — Ann Arbor, Michigan, United States
Mayo Clinic Cancer Center — Rochester, Minnesota, United States
Washington University School of Medicine — St Louis, Missouri, United States
MD Anderson Cancer Center at Cooper — Camden, New Jersey, United States
Memorial Sloan Kettering Cancer Center — New York, New York, United States
Duke Cancer Center — Durham, North Carolina, United States
OHSU Knight Cancer Institute — Portland, Oregon, United States
Vanderbilt-Ingram Cancer Center — Nashville, Tennessee, United States
Huntsman Cancer Institute — Salt Lake City, Utah, United States
Seattle Cancer Care Alliance — Seattle, Washington, United States
Chris O'Brien Lifehouse — Sydney, New South Wales, Australia
Princess Alexandra Hospital — Woolloongabba, Queensland, Australia
Peter MacCallum Cancer Centre — East Melbourne, Victoria, Australia
Princess Margaret Hospital — Toronto, Ontario, Canada
McGill University Health Centre — Montreal, Quebec, Canada
Herlev Hospital — Herlev, Denmark

Full record on ClinicalTrials.gov

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