Chiari Surgical Outcomes Trial PI: Bermans J. Iskandar and Timothy George with collaboration from John Kestle

1
Chiari Surgical Outcomes TrialPI Bermans J.
Iskandar and Timothy George with collaboration
from John Kestle

• ASAP-funded pilot study
• Initially suggested by Drs. Mihorat and Batzdorf
• Original plan led by Marcy Speer
• Study supported by the American Society of
  Pediatric Neurosurgeons (ASPN)
• The University of Wisconsin (UW) Office of
  Clinical Trials is providing scientific, clinical
  and financial support in a collaborative effort
  to bring this trial to a successful completion

2
Title Outcomes in Patients Undergoing Surgical
Intervention for Chiari Type 1 Malformation with
Syringomyelia

• Reasons for Study
• ASAP member survey regarding research needs
• ASPN member survey regarding methodology

3
Study Goals

• Direct goal To collect pilot data to evaluate
  surgical approaches and outcomes in surgery for
  patients with Chiari I malformation with
  Syringomyelia
• Indirect long-term goals
• To generate preliminary data for a large
  federally-funded study
• To initiate an organized process/database by
  which Chiari and Syringomyelia can be studied in
  an objective and consistent manner

4
Study Methods

• Prospective, multicenter trial
• Surgeons follow their own routine in diagnosis,
  decision, and treatment
• Primary endpoint
• Syrinx size postop
• Secondary endpoints
• Symptom resolution
• Cine MRI and other radiographic outcomes

5
Method Rigor and Minimizing Bias

• Independent reviewers Committee of 3 (2
  surgeons, one neuroradiologist) evaluate all
  scans blindly
• Participating Centers Neither ASAP Board members
  nor managing centers (Wisconsin, Texas, Utah)
  were allowed to participate in patient
  recruitment
• Data gathering and study coordination All
  managed by a professional office with experience
  in clinical trials - The UW Office of Clinical
  Trials (OCT)

6
UW Office of Clinical Trials(OCT)

• Provide professional clinical trial research
  experience and support to any investigator who
  requires additional expertise to bring a trial
  from a collaborative initiation to a successful
  completion

7
OCT Services Provided

• Finance
• Administration
• Regulatory compliance
• Clinical study coordination

8
Typical Trial Process Step I

• Trials originate from investigators or from
  industry sponsors
• A Confidential Disclosure Agreement (CDA) may be
  initiated
• A Clinical Trials Agreement (CTA) is executed and
  routed through our university system to obtain
  institutional approvals
• A feasibility review is conducted in
  collaboration with the PI
• Protocol is sent to Finance, Regulatory and to
  the Clinical Coordinator

9
Typical Trial Process Step 2

• Budget is negotiated, pricing is confirmed and a
  preliminary budget is prepared
• The Oncore database becomes activated
• Protocol and Subject Life Cycle Management
• Subject Safety Management
• Protocol and Subject Calendar Management
• Electronic Data Capture Data Management
• Study Information Portal
• Custom Reporting Technology

10
Typical Trial Process Step 3

• Oncore Database (contd)
• Electronic Data Capture Data Management
• Study Information Portal
• Custom Reporting Technology
• Oncore goal for the Chiari Surgical
• Outcomes Study
• To provide for a seamless transition to a larger
  trial

11
Typical Trial Process Step 4

• PI signs Office of Clinical Trials agreement and
  it is executed
• Clinical coordinator prepares the source
  documents and data collection forms for entry
  into the Oncore database
• Budget is finalized
• Protocol IRB preparation is initiated and
  submitted for approvals

12
Typical Trial Process Step 5

• IRB study approval is obtained and a recruitment
  plan is created
• A final meeting is scheduled with the PI to
  confirm accrual, recruitment and accountability

13
Chiari Surgical Outcomes Trial

• Study Objective Collect pilot data to evaluate
  surgical approaches and outcomes in surgery for
  patients with Chiari I malformation with
  Syringomyelia

14
Current Chiari Trial StatusSetup and IRB

• UW School of Medicine and Public Health received
  IRB approval to be the data collection site for
  this study on 5/4/2009.
• Our site has IRB approval to collect data from up
  to 12 clinical sites, 120 total subjects, no more
  than 10 subjects per clinical site.
• Oncore research database is complete and we are
  able to enter the data as we receive it. We ran
  a test subject through the data collection
  process March 2010. This went smoothly with no
  glitches.

15
Current Study StatusEnrollment to-date

• We currently have three active clinical sites who
  have signed Clinical Trials Agreements with our
  institution and their own IRB approvals are in
  place.
• Six clinical sites are in the clinical trials
  agreement negotiation process.
• Four enrolled subjects at this time.

16
Current Study StatusProgress and Complications

• No subject has withdrawn.
• No subject has completed participation.
• No adverse events or protocol violations
  reported.
• No changes in research plan, method or
  investigators.

17
Future Expectations

• We are anticipating a two-year timeline to
  collect data on 100-120 subjects
• We anticipate collecting enough information to
  achieve the following goals
• Publish a feasibility study that would facilitate
  obtaining NIH funding for a large study
• Provide preliminary but publishable data on
  differences in outcome between the 3 procedures
  to decompress a Chiari I