Phase 2
N=21
Lapatinib Study for Children and Adults With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors
Neurofibromatosis 2 · Vestibular Schwannoma
Bottom Line
View on ClinicalTrials.gov: NCT00973739 ↗Enrolled (actual)
21
Serious AEs
0.0%
Results posted
Mar 2016
Primary outcome: Primary: Estimated Volumetric Progression Free Survival at 12 Months — 70.6 Liklihood of PFS at 12 months
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Lapatinib (Drug)
- Age
- Pediatric, Adult, Older Adult · 4+ yrs
- Sex
- All
- Sponsor
- NYU Langone Health
- Primary completion
- Oct 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Estimated Volumetric Progression Free Survival at 12 Months |
70.6 | — |
| SECONDARY Estimated Volumetric Progression Free Survival for Hearing at 12 Months |
88.9 | — |
| SECONDARY Participants Experiencing Grades 1 or 2 Toxicities (CTCAE) |
21 | — |
| SECONDARY Participants Experiencing Grade 3 Toxicities (CTCAE) |
1 | — |
Summary
The purpose of this study is to determine if Lapatinib has any effect on tumors found in patients with Neurofibromatosis Type 2 (NF2). NF2 is a condition that mainly affects the skin and nervous system. It causes non-cancerous tumors (which are known as neuromas) to grow on the nerves around a person's body. Some signs of NF2 include a gradual loss of hearing and tumors growing on the skin, the brain and the spinal cord which can lead to complications.
Lapatinib is an oral drug that is approved by Food and Drug Administration (FDA) for other types of tumors, it is not approved by the FDA for treatment of NF2 related tumors. The investigators know a lot about how well it is tolerated, but the investigators do not know if it is effective in treating your condition, therefore it is considered to be an investigational medication. This study will test whether Lapatinib may shrink tumors commonly found in patients with NF2 or stop them from growing. This will help us to decide if Lapatinib should be used to treat NF2 patients in future. Lapatinib is a drug that has been used for over 10 years to treat various forms of cancer. It has not been studied for the treatment of tumors in NF2 patients.
Eligibility Criteria
Inclusion Criteria
- Patients must be at least 4 years of age.
- Patients must meet diagnostic criteria for NF2 and at least one volumetrically measured NF2-related brain or spinal tumor with radiographic evidence of progression over the past 12 months, designated as the primary target OR volumetrically measurable VS with ipsilateral progressive hearing loss over the past 12 months, designated as the primary target tumor.
- Significant hearing loss criteria for enrollment.
- Karnofsky (PS) OR Lansky 50-100% (>16 years of age)
- Absolute neutrophil count ≥ 1,000/mm3 g/dL
- Hemoglobin ≥ 8 g/dL
- Creatinine ≤ 1.5 times upper limit of normal (ULN) OR corrected glomerular filtration rate ≥ 70 ml/min
- Bilirubin ≤ 1.5 times ULN
- ALT ≤ 2.5 times ULN
- Fully recovered from acute toxic effects of any prior chemotherapy, biological modifiers or radiotherapy.
- Steroids are allowed for progressive symptoms but patient must be on a stable dose for at least 1 week prior to study entry.
- Any neurologic deficits must be stable for ≥ 1 week.
- Patients with the potential for pregnancy or impregnating their partner must agree to follow acceptable birth control methods to avoid conception. Women of childbearing potential must have a negative pregnancy test. The anti-proliferative activity of this experimental drug may be harmful to the developing fetus.
- Normal cardiac left ventricular ejection fraction (LVEF) by transthoracic echocardiogram.
- Able to provide written informed consent (or consent by parent/legal guardian for minors)
Exclusion Criteria
- Patients with serious concurrent infection or medical illness.
- Neurological deficits that are rapidly progressing.
- Patients who are pregnant or breast-feeding.
- Anti-tumor therapy within 4 weeks prior to enrollment.
- Radiation therapy within 2 months prior to enrollment.
- Prior therapy with agents targeting EGFR or ErbB2.
- Any surgery within 4 weeks prior to enrollment.
- Significant gastrointestinal disorder(s)
- Known cardiac disease
- Patients with a concurrent or prior malignancy are ineligible unless they are patients with curatively treated carcinoma-in-situ or basal cell carcinoma of the skin. Patients who have been free of disease (any prior malignancy) for more than five years are eligible for this study.
- Patients cannot have received cytochrome P450-inducing anticonvulsants (EIADs; e.g., phenytoin, carbamazepine, phenobarbital, primidone, oxcarbazepine) or similar agents (e.g., rifampin) or P450-inhibiting agents (Ketoconazole, Itraconazole, Clarithromycin, Atazanavir, Indinavir, Nefazodone, Nelfinavir, Ritonavir, Saquinavir, Telithromycin, Voriconazole)
Data sourced from ClinicalTrials.gov (NCT00973739). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.