Endostatin Study for Patients With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors

Phase 2

Completed

• Conditions: Vestibular Schwannoma; Neurofibromatosis Type 2
• Interventions: Drug: Endostatin

• Registration Number: NCT02104323
• Lead Sponsor: Beijing Tiantan Hospital

Brief Summary

1)Preliminarily evaluate the treatment effect of continuous vein injection of recombinant human endostatin on NF2; 2)Preliminarily evaluate the safety and the patient's tolerance of the treatment of endostatin; 3)Provide an objective basis for an enlarged randomized double-blind trial.

Detailed Description

Neurofibromatosis type 2 (NF2) is a hereditary tumor predisposition syndrome caused by mutations in the NF2 tumor suppressor gene. Individuals with NF2 have a higher likelihood to develop multiple nervous system tumors, including schwannomas, meningiomas, and ependymomas. The hallmark of NF2 is bilateral vestibular schwannomas. Historically, most NF2 patients experience complete hearing loss either from tumor progression or after treatment of the tumors with surgery or radiation. Effective treatments are urgently needed for NF2 patients with progressive hearing loss because hearing loss is associated with impairment in social, emotional,and communication function and with increased depression.

Previous studies of NF2 patients treated with bevacizumab suggested that inhibition of vascular endothelial growth factor (VEGF) could result in hearing improvement and reduction in tumor size.Recombinant human endostatin can inhibit the formation of blood vessels by inhibiting the migration of vascular endothelial cells .In this way, endostatin can inhibit the nutrition of the tumor and decrease the tumor's growth and metastasis. In vitro tests, endostatin can inhibit the cell migration and Tube formation of the microvascular endothelial cell line HHEC. Besides, it can inhibit blood vessels' formation of the chicken embryo sac membrane. Based on these studies, we perform this clinical trial to known the treatment effect and tolerability of endostatin on NF2.

Study Timeline

• Study Start: 2014-01
• Study First Submitted: 2014-03-26
• Study First Submitted QC: 2014-04-02
• Study First Posted: 2014-04-04
• Primary Completion: 2016-04
• Study Completion: 2016-04
• Status Verified: 2017-02
• Last Update Submitted: 2017-02-07
• Last Update Posted: 2017-02-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

• 1)Patients must be at the age of 16-30
• 2)Patients must meet the diagnostic criteria for NF2, with bilateral acoustic neuroma and other central nervous system tumors
• 3)Patients must not be treated with other drugs or radiation therapy recently
• 4)Patients should live in Beijing or nearby and can be treated in hospital
• 5)Patients must be healthy and not be seriously allergic with biological agents
• 6)Patients must join the clinical trial voluntarily, with good compliance, cooperate with the researchers well, sign a written informed consent.

Exclusion Criteria

• 1)Treated with other drugs, surgery or radiation therapy recently
• 2)Brainstem is compressed seriously, with hydrocephalus, need to be treated with surgery in short time
• 3)Being pregnant or try to get pregnant, lactating women
• 4)With acute or chronic infectious diseases
• 5)With heart diseases, cardiac dysfunction or abnormal ECG
• 6)With uncontrolled neural or mental diseases, poor compliance
• 7)Not available for enhanced MRI
• 8)Take part in any other clinical trial
• 9)With other conditions that are considered not suitable for this clinical trial.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Endostatin，treatment effect evaluation	Endostatin	Patients receive continuous intravenous Endostatin drug pumping during the course of treatment. The drug dosage is 7.5mg/m2/d. Every course of treatment lasts three months. Patients are designed to receive total three courses of treatment if there is no disease progression. The interval between two courses is one month.

Primary Outcome Measures

Name	Time	Method
Change from Baseline in volume of tumour after every course of the treatment	Baseline,Month 3,Month 7,Month 11	Patients in this clinical trial would receive MRI test to evaluate the volume of tumour after every course of the treatment.

Secondary Outcome Measures

Name	Time	Method
Change from Baseline in hearing ability after every course of the treatment	Baseline,Month 3,Month 7,Month 11	Patients' hearing ability would be tested after every course of the treatment.

Trial Locations

Locations (1)

Beijing Tiantan Hospital Affiliated to Capital Medical University; 🇨🇳 Beijing, Beijing, China