18 and older, any sex, with Recurrent Glioblastoma. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Summary of Response Rates in Participants on Treatment With PLX3397Primary· 6 months post dose
Response to treatment was evaluated using the Response Assessment in Neuro-Oncology (RANO) criteria. All participants were evaluated for progression free survival (PFS), and overall survival (OS). The six-month PFS rate was defined as the number of subjects with PFS of at least 6-month duration, with PFS measured from the first day of treatment (Cycle 1, Day 1) to the date of the first documented disease progression or date of death, whichever occurs first, over a 6-month period and evaluated using the Kaplan Meier method. The rate of OS was defined as the number of subjects that survived unti
Six-month PFS rate
Group
Value
95% CI
Surgical Cohort 1 (N=14)
1
Non-Surgical Cohort 2 (N=24)
2
Overall survival
Group
Value
95% CI
Surgical Cohort 1 (N=14)
10
Non-Surgical Cohort 2 (N=24)
21
Complete response rate
Group
Value
95% CI
Surgical Cohort 1 (N=14)
0
Non-Surgical Cohort 2 (N=24)
0
Partial response rate
Group
Value
95% CI
Surgical Cohort 1 (N=14)
0
Non-Surgical Cohort 2 (N=24)
0
Stable disease rate
Group
Value
95% CI
Surgical Cohort 1 (N=14)
3
Non-Surgical Cohort 2 (N=24)
4
Progressive disease rate
Group
Value
95% CI
Surgical Cohort 1 (N=14)
10
Non-Surgical Cohort 2 (N=24)
18
Not Evaluable
Group
Value
95% CI
Surgical Cohort 1 (N=14)
1
Non-Surgical Cohort 2 (N=24)
2
Mean Plasma Pharmacokinetic Parameters of PLX3397 After Oral Administration of 1000 mg/Day - Cycle 1 Day 15Primary· Pre-dose and up to 6 post dose during cycle 1, Day 15
A noncompartmental method of analysis was used to analyze the plasma concentrations of PLX3397. Mean plasma pharmacokinetic parameters, include time to maximum concentration (Tmax) and will be calculated from the Cycle 1, Day 15 values.
Group
Value
95% CI
PLX3397 - Cohort 1 (Surgical)
2.09
± 1.04
PLX3397 - Cohort 2 (Non-surgical)
1.71
± 0.90
Mean Plasma Pharmacokinetic Parameters of PLX3397 After Oral Administration of 1000 mg/Day - Cycle 1 Day 15Primary· Pre-dose and up to 6 post dose during cycle 1, Day 15
A noncompartmental method of analysis was used to analyze the plasma concentrations of PLX3397. Mean plasma pharmacokinetic parameters include maximum concentration (Cmax) and will be calculated from the Cycle 1, Day 15 values.
Group
Value
95% CI
PLX3397 - Cohort 1 (Surgical)
7760
± 2330
PLX3397 - Cohort 2 (Non-surgical)
8030
± 2790
Mean Plasma Pharmacokinetic Parameters of PLX3397 After Oral Administration of 1000 mg/Day - Cycle 1 Day 15Primary· Pre-dose and up to 6 post dose during cycle 1, Day 15
A noncompartmental method of analysis was used to analyze the plasma concentrations of PLX3397. Mean plasma pharmacokinetic parameters include an assessment of area under the curve over 0-4 hours (AUC0-4), and will be calculated from the Cycle 1, Day 15 values.
Group
Value
95% CI
PLX3397 - Cohort 1 (Surgical)
24900
± 6700
PLX3397 - Cohort 2 (Non-surgical)
26100
± 9260
Mean Plasma Pharmacokinetic Parameters of PLX3397 After Oral Administration of 1000 mg/Day - Cycle 1 Day 15Primary· Pre-dose and up to 6 post dose during cycle 1, Day 15
A noncompartmental method of analysis was used to analyze the plasma concentrations of PLX3397. Mean plasma pharmacokinetic parameters include an assessment of area under the curve over 0-6 hours (AUC0-6), and will be calculated from the Cycle 1, Day 15 values.
Group
Value
95% CI
PLX3397 - Cohort 1 (Surgical)
36100
± 10000
PLX3397 - Cohort 2 (Non-surgical)
36900
± 12200
Incidence (Number and Percentage of Participants) With Treatment-Emergent Adverse Events Related to Study Drug Occurring in ≥10% Participants During Treatment With PLX3397 (Safety Population)Secondary· Up to 1 year post dose
Any Event
Group
Value
95% CI
PLX3397 - Cohort 1 (Surgical)
12
PLX3397 - Cohort 2 (Non-surgical)
22
Fatigue
Group
Value
95% CI
PLX3397 - Cohort 1 (Surgical)
6
PLX3397 - Cohort 2 (Non-surgical)
14
Constipation
Group
Value
95% CI
PLX3397 - Cohort 1 (Surgical)
3
PLX3397 - Cohort 2 (Non-surgical)
1
Nausea
Group
Value
95% CI
PLX3397 - Cohort 1 (Surgical)
1
PLX3397 - Cohort 2 (Non-surgical)
3
Hair color changes
Group
Value
95% CI
PLX3397 - Cohort 1 (Surgical)
2
PLX3397 - Cohort 2 (Non-surgical)
4
Aspartate aminotransferase increased
Group
Value
95% CI
PLX3397 - Cohort 1 (Surgical)
3
PLX3397 - Cohort 2 (Non-surgical)
3
Alanine aminotransferase increased
Group
Value
95% CI
PLX3397 - Cohort 1 (Surgical)
2
PLX3397 - Cohort 2 (Non-surgical)
3
Decreased appetite
Group
Value
95% CI
PLX3397 - Cohort 1 (Surgical)
3
PLX3397 - Cohort 2 (Non-surgical)
3
Adverse events — posted to ClinicalTrials.gov
Time frame: Adverse event data were collected from after the first dose to 28 days after the last dose..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
PLX3397 - Cohort 1 (Surgical)
Serious: 8/14 (57%)
Deaths: 0/14
PLX3397 - Cohort 2 (Non-surgical)
Serious: 11/24 (46%)
Deaths: 1/24
Serious adverse events (22 terms)
Reaction
System
PLX3397 - Cohort 1 (Surgic…
PLX3397 - Cohort 2 (Non-su…
Convulsion
Nervous system disorders
—
—
Pneumonia
Infections and infestations
—
—
Cerebrovascular accident
Nervous system disorders
—
—
Dysarthria
Nervous system disorders
—
—
Headache
Nervous system disorders
—
—
Hydrocephalus
Nervous system disorders
—
—
Meningitis
Infections and infestations
—
—
Anaemia
Blood and lymphatic system disorders
—
—
Febrile neutropenia
Blood and lymphatic system disorders
—
—
ALT increased
Investigations
—
—
AST increased
Investigations
—
—
INR increased
Investigations
—
—
WBC count decreased
Investigations
—
—
Hypertension
Vascular disorders
—
—
Thrombosis
Vascular disorders
—
—
Nausea
Gastrointestinal disorders
—
—
Vomiting
Gastrointestinal disorders
—
—
Oedema peripheral
General disorders
—
—
Dehydration
Metabolism and nutrition disorders
—
—
Hyponatraemia
Metabolism and nutrition disorders
—
—
Muscular weakness
Musculoskeletal and connective tissue disorders
—
—
Respiratory failure
Respiratory, thoracic and mediastinal disorders
—
—
Other adverse events (117 terms — click to expand)
NCT02975700 — A Study of PLX3397 in Patients With Unresectable or Metastatic KIT-mutated Melanoma
· NA
· completed
NCT02452424 — A Combination Clinical Study of PLX3397 and Pembrolizumab To Treat Advanced Melanoma and Other Solid Tumors
· Phase 1, PHASE2
· terminated
NCT01826448 — A Phase 1b Open Label, Dose Escalation Study of PLX3397 in Combination With Vemurafenib in V600-mutated BRAF Melanoma
· Phase 1
· terminated
NCT01790503 — A Phase 1b/2 Study of PLX3397 + Radiation Therapy + Temozolomide in Patients With Newly Diagnosed Glioblastoma
· Phase 1, PHASE2
· completed
NCT01596751 — Phase Ib/II Study of PLX 3397 and Eribulin in Patients With Metastatic Breast Cancer
· Phase 1, PHASE2
· completed
Other recruiting trials for Recurrent Glioblastoma
Currently open trials in the same condition.
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· recruiting
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· Phase 1
· recruiting
NCT06910306 — Alpha Radiation Emitters Device (DaRT) for the Treatment of Recurrent Glioblastoma
· NA
· recruiting
NCT07193628 — B7H3/IL13Ra2 Bispecific Armored Chimeric Antigen Receptor T-Cell Therapy Study for Recurrent/Refractory Glioblastoma
· Phase 1
· recruiting
NCT07076472 — Sonodynamic Therapy With SONALA-001 or 5-ALA HCL and Magnetic Resonance Guided Focused Ultrasound for the Treatment of P
· EARLY_PHASE1
· recruiting
Other Daiichi Sankyo trials
Trials by the same sponsor.
NCT07474649 — A Study of Bempedoic Acid/Ezetimibe/High-intensity Statin in Patients Without Cardiovascular Events
· Phase 3
· not yet recruiting
NCT07206472 — A Study of Bempedoic Acid or Its Single-pill Combination Therapy With Ezetimibe in Patients With Primary Hypercholestero
· active not recruiting
NCT07220616 — A First-in-Human Trial of DS3790a in Participants With Hematological Malignancies
· Phase 1, PHASE2
· recruiting
NCT07268625 — Evaluating Bioequivalence of a Fixed Dose Combination Versus Individual Tablets of Bempedoic Acid / Ezetimibe, and Atorv
· Phase 1
· completed
NCT07244341 — A Study of Valemetostat (DS-3201b) in Combination With Darolutamide in Metastatic Castration Resistant Prostate Cancer (
· Phase 1
· recruiting
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Daiichi Sankyo
Last refreshed: 3 March 2020
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01349036.