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

Sponsor

Beijing Tiantan Hospital (Other)

Overall Status

Completed

CT.gov ID

NCT02104323

Collaborator

Shandong Simcere-Medgenn Bio-pharmaceutical Co., Ltd (Industry)

Enrollment

Location

Arm

Duration (Months)

0.7

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Vestibular Schwannoma Neurofibromatosis Type 2	Drug: Endostatin	Phase 2

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 Design

Study Type:

Interventional

Actual Enrollment :

20 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Recombinant Human Endostatin Injection Study for Patients With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors by Continuous Intravenous Pumping

Study Start Date :

Jan 1, 2014

Actual Primary Completion Date :

Apr 1, 2016

Actual Study Completion Date :

Apr 1, 2016

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Endostatin，treatment effect evaluation 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.	Drug: Endostatin Method of drug administration:continuous intravenous pumping; Dosage: 7.5mg/m2/d; Course of treatment: 3 months;Total three treatment courses. Other Names: Recombinant Human Endostatin

Arm

Intervention/Treatment

Experimental: Endostatin，treatment effect evaluation

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.

Drug: Endostatin

Method of drug administration:continuous intravenous pumping; Dosage: 7.5mg/m2/d; Course of treatment: 3 months;Total three treatment courses.

Other Names:

Recombinant Human Endostatin

Outcome Measures

Primary Outcome Measures

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

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.

Other Outcome Measures

Change from baseline in QOL(quality of life) score after every course of the treatment [Baseline,Month 3,Month 7,Month 11]
Patients' QOL(quality of life) would be evaluated after every course of the treatment.

Eligibility Criteria

Criteria

Ages Eligible for Study:

16 Years to 30 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.