Trial Outcomes & Findings for Lapatinib Study for Children and Adults With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors (NCT NCT00973739)

Last Updated: 2016-03-22

Results Overview

Measurements were taken every three months, up to one year. Estimated volumetric progression free survival (PFS) was measured from date of enrollment to date of volumetric progression. PFS was analyzed using the Kaplan-Meier method in terms of overall PFS (volumetric or hearing progression), volumetric progression, and hearing progression. Point estimates for PFS with 95% confidence intervals (CIs) were calculated from Kaplan-Meier curves.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

21 participants

Primary outcome timeframe

Every three months for one year

Results posted on

2016-03-22

Participant Flow

Twenty-one patients were enrolled between October 2009 and March 2011 at New York University Medical Center.

Participant milestones

Participant milestones
Measure	Lapatinib Lapatinib will be administered
Overall Study STARTED	21
Overall Study COMPLETED	17
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Lapatinib Study for Children and Adults With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Lapatinib n=21 Participants Lapatinib will be administered
Age, Categorical <=18 years	4 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	17 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	28 years STANDARD_DEVIATION 5 • n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	13 Participants n=5 Participants
Region of Enrollment United States	21 participants n=5 Participants

PRIMARY outcome

Timeframe: Every three months for one year

Population: This analysis is based on the 17 evaluable patients.

Outcome measures

Outcome measures
Measure	Lapatinib n=17 Participants Lapatinib PO dosed according to age: Children/adolescents (less than 18 years of age): 1,800 mg/m2/day PO divided into twice daily doses, to a maximum of 750 mg PO twice daily Adults (18 years of age or older): 1,500 mg PO once daily Lapatinib is available in 250 mg tablets only. For pediatric dosing, the total daily dose will be rounded up or down to the nearest 250 mg increment.
Estimated Volumetric Progression Free Survival at 12 Months	70.6 Liklihood of PFS at 12 months Interval 43.1 to 86.6

SECONDARY outcome

Timeframe: Every three months for one year

Population: This analysis is based on the 17 evaluable patients.

Measurements were taken every three months, up to one year. Estimated volumetric progression free survival (PFS) was measured from date of enrollment to date of hearing progression. PFS was analyzed using the Kaplan-Meier method in terms of overall PFS (volumetric or hearing progression), volumetric progression, and hearing progression. Point estimates for PFS with 95% confidence intervals (CIs) were calculated from Kaplan-Meier curves.

Outcome measures

Outcome measures
Measure	Lapatinib n=17 Participants Lapatinib PO dosed according to age: Children/adolescents (less than 18 years of age): 1,800 mg/m2/day PO divided into twice daily doses, to a maximum of 750 mg PO twice daily Adults (18 years of age or older): 1,500 mg PO once daily Lapatinib is available in 250 mg tablets only. For pediatric dosing, the total daily dose will be rounded up or down to the nearest 250 mg increment.
Estimated Volumetric Progression Free Survival for Hearing at 12 Months	88.9 Liklihood of PFS at 12 months Interval 43.3 to 98.4

SECONDARY outcome

Timeframe: Baseline through one year

Toxicity was assessed throughout the study, up to one year.

Outcome measures

Outcome measures
Measure	Lapatinib n=21 Participants Lapatinib PO dosed according to age: Children/adolescents (less than 18 years of age): 1,800 mg/m2/day PO divided into twice daily doses, to a maximum of 750 mg PO twice daily Adults (18 years of age or older): 1,500 mg PO once daily Lapatinib is available in 250 mg tablets only. For pediatric dosing, the total daily dose will be rounded up or down to the nearest 250 mg increment.
Participants Experiencing Grades 1 or 2 Toxicities (CTCAE)	21 participants

SECONDARY outcome

Timeframe: Baseline through one year

Toxicity was assessed throughout the study, up to one year.

Outcome measures

Outcome measures
Measure	Lapatinib n=21 Participants Lapatinib PO dosed according to age: Children/adolescents (less than 18 years of age): 1,800 mg/m2/day PO divided into twice daily doses, to a maximum of 750 mg PO twice daily Adults (18 years of age or older): 1,500 mg PO once daily Lapatinib is available in 250 mg tablets only. For pediatric dosing, the total daily dose will be rounded up or down to the nearest 250 mg increment.
Participants Experiencing Grade 3 Toxicities (CTCAE)	1 participants

Adverse Events

Lapatinib

Serious events: 0 serious events

Other events: 21 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Lapatinib n=21 participants at risk Lapatinib will be administered
Nervous system disorders Vasovagal syncope	4.8% 1/21 • Number of events 1
Blood and lymphatic system disorders Anemia	23.8% 5/21 • Number of events 5
Metabolism and nutrition disorders Anorexia	9.5% 2/21 • Number of events 2
Psychiatric disorders Anxiety	9.5% 2/21 • Number of events 2
Nervous system disorders Ataxia	4.8% 1/21 • Number of events 1
Gastrointestinal disorders Constipation	4.8% 1/21 • Number of events 1
Metabolism and nutrition disorders Decreased bicarbonate	23.8% 5/21 • Number of events 5
Skin and subcutaneous tissue disorders Dermatology/Skin (Other)	90.5% 19/21 • Number of events 19
Gastrointestinal disorders Diarrhea	71.4% 15/21 • Number of events 15
Respiratory, thoracic and mediastinal disorders Dyspnea	4.8% 1/21 • Number of events 1
Respiratory, thoracic and mediastinal disorders Epistaxis	19.0% 4/21 • Number of events 4
General disorders Fatigue	42.9% 9/21 • Number of events 9
General disorders Fever	4.8% 1/21 • Number of events 1
Gastrointestinal disorders heartburn	4.8% 1/21 • Number of events 1
Gastrointestinal disorders Hemorrhage - rectum (stools)	4.8% 1/21 • Number of events 1
Investigations Hyperbilirubinemia	9.5% 2/21 • Number of events 2
Metabolism and nutrition disorders Hyperglycemia	42.9% 9/21 • Number of events 9
Metabolism and nutrition disorders Hyperkalemia	9.5% 2/21 • Number of events 2
Metabolism and nutrition disorders Hypernatremia	9.5% 2/21 • Number of events 2
Vascular disorders Hypertension	4.8% 1/21 • Number of events 1
Metabolism and nutrition disorders Hypocalcemia	9.5% 2/21 • Number of events 2
Metabolism and nutrition disorders Hypoglycemia	14.3% 3/21 • Number of events 3
Metabolism and nutrition disorders Hypokalemia	23.8% 5/21 • Number of events 5
Metabolism and nutrition disorders Hypophosphatemia	19.0% 4/21 • Number of events 4
Investigations Increased ALT	19.0% 4/21 • Number of events 4
Investigations Increased AST	33.3% 7/21 • Number of events 7
Investigations Increased PTT	4.8% 1/21 • Number of events 1
Psychiatric disorders Insomnia	9.5% 2/21 • Number of events 2
Eye disorders Keratitis	9.5% 2/21 • Number of events 2
Blood and lymphatic system disorders Lymphopenia	23.8% 5/21 • Number of events 5
Nervous system disorders Memory Impairment	9.5% 2/21 • Number of events 2
Gastrointestinal disorders Nausea	19.0% 4/21 • Number of events 4
Nervous system disorders Numbness L-S area	4.8% 1/21 • Number of events 1
General disorders Pain-Other	38.1% 8/21 • Number of events 8
Cardiac disorders Pericardial effusion on echocardiogram	4.8% 1/21 • Number of events 1
Cardiac disorders Possible ectopic atrial bradycardia on EKG	4.8% 1/21 • Number of events 1
Cardiac disorders Premature supraventricular complexes on EKG	4.8% 1/21 • Number of events 1
Cardiac disorders Sinus Bradycardia	9.5% 2/21 • Number of events 2
Skin and subcutaneous tissue disorders Stomatitis (oral cavity ulceration)	4.8% 1/21 • Number of events 1
Gastrointestinal disorders Vomiting	14.3% 3/21 • Number of events 3

Additional Information

Matthias Karajannis, MD

New York University Langone Medical Center

Phone: 212-263-8400

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place