Regulatory Sciences Community

Dear all,

I'm looking to the possibility for patients recruited in a Phase II study to perform subcutaneous "self-administration" of a test monoclonal antibody (all active and placebo arms) with Prefilled Syringes (PFS). Theoretically, this could be a way for increasing patients' compliance and to improve recruitment rate due to the more comfortable environment, I mean by self-administration patients can avoid to reach clinical facility for receiving the dose. 

This study should be planned as a classical multicentric Phase 2, multiple dose, double blind, placebo controlled, study.

Is this feasible?

Have you some experience about it?

Have you had any experience to manage a Phase 2 study in this way in the past?

Are there intrinsic risks to invalidate the study by not compliance and/or of generation of unmanageable deviations, unexplained exposure, efficacy and safety data, etc.?

Honestly, my main concerns are both GCP compliance by itself, mainly related to the inclusion of a procedure only partially controlled or not directly controlled by study responsible personnel (i.e. investigators), and of self-administration by itself, since patients are requested to administers subcutaneously by themself with possible issues about.

Many thanks for your support, any consideration is greatly appreciated!

Best

Stefano

Hi Stefano

I am clinical pharmacologist and worked on biologics studies including self-administration. I have not managed the studies, provided feedback for their conduct and control of dosing.

It is possible to conduct a steady with self-administration.

The patients will have to have intense training on how to administer the drug and possibly first doses can be adminstered by a nurse and then by a patient in the presence of nurse.

The dosing diary will be required which will have a step by step refresher on how to conduct the dosing. They would have a checklist for all steps required for dosing to fill at every dose as well as date and clock time for dose completion. They would have to return the prefilled syringes to the clinical sites for reconciliation where the syringes can be examined for any issues - leftover dose, bent needles, etc.

The human factor studies test self-administration in patients and provide troubleshooting.

Possibly a short human factor study ahead of Phase II can help develop a training and troubleshooting for self-administration.

The troubleshooting list and related actions would need to be included in protocol, ICF and dosing diary - for example, if the plunger does not move under reasonable pressure, discard the syringe and notify the site, etc.

PK sampling would be needed to evaluate the compliance - would be best to take samples after the first dose administered by nurse or at the site and compare with follow-up sampling for doses administered by patient at home. The PK sampling can be done at pre-specified safety visits, not intense. 

Population PK modeling can help with comparison of effects of self-administration using sparse PK sampling.

Hope it helps. Best regards,

Dear all,

I'm looking to the possibility for patients recruited in a Phase II study to perform subcutaneous "self-administration" of a test monoclonal antibody (all active and placebo arms) with Prefilled Syringes (PFS). Theoretically, this could be a way for increasing patients' compliance and to improve recruitment rate due to the more comfortable environment, I mean by self-administration patients can avoid to reach clinical facility for receiving the dose. 

This study should be planned as a classical multicentric Phase 2, multiple dose, double blind, placebo controlled, study.

Is this feasible?

Have you some experience about it?

Have you had any experience to manage a Phase 2 study in this way in the past?

Are there intrinsic risks to invalidate the study by not compliance and/or of generation of unmanageable deviations, unexplained exposure, efficacy and safety data, etc.?

Honestly, my main concerns are both GCP compliance by itself, mainly related to the inclusion of a procedure only partially controlled or not directly controlled by study responsible personnel (i.e. investigators), and of self-administration by itself, since patients are requested to administers subcutaneously by themself with possible issues about.

Many thanks for your support, any consideration is greatly appreciated!

Best

Stefano