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CompletedNCT02734433Updated Apr 25, 2022Results posted

Study of Pexidartinib in Asian Subjects With Advanced Solid Tumors

A Phase 1 interventional study of Pexidartinib in Advanced Solid Tumors, sponsored by Daiichi Sankyo Co., Ltd.. Completed at 1 site in Taiwan. Open to participants aged 20 Years and older. Per ClinicalTrials.gov, last updated 2022-04-25.

Sponsored by Daiichi Sankyo Co., Ltd. · Phase 1, Interventional, and Treatment

Phase
Phase 1
Study type
Interventional
Enrollment
12
Allocation
Not applicable
Ages
20 Years and older
Sex
All
01

Study summary

This is a Phase I, non-randomized, open-label, multiple dose study of pexidartinib in Asian subjects with advanced solid tumors. The study will be conducted in a dose escalation to assess the safety and tolerability, pharmacokinetics (PK) and pharmacodynamics (PD), and preliminary antitumor activity of pexidartinib.

02

Conditions studied

  • Advanced Solid Tumors

Keywords

  • Advanced solid tumors
  • Asian subjects
  • Developmental Phase I
03

In context

Lead sponsor

Daiichi Sankyo Co., Ltd. is the lead sponsor of 73 studies on the registry; none are open to participants now.

Of its 10 completed or terminated interventional studies of FDA-regulated products, 8 (80%) have results posted.

Browse Daiichi Sankyo studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
20 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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.

Inclusion criteria

  • Age should be ≥ 20 years
  • Subjects must have a pathologically documented solid tumor that has relapsed from, or is refractory to standard treatment, or for which no standard treatment is available
  • All associated toxicity from previous cancer therapy must have been resolved (to ≤ Grade 1) prior to administration of pexidartinib
  • Eastern Cooperative Oncology Group (ECOG) score of 0 or 1
  • Adequate hematologic, hepatic, and renal function tests

  • Adequate treatment washout period before registration defined as:

    1. Major surgery: ≥ 4 weeks (2 weeks for less invasive surgery, such as colostomy)
    2. Radiation therapy (eg, whole brain radiotherapy): ≥ 4 weeks (if palliative stereotactic radiation therapy, ≥ 2 weeks)
    3. Chemotherapy or immunotherapy (including targeted therapy with antibody or small molecule, retinoid therapy, and hormonal therapy): 4 weeks or 5 half-lives of the agent, whichever is shorter (if the regimen has contained nitrosoureas or mitomycin C, ≥ 6 weeks)
    4. Other investigational drug therapy: ≥ 4 weeks

Exclusion criteria

Exclusion Criteria:

  • Refractory nausea and vomiting, malabsorption, external biliary shunt, or significant small bowel resection that would have precluded adequate absorption
  • Previous use of pexidartinib or any biologic treatment targeting colony stimulating factor-1 (CSF-1) or the receptor for colony-stimulating factor-1 (CSF1R); previous use of oral tyrosine kinase inhibitors, eg, imatinib or nilotinib, is allowed
  • Clinically active primary central nervous system tumors or brain metastasis, defined as untreated and symptomatic, or requiring therapy with steroids or anticonvulsants to control associated symptoms
  • Active or chronic infection with hepatitis C or known positive hepatitis B surface. antigen, or known active or chronic infection with human immunodeficiency virus
  • A screening Fridericia-corrected time between the start of the Q wave and the end of the T wave in the heart's electrical cycle (QTcF) ≥ 450 ms (in men) or ≥ 470 ms (in women).
  • A medical history or complications of clinically significant lung disease (eg, interstitial pneumonia, pneumonitis, pulmonary fibrosis, and severe radiation pneumonitis)
  • A history of symptomatic congestive heart failure (CHF) [New York Heart Association (NYHA) Classes II to IV] or serious cardiac arrhythmia requiring treatment
  • A history of myocardial infarction or unstable angina within 6 months before enrollment
  • An uncontrolled infection requiring intravenous injection of antibiotics, antivirals, or antifungals
05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
12 participants (actual)

Study arms

  • Experimental
    Pexidartinib

    Pexidartinib capsules administered twice daily in the morning and evening. Each cycle of treatment is 28 days in duration. The cycle of treatment is continued until disease progression, unacceptable toxicity, or consent withdrawal.

    Drug: Pexidartinib

Interventions

06

What researchers measure

Primary outcomes

  1. Overall Response Based on RECIST V1.1 Following Oral Doses of Pexidartinib (Efficacy Analysis Set)

    For the assessment of tumor response, participants were classified into the best of the following tumor response categories by RECIST v1.1: complete response (CR), disappearance of all target lesions and normalization of tumor marker level; partial response (PR), at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters; stable disease (SD); neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease, taking as reference the smallest sum diameters while on study; progressive disease (PD), at least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study and the sum must also demonstrate an absolute increase of at least 5 mm; or not evaluable (NE) for analysis. Objective response rate was defined as CR or PR and disease control rate was defined as CR, PR, or SD.

    Time frame: Day 1 through Day 28 after last dose (within 18 months)

Secondary outcomes

  1. Duration of Response or Duration of Stable Disease Following Oral Doses of Pexidartinib (Efficacy Analysis Set)

    Based on RECIST v1.1, complete response (CR), disappearance of all target lesions and normalization of tumor marker level; partial response (PR), at least a 30% decrease in sum of diameters of target lesions, with reference to baseline sum diameters; stable disease (SD); neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease (PD), with reference to smallest sum diameters; PD, at least a 20% increase in the sum of diameters of target lesions, with reference to smallest sum on study and sum must also demonstrate an absolute increase of at least 5 mm; Duration of response for responders (CR or PR) is defined as the time interval between the date of the earliest qualifying response and the date of PD or death for any cause, whichever occurs earlier. Duration of SD is defined as the time interval, in the absence of either CR or PR that will be calculated between the date of the first administration of the study drug and the date of PD.

    Time frame: Day 1 through Day 28 after last dose (within 18 months)

  2. A Summary of Pexidartinib Pharmacokinetic Parameter (Cmax) by Cohort and Day Following Oral Doses of Pexidartinib

    Maximum concentration (Cmax) of pexidartinib was assessed using standard non-compartmental methods.

    Time frame: Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)

  3. A Summary of Pexidartinib Pharmacokinetic Parameter (AUC[0-8h]) by Cohort and Day Following Oral Doses of Pexidartinib

    Area under the curve from 0 to 8 hours (AUC\[0-8h\]) was assessed using standard non-compartmental methods.

    Time frame: Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)

  4. A Summary of Pexidartinib Pharmacokinetic Parameter (Tmax) by Cohort and Day Following Oral Doses of Pexidartinib

    Time at maximum pexidartinib concentration (Tmax) was assessed using standard non-compartmental methods.

    Time frame: Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)

  5. A Summary of Pexidartinib Pharmacokinetic Parameter (R[AUC]) by Cohort Following Oral Doses of Pexidartinib

    Accumulation ratio of area under the curve (R\[AUC\]) was assessed using standard non-compartmental methods.

    Time frame: Cycle 1, Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)

  6. A Summary of Pexidartinib Pharmacokinetic Parameter (R[Cmax]) by Cohort Following Oral Doses of Pexidartinib

    Accumulation ratio of maximum concentration of pexidartinib (R\[Cmax\]) was assessed using standard non-compartmental methods.

    Time frame: Cycle 1, Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)

  7. A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (AUC[0-8h]) Following Oral Doses of Pexidartinib

    Area under the curve from 0 to 8 hours (AUC\[0-8h\]) was assessed using standard non-compartmental methods.

    Time frame: Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)

  8. A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (Cmax) Following Oral Doses of Pexidartinib

    Maximum concentration (Cmax) of pexidartinib was assessed using standard non-compartmental methods.

    Time frame: Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)

  9. A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (Tmax) Following Oral Doses of Pexidartinib

    Time at maximum pexidartinib concentration (Tmax) was assessed using standard non-compartmental methods.

    Time frame: Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)

  10. A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (MR AUC[0-8h]) Following Oral Doses of Pexidartinib

    Metabolite to parent drug ratio of area under the curve from 0-8 h (MR AUC\[0-8h\]) was assessed using standard non-compartmental methods.

    Time frame: Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)

  11. A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (MR Cmax) Following Oral Doses of Pexidartinib

    Metabolite to parent drug ratio of maximum pexidartinib concentration (MR Cmax) was assessed using standard non-compartmental methods.

    Time frame: Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)

  12. Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)

    An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment. All TEAEs were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Event (NCI-CTCAE) Version 4.0.

    Time frame: Baseline through study completion, up to 18 months

  13. Number and Percentage of Participants With Treatment-related TEAEs by Preferred Term (Safety Analysis Set)

    An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment. All TEAEs were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Event (NCI-CTCAE) Version 4.0.

    Time frame: Baseline through study completion, up to 18 months

07

Results

Posted Apr 24, 2020

Participant flow

A total of 12 participants who met all inclusion and no exclusion criteria were enrolled in the study; 11 participants were treated.

Participant flow — Overall Study
MilestoneCohort 1 - 600 mg/DayCohort 2 - 1000 mg/Day
Started38
Completed10
Not completed28
Withdrew: Disease progression15
Withdrew: Clinical progression01
Withdrew: Withdrawal by subject12

Outcome measures

PrimaryOverall Response Based on RECIST V1.1 Following Oral Doses of Pexidartinib (Efficacy Analysis Set)

For the assessment of tumor response, participants were classified into the best of the following tumor response categories by RECIST v1.1: complete response (CR), disappearance of all target lesions and normalization of tumor marker level; partial response (PR), at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters; stable disease (SD); neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease, taking as reference the smallest sum diameters while on study; progressive disease (PD), at least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study and the sum must also demonstrate an absolute increase of at least 5 mm; or not evaluable (NE) for analysis. Objective response rate was defined as CR or PR and disease control rate was defined as CR, PR, or SD.

Time frame:
Day 1 through Day 28 after last dose (within 18 months)
Reported as:
Number · Number of participants
Overall Response Based on RECIST V1.1 Following Oral Doses of Pexidartinib (Efficacy Analysis Set)
Number of participantsCohort 1 - 600 mg/DayCohort 2 - 1000 mg/Day
Complete response (CR)00
Partial response (PR)10
Stable disease (SD)13
Progressive disease (PD)12
Not evaluable (NE)00
Objective response rate (CR or PR)10
Disease control rate (CR or PR or SD)23
SecondaryDuration of Response or Duration of Stable Disease Following Oral Doses of Pexidartinib (Efficacy Analysis Set)

Based on RECIST v1.1, complete response (CR), disappearance of all target lesions and normalization of tumor marker level; partial response (PR), at least a 30% decrease in sum of diameters of target lesions, with reference to baseline sum diameters; stable disease (SD); neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease (PD), with reference to smallest sum diameters; PD, at least a 20% increase in the sum of diameters of target lesions, with reference to smallest sum on study and sum must also demonstrate an absolute increase of at least 5 mm; Duration of response for responders (CR or PR) is defined as the time interval between the date of the earliest qualifying response and the date of PD or death for any cause, whichever occurs earlier. Duration of SD is defined as the time interval, in the absence of either CR or PR that will be calculated between the date of the first administration of the study drug and the date of PD.

Time frame:
Day 1 through Day 28 after last dose (within 18 months)
Reported as:
Mean · months
Duration of Response or Duration of Stable Disease Following Oral Doses of Pexidartinib (Efficacy Analysis Set)
monthsCohort 1 - 600 mg/DayCohort 2 - 1000 mg/Day
Duration of response (n=1,0)7.62 ± 0—
Duration of stable disease (n=1,3)1.87 ± 04.62 ± 1.6
SecondaryA Summary of Pexidartinib Pharmacokinetic Parameter (Cmax) by Cohort and Day Following Oral Doses of Pexidartinib

Maximum concentration (Cmax) of pexidartinib was assessed using standard non-compartmental methods.

Time frame:
Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)
Reported as:
Mean · ng/mL
A Summary of Pexidartinib Pharmacokinetic Parameter (Cmax) by Cohort and Day Following Oral Doses of Pexidartinib
ng/mLCohort 1 - 600 mg/DayCohort 2 - 1000 mg/Day
Day 13210 ± 13203650 ± 1170
Day 158490 ± 143010800 ± 2860
SecondaryA Summary of Pexidartinib Pharmacokinetic Parameter (AUC[0-8h]) by Cohort and Day Following Oral Doses of Pexidartinib

Area under the curve from 0 to 8 hours (AUC\[0-8h\]) was assessed using standard non-compartmental methods.

Time frame:
Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)
Reported as:
Mean · ng*h/mL
A Summary of Pexidartinib Pharmacokinetic Parameter (AUC[0-8h]) by Cohort and Day Following Oral Doses of Pexidartinib
ng*h/mLCohort 1 - 600 mg/DayCohort 2 - 1000 mg/Day
Day 113300 ± 472016500 ± 4760
Day 1550900 ± 1830064600 ± 15900
SecondaryA Summary of Pexidartinib Pharmacokinetic Parameter (Tmax) by Cohort and Day Following Oral Doses of Pexidartinib

Time at maximum pexidartinib concentration (Tmax) was assessed using standard non-compartmental methods.

Time frame:
Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)
Reported as:
Mean · hours
A Summary of Pexidartinib Pharmacokinetic Parameter (Tmax) by Cohort and Day Following Oral Doses of Pexidartinib
hoursCohort 1 - 600 mg/DayCohort 2 - 1000 mg/Day
Day 13.95 ± 3.512.48 ± 1.18
Day 151.00 ± 1.001.26 ± 0.94
SecondaryA Summary of Pexidartinib Pharmacokinetic Parameter (R[AUC]) by Cohort Following Oral Doses of Pexidartinib

Accumulation ratio of area under the curve (R\[AUC\]) was assessed using standard non-compartmental methods.

Time frame:
Cycle 1, Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)
Reported as:
Mean · accumulation ratio of AUC
A Summary of Pexidartinib Pharmacokinetic Parameter (R[AUC]) by Cohort Following Oral Doses of Pexidartinib
accumulation ratio of AUCCohort 1 - 600 mg/DayCohort 2 - 1000 mg/Day
A Summary of Pexidartinib Pharmacokinetic Parameter (R[AUC]) by Cohort Following Oral Doses of Pexidartinib4.29 ± 2.294.58 ± 2.23
SecondaryA Summary of Pexidartinib Pharmacokinetic Parameter (R[Cmax]) by Cohort Following Oral Doses of Pexidartinib

Accumulation ratio of maximum concentration of pexidartinib (R\[Cmax\]) was assessed using standard non-compartmental methods.

Time frame:
Cycle 1, Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)
Reported as:
Mean · accumulation ratio of Cmax
A Summary of Pexidartinib Pharmacokinetic Parameter (R[Cmax]) by Cohort Following Oral Doses of Pexidartinib
accumulation ratio of CmaxCohort 1 - 600 mg/DayCohort 2 - 1000 mg/Day
A Summary of Pexidartinib Pharmacokinetic Parameter (R[Cmax]) by Cohort Following Oral Doses of Pexidartinib2.94 ± 1.194.58 ± 2.23
SecondaryA Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (AUC[0-8h]) Following Oral Doses of Pexidartinib

Area under the curve from 0 to 8 hours (AUC\[0-8h\]) was assessed using standard non-compartmental methods.

Time frame:
Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)
Reported as:
Mean · ng*h/mL
A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (AUC[0-8h]) Following Oral Doses of Pexidartinib
ng*h/mLCohort 1 - 600 mg/DayCohort 2 - 1000 mg/Day
Day 114800 ± 801015600 ± 9130
Day 1591600 ± 37500120000 ± 61100
SecondaryA Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (Cmax) Following Oral Doses of Pexidartinib

Maximum concentration (Cmax) of pexidartinib was assessed using standard non-compartmental methods.

Time frame:
Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)
Reported as:
Mean · ng/mL
A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (Cmax) Following Oral Doses of Pexidartinib
ng/mLCohort 1 - 600 mg/DayCohort 2 - 1000 mg/Day
Day 12930 ± 9353200 ± 1630
Day 1513200 ± 512017800 ± 9630
SecondaryA Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (Tmax) Following Oral Doses of Pexidartinib

Time at maximum pexidartinib concentration (Tmax) was assessed using standard non-compartmental methods.

Time frame:
Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)
Reported as:
Mean · hours
A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (Tmax) Following Oral Doses of Pexidartinib
hoursCohort 1 - 600 mg/DayCohort 2 - 1000 mg/Day
Day 16.55 ± 2.245.00 ± 2.87
Day 151.5 ± 2.181.43 ± 0.87
SecondaryA Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (MR AUC[0-8h]) Following Oral Doses of Pexidartinib

Metabolite to parent drug ratio of area under the curve from 0-8 h (MR AUC\[0-8h\]) was assessed using standard non-compartmental methods.

Time frame:
Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)
Reported as:
Mean · metabolite to parent drug ratio
A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (MR AUC[0-8h]) Following Oral Doses of Pexidartinib
metabolite to parent drug ratioCohort 1 - 600 mg/DayCohort 2 - 1000 mg/Day
Day 10.801 ± 0.4540.658 ± 0.298
Day 151.27 ± 0.4161.33 ± 0.711
SecondaryA Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (MR Cmax) Following Oral Doses of Pexidartinib

Metabolite to parent drug ratio of maximum pexidartinib concentration (MR Cmax) was assessed using standard non-compartmental methods.

Time frame:
Cycle 1, Day 1 and Day 15 at predose, 0.5h, 1h, 2h, 4h, and 8h postdose (each cycle was 28 days)
Reported as:
Mean · metabolite to parent drug ratio
A Summary of Plasma ZAAD-1006a Pharmacokinetic Parameter (MR Cmax) Following Oral Doses of Pexidartinib
metabolite to parent drug ratioCohort 1 - 600 mg/DayCohort 2 - 1000 mg/Day
Day 10.669 ± 0.1890.639 ± 0.314
Day 151.10 ± 0.4401.18 ± 0.695
SecondaryNumber and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)

An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment. All TEAEs were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Event (NCI-CTCAE) Version 4.0.

Time frame:
Baseline through study completion, up to 18 months
Reported as:
Count of participants · Participants
Number and Percentage of Participants With Common Treatment-emergent Adverse Events (TEAEs) (≥20%) Classified by Preferred Term (Safety Analysis Set)
ParticipantsCohort 1 - 600 mg/DayCohort 2 - 1000 mg/Day
Any TEAE38
Chronic gastritis01
Constipation02
Diarrhoea03
Nausea01
Stomatitis01
Face oedema01
Fatigue13
Malaise10
Alanine aminotransferase increased13
Aspartate aminotransferase increased23
Blood alkaline phosphatase increased13
Blood bilirubin increased11
Blood cholesterol increased10
Gamma-glutamyltransferase increased02
Weight increased10
Dysuria01
Proteinuria10
Urinary retention01
Cough12
Haemoptysis01
Hair colour changes22
Pruritus generalised10
Rash generalised10
Skin hypopigmentation01
SecondaryNumber and Percentage of Participants With Treatment-related TEAEs by Preferred Term (Safety Analysis Set)

An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product that does not necessarily have to have a causal relationship with this treatment. All TEAEs were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Event (NCI-CTCAE) Version 4.0.

Time frame:
Baseline through study completion, up to 18 months
Reported as:
Count of participants · Participants
Number and Percentage of Participants With Treatment-related TEAEs by Preferred Term (Safety Analysis Set)
ParticipantsCohort 1 - 600 mg/DayCohort 2 - 1000 mg/Day
Any treatment-related TEAE36
Diarrhoea03
Face oedema01
Fatigue13
Alanine aminotransferase increased13
Aspartate aminotransferase increased23
Blood alkaline phosphatase increased13
Blood bilirubin increased10
Blood cholesterol increased10
Gamma-glutamyltransferase increased02
Proteinuria10
Hair colour changes22
Pruritus generalised10
Rash generalised10
Skin hypopigmentation01

Adverse events

Collected over Adverse event data were collected after the first dose up 28 days after the last dose through study completion, up to 18 months postdose.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Cohort 1 - 600 mg/Day0/3 (0%)2/3 (66.7%)3/3 (100%)
Cohort 2 - 1000 mg/Day1/8 (12.5%)1/8 (12.5%)8/8 (100%)
Most frequent serious events
Most frequent serious events
EventCohort 1 - 600 mg/DayCohort 2 - 1000 mg/Day
Aspartate aminotransferase increasedInvestigations2/30/8
Alanine aminotransferase increasedInvestigations1/30/8
Blood alkaline phosphatase increasedInvestigations1/30/8
Blood bilirubin increasedInvestigations1/30/8
AnaemiaBlood and lymphatic system disorders0/31/8
Malignant neoplasm progressionNeoplasms benign, malignant and unspecified (incl cysts and polyps)0/31/8
Most frequent other events
Showing 10 of 30
Most frequent other events
EventCohort 1 - 600 mg/DayCohort 2 - 1000 mg/Day
Aspartate aminotransferase increasedInvestigations2/33/8
Hair colour changesSkin and subcutaneous tissue disorders2/32/8
DiarrhoeaGastrointestinal disorders0/33/8
FatigueGeneral disorders1/33/8
Alanine aminotransferase increasedInvestigations1/33/8
Blood alkaline phosphatase increasedInvestigations1/33/8
MalaiseGeneral disorders1/30/8
Blood bilirubin increasedInvestigations1/31/8
Blood cholesterol increasedInvestigations1/30/8
Weight increasedInvestigations1/30/8

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Cohort 1 - 600 mg/DayCohort 2 - 1000 mg/DayTotal
<=18 years000
Between 18 and 65 years156
>=65 years235
Age, Continuous
Age, Continuous(years)Cohort 1 - 600 mg/DayCohort 2 - 1000 mg/DayTotal
Mean55.0 ± 27.962.4 ± 12.260.4 ± 16.5
Sex: Female, Male
Sex: Female, Male(Participants)Cohort 1 - 600 mg/DayCohort 2 - 1000 mg/DayTotal
Female145
Male246
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Cohort 1 - 600 mg/DayCohort 2 - 1000 mg/DayTotal
American Indian or Alaska Native000
Asian3811
Native Hawaiian or Other Pacific Islander000
Black or African American000
White000
More than one race000
Unknown or Not Reported000
Region of Enrollment
Region of Enrollment(participants)Cohort 1 - 600 mg/DayCohort 2 - 1000 mg/DayTotal
Taiwan3811
08

Study locations

1 site
  • Taipei, Taiwan
09

References and documents

Publications

  • Lewis JH, Gelderblom H, van de Sande M, Stacchiotti S, Healey JH, Tap WD, Wagner AJ, Pousa AL, Druta M, Lin CC, Baba HA, Choi Y, Wang Q, Shuster DE, Bauer S. Pexidartinib Long-Term Hepatic Safety Profile in Patients with Tenosynovial Giant Cell Tumors. Oncologist. 2021 May;26(5):e863-e873. doi: 10.1002/onco.13629. Epub 2020 Dec 24. PubMed 33289960 ↗
  • Lee JH, Chen TW, Hsu CH, Yen YH, Yang JC, Cheng AL, Sasaki SI, Chiu LL, Sugihara M, Ishizuka T, Oguma T, Tajima N, Lin CC. A phase I study of pexidartinib, a colony-stimulating factor 1 receptor inhibitor, in Asian patients with advanced solid tumors. Invest New Drugs. 2020 Feb;38(1):99-110. doi: 10.1007/s10637-019-00745-z. Epub 2019 Mar 2. PubMed 30825104 ↗

Study documents

  • Protocol and statistical analysis plan · Feb 25, 2020

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No

10

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Ran from Jun 2016 to primary completion in Jun 2017, 12 months.

Results were first posted in Apr 2020, 2 years 10 months after primary completion.

How these figures are worked out, and which studies are left out of them.

Registered
Mar 22, 2016
First posted
Apr 12, 2016
StartActual
Jun 2016
Primary completionActual
Jun 13, 2017
CompletionActual
May 28, 2021
Results first posted
Apr 24, 2020
Last update
Apr 25, 2022
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Apr 25, 2022, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT02734433
Lead sponsor
Daiichi Sankyo Co., Ltd.
Responsible party
Sponsor
First posted
Apr 12, 2016
Start date
Jun 2016
Primary completion
Jun 13, 2017
Completion
May 28, 2021
Results posted
Apr 24, 2020
Last update
Apr 25, 2022

Study contacts

Global Clinical Leader
study director · Daiichi Sankyo

Oversight

Data monitoring committee
No
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This study is completed, as verified in Mar 2022. You cannot join it, but the record below documents what was studied.

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Studies of the drug tested here: Pexidartinib

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Daiichi Sankyo Co., Ltd. →

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