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INTRODUCTION Viral shedding is crucial for Gene Therapy Products - PowerPoint PPT Presentation

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INTRODUCTION Viral shedding is crucial for Gene Therapy Products Safety linked with shedding data For the patient For families and friends For the environment No treatment possible without good safety results Shedding studies must

  2. INTRODUCTION Viral shedding is crucial for Gene Therapy Products Safety linked with shedding data – For the patient – For families and friends – For the environment No treatment possible without good safety results Shedding studies must be conducted properly

  3. THE STRATEGY Viral shedding must be evaluated early in Phase I and through all phases Launch requires strong data Viral shedding evaluation is mandatory and costly What is the best approach? A clinical trial dedicated to viral shedding evaluation?

  4. VIRAL SHEDDING EVALUATION Test selection is very complex Multiple parameters have to be considered to obtain strong data – Sample type – Sampling times – Logistical issues – Analysis and method validation

  5. SHIPMENT SAMPLES VIRAL SHEDDING TESTS AND METHODS COLLECTION STORAGE

  6. SHIPMENT SAMPLES VIRAL SHEDDING TESTS AND METHODS COLLECTION STORAGE TEST AND METHODS TEST AND METHODS No routine testing Methods developed according to viral construct Very sensitive tests using cellular and molecular methods Test validation is crucial Lab selection based on strong partnership

  7. SHIPMENT SAMPLES VIRAL SHEDDING TESTS AND METHODS COLLECTION STORAGE Multiple sample types for full evaluation SAMPLING SAMPLING Multiple sampling points requested Test results depend on the quality of the samples Sample collection process is crucial

  8. SHIPMENT SAMPLES VIRAL SHEDDING TESTS AND METHODS COLLECTION STORAGE COLLECTION COLLECTION Collection procedures Clinical site training Sampling kits Clinical site monitoring Labeling

  9. EXAMPLES Collection procedure Sampling kits COLLECTION COLLECTION Issues – Preparation issues – Labeling issues • Mix up between patient and household member • Tubes not labeled – Timing issues • Tubes not collected at baseline but after treatment • Tubes not collected

  10. COLLECTION PROCEDURE COLLECTION PROCEDURE Draw blood slowly, using a syringe if possible, and the largest-bore catheter that is feasible for the patient. Transfer blood to a CTAD vacutainer tube (Hemogard, sedimentation rate determination 4.5 mL tubes), being careful to minimize agitation of the sample. Gently mix tube by inverting 4 times Centrifuge at 2000 X g for 15 minutes at room temperature within 1 hour of blood draw. Within 30 minutes after centrifugation, draw off plasma very slowly with transfer pipette to within 0.5 cm of the buffy coat, taking great care not to disturb the buffy coat (any contamination may invalidate the assay). Pipette the plasma specimen into two separate plastic cryovials. Complete corresponding lines for each blood draw on the shipping log. Include patient identification number, initials, and date of specimen collection. All specimens must be stored frozen at -20°C (or colder) in a freezer The Specimen Log Sheets must be completed and included along with all shipments Only the primary samples of each patient should be sent with the first shipment to ensure that duplicates are available at the site for backup in case of damage during shipment.

  11. SHIPMENT SAMPLES VIRAL SHEDDING TESTS AND METHODS COLLECTION STORAGE STORAGE STORAGE Qualified Freezer / Refrigerator Restricted area Monitored temperature equipment Storage documentation requirements

  12. SHIPMENT SAMPLES VIRAL SHEDDING TESTS AND METHODS COLLECTION STORAGE Select the best shipping temperature Courier selection SHIPMENT SHIPMENT Cold chain management Shipping materials Shipping requirements Shipping issues

  13. SHIPMENT - SELECT THE BEST SHIPPING TEMPERATURE Select the best shipping temperature Depends on the stability of the viral construct Courier selection – The best but also the easiest to manage Cold chain • -20°C versus dry ice Management – Viral construct is temperature sensitive Shipping • Impact of Thawing / Freezing issues Materials Shipping Requirements Shipping issues

  14. SHIPMENT – COURIER SELECTION Select the best shipping temperature Courier Some parameters will impact the selection selection – The nature of the samples – The clinical site and testing lab locations Cold chain Management – The level of internal expertise – The QA requirements Shipping – The budget Materials Different courier types for different needs – Support Shipping Requirements – Training Shipping issues

  15. SHIPMENT – COLD CHAIN MANAGEMENT Select the best shipping temperature Courier Viral products are temperature sensitive selection Most of the time, frozen shipment under Cold chain Management dry ice, or at +2°C+8°C with gel packs – Cold chain must be well managed Shipping – Packaging selection is crucial Materials – Temperature probe – Strong partnership with a courier Shipping Requirements Shipping issues

  16. SHIPMENT – COLD CHAIN MANAGEMENT EXAMPLE: Temperature impact Shipment under dry ice Biological samples containing adenoviral construct Lack of cold chain management Freeze / Thaw accident – Decrease in viral concentration – False negative shedding results

  17. SHIPMENT – COLD CHAIN MANAGEMENT Shipment of biological samples: -80°C

  18. SHIPMENT – COLD CHAIN MANAGEMENT Shipment of biological samples: -80°C

  19. SHIPMENT – SHIPPING MATERIAL Select the Packaging validation best shipping temperature Courier selection Cold chain Management Ambient temperature Shipping Materials Dry ice probe N ° 1 Shipping Plastic Tube Probe N ° 2 Requirements Probe N ° 3 Shipping issues

  20. Product -60°C

  21. SHIPMENT – SHIPPING MATERIAL Select the best SOLUTIONS shipping temperature ALL PACKED IN ONE Courier selection Cold chain Management Shipping Materials Shipping Requirements Shipping issues

  22. SHIPMENT – SHIPPING REQUIREMENTS Select the best Customs, documents, shipping classification of the viral construct temperature Courier selection Cold chain Management Shipping Materials Shipping CDC Permit, others… Requirements Shipping issues

  23. SHIPMENT – SHIPPING ISSUES Select the best shipping temperature Courier selection Packaging Cold chain Management Packaging instructions Shipping Materials Initial sample temperature Shipping Replenishment of refrigerant Requirements Shipping issues

  24. SHIPMENT – SHIPPING ISSUES Packaging Perforation occurred during shipment Packaging Refused by airline company Samples blocked

  25. SHIPMENT – SHIPPING ISSUES Selected option: Replace the shipping box QUESTION: • HOW to find a certified company? • HOW to transfer the responsibility of the shipment? • HOW to verify that everything is properly done? • HOW to be sure that the cold chain will be maintain

  26. SHIPMENT – SHIPPING ISSUES Packing instructions have been followed

  27. SHIPMENT – SHIPPING ISSUE Packing instructions have not been followed

  28. SHIPMENT – SHIPPING ISSUE Initial product temperature: same as shipping temperature PRE-SHIPMENT 8 2

  29. SHIPMENT – SHIPPING ISSUE Shipment Pre-Shipment Initial product temperature: different as shipping temperature : The samples have been collected few minutes before the pick-up

  30. SHIPMENT – SHIPPING ISSUE Appropriate replenishment First replenishment Second replenishment

  31. SHIPMENT – SHIPPING ISSUE Without replenishment Not sufficient or too late replenishment

  32. CONCLUSION Multiple parameters could interfere in the viral shedding evaluation Strong expertise requires in all fields Harmonization through the process How to reduce shedding evaluation costs Could we use the data through different constructs / indications Specific guidelines needed

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