Trial Outcomes & Findings for Concentration and Activity of Lapatinib in Vestibular Schwannomas (NCT NCT00863122)

NCT ID: NCT00863122

Last Updated: 2021-01-12

Results Overview

Steady-state plasma concentrations of lapatinib (ng/mL) at time of surgery, 10-13 days from starting drug.

Recruitment status

COMPLETED

Study phase

EARLY_PHASE1

Target enrollment

26 participants

Primary outcome timeframe

At time of surgery, 10-13 days from starting drug.

Results posted on

2021-01-12

Participant Flow

All patients undergoing surgery for vestibular schwannoma resection who met eligibility criteria were invited to participate

Patients could enroll on control (no drug) or lapatinib arms. 20 people were consented for the the lapatinib arm, but one patient withdrew consent prior to completion of eligibility screening and enrollment. So 19 patients enrolled on the lapatinib arm.

Participant milestones

Participant milestones
Measure
Lapatinib
Subjects received lapatinib 1500 mg by mouth for 10 days prior to surgery for vestibular schwannoma resection.
Control
Control subjects did not receive any intervention prior to surgery for vestibular schwannoma resection.
Overall Study
STARTED
19
7
Overall Study
COMPLETED
19
7
Overall Study
NOT COMPLETED
0
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Per protocol, KPS was not recorded for control participants. Control participants agreed to donate tissue only. No clinical data was collected.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Lapatinib
n=19 Participants
Subjects received lapatinib for 1500 mg by mouth daily for 10 days prior to surgery for vestibular schwannoma resection
Control
n=7 Participants
Control subjects did not receive any intervention prior to surgery for vestibular schwannoma resection
Total
n=26 Participants
Total of all reporting groups
Age, Categorical
<=18 years
0 Participants
n=19 Participants
0 Participants
n=7 Participants
0 Participants
n=26 Participants
Age, Categorical
Between 18 and 65 years
19 Participants
n=19 Participants
7 Participants
n=7 Participants
26 Participants
n=26 Participants
Age, Categorical
>=65 years
0 Participants
n=19 Participants
0 Participants
n=7 Participants
0 Participants
n=26 Participants
Age, Continuous
44 years
n=19 Participants
40 years
n=7 Participants
43.5 years
n=26 Participants
Sex: Female, Male
Female
15 Participants
n=19 Participants
6 Participants
n=7 Participants
21 Participants
n=26 Participants
Sex: Female, Male
Male
4 Participants
n=19 Participants
1 Participants
n=7 Participants
5 Participants
n=26 Participants
Region of Enrollment
United States
19 participants
n=19 Participants
7 participants
n=7 Participants
26 participants
n=26 Participants
Karnofsky Performance Status (KPS)
90 units on a scale (percent)
n=19 Participants • Per protocol, KPS was not recorded for control participants. Control participants agreed to donate tissue only. No clinical data was collected.
90 units on a scale (percent)
n=19 Participants • Per protocol, KPS was not recorded for control participants. Control participants agreed to donate tissue only. No clinical data was collected.
Patients with a diagnosis of NF2
9 participants
n=19 Participants
0 participants
n=7 Participants
9 participants
n=26 Participants

PRIMARY outcome

Timeframe: At time of surgery, 10-13 days from starting drug.

Population: In the lapatinib group, 10 tissue and plasma samples were lost from analysis due to a freezer failure during a natural disaster denaturing the samples. 1 blood sample was contaminated, but tissue was available. Control group did not get any drug. A total of 8 participants of 19 given drug had data for analysis.

Steady-state plasma concentrations of lapatinib (ng/mL) at time of surgery, 10-13 days from starting drug.

Outcome measures

Outcome measures
Measure
Lapatinib
n=8 Participants
Subjects received lapatinib 1500 mg by mouth for 10 days prior to surgery for vestibular schwannoma resection
Control
Control subjects will not receive any intervention prior to surgery for vestibular schwannoma resection. Control subjects donate tissue at the time of surgery.
Median Steady-state Lapatinib Plasma Concentrations at the Time of Surgical Resection
3149 ng/mL
Interval 786.0 to 5830.0

PRIMARY outcome

Timeframe: one year

Population: In the lapatinib group, 10 tissue and plasma samples were lost from analysis due to a freezer failure during a natural disaster denaturing the samples. Nine samples were available for tissue concentration assessment.

Count of tissue samples with lapatinib concentration \>3uM

Outcome measures

Outcome measures
Measure
Lapatinib
n=9 Participants
Subjects received lapatinib 1500 mg by mouth for 10 days prior to surgery for vestibular schwannoma resection
Control
Control subjects will not receive any intervention prior to surgery for vestibular schwannoma resection. Control subjects donate tissue at the time of surgery.
To Assess Whether Lapatinib Can Reach a Minimum Tumor Concentration Level of >3uM in VS After Oral Dosing.
7 Participants

SECONDARY outcome

Timeframe: at time of surgery

Population: 10 lapatinib participants and 3 control participants did not have adequate tissue for analysis.

Assessed number of samples with high expression of phospho-ErbB2 in tissue at time of surgery

Outcome measures

Outcome measures
Measure
Lapatinib
n=9 Participants
Subjects received lapatinib 1500 mg by mouth for 10 days prior to surgery for vestibular schwannoma resection
Control
n=4 Participants
Control subjects will not receive any intervention prior to surgery for vestibular schwannoma resection. Control subjects donate tissue at the time of surgery.
Assess the Level of ErbB2 Phosphorylation in VS.
5 Participants
2 Participants

SECONDARY outcome

Timeframe: At time of surgery

Population: The experiment to assess this outcome measure failed and no interpretable data could be collected.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: one year

Population: Of the nine participants in the lapatinib who had tumor available for analysis, four had a vestibular schwnannoma associated with a diagnosis of neurofibromatosis type 2 (NF2); five had sporadic vestibular schwannomas.

A comparison in the median lapatinib concentration (ng/g) in vestibular schwannomas associated with neurofibromatosis type 2 and sporadic vestibular schwannomas

Outcome measures

Outcome measures
Measure
Lapatinib
n=4 Participants
Subjects received lapatinib 1500 mg by mouth for 10 days prior to surgery for vestibular schwannoma resection
Control
n=5 Participants
Control subjects will not receive any intervention prior to surgery for vestibular schwannoma resection. Control subjects donate tissue at the time of surgery.
Explore the Difference in the Concentration of Lapatinib Achieved in NF2-related Versus Idiopathic VS.
8367 ng/g
Interval 314.0 to 17821.0
4502 ng/g
Interval 1737.0 to 11712.0

SECONDARY outcome

Timeframe: at time of surgery

Population: 6 of the 7 participants had sporadic VS; 1 of 7 had NF2-associated VS. As such, we were unable to explore differences between the groups.

Due to the sample sizes, a comparison between sporadic and NF2-related vestibular schwannomas could not be made. Instead we report the mutational status.

Outcome measures

Outcome measures
Measure
Lapatinib
n=7 Participants
Subjects received lapatinib 1500 mg by mouth for 10 days prior to surgery for vestibular schwannoma resection
Control
Control subjects will not receive any intervention prior to surgery for vestibular schwannoma resection. Control subjects donate tissue at the time of surgery.
Perform NF2 Gene Mutation Analysis Via Exon Scanning and MLPA as Well as Protein Expression in All VS and Explore Differences Between Sporadic and NF2 Related VS.
Hemizygous deletion in NF2 gene
4 Participants
Perform NF2 Gene Mutation Analysis Via Exon Scanning and MLPA as Well as Protein Expression in All VS and Explore Differences Between Sporadic and NF2 Related VS.
Homozygous deletion in NF2 gene
1 Participants
Perform NF2 Gene Mutation Analysis Via Exon Scanning and MLPA as Well as Protein Expression in All VS and Explore Differences Between Sporadic and NF2 Related VS.
No deletion in NF2 gene
2 Participants

Adverse Events

Lapatinib

Serious events: 0 serious events
Other events: 12 other events
Deaths: 0 deaths

Control

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Lapatinib
n=19 participants at risk
Subjects received lapatinib 1500 mg by mouth for 10 days prior to surgery for vestibular schwannoma resection.
Control
Control subjects will not receive any intervention prior to surgery for vestibular schwannoma resection. Control subjects donate tissue at the time of surgery. AEs were not collected in this group as there was no study intervention (i.e. they did not take the drug).
Skin and subcutaneous tissue disorders
Rash
31.6%
6/19 • Number of events 6
Zero participants were At Risk from the Control arm since they did not receive any intervention.
0/0
Zero participants were At Risk from the Control arm since they did not receive any intervention.
Gastrointestinal disorders
Diarrhea
36.8%
7/19 • Number of events 7
Zero participants were At Risk from the Control arm since they did not receive any intervention.
0/0
Zero participants were At Risk from the Control arm since they did not receive any intervention.
General disorders
Fatigue
21.1%
4/19 • Number of events 4
Zero participants were At Risk from the Control arm since they did not receive any intervention.
0/0
Zero participants were At Risk from the Control arm since they did not receive any intervention.
Gastrointestinal disorders
Nausea
21.1%
4/19 • Number of events 4
Zero participants were At Risk from the Control arm since they did not receive any intervention.
0/0
Zero participants were At Risk from the Control arm since they did not receive any intervention.
Skin and subcutaneous tissue disorders
Acne
10.5%
2/19 • Number of events 2
Zero participants were At Risk from the Control arm since they did not receive any intervention.
0/0
Zero participants were At Risk from the Control arm since they did not receive any intervention.

Additional Information

Jaishri Blakeley

Johns Hopkins School of Medicine

Phone: 410-955-6827

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place

Restriction type: LTE60