• Decrease font size
  • Return font size to normal
  • Increase font size
U.S. Department of Health and Human Services

Post-Approval Studies (PAS) Database

  • Print
  • Share
  • E-mail
-

The FDA has the authority to require sponsors to perform a post-approval study (or studies) at the time of approval of a premarket approval (PMA), humanitarian device exemption (HDE), or product development protocol (PDP) application. Post-approval studies can provide patients, health care professionals, the device industry, the FDA and other stakeholders information on the continued safety and effectiveness (or continued probable benefit, in the case of an HDE) of approved medical devices. This database allows you to search Post-Approval Study information by applicant or device information.

Learn more...


           

Registry Study for Optune System


Suggest Enhancement / Report Issue | export reports to excelExport to Excel
General
Study Status Completed
Application Number P100034 / PAS002
Date Current Protocol Accepted 02/09/2016
Study Name Registry Study for Optune System
General Study Protocol Parameters
Study Design Prospective & Retrospective Study
Data Source Sponsor Registry
Comparison Group Historical Control
Analysis Type Analytical
Study Population Adult: >21
Detailed Study Protocol Parameters
Study Design Description The study is a new enrollment, non-randomized, open label, post-approval registry study of Optunein recurrent glioblastoma multiforme (GBM) patients. Treatment group is prospectively enrolled, the historica control ground is from the IDE study.

Primary objective:

To confirm that the efficacy of Optunein patients with recurrent GBM treated in a real life setting following approval is comparable to that of Best Standard of Care (BSC) chemotherapy patients in the pivotal trial.

Secondary objective:

•To collect additional data on the safety profile of the Optune in a real life setting

•To define Optune overall survival (months) by MGMT methylation status (where available).

•To define Optune overall survival (months) by baseline MMSE score (where available)

•To compare time to treatment failure (months) on Optune to that of BSC chemotherapy patients.

•To evaluate Optune functional impairment using Karnofsky Performance Score (KPS) (where available)

Study Population Description The treatment group will consist of 192 patients who will receive Optune monotherapyuntil clinical disease progression. Minimal recommended treatment duration – 4 weeks. Patients in the Optune group will be encouraged to use the device for at least 18 hours a day(monthly average). Patients will be enrolled through the PRiDe registry, sponsored by Novocure.

Best standard of care chemotherapy (control) group from the EF-11 pivotal study control group. The control arm consisted of the 117 BSC group patients. These patients were treated with the best standard of care chemotherapy available in the US at the time of the study.

Inclusion Criteria:

•22 years of age or older

•Histological diagnosis of GBM (WHO grade IV)

•Tumor located in the supra-tentorial region of the brain

•Received maximal, safe, surgical resection

•Received maximal radiation therapy (45-70Gy)

•Received concomitant Temozolomide (75mg/m^2/day for 6 weeks)

•Received maintenance Temozolomide (150-200 mg/m^2 daily for 5 days followed by 23 days without treatment for 6 cycles or until disease progression)

•Any recurrence (based on radiological or histological evidence of recurrence)

•Karnofsky performance score 70 or above

•Women of childbearing age must be on effective contraception

•Signed consent to use PHI in the Optune registry

Exclusion Criteria

•Implanted electronic medical device in the brain:

oDeep brain stimulator

oVagus nerve stimulator

oProgrammable shunt

•Skull defect without replacement

•Receiving concomitant chemotherapy

•Unable to comply with treatment with Optune

•Pregnant

•Actively participating in another therapeutic clinical trial

•Radiological suspicion of pseudoprogression or radionecrosis (a cold PET scan or negative biopsy are required in order to rule out these conditions if radiological suspicion exists)

•Any serious co-morbidity which is expected to affect survival more adversely than GBM

Sample Size Treatment arm: N1=192 Optunepatients

Comparator arm: N2=117 best standard of care control patients from the EF-11 pivotal study

Assumptions: ¿2 = 0.1083 (hazard of death in the recurrent GBM pivotal study BSC control group)

Type I error: 0.05

Power: 0.80

Test statistics: Log-rank test

Lost to follow-up: 10% in the Optune arm only

Data Collection Primary endpoint: Overall survival

Secondary endpoints:

• Adverse event incidence by body system and term,

• including:

o Incidence of seizures and headaches

• Overall survival (months) by MGMT methylation status (where available)

• Overall survival (months by baseline MMSE score (where available)

• Time to treatment failure (months)

Follow-up Visits and Length of Follow-up The range of follow-up is from 12 months to three years. Patients will be followed until death or the end of the study (up to three years). The last patient enrolled will be followed up to 12 months.



Interim or Final Data Summary
Actual Number of Patients Enrolled 192
Actual Number of Sites Enrolled Registry study with all optune certified sites in the U.S.
Patient Follow-up Rate 26 out of 192 subjects censored at 12-months follow-up. Follow-up rate is unavailable for survival study.
Final Safety Findings A total of 33 patients experienced serious adverse events (SAEs) in the Optune registry population, compared to 41 patients in the EF-11 Best Standard of Care (BSC) population, respectively. The incidence of patients with SAEs was lower in Optune registry arm compared to the EF-11 BSC arm (17% vs. 45%, respectively). No Optune-related SAEs were reported in the registry dataset.
Final Effect Findings The study used historical control, which is associated with potential limitations such as selection bias, differences in baseline characteristics, diagnosis, and outcome assessment. The length of median 12-month overall survival (OS) in the Optune registry arm was numerically longer than that in the BSC group but does not meet statistical significance (log-rank p=0.053), suggesting Optune treatment is not worse (or non-inferior) than the BSC treatment.
Study Strengths & Weaknesses Study Strength: The registry Study for Optune System is a prospective registry study with subjects enrolled from all Optune certified oncology centers in the U.S. The study has a prespecified statistical analysis plan to assess non-inferiority of the Optune treated group compared to the BSC group.

Study Weaknesses: 12-months overall survival (12- months) by baseline MMSE score is not available due to limited information on MMSE score at baseline. The study does not provide adequate evaluation on the change of KPS score overtime due to limited KPS reported at follow-up.
Recommendations for Labeling Changes Yes


Registry Study for Optune System Schedule

Report Schedule
Report
Date Due
FDA Receipt
Date
Applicant's Reporting Status
six month report 08/08/2016 08/08/2016 On Time
one year report 02/09/2017 02/08/2017 On Time
18 month report 08/08/2017 08/08/2017 On Time
two year report 02/09/2018 02/09/2018 On Time
three year report/final 02/09/2019 07/10/2019 Overdue/Received


Contact Us

Mandated Studies Program
Food and Drug Administration
10903 New Hampshire Ave.
Silver Spring, MD 20993-0002
Email: MandatedStudiesPrograms@fda.hhs.gov

Additional Resources

-
-