Study Operations
ION373-CS1 is a Phase 1-3 study. What phase this study is currently in?
A) Phase I
B) Phase II
C) Phase III
C) Phase III
Can I find the latest protocol in Veeva?
A) True
B) False
A) True
What is the number of days that the sites should enter data after a subject visit?
A) Before the next monitoring visit
B) 3 days
C) 5 days
D) Who knows
The recommendation is B) 3 days.
Data should be entered as it is collected.
Can assessments be performed remotely?
A) Yes
B) No
B) No
Should a 1.5 day monitoring visit be entered in PSO as 2 days on site?
A)True
B) False
B) False, must be entered as 1.5 days on site
A CRA must attach a copy of the site’s PD listing converted to "pdf format" and attached to the FUL to send to the site and filed in the eTMF.
A) True
B) False
A) True
If a subject's LP is unsuccessfully attempted using atraumatic (non-cutting) needles, can the site switch to traumatic (cutting) needles to perform the LP?
A) True
B) False
B) False
If the LP cannot be performed with atraumatic (non-cutting) needle the procedure and dosing should be aborted and rescheduled.
Additional Note: 14Oct24 PD Guidance- Unauthorized use of spinal needle other than approved atraumatic spinal needles (listed below), without prior written sponsor approval is not permitted (traumatic vs atraumatic needle). Quincke needles are not permitted. If sites cannot perform lumbar puncture (LP) with atraumatic needle, or if they need to switch to a traumatic spinal needle after an atraumatic spinal needle has been unsuccessful, the procedure and investigational product (IP) dosing need to be aborted (site cannot proceed with the LP procedure and dosing with a Quincke or traumatic needles). Sponsor provided the following needles within each CSF kit Whitacre 24G x 3 1/2” spinal needles with introducer (Main study) Pajunk Sprotte 24G x 1 1/3” spinal needles (Sub-study) The following are also available upon request (order via MRL resupply form/ClinTrak)
In cases where an assessment within a visit was not completed, should the site:
A) Mark the CRF page as Intentionally Left Blank (ILB)
B) Indicate "No" in response to the question regarding whether the assessment was performed
A) Mark the CRF page as Intentionally Left Blank (ILB)
If a specific assessment/procedure was not completed.
Additional Note: The site must fill out the relevant CRF by indicating "No" in response to the question regarding whether the assessment was performed and provide a reason for why it was not done
During the 10MWT, a subject sits down due to fatigue. Should this 10MWT be recorded as a completed trial?
A) Yes
B) No
B) No
IMPORTANT NOTE: The site should add a comment in EDC why the trial was not completed.
The site has added new Sub-I to the study. What documents should you remind the site to update or collect?
A) 1572
B ) Delegation Log
C) Financial Disclosure Form
D) CV and Medical License (as needed)
D) All of the above
B) Delegation Log.
The resource must also undergo training from Sitero, and the training certificate must also be filed in the Investigator Site File. Also, update Daus so the Site Contact List can be updated in PSO.
What tool(s) can a CRA/CTM use to determine if a site(s)/subject(s) have open SAE queries?
A) EDC
B) ION373-CS1 Safety Queries Report
C) Firecrest
D) All of the above
A) EDC. (PPD will enter a query into EDC noted by "PPD PVG QUERY")
B) ION373-CS1 Safety Queries Report sent weekly
If there are difficulties collecting the specified volume of CSF, what is the Minimum
CSF Volume that be collected for ≥8 yo at screening?
A) 15mL
B) 11mL
C) 7.5mL
A) ≥8 yo = 15 mL
IMPORTANT NOTE: the site must document the difficulties in their source and within the comment field of the EDC.
If not, documentation of a PD must be issued.
Which of the following Day 85 pK Sample Code is a correct entry in EDC?
A. P85-0
B. C85-0
C. J013BIL1C (barcode from the requisition form for the pK sample)
A. P85-0
Per CRF Completion Guidelines, PK SAMPLE CODE: Enter the sample code. This should be in the format of P#-# (e.g. P1-0, P1-4) and can be found on the lab requisition form and on the tube label for the PK sample.
As you are monitoring, you noted that the site's Vineland assessor has changed to someone new. What do you do?
A) check to see if the assessor has been approved to perform Vineland
B) check the delegation log to see if they have been added
C) Record a protocol deviation if the assessor was not previously approved
D) All of the above
D) All of the above
Does an experienced Physical Therapist (PT), who has been performing the assessments for years in a clinical setting or other studies, need to complete ION373-CS1 study specific training before they can assess patients in our study?
A) Yes
B) No
A) Yes, any incoming PT must be self-trained on reviewing the following recording and return the completed GMFM Training Record Form to Ionis.
a. GMFCS
b. 10MWT
c. 9HPT
d. GMFM
The ION373-CS1 Study Plan Tracker is a study tool that indicates what?
A) All issued Study Newsletters, DSMB communications, global email blast to site
B) List of Study Plan and their current versions
C) PI/SC call dates and associated communications
D) All of the above
D) All of the above
Study Plan Tracker
Link: https://iconplc.box.com/s/9p4n31icq1v391njyyo4k8v9m377g6yy
How can fundus photographs be taken?
A) Tabletop fundus camera
B) Portable ophthalmoscope
C) Smartphone attachment specifically designed for capturing fundus images
D) All of the above
D) All of the above
In the absence of a tabletop fundus camera, the physician may use a portable ophthalmoscope equipped to take fundus photographs or a smartphone attachment specifically designed for capturing fundus images.
Wihtin EDC, can the Dosing Date and Visit Date be different dates?
A) True
B) False
B) False
Per CCG: Visit Date for Dosing visits must match the date of the dose.
If patient comes in a day earlier for other study assessments/blood draw, enter that information in the corresponding CRF pages.
Example: LAB SAMPLE COLLECTION DATE: Enter the date of the lab draw. If this date is not the same as the visit date, create a COMMENTS CRF stating why the labs were not drawn at the visit.
The protocol suggests efforts should be made for the performance outcome assessments and the patient reported outcomes to be completed before dosing if possible.
Is it recommended for the clinician reported outcomes to be completed after dosing?
A) True
B) False
B) False
As with the Patient-Reported Outcomes, efforts should be made to complete all Reported Outcomes before all study procedures including performance outcome assessments, blood draws, physical exams and dosing.
Can a pencil be used complete source document (including assessments)?
A) Yes
B) No
B) No, doesn't meet ICP GCP Requirements
Ionis has released a LP Safety Assessment and Dosing Assessment Algorithms (i.e CSF pressure monitoring) to ensure that the safety assessments are performed during the LP procedure. In what manual/plan can I find the new LP Safety Assessment and Dosing Algorithm?
Study Drug Manual
US v8.0
ROW v7.0
UK v7.0
Japan v7.0
Who can take the fundus photographs if they are not being done by an ophthalmologist?
A) Any capable person
B) Study Nurse
C) PI or sub-I
D) Study Coordinator
E) All of the above
Daily Double: Who must review the subjects photos?
A) PI
B) PI or sub-I
C) ophthalmologist
A) Any capable person or E) All of the above
The protocol does not specifically note who takes the photos. Any capable person including an ophthalmology technician can take the fundus photos.
Daily double answer: The protocol specifies they should be reviewed by an ophthalmologist and must be reviewed by the PI.
What month will the interim data cut be performed?
Oct 2025
Neurological examination: if all components of the exam can not completed due to non-cooperative child.
What parts of the exams are required to be attempted/completed at minimum?
A) Missing level of consciousness, lower extremity strength, lower extremity reflexes, usual gait
B) Missing level of consciousness and usual gait
C) Lower extremity, lower strength, and usual gait
D) None of the above
A) Missing level of consciousness, lower extremity strength, lower extremity reflexes, usual gait
What documentation should be on-site to verify the study site has completed annual phantom requirement and received approval?
A) Invirco Certificate
B) Nothing, need to review the Invicro tracker for verification
C) Invirco email notification
D) All of the above
B) Invirco email notification
Invirco will provided the site an email. This email should be filed in the ISF and TMF.
In addition, there is a Invirco tracker listing sites who received annual approves.