CompletedNCT03291288Updated May 14, 2021Results posted

Effect of Pexidartinib on the Way the Body Processes CYP3A4 and CYP2C9 Substrates (Pharmacokinetics)

A Phase 1 interventional study of Tolbutamide and Midazolam in Drug Interaction Potential, sponsored by Daiichi Sankyo. Completed at 11 sites in 4 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2021-05-14.

Sponsored by Daiichi Sankyo · Phase 1, Interventional, and Treatment

Phase: Phase 1
Study type: Interventional
Enrollment: 32
Allocation: Not applicable
Ages: 18 Years and older
Sex: All

Study summary

This study has two parts.

Part 1 will evaluate how pexidartinib affects the way the body processes CYP3A4 and CYP2C9 substrates using midazolam and tolbutamide, respectively, as probe agents.

Part 2 will test the efficacy and safety of pexidartinib treatment in various tumor types.

In Part 2, the same participants will continue to receive pexidartinib twice daily.

Participants will be allowed to continue using pexidartinib as long as the participant derives benefit.

Conditions studied

Drug Interaction Potential

Keywords

Tenosynovial Giant Cell Tumors (TGCT)
Kit-mutant melanoma
Kit-mutant gastrointestinal stromal tumor (GIST)
Cocktail drug-drug interaction (DDI)

Who can participate

Ages eligible: 18 Years and older
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

Is the age of majority in country of residence
Has a diagnosis of:
1. tenosynovial giant cell tumor (TGCT), which is associated with severe morbidity or functional limitations and for whom surgery is not an option (prior pexidartinib is permitted for TGCT patients unless ineffective or not tolerated and there has been a washout period of at least 4 weeks)
2. KIT-mutant tumor, including melanoma or gastrointestinal stromal tumor (GIST), for which there is no standard systemic therapy, or
3. other solid tumors (all comers) for which there is no standard systemic therapy and there is a rationale for use of pexidartinib at the Investigator's discretion
If a female of childbearing potential, had a negative serum pregnancy test within 14 days before enrollment, or within 72 hours before enrollment where required
Is a non-sterile male or female willing to use of one of the protocol-defined highly effective contraception methods:
1. intra-uterine device (nonhormonal or hormonal)
2. sexual abstinence (only if this is in line with the patient's current lifestyle)
3. barrier methods (eg, condom, diaphragm) used in combination with hormonal methods associated with inhibition of ovulation
Is a surgically sterile male or female, or is postmenopausal for at least 1 year, at least 50 years of age, with a follicle-stimulating hormone level > 40 milli-International units per mL (mIU/mL)
Has adequate hematologic, hepatic, and renal function as defined by the protocol
Is able and willing to follow all study procedures
Has provided a signed informed consent

Exclusion criteria

Exclusion Criteria:

Is pregnant or breastfeeding
Is unable to swallow oral medication
Is unable to follow study procedures
Is taking or has taken any medications or therapies outside of protocol-defined parameters
Has any disease or condition that, per protocol or in the opinion of the investigator, might affect:
1. safety and well-being of the participant or offspring
2. safety of study staff
3. analysis of results

Study design

Phase: Phase 1
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Sequential assignment
Masking: None (open label)
Enrollment: 32 participants (actual)

Study arms

Experimental
Pexidartinib
Part 1 (Drug-drug Interaction Phase): On Day 1, all participants will receive a single oral dose each of midazolam (2 mg) and tolbutamide (500 mg). On Day 3, pexidartinib (800 mg/d) in twice daily (400 mg BID) dosing will be initiated and continue throughout the remainder of Part 1 and into Part 2. On the first day of pexidartinib treatment (Day 3), a single dose of midazolam (2 mg) and tolbutamide (500 mg) will be co-administered with the morning pexidartinib dose (400 mg). On Day 13, a single dose of midazolam (2 mg) and tolbutamide (500 mg) will be co-administered with the morning dose of pexidartinib (400 mg). Part 2 (Efficacy and Safety Phase): All participants will continue to receive pexidartinib 400 mg BID.
Drug: Tolbutamide · Drug: Midazolam · Drug: Pexidartinib

Interventions

DrugTolbutamide
Commercially available tolbutamide
Also known as: Orinase
DrugMidazolam
Commercially available midazolam
Also known as: Dormicum, Hypnovel, Versed, Others
DrugPexidartinib
Pexidartinib is formulated as opaque, white, 200-mg capsules
Also known as: PLX-3397

What researchers measure

Primary outcomes

Pharmacokinetic Analysis: Maximum Concentration (Cmax) for Midazolam
Plasma samples for midazolam were collected at predose, 0.5, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 10, 24, and 48 hours (h) (±10 minutes up to 1 h, ±10% thereafter) on Days 1 to 3, and also when co-administered with pexidartinib on Days 3 (to 5) and Days 13 (to 15).
Time frame: Baseline to 15 days post treatment
Pharmacokinetic Analysis: Maximum Concentration (Cmax) for Tolbutamide
Plasma samples for tolbutamide were collected at predose, 0.5, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 10, 24, and 48 h (±10 min up to 1 h, ±10% thereafter) on Days 1 to 3, and also when co-administered with pexidartinib on Days 3 (to 5) and Days 13 (to 15).
Time frame: Baseline to 15 days post treatment
Pharmacokinetic Analysis: Time to Maximum Concentration (Tmax) for Midazolam
Plasma samples for midazolam were collected at predose, 0.5, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 10, 24, and 48 hours (h) (±10 minutes up to 1 h, ±10% thereafter) on Days 1 to 3, and also when co-administered with pexidartinib on Days 3 (to 5) and Days 13 (to 15).
Time frame: Baseline to 15 days post treatment
Pharmacokinetic Analysis: Time to Maximum Concentration (Tmax) for Tolbutamide
Plasma samples for tolbutamide were collected at predose, 0.5, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 10, 24, and 48 h (±10 min up to 1 h, ±10% thereafter) on Days 1 to 3, and also when co-administered with pexidartinib on Days 3 (to 5) and Days 13 (to 15).
Time frame: Baseline to 15 days post treatment
Pharmacokinetic Analysis: Area Under the Curve to the Last Observable Concentration (AUClast) for Midazolam
Plasma samples for midazolam were collected at predose, 0.5, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 10, 24, and 48 hours (h) (±10 minutes up to 1 h, ±10% thereafter) on Days 1 to 3, and also when co-administered with pexidartinib on Days 3 (to 5) and Days 13 (to 15).
Time frame: Baseline to 15 days post treatment
Pharmacokinetic Analysis: Area Under the Curve to the Last Observable Concentration (AUClast) for Tolbutamide
Plasma samples for tolbutamide were collected at predose, 0.5, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 10, 24, and 48 h (±10 min up to 1 h, ±10% thereafter) on Days 1 to 3, and also when co-administered with pexidartinib on Days 3 (to 5) and Days 13 (to 15).
Time frame: Baseline to 15 days post treatment
Overall Summary of Treatment-emergent Adverse Events
Adverse events that emerge during treatment, having been absent pre-treatment, or worsens relative to the pre-treatment state.
Time frame: Baseline to 1 year post treatment

Secondary outcomes

Pharmacokinetic Analysis: Maximum Concentration (Cmax) for Pexidartinib and ZAAD-1006a Metabolite
Plasma samples for pexidartinib and its metabolite were collected at predose, 0.5, 1, 2, 2.5, 3, 4, 6, 8, and 10 (±1) h after the first dose on Day 3, and at steady state when co-administered with midazolam and tolbutamide on Day 13.
Time frame: Baseline to 13 days post treatment
Pharmacokinetic Analysis: Time to Maximum Concentration (Tmax) for Pexidartinib and ZAAD-1006a Metabolite
Plasma samples for pexidartinib and its metabolite were collected at predose, 0.5, 1, 2, 2.5, 3, 4, 6, 8, and 10 (±1) h after the first dose on Day 3, and at steady state when co-administered with midazolam and tolbutamide on Day 13.
Time frame: Baseline to 13 days post treatment
Pharmacokinetic Analysis: Area Under the Curve to the Last Observable Concentration (AUClast) for Pexidartinib and ZAAD-1006a Metabolite
Plasma samples for pexidartinib and its metabolite were collected at predose, 0.5, 1, 2, 2.5, 3, 4, 6, 8, and 10 (±1) h after the first dose on Day 3, and at steady state when co-administered with midazolam and tolbutamide on Day 13.
Time frame: Baseline to 13 days post treatment
Pharmacokinetic Analysis: Maximum Concentration (Cmax) for Midazolam Metabolite, 1-Hydroxy Midazolam
Plasma samples for midazolam and 1-hydroxy midazolam were to be collected at predose, 0.5, 1, 1.5, 2, 2.5, 3, 4, 6, 8 (±10 min up to 8 h), 10 (±2), 24 (±2), and 48 (±2) hours on Days 1 to 3 and also when co-administered with pexidartinib on Days 3 to 5 and Days 13 to 15.
Time frame: Baseline to 13 days post treatment
Pharmacokinetic Analysis: Time to Maximum Concentration (Tmax) for Midazolam Metabolite, 1-Hydroxy Midazolam
Plasma samples for midazolam and 1-hydroxy midazolam were to be collected at predose, 0.5, 1, 1.5, 2, 2.5, 3, 4, 6, 8 (±10 min up to 8 h), 10 (±2), 24 (±2), and 48 (±2) hours on Days 1 to 3 and also when co-administered with pexidartinib on Days 3 to 5 and Days 13 to 15.
Time frame: Baseline to 13 days post treatment
Pharmacokinetic Analysis: Area Under the Curve to the Last Observable Concentration (AUClast) for Midazolam Metabolite, 1-Hydroxy Midazolam
Plasma samples for midazolam and 1-hydroxy midazolam were to be collected at predose, 0.5, 1, 1.5, 2, 2.5, 3, 4, 6, 8 (±10 min up to 8 h), 10 (±2), 24 (±2), and 48 (±2) hours on Days 1 to 3 and also when co-administered with pexidartinib on Days 3 to 5 and Days 13 to 15.
Time frame: Baseline to 13 days post treatment
Pharmacokinetic Analysis: Metabolite to Parent Ratio (MPR) for Midazolam
Plasma samples for midazolam were collected at predose, 0.5, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 10, 24, and 48 hours (h) (±10 minutes up to 1 h, ±10% thereafter) on Days 1 to 3, and also when co-administered with pexidartinib on Days 3 (to 5) and Days 13 (to 15). Plasma pharmacokinetic parameters calculated for Midazolam metabolite. 1-hydroxy midazolam and midazolam for the MPR value.
Time frame: Baseline to 13 days post treatment

Results

Posted Mar 19, 2020

Participant flow

A total of 32 participants who met all inclusion and no exclusion criteria were enrolled in the study; 2 participants did not receive pexidartinib treatment.

Drug-drug Interaction Phase

Participant flow — Drug-drug Interaction Phase
Milestone	Part 1: Drug-drug Interaction Phase	Part 2: Pexidartinib Only
Started	32	0
Completed	26	0
Not completed	6	0
Withdrew: Adverse event	2	0
Withdrew: Disease progression	1	0
Withdrew: Other	3	0

Pexidartinib Only

Participant flow — Pexidartinib Only
Milestone	Part 1: Drug-drug Interaction Phase	Part 2: Pexidartinib Only
Started	0	26
Completed	0	24
Not completed	0	2
Withdrew: Ongoing	0	2

Outcome measures

PrimaryPharmacokinetic Analysis: Maximum Concentration (Cmax) for Midazolam

Time frame:: Baseline to 15 days post treatment
Reported as:: Mean · ng/mL

Pharmacokinetic Analysis: Maximum Concentration (Cmax) for Midazolam
ng/mL	Part 1: Midazolam Only	Part 1: Pexidartinib + Midazolam (Cycle 1, Day 1)	Part 1: Pexidartinib + Midazolam (Cycle 1, Day 11)
Pharmacokinetic Analysis: Maximum Concentration (Cmax) for Midazolam	13.6 ± 6.8	12.0 ± 4.9	9.7 ± 4.1

PrimaryPharmacokinetic Analysis: Maximum Concentration (Cmax) for Tolbutamide

Plasma samples for tolbutamide were collected at predose, 0.5, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 10, 24, and 48 h (±10 min up to 1 h, ±10% thereafter) on Days 1 to 3, and also when co-administered with pexidartinib on Days 3 (to 5) and Days 13 (to 15).

Time frame:: Baseline to 15 days post treatment
Reported as:: Mean · ng/mL

Pharmacokinetic Analysis: Maximum Concentration (Cmax) for Tolbutamide
ng/mL	Part 1: Tolbutamide Only	Part 1: Pexidartinib + Tolbutamide (Cycle 1, Day 1)	Part 1: Pexidartinib + Tolbutamide (Cycle 1, Day 11)
Pharmacokinetic Analysis: Maximum Concentration (Cmax) for Tolbutamide	44400 ± 13100	46700 ± 16300	44400 ± 12200

PrimaryPharmacokinetic Analysis: Time to Maximum Concentration (Tmax) for Midazolam

Time frame:: Baseline to 15 days post treatment
Reported as:: Median · hours

Pharmacokinetic Analysis: Time to Maximum Concentration (Tmax) for Midazolam
hours	Part 1: Midazolam Only	Part 1: Pexidartinib + Midazolam (Cycle 1, Day 1)	Part 1: Pexidartinib + Midazolam (Cycle 1, Day 11)
Pharmacokinetic Analysis: Time to Maximum Concentration (Tmax) for Midazolam	0.525 (0.333 to 1.37)	0.5 (0.417 to 1.42)	0.5 (0.333 to 0.917)

PrimaryPharmacokinetic Analysis: Time to Maximum Concentration (Tmax) for Tolbutamide

Time frame:: Baseline to 15 days post treatment
Reported as:: Median · hours

Pharmacokinetic Analysis: Time to Maximum Concentration (Tmax) for Tolbutamide
hours	Part 1: Tolbutamide Only	Part 1: Pexidartinib + Tolbutamide (Cycle 1, Day 1)	Part 1: Pexidartinib + Tolbutamide (Cycle 1, Day 11)
Pharmacokinetic Analysis: Time to Maximum Concentration (Tmax) for Tolbutamide	2.90 (1 to 8.2)	2.94 (1.52 to 8.08)	3.17 (2.45 to 6.02)

PrimaryPharmacokinetic Analysis: Area Under the Curve to the Last Observable Concentration (AUClast) for Midazolam

Time frame:: Baseline to 15 days post treatment
Reported as:: Mean · ng*hour/mL

Pharmacokinetic Analysis: Area Under the Curve to the Last Observable Concentration (AUClast) for Midazolam
ng*hour/mL	Part 1: Midazolam Only	Part 1: Pexidartinib + Midazolam (Cycle 1, Day 1)	Part 1: Pexidartinib + Midazolam (Cycle 1, Day 11)
Pharmacokinetic Analysis: Area Under the Curve to the Last Observable Concentration (AUClast) for Midazolam	43.7 ± 34.1	31.6 ± 19.6	18.5 ± 8.2

PrimaryPharmacokinetic Analysis: Area Under the Curve to the Last Observable Concentration (AUClast) for Tolbutamide

Time frame:: Baseline to 15 days post treatment
Reported as:: Mean · ng*hour/mL

Pharmacokinetic Analysis: Area Under the Curve to the Last Observable Concentration (AUClast) for Tolbutamide
ng*hour/mL	Part 1: Tolbutamide Only	Part 1: Pexidartinib + Tolbutamide (Cycle 1, Day 1)	Part 1: Pexidartinib + Tolbutamide (Cycle 1, Day 11)
Pharmacokinetic Analysis: Area Under the Curve to the Last Observable Concentration (AUClast) for Tolbutamide	500000 ± 196000	585000 ± 234000	700000 ± 207000

PrimaryOverall Summary of Treatment-emergent Adverse Events

Adverse events that emerge during treatment, having been absent pre-treatment, or worsens relative to the pre-treatment state.

Time frame:: Baseline to 1 year post treatment
Reported as:: Count of participants · Participants

Overall Summary of Treatment-emergent Adverse Events
Participants	Part 1: Drug-drug Interaction Phase	Part 2: Pexidartinib Only
TEAEs	24	23
Pexidartinib-related TEAEs	16	20
Treatment-emergent serious adverse events (SAEs)	5	5
Pexidartinib-related SAEs	1	1

SecondaryPharmacokinetic Analysis: Maximum Concentration (Cmax) for Pexidartinib and ZAAD-1006a Metabolite

Plasma samples for pexidartinib and its metabolite were collected at predose, 0.5, 1, 2, 2.5, 3, 4, 6, 8, and 10 (±1) h after the first dose on Day 3, and at steady state when co-administered with midazolam and tolbutamide on Day 13.

Time frame:: Baseline to 13 days post treatment
Reported as:: Mean · ng/mL

Pharmacokinetic Analysis: Maximum Concentration (Cmax) for Pexidartinib and ZAAD-1006a Metabolite
ng/mL	Part 1: Pexidartinib (Cycle 1, Day 1)	Part 1: Pexidartinib (Cycle 1, Day 11)
Pexidartinib	3140 ± 1250	8320 ± 2530
ZAAD-1006a	3330 ± 1520	13500 ± 5980

SecondaryPharmacokinetic Analysis: Time to Maximum Concentration (Tmax) for Pexidartinib and ZAAD-1006a Metabolite

Time frame:: Baseline to 13 days post treatment
Reported as:: Median · hours

Pharmacokinetic Analysis: Time to Maximum Concentration (Tmax) for Pexidartinib and ZAAD-1006a Metabolite
hours	Part 1: Pexidartinib (Cycle 1, Day 1)	Part 1: Pexidartinib (Cycle 1, Day 11)
Pexidartinib	2.03 (1.08 to 7.83)	1.93 (0.92 to 8.03)
ZAAD-1006a	6.04 (2.37 to 9.07)	2.53 (0 to 8.25)

SecondaryPharmacokinetic Analysis: Area Under the Curve to the Last Observable Concentration (AUClast) for Pexidartinib and ZAAD-1006a Metabolite

Time frame:: Baseline to 13 days post treatment
Reported as:: Mean · ng*hour/mL

Pharmacokinetic Analysis: Area Under the Curve to the Last Observable Concentration (AUClast) for Pexidartinib and ZAAD-1006a Metabolite
ng*hour/mL	Part 1: Pexidartinib (Cycle 1, Day 1)	Part 1: Pexidartinib (Cycle 1, Day 11)
Pexidartinib	14400 ± 5870	53200 ± 17800
ZAAD-1006a	18300 ± 7240	102000 ± 44100

SecondaryPharmacokinetic Analysis: Maximum Concentration (Cmax) for Midazolam Metabolite, 1-Hydroxy Midazolam

Plasma samples for midazolam and 1-hydroxy midazolam were to be collected at predose, 0.5, 1, 1.5, 2, 2.5, 3, 4, 6, 8 (±10 min up to 8 h), 10 (±2), 24 (±2), and 48 (±2) hours on Days 1 to 3 and also when co-administered with pexidartinib on Days 3 to 5 and Days 13 to 15.

Time frame:: Baseline to 13 days post treatment
Reported as:: Mean · ng/mL

Pharmacokinetic Analysis: Maximum Concentration (Cmax) for Midazolam Metabolite, 1-Hydroxy Midazolam
ng/mL	Part 1: Midazolam Only	Part 1: Pexidartinib + Midazolam (Cycle 1, Day 1)	Part 1: Pexidartinib + Midazolam (Cycle 1, Day 11)
Pharmacokinetic Analysis: Maximum Concentration (Cmax) for Midazolam Metabolite, 1-Hydroxy Midazolam	49.7 ± 17.9	52.1 ± 17.4	55.7 ± 17.4

SecondaryPharmacokinetic Analysis: Time to Maximum Concentration (Tmax) for Midazolam Metabolite, 1-Hydroxy Midazolam

Time frame:: Baseline to 13 days post treatment
Reported as:: Median · hours

Pharmacokinetic Analysis: Time to Maximum Concentration (Tmax) for Midazolam Metabolite, 1-Hydroxy Midazolam
hours	Part 1: Midazolam Only	Part 1: Pexidartinib + Midazolam (Cycle 1, Day 1)	Part 1: Pexidartinib + Midazolam (Cycle 1, Day 11)
Pharmacokinetic Analysis: Time to Maximum Concentration (Tmax) for Midazolam Metabolite, 1-Hydroxy Midazolam	1.0 (0.333 to 1.45)	0.933 (0.417 to 1.37)	0.833 (0.383 to 1.55)

SecondaryPharmacokinetic Analysis: Area Under the Curve to the Last Observable Concentration (AUClast) for Midazolam Metabolite, 1-Hydroxy Midazolam

Time frame:: Baseline to 13 days post treatment
Reported as:: Mean · ng*hour/mL

Pharmacokinetic Analysis: Area Under the Curve to the Last Observable Concentration (AUClast) for Midazolam Metabolite, 1-Hydroxy Midazolam
ng*hour/mL	Part 1: Midazolam Only	Part 1: Pexidartinib + Midazolam (Cycle 1, Day 1)	Part 1: Pexidartinib + Midazolam (Cycle 1, Day 11)
Pharmacokinetic Analysis: Area Under the Curve to the Last Observable Concentration (AUClast) for Midazolam Metabolite, 1-Hydroxy Midazolam	200 ± 93	206 ± 95.5	212 ± 99.6

SecondaryPharmacokinetic Analysis: Metabolite to Parent Ratio (MPR) for Midazolam

Plasma samples for midazolam were collected at predose, 0.5, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 10, 24, and 48 hours (h) (±10 minutes up to 1 h, ±10% thereafter) on Days 1 to 3, and also when co-administered with pexidartinib on Days 3 (to 5) and Days 13 (to 15). Plasma pharmacokinetic parameters calculated for Midazolam metabolite. 1-hydroxy midazolam and midazolam for the MPR value.

Time frame:: Baseline to 13 days post treatment
Reported as:: Mean · Ratio

Pharmacokinetic Analysis: Metabolite to Parent Ratio (MPR) for Midazolam
Ratio	Part 1: Midazolam Only	Part 1: Pexidartinib + Midazolam (Cycle 1, Day 1)	Part 1: Pexidartinib + Midazolam (Cycle 1, Day 11)
Pharmacokinetic Analysis: Metabolite to Parent Ratio (MPR) for Midazolam	587 ± 359	750 ± 391	1220 ± 657

Adverse events

Collected over Adverse events were collected after first dose up to 28 days after the last dose of study drug, up to 1 year.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
Part 1: Drug-drug Interaction Phase	1/30 (3.3%)	5/30 (16.7%)	24/30 (80%)
Part 2: Pexidartinib Only	0/25 (0%)	5/25 (20%)	23/25 (92%)

Most frequent serious events

Showing 10 of 14

Most frequent serious events
Event	Part 1: Drug-drug Interaction Phase	Part 2: Pexidartinib Only
Pericardial effusionCardiac disorders	0/30	1/25
Cardiac tamponadeCardiac disorders	0/30	1/25
OesophagitisGastrointestinal disorders	0/30	1/25
Gastrointestinal haemorrhageGastrointestinal disorders	0/30	1/25
CholestasisHepatobiliary disorders	0/30	1/25
PneumoniaImmune system disorders	0/30	1/25
Subdural haematomaInjury, poisoning and procedural complications	0/30	1/25
Chest wall haematomaMusculoskeletal and connective tissue disorders	0/30	1/25
Malignant pleural effusionNeoplasms benign, malignant and unspecified (incl cysts and polyps)	0/30	1/25
Malignant bowel obstructionGastrointestinal disorders	1/30	0/25

Most frequent other events

Showing 10 of 30

Most frequent other events
Event	Part 1: Drug-drug Interaction Phase	Part 2: Pexidartinib Only
Hair colour changesSkin and subcutaneous tissue disorders	0/30	10/25
FatigueGeneral disorders	4/30	7/25
White blood cell count decreasedInvestigations	1/30	6/25
AnemiaBlood and lymphatic system disorders	5/30	4/25
Aspartate aminotransferase increasedInvestigations	5/30	1/25
VomitingGastrointestinal disorders	2/30	4/25
Neutrophil count decreasedInvestigations	0/30	4/25
HypophosphataemiaMetabolism and nutrition disorders	1/30	4/25
Decreased appetiteMetabolism and nutrition disorders	2/30	4/25
DiarrheaGastrointestinal disorders	4/30	0/25

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)	All Participants
<=18 years	2
Between 18 and 65 years	19
>=65 years	9

Age, Continuous
Age, Continuous(years)	All Participants
Mean	50.8 ± 19.3

Sex: Female, Male
Sex: Female, Male(Participants)	All Participants
Female	15
Male	15

Race (NIH/OMB)
Race (NIH/OMB)(Participants)	All Participants
American Indian or Alaska Native	0
Asian	5
Native Hawaiian or Other Pacific Islander	0
Black or African American	0
White	23
More than one race	0
Unknown or Not Reported	2

Study locations

11 sites

HonorHealth
Scottsdale, Arizona 85258, United States
University of Arizona
Tucson, Arizona 85719, United States
Stanford University
Palo Alto, California 94304, United States
University of Kansas Cancer Center
Westwood, Kansas 66205, United States
Dana-Farber Cancer Institute
Boston, Massachusetts 02215, United States
Karmanos Cancer Center
Detroit, Michigan 48201, United States
Northwell Health
Lake Success, New York 10042, United States
Mary Crowley Cancer Research
Dallas, Texas 75230, United States
Leids Universitair Medisch Centrum
Leiden, 2333 ZA, Netherlands
Christchurch Hospital NZ
Christchurch, 8011, New Zealand
National Taiwan University Hospital
Taipei, 10002, Taiwan

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 ↗

Study documents

Protocol and statistical analysis plan · Apr 28, 2017

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

Individual participant data

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, Sap, Csr

Registry details

Key details

Study ID: NCT03291288
Lead sponsor: Daiichi Sankyo
Responsible party: Sponsor
First posted: Sep 25, 2017
Start date: Feb 26, 2018
Primary completion: Sep 26, 2018
Completion: Apr 16, 2021
Results posted: Mar 19, 2020
Last update: May 14, 2021

Study contacts

Global Clinical Leader

study chair · Daiichi Sankyo

Oversight

Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Apr 2021. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies from this sponsor

Daiichi Sankyo

Start the discussion