Clinical efficacy of Bevacizumab in the treatment of Type II Neurofibromatosis

• Register: ChiCTR
• Last refreshed on: 14 May 2018
• Main ID: ChiCTR1800016040
• Date of registration: 2018-05-08
• Prospective Registration: Yes
• Primary sponsor: Shanghai Ninth People's Hospital affiliated to Shanghai JiaoTong University, School of Medicine
• Public title: Clinical efficacy of Bevacizumab in the treatment of Type II Neurofibromatosis
• Scientific title: Clinical efficacy of Bevacizumab in the treatment of Type II Neurofibromatosis
• Date of first enrolment: 2018-05-09
• Target sample size: Case series:30;
• Recruitment status: Recruiting
• URL: http://www.chictr.org.cn/showproj.aspx?proj=25322
• Study type: Interventional study
• Study design: Before-After control
• Phase: Other

Countries of recruitment

China

Contacts

• Name: WANG Zhaoyan
• Affiliation: Shanghai Ninth People's Hospital affiliated to Shanghai JiaoTong University, School of Medicine

• Name: WANG Zhaoyan
• Affiliation: Shanghai Ninth People's Hospital affiliated to Shanghai JiaoTong University, School of Medicine

Key inclusion & exclusion criteria

Inclusion criteria: 1. Aged 6 years or greater;
2. Patients must have a confirmed diagnosis of type II neurofibromatosis;
3. Life expectancy of greater than 1 year;
4. Evidence of progressive hearing loss (with decrease in speech discrimination score) related to VS with a speech discrimination score of < 90% in the target ear;and evidence of moderate or severe or profound sensorineural hearing loss;
5. Participants must have normal organ and marrow function be defined as Leukocytes > 3,000/mcL; Absolute neutrophil count > 1,500/mcL; Platelets > 100,000/mcL; Total bilirubin within normal institutional limits; AST (SGOT)/ALT (SGPT) <2.5 X institutional upper limit of normal; Creatinine clearance=60ml/min/1.73 m2 or a normal serum creatinine; 24-hour urine protein level should be <1000 mg;
6. Subjects must have a VS not amenable to surgery or have refused surgery due to high risk for permanent complications related to surgery as determined by a surgeon with experience in management of NF2;
7. Subjects must have had a discussion of all available treatment options including surgery, radiation therapy, observation, other clinical trials and expressed their preference for participation in this trial in the informed consent process.
Exclusion criteria: 1. Patients who have had chemotherapy or radiotherapy within 4 weeks prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier;
2. Patients with known hypersensitivity of Chinese hamster ovary cell products, other recombinant human antibodies, or compounds of similar chemical or biologic composition to bevacizumab;
3. Inability to tolerate periodic MRI scans;
4. Uncontrolled intercurrent illness including ongoing or active infection, or psychiatric illness/social situations that would limit compliance with study requirements;
5. Pregnant or fertile women;
6. Participating in any other clinical trial;
7. History of cardiovascular disease,such as: Inadequately controlled HTN; History of CVA within 12 months; Myocardial infarction or unstable angina within 12 months; New York heart association grade II or greater congestive heart failure; Serious and inadequately controlled cardiac arrhythmia; Significant vascular disease (e.g., aortic aneurysm, history of aortic dissection); Clinically significant peripheral vascular disease;
8. Concurrent use of anti-coagulant drugs (not including prophylactic doses), history of coagulopathy, or evidence of bleeding diathesis or coagulopathy;
9. Imaging (CT or MRI) evidence of newly identified hemorrhage (new within the last in the 6 months prior to enrollment), any history of symptomatic intracranial hemorrhage, or any history of spontaneous intracranial hemorrhage;
10. Serious or non-healing wound, ulcer or bone fracture;
11. History of abdominal fistula, gastrointestinal perforation or intra-abdominal abscess within 6 months;
12. Invasive procedures be defined as Major surgical procedure; Brain biopsy within 28 days; Anticipation of need for major surgical procedures during the course of the study; Core biopsy within 7 days;
13. Prior treatment with bevacizumab or other VEGF targeting therapies;
14. Personal history of autoimmune coagulopathy, including idiopathic thrombocytopenia purpura (ITP);
15. Patients couldn't finish follow-up.

Age minimum: 6
Age maximum:
Gender: Both

Health Condition(s) or Problem(s) studied

Type II Neurofibromatosis

Intervention(s)

Case series:Bevacizumab therapy;

Primary Outcome(s)

Speech Discrimination Score in the target ear;

Secondary Outcome(s)

0??13??25??49??72?;Volume Decreased Rate of vestibular schwannomas in the contralateral ear;Volume Decreased Rate of other tumors;

Source(s) of Monetary Support

Self-financing

Ethics review

Status: Approved
Approval date: 16/04/2018
Contact:

Zhen Hong

[phone]-5576