CompletedNCT03027388Updated Nov 22, 2023Results posted

Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma

A Phase 2 interventional study of LB-100 in Astrocytoma, Grades II, III and IV, Glioblastoma Multiforme and Giant Cell Glioblastoma, sponsored by National Cancer Institute (NCI). Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2023-11-22.

Sponsored by National Cancer Institute (NCI) · Phase 2, Interventional, and Basic science

Phase: Phase 2
Study type: Interventional
Enrollment: 7
Allocation: Not applicable
Ages: 18 Years and older
Sex: All

Study summary

Background:

The brain is separated from the rest of the blood stream by the blood-brain barrier. This is like a filter that protects the brain. But is also a challenge when medicines need to get into the brain. Researchers want to give the new drug LB100 to people before brain tumor surgery. They will measure how much LB100 is in the blood and how much gets into the brain. This may help with the use of LB100 to treat brain tumors in the future.

Objective:

To see if LB100 can pass into the brain.

Eligibility:

People at least 18 years old with a brain tumor that requires surgery.

Design:

Participants will be screened with:

Physical exam

Medical history

Blood tests

Neurosurgery evaluation

Scans

Heart tests

Tumor sample. This can be from a previous procedure.

Participants will have their brain surgery at the Clinical Center.

Participants will get a dose of the study drug through a plastic tube in a vein for 2 hours during surgery.

Participants will have blood taken 7 times in the 8 hours after getting the study drug.

Tumor samples will be taken during surgery.

Participants will have a heart test after getting the study drug. Sticky pads on the skin will measure electrical activity of the heart.

Two-three weeks after leaving the hospital, participants will have a follow-up visit. They will have a physical exam and blood tests.

One month after surgery, they will be contacted in person or by phone to see how they are doing.

Read the detailed description

Background:

Primary gliomas are an incurable disease in spite of aggressive multimodality therapy consisting of craniotomy, irradiation, and chemotherapy. Therapeutic options for patients with recurrent glioma are limited, and there is an unmet need to identify more effective agents.
LB100, a water-soluble small molecule novel protein phosphatase 2A (PP2A) inhibitor, was commercially developed through a Cooperative Research and Development Agreement (CRADA) based on our previous intramural research. This compound has shown to be effective in a variety of cancer types in both in vitro and in vivo models. Preclinical studies indicate LB100 has in vitro and in vivo activity as a single agent as well as potentiating the effect of cytotoxic agents including temozolomide, docetaxel, doxorubicin, and ionizing radiation. LB100 is active in combination with temozolomide or doxorubicin against xenografts of glioblastoma, neuroblastoma, pheochromocytoma, breast cancer, fibrosarcoma, and melanoma.
A complete phase I study of LB100 has established its safety and the recommended phase II dose (2.33 mg/m\^2, daily for three days every 3 weeks).
Although it is a polar compound, rodent studies suggest LB100 has activity in the brain.
Whether LB100 can across the human blood brain barrier (BBB), and at what concentration relative to the plasma level is not known. Characterizing these parameters is important because:
- 1) Our ongoing in vitro studies indicate that LB100 has distinct mechanisms of action at different drug concentrations (e.g., nM versus uM);
- 2) There are other brain tumors lacking effective medical therapies but without a BBB. Characterizing the LB100 BBB penetration profile will assist in defining its optimal clinical indication.

Objective:

-To determine the pharmacokinetic (PK) properties of LB100 in glioma tumor tissues.

Eligibility:

Patients with histologically proven glioblastoma and grades II-III astrocytomas and oligodendrogliomas.
A clear clinical indication for another surgical resection must be present.
Subjects must be greater than or equal to 18 years old.
Karnofsky performance status of greater than or equal to 60%.
Patients must have adequate organ function.

Design:

This is a two stage Phase II, open label, single institution study to determine the PK and pharmacodynamic (PD) profile of LB100.
The dose (established from a Phase I study) will be 2.33 mg/m\^2 delivered intravenously over 2 hours.
PK and PD effect of LB100 treated tissues will only be evaluated with pathologic confirmation of recurrent tumor. Resected material demonstrating chemoradiation treatment effect or inflammatory response will not be included in the analysis.
PK will be determined by quantitating LB100 in tumor tissues removed at various time points.
The primary endpoint is PK response, defined as a binary variable indicating the presence/absence of LB100 in tumor tissues.
PD effect is defined as statistically significant elevation of phospho-proteins in treated tumor tissues compared to untreated glioma specimens. Untreated inter-patient baseline variance and standard deviation (SD) will be calculated. Post-treatment PD effect difference greater than 2.5 times the baseline SD is statistically significant at the .05 significance level. Due to relatively small sample size, t-distribution is to be used to calculate the cutoff defining the PD response.
Up to 25 patients may be enrolled to obtain 8 evaluable subjects. A two-stage design will be used. Five patients will be initially treated. If at least one of five demonstrates PK activity, 3 additional subjects will be enrolled. PK effect will be declared to be significant if at least 2 of the 8 patients demonstrate a PK response (presence of LB100 in tumor tissue).

Conditions studied

Astrocytoma, Grades II, III and IV
Glioblastoma Multiforme
Giant Cell Glioblastoma
Glioma
Oligodendrogliomas

Browse trials for

Keywords

Brain Tumor
Blood Brain Barrier
Pharmacokinetics
Pharmacodynamics

In context

Astrocytoma

434 studies on the registry are indexed under Astrocytoma; 79 are open to participants now.

This study's enrollment of 7 is below the median of 30 across 359 interventional studies indexed under Astrocytoma.

Browse Astrocytoma studies →

Lead sponsor

National Cancer Institute (NCI) is the lead sponsor of 3,554 studies on the registry; 334 are open to participants now.

Of its 407 completed or terminated interventional studies of FDA-regulated products, 370 (91%) have results posted.

Browse National Cancer Institute (NCI) studies →

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

Who can participate

Ages eligible: 18 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

Patients must have histologically confirmed glioblastoma/gliosarcoma, grades II-III astrocytoma and oligodendroglioma.
Patients must have recurrent disease for which there is a clinical indication for resection.
Age greater than or equal to18 years.
Karnofsky greater than or equal to 60%.
Patients must have adequate bone marrow function (white blood count (WBC) greater than or equal to 3,000/uL absolute neutrophil count (ANC) greater than or equal to 1,500/mm\^3, platelet count of greater than or equal to 100,000/mm\^3, and hemoglobin greater than or equal to 10 gm/dL), adequate liver function (SGOT and bilirubin \< 2 times upper limit of normal (ULN). These tests must be performed within 28 days prior to receiving drug. Eligibility level for hemoglobin may be reached by transfusion.
Patients must have a serum creatinine of \<=1.7 mg/dL. If the serum creatinine is greater than 1.7 mg/dl, a 24-hour urine creatinine clearance will be obtained and if the result of this study is within normal limits*, the patient would be eligible to enroll onto study. This test must be performed within 28 days prior to registrationreceiving drug. (*Normal Creatinine Clearance Range: Male: 90 - 130 ml/min; Female: 80 - 125 ml/min)
Patients must be in adequate general medical health to safely tolerate a craniotomy.
At the time of registration, all subjects must be removed greater than or equal to 28 days from any investigational agents.
The effects of LB100 on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
Ability of subject to understand and the willingness to sign a written informed consent document indicating that they are aware of the investigational nature of this study, and that this is not a therapeutic clinical trial.

Exclusion criteria

EXCLUSION CRITERIA:

Patients who are receiving any other investigational agents.
Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
Patients unwilling to undergo craniotomy.
Pregnant women are excluded from this study because the safety of Protein Phosphatase 2A (PP2A) inhibition on a developing fetus has not been established. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with LB100, breastfeeding should be discontinued if the mother is treated with LB100.
Patients may not have had prior chemotherapy or biologic therapy in the 4 weeks prior to study entry. For patients who have been treated with targeted therapy, 5 half-lives of that therapy (or 28 days, whichever is shorter) must have passed prior to enrollment in the study.
Known human immunodeficiency virus (HIV)-positive patients on combination antiretroviral therapy are ineligible because of the potential for pharmacokinetic interactions with LB100. Appropriate studies will be undertaken in patients receiving combination antiretroviral therapy when indicated.
Patients who are receiving strong cytochromes P450 (CYP450) inducers or inhibitors are ineligible
Recruitment Strategies

Patients with recurrent disease will be identified by the Neuro-Oncology Branch, Clinical Center. This study will be posted on National Institutes of Health (NIH) websites and on NIH Social media forums.

Study design

Phase: Phase 2
Primary purpose: Basic science
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrollment: 7 participants (actual)

Study arms

Experimental
1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
Treatment with LB100
Drug: LB-100

Interventions

DrugLB-100
LB-100 will be infused over 2 hours via intravenous (IV) infusion 2 to 4 hours before surgery. The dose established from a Phase I study will be 2.33 mg/m\^2.

What researchers measure

Primary outcomes

Number of Participants With the Presence of Drug in the Tumor Tissue
Participants tumor tissue was examined to determine the presence of drug in the tumor tissue.
Time frame: an average of 5.5 hours after drug administration

Secondary outcomes

Plasma Concentration and Calculated LB100
Blood samples for the determination of plasma levels of LB-100 will be obtained from each participant. Plasma concentrations of LB100 will be determined at each time point. LC-MS-MS assay that can measure LB-100 in human plasma. Assay range is 2.0 -1000 ng/mL. A higher value is better because it would have made it more likely drug could penetrate the blood brain barrier at therapeutic levels.
Time frame: Pre-dose; End of infusion (2 hours post-start); 30 minutes post LB100 infusion completion; 1 hour post LB-100 infusion completion; 2 hours post LB100 infusion completion; 4 hours post LB100 infusion completion; 8 hours post LB100 infusion completion
Concentration of LB100
Concentration of LB100 in glioma tumor tissue when a known non-toxic dose of LB100 is delivered intravenously. Glioma tissue sampling to detect and quantify LB100 will be performed by assaying phospho-protein expression in glioma tissue resected prior to and after infusion of LB100. These are values for the LB100 in the tumor which was done at one timepoint.
Time frame: average of 5.5 hours after infusion
Changes in Phospho-protein Expression in Circulating Peripheral Blood Mononuclear Cells (PBMC)
Changes in phospho-protein expression in circulating PBMC. Pharmacodynamic (PD) response is defined as statistically significant elevation of phospho-protein expression in treated tumor tissues compared to untreated glioma specimens. Post-treatment PD effect (as measured by increase in tumor tissue phospho-protein expression) difference greater than 2.5 times the baseline standard deviation (SD) is statistically significant at the .05 significance level.
Time frame: Pre-dose; End of infusion (2 hours post-start); 30 minutes post LB100 infusion completion; 1 hour post LB-100 infusion completion; 2 hours post LB100 infusion completion; 4 hours post LB100 infusion completion; 8 hours post LB100 infusion completion
Maximum Observed Plasma Concentration of LB100 (Cmax)
The maximum observed analyte concentration in serum was reported.
Time frame: Pre-dose; End of infusion (2 hours post-start); 30 minutes post LB100 infusion completion; 1 hour post LB-100 infusion completion; 2 hours post LB100 infusion completion; 4 hours post LB100 infusion completion; 8 hours post LB100 infusion completion
Area Under the Plasma Concentration vs. Time Curve Extrapolated to Infinity (AUC(INF)
AUC is a measure of the serum concentration of LMB100 over time. It is used to characterize drug absorption.
Time frame: Pre-dose; End of infusion (2 hours post-start); 30 minutes post LB100 infusion completion; 1 hour post LB-100 infusion completion; 2 hours post LB100 infusion completion; 4 hours post LB100 infusion completion; 8 hours post LB100 infusion completion
Plasma Half-Life of Drug LB100
Plasma decay half-life is the time measured for the plasma concentration of the drug to decrease by one half.
Time frame: Pre-dose; End of infusion (2 hours post-start); 30 minutes post LB100 infusion completion; 1 hour post LB-100 infusion completion; 2 hours post LB100 infusion completion; 4 hours post LB100 infusion completion; 8 hours post LB100 infusion completion
Time to Maximum Observed Plasma Concentration of LMB100 (Tmax)
Time to maximum observed plasma concentration of LMB100 (Tmax).
Time frame: Pre-dose; End of infusion (2 hours post-start); 30 minutes post LB100 infusion completion; 1 hour post LB-100 infusion completion; 2 hours post LB100 infusion completion; 4 hours post LB100 infusion completion; 8 hours post LB100 infusion completion
Total Clearance (CL) of LMB100
The CL is a quantitative measure of the rate at which a drug substance is removed from the body.
Time frame: Pre-dose; End of infusion (2 hours post-start); 30 minutes post LB100 infusion completion; 1 hour post LB-100 infusion completion; 2 hours post LB100 infusion completion; 4 hours post LB100 infusion completion; 8 hours post LB100 infusion completion
Volume of Distribution (Vd) of LMB100
Volume of distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug.
Time frame: Pre-dose; End of infusion (2 hours post-start); 30 minutes post LB100 infusion completion; 1 hour post LB-100 infusion completion; 2 hours post LB100 infusion completion; 4 hours post LB100 infusion completion; 8 hours post LB100 infusion completion
Brain Concentration
Drug amount in brain as a percent of drug in plasma.
Time frame: Pre-dose; End of infusion (2 hours post-start); 30 minutes post LB100 infusion completion; 1 hour post LB-100 infusion completion; 2 hours post LB100 infusion completion; 4 hours post LB100 infusion completion; 8 hours post LB100 infusion completion
Brain Penetration
Concentration of drug in brain tumor tissue. Brain penetration is measured as "drug amount in a brain as a percent of drug in plasma.
Time frame: Pre-dose; End of infusion (2 hours post-start); 30 minutes post LB100 infusion completion; 1 hour post LB-100 infusion completion; 2 hours post LB100 infusion completion; 4 hours post LB100 infusion completion; 8 hours post LB100 infusion completion

Other outcomes

Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)
Here is the number of participants with serious and/or non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.
Time frame: Date treatment consent signed to date off study, approximately 39 months and 6 days.

Results

Posted Nov 22, 2023

Participant flow

Participant flow — Overall Study
Milestone	1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
Started	7
Completed	7
Not completed	0

Outcome measures

PrimaryNumber of Participants With the Presence of Drug in the Tumor Tissue

Participants tumor tissue was examined to determine the presence of drug in the tumor tissue.

Time frame:: an average of 5.5 hours after drug administration
Reported as:: Count of participants · Participants

Number of Participants With the Presence of Drug in the Tumor Tissue
Participants	1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
Number of Participants With the Presence of Drug in the Tumor Tissue	2

SecondaryPlasma Concentration and Calculated LB100

Blood samples for the determination of plasma levels of LB-100 will be obtained from each participant. Plasma concentrations of LB100 will be determined at each time point. LC-MS-MS assay that can measure LB-100 in human plasma. Assay range is 2.0 -1000 ng/mL. A higher value is better because it would have made it more likely drug could penetrate the blood brain barrier at therapeutic levels.

Time frame:: Pre-dose; End of infusion (2 hours post-start); 30 minutes post LB100 infusion completion; 1 hour post LB-100 infusion completion; 2 hours post LB100 infusion completion; 4 hours post LB100 infusion completion; 8 hours post LB100 infusion completion
Reported as:: Median · (rounded)nM

Plasma Concentration and Calculated LB100
(rounded)nM	1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
Pre-dose	0.0 (0 to 0)
End of infusion (2 hours post-start)	575 (354 to 772)
30 minutes post LB100 infusion completion	534 (291 to 626)
1 hour post LB-100 infusion completion	355 (251 to 633)
2 hours post LB100 infusion completion	192 (174 to 263)
4 hours post LB100 infusion completion	80 (30 to 150)
8 hours post LB100 infusion completion	16 (07 to 71)

SecondaryConcentration of LB100

Concentration of LB100 in glioma tumor tissue when a known non-toxic dose of LB100 is delivered intravenously. Glioma tissue sampling to detect and quantify LB100 will be performed by assaying phospho-protein expression in glioma tissue resected prior to and after infusion of LB100. These are values for the LB100 in the tumor which was done at one timepoint.

Time frame:: average of 5.5 hours after infusion
Reported as:: Median · nM

Concentration of LB100
nM	1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
Concentration of LB100	0.18 (0 to 0.67)

SecondaryChanges in Phospho-protein Expression in Circulating Peripheral Blood Mononuclear Cells (PBMC)

Changes in phospho-protein expression in circulating PBMC. Pharmacodynamic (PD) response is defined as statistically significant elevation of phospho-protein expression in treated tumor tissues compared to untreated glioma specimens. Post-treatment PD effect (as measured by increase in tumor tissue phospho-protein expression) difference greater than 2.5 times the baseline standard deviation (SD) is statistically significant at the .05 significance level.

Time frame:: Pre-dose; End of infusion (2 hours post-start); 30 minutes post LB100 infusion completion; 1 hour post LB-100 infusion completion; 2 hours post LB100 infusion completion; 4 hours post LB100 infusion completion; 8 hours post LB100 infusion completion

No measurements were reported for this outcome.

SecondaryMaximum Observed Plasma Concentration of LB100 (Cmax)

The maximum observed analyte concentration in serum was reported.

Time frame:: Pre-dose; End of infusion (2 hours post-start); 30 minutes post LB100 infusion completion; 1 hour post LB-100 infusion completion; 2 hours post LB100 infusion completion; 4 hours post LB100 infusion completion; 8 hours post LB100 infusion completion
Reported as:: Median · ng/mL

Maximum Observed Plasma Concentration of LB100 (Cmax)
ng/mL	1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
Maximum Observed Plasma Concentration of LB100 (Cmax)	156 (95 to 207)

SecondaryArea Under the Plasma Concentration vs. Time Curve Extrapolated to Infinity (AUC(INF)

AUC is a measure of the serum concentration of LMB100 over time. It is used to characterize drug absorption.

Time frame:: Pre-dose; End of infusion (2 hours post-start); 30 minutes post LB100 infusion completion; 1 hour post LB-100 infusion completion; 2 hours post LB100 infusion completion; 4 hours post LB100 infusion completion; 8 hours post LB100 infusion completion
Reported as:: Median · hr*ng/mL

Area Under the Plasma Concentration vs. Time Curve Extrapolated to Infinity (AUC(INF)
hr*ng/mL	1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
Area Under the Plasma Concentration vs. Time Curve Extrapolated to Infinity (AUC(INF)	438 (319 to 585)

SecondaryPlasma Half-Life of Drug LB100

Plasma decay half-life is the time measured for the plasma concentration of the drug to decrease by one half.

Time frame:: Pre-dose; End of infusion (2 hours post-start); 30 minutes post LB100 infusion completion; 1 hour post LB-100 infusion completion; 2 hours post LB100 infusion completion; 4 hours post LB100 infusion completion; 8 hours post LB100 infusion completion
Reported as:: Median · Hours

Plasma Half-Life of Drug LB100
Hours	1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
Plasma Half-Life of Drug LB100	1.20 (1.09 to 1.46)

SecondaryTime to Maximum Observed Plasma Concentration of LMB100 (Tmax)

Time to maximum observed plasma concentration of LMB100 (Tmax).

Time frame:: Pre-dose; End of infusion (2 hours post-start); 30 minutes post LB100 infusion completion; 1 hour post LB-100 infusion completion; 2 hours post LB100 infusion completion; 4 hours post LB100 infusion completion; 8 hours post LB100 infusion completion
Reported as:: Median · Hours

Time to Maximum Observed Plasma Concentration of LMB100 (Tmax)
Hours	1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
Time to Maximum Observed Plasma Concentration of LMB100 (Tmax)	2.10 (2.0 to 2.63)

SecondaryTotal Clearance (CL) of LMB100

The CL is a quantitative measure of the rate at which a drug substance is removed from the body.

Time frame:: Pre-dose; End of infusion (2 hours post-start); 30 minutes post LB100 infusion completion; 1 hour post LB-100 infusion completion; 2 hours post LB100 infusion completion; 4 hours post LB100 infusion completion; 8 hours post LB100 infusion completion
Reported as:: Median · L/hr

Total Clearance (CL) of LMB100
L/hr	1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
Total Clearance (CL) of LMB100	10.4 (7.13 to 14.7)

SecondaryVolume of Distribution (Vd) of LMB100

Volume of distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug.

Time frame:: Pre-dose; End of infusion (2 hours post-start); 30 minutes post LB100 infusion completion; 1 hour post LB-100 infusion completion; 2 hours post LB100 infusion completion; 4 hours post LB100 infusion completion; 8 hours post LB100 infusion completion
Reported as:: Median · L

Volume of Distribution (Vd) of LMB100
L	1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
Volume of Distribution (Vd) of LMB100	20.1 (12.2 to 31.3)

SecondaryBrain Concentration

Drug amount in brain as a percent of drug in plasma.

Time frame:: Pre-dose; End of infusion (2 hours post-start); 30 minutes post LB100 infusion completion; 1 hour post LB-100 infusion completion; 2 hours post LB100 infusion completion; 4 hours post LB100 infusion completion; 8 hours post LB100 infusion completion
Reported as:: Median · percent

Brain Concentration
percent	1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
Brain Concentration	0.30 (0.0 to 0.45)

SecondaryBrain Penetration

Concentration of drug in brain tumor tissue. Brain penetration is measured as "drug amount in a brain as a percent of drug in plasma.

Time frame:: Pre-dose; End of infusion (2 hours post-start); 30 minutes post LB100 infusion completion; 1 hour post LB-100 infusion completion; 2 hours post LB100 infusion completion; 4 hours post LB100 infusion completion; 8 hours post LB100 infusion completion
Reported as:: Median · Percent

Brain Penetration
Percent	1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
Brain Penetration	0.26 (0 to 0.7)

Other pre-specifiedNumber of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)

Here is the number of participants with serious and/or non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned.

Time frame:: Date treatment consent signed to date off study, approximately 39 months and 6 days.
Reported as:: Count of participants · Participants

Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)
Participants	1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)	7

Adverse events

Collected over Date treatment consent signed to date off study, approximately 39 months and 6 days.. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma	0/7 (0%)	1/7 (14.3%)	7/7 (100%)

Most frequent serious events

Most frequent serious events
Event	1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
Back painMusculoskeletal and connective tissue disorders	1/7
FeverGeneral disorders	1/7
MyalgiaMusculoskeletal and connective tissue disorders	1/7
NauseaGastrointestinal disorders	1/7

Most frequent other events

Showing 10 of 15

Most frequent other events
Event	1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
HeadacheNervous system disorders	5/7
HypophosphatemiaMetabolism and nutrition disorders	3/7
AgitationPsychiatric disorders	2/7
HiccupsRespiratory, thoracic and mediastinal disorders	2/7
HypomagnesemiaMetabolism and nutrition disorders	2/7
Back painMusculoskeletal and connective tissue disorders	1/7
DysphasiaNervous system disorders	1/7
Ear and labyrinth disorders - Other, impacted cerumenEar and labyrinth disorders	1/7
Ear painEar and labyrinth disorders	1/7
Facial muscle weaknessNervous system disorders	1/7

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)	1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
<=18 years	0
Between 18 and 65 years	7
>=65 years	0

Age, Continuous
Age, Continuous(years)	1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
Mean	57.17 ± 7.54

Sex: Female, Male
Sex: Female, Male(Participants)	1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
Female	1
Male	6

Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)	1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
Hispanic or Latino	0
Not Hispanic or Latino	7
Unknown or Not Reported	0

Race (NIH/OMB)
Race (NIH/OMB)(Participants)	1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
American Indian or Alaska Native	0
Asian	0
Native Hawaiian or Other Pacific Islander	0
Black or African American	0
White	7
More than one race	0
Unknown or Not Reported	0

Region of Enrollment
Region of Enrollment(participants)	1/LB100 Protein Phosphatase 2A Inhibitor, in Recurrent Glioblastoma
United States	7

Study locations

1 site

National Institutes of Health Clinical Center
Bethesda, Maryland 20892, United States

References and documents

Study documents

Protocol and statistical analysis plan · Jun 2, 2022
Informed consent form · Jul 13, 2022

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

Individual participant data

Plan to share: Yes — All individual participant data (IPD) recorded in the medical record will be shared with intramural investigators upon request.

Supporting information: Study protocol, Statistical analysis plan, Informed consent form

Time frame: Clinical data available during the study and indefinitely.

Access criteria: Clinical data will be made available via subscription to Biomedical Translational Research Information System (BTRIS) and with the permission of the study principal investigator (PI).

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Nov 22, 2023, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT03027388
Lead sponsor: National Cancer Institute (NCI)
Responsible party: Eric Burton (Principal Investigator, National Cancer Institute (NCI)) — Principal investigator
First posted: Jan 23, 2017
Start date: Jan 9, 2019
Primary completion: Aug 15, 2022
Completion: Aug 31, 2022
Results posted: Nov 22, 2023
Last update: Nov 22, 2023

Study contacts

Eric C Burton, M.D.

principal investigator · National Cancer Institute (NCI)

Oversight

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

View the source record on ClinicalTrials.gov ↗

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