A Phase 3 interventional study of Pexidartinib and Placebo in Pigmented Villonodular Synovitis, Giant Cell Tumors of the Tendon Sheath and Tenosynovial Giant Cell Tumor, sponsored by Daiichi Sankyo. Completed at 39 sites in 12 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-05-11.
Sponsored by Daiichi Sankyo · Phase 3, Interventional, and Treatment
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.
20 studies on the registry are indexed under Synovitis, Pigmented Villonodular; 2 are open to participants now.
This study's enrollment of 120 is above the median of 36 across 17 interventional studies indexed under Synovitis, Pigmented Villonodular.
Browse Synovitis, Pigmented Villonodular studies →Daiichi Sankyo is the lead sponsor of 322 studies on the registry; 37 are open to participants now.
Of its 51 completed or terminated interventional studies of FDA-regulated products, 38 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Symptomatic disease because of active PVNS or GCT-TS, defined as one or more of the following:
Adequate hematologic, hepatic, and renal function, defined by:
Exclusion Criteria
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
The standard rules for measuring tumours on CT or MRI scans, to tell whether a cancer is shrinking, stable or growing during treatment.
“Measurable disease per RECIST 1.1” usually means at least one tumour 10 mm or more at its longest on a CT scan, or a lymph node 15 mm or more across its short axis.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Histologically or cytologically confirmed
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Measurable disease
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
RECIST
The standard rules for measuring tumours on CT or MRI scans, to tell whether a cancer is shrinking, stable or growing during treatment.
“Measurable disease per RECIST 1.1” usually means at least one tumour 10 mm or more at its longest on a CT scan, or a lymph node 15 mm or more across its short axis.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Postmenopausal
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants received blinded treatment of pexidartinib,1000 mg (5 capsules per day ) for 2 weeks, then 800 mg (4 capsules per day) for 22 weeks
Drug: Pexidartinib
Participants received blinded treatment of matching placebo (5 capsules per day) for 2 weeks, then matching placebo (4 capsules per day) for 22 weeks
Drug: Placebo
Participants received pexidartinib in Part 1 and in Part 2 at their prescribed dose
Drug: Pexidartinib
Participants received placebo in Part 1 and pexidartinib in Part 2 at their prescribed dose
Drug: Pexidartinib · Drug: Placebo
Each capsule contains 200 mg of pexidartinib for oral administration
Also known as: PLX3397, Pexidartinib hydrochloride (HCl)
Placebo capsule matching pexidartinib capsule for oral administration
Also known as: Placebo Capsule
Percentage of Participants With Symptomatic, Locally Advanced Tenosynovial Giant Cell Tumor (TGCT) Achieving Complete or Partial Response to Pexidartinib Compared With That of Placebo Per Response Evaluation Criteria in Solid Tumors Version 1.1 at Week 25
Complete response (CR) and partial responses (PR) were assessed based on centrally-read magnetic resonance imaging (MRI) scans and Response Evaluation Criteria in Solid Tumors, version 1.1 (RECIST 1.1). A CR was defined as disappearance of all tumors and a PR was defined as at least a 30% decrease in the sum of diameters of target tumors using the baseline sum diameters as the reference.
Time frame: Week 25
Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25
Range of motion (ROM) of the joint was assessed by a qualified, independent, and blinded or third-party assessors at the clinical site. Measurements were recorded in degrees. At baseline, the plane of movement with the smallest relative value (worst) was identified and this plane was used for evaluating the relative change of motion subsequently. Only the plane with the worst impaired ROM at baseline was selected for subsequent analyses.
Time frame: Baseline, Week 13, and Week 25
Percentage of Participants With Symptomatic, Locally Advanced Tenosynovial Giant Cell Tumor (TGCT) Achieving Complete or Partial Response Based on Tumor Volume Score (TVS) After Receiving Pexidartinib Compared With Those on Placebo at Week 25
Complete response (CR) and partial response (PR) were assessed using tumor volume score (TVS). A CR was defined as disappearance of all tumors and a PR was defined as at least a 30% decrease in the sum of diameters of target tumors using the baseline sum diameters as the reference. TVS is a semi-quantitative MRI scoring system that describes tumor mass and is based on 10% increments of the estimated volume of the maximally distended synovial cavity or tendon sheath involved. A tumor that is equal in volume to that of a maximally distended synovial cavity or tendon sheath was scored 10; a score of 0 indicated no evidence of tumor.
Time frame: Week 25
Mean Change From Baseline in the Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Score in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25
The Patient-reported Outcomes Measurement Information System (PROMIS) physical function scale was used to assess physical function of the upper and lower limbs. The scale ranged from 1 defined as 'unable to do' or 'cannot do' to 5 defined as 'without any difficulty' or 'not at all', where higher scores represent better outcomes.
Time frame: at Week 9 , Week 17, and Week 25
Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo Up to Week 25
The Worst Stiffness Numeric Rating Scale (NRS) was a 1-item, self-administered questionnaire assessing the "worst" stiffness in the last 24 hours. The NRS for this item ranged from 0 (no stiffness) to 10 (stiffness as bad as you can imagine).
Time frame: Baseline, Week 9, Week 17, and Week 25
Percentage of Participants Who Responded With a Decrease of at Least 30% in the Mean Brief Pain Inventory Worst Pain Numeric Rating Scale Score Among Participants Receiving Pexidartinib Compared With Those on Placebo at Week 25
The Brief Pain Inventory (BPI) Worst Pain Numeric Rating Scale Score (NRS) was a 1-item, self-administered questionnaire assessing the "worst" pain in the last 24 hours. The NRS for this item ranged from 0 (no pain) to 10 (pain as bad as you can imagine).
Time frame: Week 25
Number of Responders to Pexidartinib With and Without Disease Progression
Duration of response (DOR) based on RECIST 1.1 is defined as the date of the first recorded response to the first date of documented disease progression. The overall number of responses and the number of participants with and without disease progression was assessed.
Time frame: By Week 96
Number of Responders to Pexidartinib With and Without Disease Progression Based on Tumor Volume Score
Tumor Volume Score (TVS) is a semi-quantitative MRI scoring system that describes tumor mass and is based on 10% increments of the estimated volume of the maximally distended synovial cavity or tendon sheath involved. A tumor that is equal in volume to that of a maximally distended synovial cavity or tendon sheath was scored 10; a score of 0 indicated no evidence of tumor. The overall number of responses and the number of participants with and without disease progression was assessed.
Time frame: By Week 120
Duration of Response (DOR) Based on RECIST 1.1
Duration of response (DOR) based on RECIST 1.1 is defined from the date of the first recorded evidence of response to the first date of documented disease progression.
Time frame: Date of first documentation of objective response up to date of first documentation of progressive disease, assessed up to end of study (approximately 71 months)
Duration of Response (DOR) Based on Tumor Volume Score (TVS)
Duration of response (DOR) based on TVS is defined from the date of the first recorded evidence of response to the first date of documented disease progression.
Time frame: Date of first documentation of objective response up to date of first documentation of progressive disease, assessed up to end of study (approximately 71 months)
Percentage of Participants Reporting Frequent (≥10%) Treatment-Emergent Adverse Events by Preferred Term
Treatment-emergent adverse events (TEAEs) were defined as adverse events that started or worsened after the first dose of treatment and within 28 days after the last dose. The National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0 was used to grade adverse events. Any Grade and Grade ≥3 (severe) TEAEs are reported. TEAEs were coded using MedDRA version 17.1.
Time frame: After the first dose of treatment up to 28 days after the last dose
Percentage of Participants With Symptomatic, Locally Advanced Tenosynovial Giant Cell Tumor (TGCT) Achieving Complete or Partial Response to Pexidartinib Compared With That of Placebo Per Response Evaluation Criteria in Solid Tumors Version 1.1 by Week 49
Complete (CR) and partial responses (PR) were assessed based on centrally-read magnetic resonance imaging (MRI) scans and Response Evaluation Criteria in Solid Tumors, version 1.1 (RECIST 1.1). A CR was defined as disappearance of all tumors and a PR was defined as at least a 30% decrease in the sum of diameters of target tumors using the baseline sum diameters as the reference.
Time frame: By Week 49
Mean Change From Baseline For Range of Motion (ROM) Score in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49
Range of motion (ROM) of the joint was assessed by a qualified, independent, and blinded or third-party assessors at the clinical site. Measurements were recorded in degrees. At baseline, the plane of movement with the smallest relative value (worst) was identified and this plane was used for evaluating the relative change of motion subsequently. Only the plane with the worst impaired ROM at baseline was selected for subsequent analyses.
Time frame: By Week 49
Mean Change From Baseline in the Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function Score in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49
The Patient-reported Outcomes Measurement Information System (PROMIS) physical function scale was used to assess physical function of the upper and lower limbs. The scale ranged from 1 defined as 'unable to do' or 'cannot do' to 5 defined as 'without any difficulty' or 'not at all', where higher scores represent better outcomes.
Time frame: By Week 49
Mean Change From Baseline for Worst Stiffness Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49
The Worst Stiffness Numeric Rating Scale (NRS) was a 1-item, self-administered questionnaire assessing the "worst" stiffness in the last 24 hours. The NRS for this item ranged from 0 (no stiffness) to 10 (stiffness as bad as you can imagine).
Time frame: Baseline, Week 25, and Week 49
Mean Change From Baseline for Worst Pain Numeric Rating Scale Score (NRS) in Participants Receiving Pexidartinib Compared With Those on Placebo by Week 49
The Brief Pain Inventory (BPI) Worst Pain NRS was a 1-item, self-administered questionnaire assessing the "worst" pain in the last 24 hours. The NRS for this item ranged from 0 (no pain) to 10 (pain as bad as you can imagine).
Time frame: By Week 49
Percentage of Participants With Symptomatic, Locally Advanced Tenosynovial Giant Cell Tumor (TGCT) Achieving Complete or Partial Response Based on Tumor Volume Score (TVS) After Receiving Pexidartinib Compared With Those on Placebo by Week 49
Best overall response (CR or PR) was assessed using tumor volume score (TVS) in the ITT population. Tumor Volume Score is a semi-quantitative MRI scoring system that describes tumor mass and is based on 10% increments of the estimated volume of the maximally distended synovial cavity or tendon sheath involved. A tumor that is equal in volume to that of a maximally distended synovial cavity or tendon sheath was scored 10; a score of 0 indicated no evidence of tumor.
Time frame: By Week 49
Part 1 was a double-blind, randomized, Pexidartinib or placebo in participants with symptomatic TGCT for whom surgical resection would be associated with potentially worsening functional limitation or severe morbidity. Part 2 is a long-term treatment phase in which all eligible participants received open-label Pexidartinib.
| Milestone | Pexidartinib Part 1, Then Pexidartinib Part 2 | Placebo Part 1, Then Pexidartinib Part 2 |
|---|---|---|
| Started | 61 | 59 |
| Completed | 52 | 48 |
| Not completed | 9 | 11 |
| Withdrew: Physician decision | 0 | 3 |
| Withdrew: Adverse event | 8 | 0 |
| Withdrew: Withdrawal by subject | 1 | 6 |
| Withdrew: Disease progression | 0 | 1 |
| Withdrew: Protocol violation | 0 | 1 |
| Milestone | Pexidartinib Part 1, Then Pexidartinib Part 2 | Placebo Part 1, Then Pexidartinib Part 2 |
|---|---|---|
| Started | 48 | 30 |
| Completed | 0 | 0 |
| Not completed | 48 | 30 |
| Withdrew: Adverse event | 6 | 5 |
| Withdrew: Withdrawal by subject | 16 | 6 |
| Withdrew: Investigator decision | 1 | 2 |
| Withdrew: Death | 0 | 1 |
| Withdrew: Disease progression | 1 | 0 |
| Withdrew: Subject noncompliance | 1 | 0 |
| Withdrew: Lost to follow-up | 1 | 1 |
| Withdrew: Subject transitioned to commercial supply | 6 | 4 |
| Withdrew: Subject transitioned to another ds pexidartinib protocol | 15 | 9 |
| Withdrew: Surgical resection of tumor | 1 | 1 |
| Withdrew: Other | 0 | 1 |
Complete response (CR) and partial responses (PR) were assessed based on centrally-read magnetic resonance imaging (MRI) scans and Response Evaluation Criteria in Solid Tumors, version 1.1 (RECIST 1.1). A CR was defined as disappearance of all tumors and a PR was defined as at least a 30% decrease in the sum of diameters of target tumors using the baseline sum diameters as the reference.
| Percentage of participants | Pexidartinib Part 1 | Placebo Part 1 |
|---|---|---|
| Complete Response (CR) | 14.8 | 0 |
| Partial Response (PR) | 24.6 | 0 |
| Response (CR or PR) | 39.3 | 0 |
Range of motion (ROM) of the joint was assessed by a qualified, independent, and blinded or third-party assessors at the clinical site. Measurements were recorded in degrees. At baseline, the plane of movement with the smallest relative value (worst) was identified and this plane was used for evaluating the relative change of motion subsequently. Only the plane with the worst impaired ROM at baseline was selected for subsequent analyses.
| degrees | Pexidartinib Part 1 | Placebo Part 1 |
|---|---|---|
| Baseline | 62.5 ± 3.2 | 62.9 ± 2.9 |
| Week 13 | 13.0 ± 2.3 | 4.8 ± 2.6 |
| Week 25 | 15.1 ± 2.1 | 6.2 ± 2.4 |
Complete response (CR) and partial response (PR) were assessed using tumor volume score (TVS). A CR was defined as disappearance of all tumors and a PR was defined as at least a 30% decrease in the sum of diameters of target tumors using the baseline sum diameters as the reference. TVS is a semi-quantitative MRI scoring system that describes tumor mass and is based on 10% increments of the estimated volume of the maximally distended synovial cavity or tendon sheath involved. A tumor that is equal in volume to that of a maximally distended synovial cavity or tendon sheath was scored 10; a score of 0 indicated no evidence of tumor.
| Percentage of participants | Pexidartinib Part 1 | Placebo Part 1 |
|---|---|---|
| Complete Response (CR) | 4.9 | 0 |
| Partial Response (PR) | 50.8 | 0 |
| Response (CR or PR) | 55.7 | 0 |
The Patient-reported Outcomes Measurement Information System (PROMIS) physical function scale was used to assess physical function of the upper and lower limbs. The scale ranged from 1 defined as 'unable to do' or 'cannot do' to 5 defined as 'without any difficulty' or 'not at all', where higher scores represent better outcomes.
| units on a scale | Pexidartinib Part 1 | Placebo Part 1 |
|---|---|---|
| Week 9 | 2.8 ± 1.0 | -0.4 ± 0.8 |
| Week 17 | 3.2 ± 1.1 | 0.2 ± 1.0 |
| Week 25 | 4.1 ± 1.1 | -0.9 ± 1.0 |
The Worst Stiffness Numeric Rating Scale (NRS) was a 1-item, self-administered questionnaire assessing the "worst" stiffness in the last 24 hours. The NRS for this item ranged from 0 (no stiffness) to 10 (stiffness as bad as you can imagine).
| units on a scale | Pexidartinib Part 1 | Placebo Part 1 |
|---|---|---|
| Baseline | 5.6 ± 0.2 | 5.9 ± 0.3 |
| Week 9 | -1.5 ± 0.3 | -0.5 ± 0.3 |
| Week 17 | -2.4 ± 0.3 | -0.4 ± 0.3 |
| Week 25 | -2.5 ± 0.3 | -0.3 ± 0.3 |
The Brief Pain Inventory (BPI) Worst Pain Numeric Rating Scale Score (NRS) was a 1-item, self-administered questionnaire assessing the "worst" pain in the last 24 hours. The NRS for this item ranged from 0 (no pain) to 10 (pain as bad as you can imagine).
| percentage of participants | Pexidartinib Part 1 | Placebo Part 1 |
|---|---|---|
| Percentage of Participants Who Responded With a Decrease of at Least 30% in the Mean Brief Pain Inventory Worst Pain Numeric Rating Scale Score Among Participants Receiving Pexidartinib Compared With Those on Placebo at Week 25 | 31.1 | 15.3 |
Duration of response (DOR) based on RECIST 1.1 is defined as the date of the first recorded response to the first date of documented disease progression. The overall number of responses and the number of participants with and without disease progression was assessed.
| participants | Pexidartinib Part 1 and Part 2 | Placebo Part 1, Pexidartinib Part 2 | All Pexidartinib Treated |
|---|---|---|---|
| Number of responses | 23 | 12 | 35 |
| Week 12 (Day 84); Without disease progression | 23 | 12 | 35 |
| Week 12 (Day 84); With disease progression | 0 | 0 | 0 |
| Week 24 (Day 168); Without disease progression | 23 | 12 | 35 |
| Week 24 (Day 168); With disease progression | 0 | 0 | 0 |
| Week 48 (Day 336); Without disease progression | 15 | 9 | 24 |
| Week 48 (Day 336); With disease progression | 1 | 0 | 1 |
| Week 72 (Day 504); Without disease progression | 9 | 3 | 12 |
| Week 72 (Day 504); With disease progression | 1 | 0 | 1 |
| Week 96 (Day 672); Without disease progression | 2 | 1 | 3 |
| Week 96 (Day 672); With disease progression | 1 | 0 | 1 |
Tumor Volume Score (TVS) is a semi-quantitative MRI scoring system that describes tumor mass and is based on 10% increments of the estimated volume of the maximally distended synovial cavity or tendon sheath involved. A tumor that is equal in volume to that of a maximally distended synovial cavity or tendon sheath was scored 10; a score of 0 indicated no evidence of tumor. The overall number of responses and the number of participants with and without disease progression was assessed.
| participants | Pexidartinib in Part 1 and Part 2 | Placebo Part 1, Pexidartinib Part 2 | All Pexidartinib Treated |
|---|---|---|---|
| Number of responders | 34 | 18 | 52 |
| Week 12 (Day 84); Without disease progression | 33 | 18 | 51 |
| Week 12 (Day 84); With disease progression | 0 | 0 | 0 |
| Week 24 (Day 168); Without disease progression | 32 | 18 | 50 |
| Week 24 (Day 168); With disease progression | 0 | 0 | 0 |
| Week 48 (Day 336); Without disease progression | 22 | 13 | 35 |
| Week 48 (Day 336); With disease progression | 3 | 1 | 4 |
| Week 72 (Day 504); Without disease progression | 13 | 3 | 16 |
| Week 72 (Day 504); With disease progression | 3 | 1 | 4 |
| Week 96 (Day 672); Without disease progression | 3 | 1 | 4 |
| Week 96 (Day 672); With disease progression | 3 | 1 | 4 |
| Week 120 (Day 840); Without disease progression | 1 | 0 | 1 |
| Week 120 (Day 840); With disease progression | 3 | 1 | 4 |
Duration of response (DOR) based on RECIST 1.1 is defined from the date of the first recorded evidence of response to the first date of documented disease progression.
| months | Pexidartinib Part 1 and Part 2 | Placebo Part 1, Pexidartinib Part 2 |
|---|---|---|
| Duration of Response (DOR) Based on RECIST 1.1 | NA (31.01 to NA) | NA (39.0 to NA) |
Duration of response (DOR) based on TVS is defined from the date of the first recorded evidence of response to the first date of documented disease progression.
| months | Pexidartinib Part 1 and Part 2 | Placebo Part 1, Pexidartinib Part 2 |
|---|---|---|
| Duration of Response (DOR) Based on Tumor Volume Score (TVS) | 52.70 (38.60 to NA) | NA (NA to NA) |
Treatment-emergent adverse events (TEAEs) were defined as adverse events that started or worsened after the first dose of treatment and within 28 days after the last dose. The National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0 was used to grade adverse events. Any Grade and Grade ≥3 (severe) TEAEs are reported. TEAEs were coded using MedDRA version 17.1.
| Percentage of participants | Pexidartinib Part 1 | Placebo Part 1 | Pexidartinib Part 1 and Part 2 | Placebo Part 1, Pexidartinib Part 2 | All Pexidartinib Treated |
|---|---|---|---|---|---|
| Any Hair color changes | 67.2 | 3.4 | 73.8 | 83.3 | 76.9 |
| Grade ≥3 Hair color changes | 0 | 0 | 0 | 0 | 0 |
| Any Pruritis | 9.8 | 3.4 | 16.4 | 20.0 | 17.6 |
| Grade ≥3 Pruritis | 0 | 0 | 1.6 | 0 | 1.1 |
| Any Rash maculopapular | 9.8 | 1.7 | 14.8 | 10.0 | 13.2 |
| Grade ≥3 Rash maculopapular | 0 | 0 | 1.6 | 0 | 1.1 |
| Any Pruritis generalized | 8.2 | 0 | 8.2 | 10.0 | 8.8 |
| Grade ≥3 Pruritis generalized | 0 | 0 | 0 | 0 | 0 |
| Any Erythema | 1.6 | 0 | 3.3 | 20.0 | 8.8 |
| Grade ≥3 Erythema | 0 | 0 | 0 | 0 | 0 |
| Any Dry skin | 3.3 | 3.4 | 6.6 | 10.0 | 7.7 |
| Grade ≥3 Dry skin | 0 | 0 | 0 | 3.3 | 1.1 |
| Any Photosensitivity reaction | 0 | 0 | 1.6 | 10.0 | 4.4 |
| Grade ≥3 Photosensitivity reaction | 0 | 0 | 0 | 0 | 0 |
| Any Nausea | 37.7 | 40.7 | 44.3 | 20.0 | 36.3 |
| Grade ≥3 Nausea | 0 | 0 | 0 | 0 | 0 |
| Any Diarrhea | 19.7 | 25.4 | 26.2 | 30.0 | 27.5 |
| Grade ≥3 Diarrhea | 0 | 0 | 0 | 0 | 0 |
| Any Vomiting | 19.7 | 5.1 | 23.0 | 6.7 | 17.6 |
| Grade ≥3 Vomiting | 1.6 | 0 | 1.6 | 0 | 1.1 |
| Any Abdominal Pain | 16.4 | 10.2 | 21.3 | 6.7 | 16.5 |
| Grade ≥3 Abdominal Pain | 0 | 0 | 0 | 0 | 0 |
| Any Dry mouth | 9.8 | 3.4 | 13.1 | 13.3 | 13.2 |
| Grade ≥3 Dry mouth | 0 | 0 | 0 | 0 | 0 |
| Any Constipation | 11.5 | 5.1 | 14.8 | 10.0 | 13.2 |
| Grade ≥3 Constipation | 0 | 0 | 0 | 0 | 0 |
| Any Stomatitis | 6.6 | 1.7 | 8.2 | 10.0 | 8.8 |
| Grade ≥3 Stomatitis | 0 | 0 | 0 | 0 | 0 |
| Any Fatigue | 54.1 | 35.6 | 55.7 | 26.7 | 46.2 |
| Grade ≥3 Fatigue | 0 | 0 | 0 | 0 | 0 |
| Any Edema peripheral | 13.1 | 3.4 | 16.4 | 20.0 | 17.6 |
| Grade ≥3 Edema peripheral | 0 | 0 | 0 | 0 | 0 |
| Any Face edema | 13.1 | 1.7 | 14.8 | 20.0 | 16.5 |
| Grade ≥3 Face edema | 0 | 0 | 1.6 | 3.3 | 2.2 |
| Any Asthenia | 9.8 | 5.1 | 11.5 | 20.0 | 14.3 |
| Grade ≥3 Asthenia | 0 | 0 | 0 | 0 | 0 |
| Any Pyrexia | 6.6 | 1.7 | 8.2 | 13.3 | 9.9 |
| Grade ≥ Pyrexia | 0 | 0 | 0 | 0 | 0 |
| Any AST increased | 39.3 | 0 | 44.3 | 16.7 | 35.2 |
| Grade ≥3 AST increased | 9.8 | 0 | 9.8 | 6.7 | 8.8 |
| Any ALT increased | 27.9 | 1.7 | 31.1 | 23.3 | 28.6 |
| Grade ≥3 ALT increased | 9.8 | 0 | 9.8 | 10.0 | 9.9 |
| Any ALP increased | 14.8 | 0 | 14.8 | 3.3 | 11.0 |
| Grade ≥3 ALP increased | 6.6 | 0 | 6.6 | 3.3 | 5.5 |
| Any LDH increased | 11.5 | 0 | 11.5 | 10.0 | 11.0 |
| Grade ≥3 LDH increased | 1.6 | 0 | 1.6 | 0 | 1.1 |
| Any Weight increased | 3.3 | 0 | 4.9 | 10.0 | 6.6 |
| Grade ≥3 Weight increased | 0 | 0 | 0 | 0 | 0 |
| Any Dysgeusia | 24.6 | 1.7 | 27.9 | 23.3 | 26.4 |
| Grade ≥3 Dysgeusia | 0 | 0 | 0 | 0 | 0 |
| Any Headache | 19.7 | 18.6 | 23.0 | 20.0 | 22.0 |
| Grade ≥3 Headache | 0 | 0 | 1.6 | 0 | 1.1 |
| Any Dizziness | 9.8 | 15.3 | 13.1 | 13.3 | 13.2 |
| Grade ≥3 Dizziness | 1.6 | 0 | 1.6 | 0 | 1.1 |
| Any Paresthesia | 1.6 | 1.7 | 8.2 | 10.0 | 8.8 |
| Grade ≥3 Paresthesia | 0 | 0 | 0 | 0 | 0 |
| Any Memory impairment | 0 | 1.7 | 1.6 | 10.0 | 4.4 |
| Grade ≥3 Memory impairment | 0 | 0 | 0 | 0 | 0 |
| Any Arthralgia | 23.0 | 25.4 | 27.9 | 30.0 | 28.6 |
| Grade ≥3 Arthralgia | 3.3 | 1.7 | 3.3 | 0 | 2.2 |
| Any Pain in extremity | 6.6 | 6.8 | 9.8 | 13.3 | 11.0 |
| Grade ≥3 Pain in extremity | 0 | 1.7 | 0 | 0 | 0 |
| Any Periorbital edema | 18.0 | 1.7 | 24.6 | 13.3 | 20.0 |
| Grade ≥3 Periorbital edema | 1.6 | 0 | 1.6 | 0 | 1.1 |
| Any Eyelid edema | 3.3 | 0 | 4.9 | 10.0 | 6.6 |
| Grade ≥3 Eyelid edema | 0 | 0 | 0 | 0 | 0 |
| Any Decreased appetite | 16.4 | 10.2 | 18.0 | 10.0 | 15.4 |
| Grade ≥3 Decreased appetite | 0 | 0 | 0 | 0 | 0 |
| Any Hypertension | 14.8 | 10.2 | 19.7 | 30.0 | 13.1 |
| Grade ≥3 Hypertension | 4.9 | 0 | 4.9 | 6.7 | 5.5 |
| Any Upper respiratory tract infection | 1.6 | 0 | 11.5 | 3.3 | 8.8 |
| Grade ≥3 Upper respiratory tract infection | 0 | 0 | 0 | 0 | 0 |
| Any Cough | 4.9 | 5.1 | 6.6 | 10.0 | 7.7 |
| Grade ≥3 Cough | 0 | 0 | 0 | 0 | 0 |
| Any Dyspnea | 1.6 | 0 | 4.9 | 10.0 | 7.7 |
| Grade ≥3 Dyspnea | 0 | 0 | 0 | 0 | 0 |
| Any Insomnia | 4.9 | 3.4 | 4.9 | 10.0 | 6.6 |
| Grade ≥3 Insomnia | 0 | 0 | 0 | 0 | 0 |
| Any Rash | 14.8 | 5.1 | 27.9 | 23.3 | 26.4 |
| Grade ≥3 Rash | 1.6 | 0 | 1.6 | 0 | 1.1 |
Complete (CR) and partial responses (PR) were assessed based on centrally-read magnetic resonance imaging (MRI) scans and Response Evaluation Criteria in Solid Tumors, version 1.1 (RECIST 1.1). A CR was defined as disappearance of all tumors and a PR was defined as at least a 30% decrease in the sum of diameters of target tumors using the baseline sum diameters as the reference.
| Percentage of participants | Pexidartinib Part 1 and Part 2 | Placebo Part 1, Pexidartinib Part 2 | All Pexidartinib Treated |
|---|---|---|---|
| Complete Response (CR) | 24.6 | 23.3 | 24.2 |
| Partial Response (PR) | 29.5 | 30.0 | 29.7 |
| Response (CR or PR) | 54.1 | 53.3 | 53.8 |
Range of motion (ROM) of the joint was assessed by a qualified, independent, and blinded or third-party assessors at the clinical site. Measurements were recorded in degrees. At baseline, the plane of movement with the smallest relative value (worst) was identified and this plane was used for evaluating the relative change of motion subsequently. Only the plane with the worst impaired ROM at baseline was selected for subsequent analyses.
| degrees | Pexidartinib Part 1 and Part 2 | Placebo Part 1, Pexidartinib Part 2 | All Pexidartinib Treated |
|---|---|---|---|
| Baseline | 62.5 ± 24.8 | 66.5 ± 22.9 | 63.8 ± 24.2 |
| Week 25 | 15.6 ± 14.9 | 13.1 ± 12.9 | 14.8 ± 14.2 |
| Week 49 | 14.4 ± 19.5 | 12.0 ± 13.4 | 13.4 ± 17.3 |
The Patient-reported Outcomes Measurement Information System (PROMIS) physical function scale was used to assess physical function of the upper and lower limbs. The scale ranged from 1 defined as 'unable to do' or 'cannot do' to 5 defined as 'without any difficulty' or 'not at all', where higher scores represent better outcomes.
| units on a scale | Pexidartinib Part 1 and Part 2 | Placebo Part 1, Pexidartinib Part 2 | All Pexidartinib Treated |
|---|---|---|---|
| Week 25 | 3.6 ± 4.9 | 4.9 ± 6.3 | 4.0 ± 5.4 |
| Week 49 | 4.7 ± 4.4 | 7.6 ± 6.3 | 5.8 ± 5.2 |
The Worst Stiffness Numeric Rating Scale (NRS) was a 1-item, self-administered questionnaire assessing the "worst" stiffness in the last 24 hours. The NRS for this item ranged from 0 (no stiffness) to 10 (stiffness as bad as you can imagine).
| units on a scale | Pexidartinib Part 1 and Part 2 | Placebo Part 1, Pexidartinib Part 2 | All Pexidartinib Treated |
|---|---|---|---|
| Baseline | 5.6 ± 1.7 | 5.7 ± 2.3 | 5.6 ± 1.9 |
| Week 25 | -2.7 ± 2.2 | -3.0 ± 3.1 | -2.8 ± 2.5 |
| Week 49 | -3.5 ± 1.9 | -2.2 ± 2.8 | -3.1 ± 2.3 |
The Brief Pain Inventory (BPI) Worst Pain NRS was a 1-item, self-administered questionnaire assessing the "worst" pain in the last 24 hours. The NRS for this item ranged from 0 (no pain) to 10 (pain as bad as you can imagine).
| units on a scale | Pexidartinib Part 1 and Part 2 | Placebo Part 1, Pexidartinib Part 2 | All Pexidartinib Treated |
|---|---|---|---|
| Baseline | 5.6 ± 1.6 | 5.2 ± 2.5 | 5.5 ± 1.9 |
| Week 25 | -2.7 ± 2.2 | -2.6 ± 3.1 | -2.7 ± 2.5 |
| Week 49 | -3.3 ± 1.7 | -2.8 ± 3.4 | -3.2 ± 2.3 |
Best overall response (CR or PR) was assessed using tumor volume score (TVS) in the ITT population. Tumor Volume Score is a semi-quantitative MRI scoring system that describes tumor mass and is based on 10% increments of the estimated volume of the maximally distended synovial cavity or tendon sheath involved. A tumor that is equal in volume to that of a maximally distended synovial cavity or tendon sheath was scored 10; a score of 0 indicated no evidence of tumor.
| Percentage of participants | Pexidartinib Part 1 and Part 2 | Placebo Part 1, Pexidartinib Part 2 | All Pexidartinib Treated |
|---|---|---|---|
| Percentage of Participants With Symptomatic, Locally Advanced Tenosynovial Giant Cell Tumor (TGCT) Achieving Complete or Partial Response Based on Tumor Volume Score (TVS) After Receiving Pexidartinib Compared With Those on Placebo by Week 49 | 63.9 | 66.7 | 64.8 |
Collected over Adverse events were monitored throughout the study from the time the participant signed the informed consent form to 28 days after the final treatment dose, up to 71 months.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Pexidartinib (Part 1) | 0/61 (0%) | 8/61 (13.1%) | 60/61 (98.4%) |
| Placebo (Part 1) | 0/59 (0%) | 1/59 (1.7%) | 55/59 (93.2%) |
| Pexidartinib (Parts 1 and 2) | 0/61 (0%) | 12/61 (19.7%) | 61/61 (100%) |
| Placebo (Part 1), Crossover Pexidartinib (Part 2) | 1/30 (3.3%) | 9/30 (30%) | 30/30 (100%) |
| All Pexidartinib Treated | 1/91 (1.1%) | 21/91 (23.1%) | 91/91 (100%) |
| Event | Pexidartinib (Part 1) | Placebo (Part 1) | Pexidartinib (Parts 1 and 2) | Placebo (Part 1), Crossover Pexidartinib (Part 2) | All Pexidartinib Treated |
|---|---|---|---|---|---|
| Adenosquamous carcinoma of the cervixNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 0/61 | 0/59 | 0/61 | 1/30 | 1/91 |
| Rectal adenocarcinomaNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 0/61 | 0/59 | 0/61 | 1/30 | 1/91 |
| Squamous cell carcinoma of skinNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 0/61 | 0/59 | 0/61 | 1/30 | 1/91 |
| Cardiac arrestCardiac disorders | 0/61 | 0/59 | 0/61 | 1/30 | 1/91 |
| Local swellingGeneral disorders | 0/61 | 0/59 | 0/61 | 1/30 | 1/91 |
| Non-cardiac chest painGeneral disorders | 0/61 | 0/59 | 0/61 | 1/30 | 1/91 |
| Neck painMusculoskeletal and connective tissue disorders | 0/61 | 0/59 | 0/61 | 1/30 | 1/91 |
| LymphangitisInfections and infestations | 0/61 | 0/59 | 0/61 | 1/30 | 1/91 |
| Rectal cancer stage IINeoplasms benign, malignant and unspecified (incl cysts and polyps) | 0/61 | 0/59 | 0/61 | 1/30 | 1/91 |
| Joint injuryInjury, poisoning and procedural complications | 0/61 | 0/59 | 0/61 | 1/30 | 1/91 |
| Event | Pexidartinib (Part 1) | Placebo (Part 1) | Pexidartinib (Parts 1 and 2) | Placebo (Part 1), Crossover Pexidartinib (Part 2) | All Pexidartinib Treated |
|---|---|---|---|---|---|
| Hair color changesSkin and subcutaneous tissue disorders | 41/61 | 2/59 | 44/61 | 25/30 | 69/91 |
| FatigueGeneral disorders | 33/61 | 21/59 | 35/61 | 8/30 | 43/91 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 14/61 | 15/59 | 19/61 | 15/30 | 34/91 |
| Aspartate aminotransferase increasedInvestigations | 24/61 | 0/59 | 28/61 | 6/30 | 34/91 |
| NauseaGastrointestinal disorders | 23/61 | 24/59 | 28/61 | 7/30 | 35/91 |
| HypertensionVascular disorders | 9/61 | 6/59 | 14/61 | 12/30 | 26/91 |
| DiarrheaGastrointestinal disorders | 13/61 | 15/59 | 19/61 | 10/30 | 29/91 |
| Alanine aminotransferase increasedInvestigations | 17/61 | 1/59 | 19/61 | 7/30 | 26/91 |
| Oedema peripheralGeneral disorders | 8/61 | 2/59 | 15/61 | 9/30 | 24/91 |
| PruritisSkin and subcutaneous tissue disorders | 10/61 | 2/59 | 10/61 | 9/30 | 19/91 |
| Age, Categorical(Participants) | Pexidartinib Part 1, Then Pexidartinib Part 2 | Placebo Part 1, Then Pexidartinib Part 2 | Total |
|---|---|---|---|
| <=18 years | 0 | 0 | 0 |
| Between 18 and 65 years | 57 | 56 | 113 |
| >=65 years | 4 | 3 | 7 |
| Age, Continuous(years) | Pexidartinib Part 1, Then Pexidartinib Part 2 | Placebo Part 1, Then Pexidartinib Part 2 | Total |
|---|---|---|---|
| Mean | 44.6 ± 13.2 | 44.3 ± 13.6 | 44.5 ± 13.4 |
| Sex: Female, Male(Participants) | Pexidartinib Part 1, Then Pexidartinib Part 2 | Placebo Part 1, Then Pexidartinib Part 2 | Total |
|---|---|---|---|
| Female | 35 | 36 | 71 |
| Male | 26 | 23 | 49 |
| Race (NIH/OMB)(Participants) | Pexidartinib Part 1, Then Pexidartinib Part 2 | Placebo Part 1, Then Pexidartinib Part 2 | Total |
|---|---|---|---|
| American Indian or Alaska Native | 2 | 0 | 2 |
| Asian | 1 | 2 | 3 |
| Native Hawaiian or Other Pacific Islander | 2 | 2 | 4 |
| Black or African American | 3 | 1 | 4 |
| White | 52 | 54 | 106 |
| More than one race | 1 | 0 | 1 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Region of Enrollment(participants) | Pexidartinib Part 1, Then Pexidartinib Part 2 | Placebo Part 1, Then Pexidartinib Part 2 | Total |
|---|---|---|---|
| Canada | 2 | 3 | 5 |
| Netherlands | 7 | 4 | 11 |
| Hungary | 2 | 1 | 3 |
| United States | 23 | 22 | 45 |
| Denmark | 1 | 2 | 3 |
| Poland | 2 | 0 | 2 |
| Italy | 8 | 9 | 17 |
| United Kingdom | 0 | 1 | 1 |
| Australia | 5 | 7 | 12 |
| France | 2 | 5 | 7 |
| Germany | 4 | 2 | 6 |
| Spain | 5 | 3 | 8 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — 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 information: Study protocol, Statistical analysis plan, Clinical study report
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.
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Studies of the drug tested here: Pexidartinib
Synovitis, Pigmented Villonodular →