Individual Patient Exposure and Response in Pediatric Lupus

Study Overview
=================
Brief Summary
-----------------
The purpose of this research study is to see if an electronic pill bottle cap can help children and teens with systemic lupus better remember to take their medicine. It will also gather information on the best dose of hydroxychloroquine (Plaquenil®) for children and teens. Participants in this study will continue to take their usual medication as prescribed by their doctors. Participants will receive an electronic pill bottle cap, a smartphone, and a Fitbit. Over 6 months, a nurse will visit each participant 4 times to ask questions about symptoms, draw blood, and take a urine sample. After the study, participants will be able to keep the electronic pill bottle cap and Fitbit, but will return the smartphone.

Detailed Description
-----------------
This is an exploratory Phase 2, single site, open-label, direct-to-family, adherence and exposure-response study of hydroxychloroquine (HCQ) in pediatric systemic lupus. The study will measure a participant's adherence to HCQ self-administration using an electronic pill bottle cap that records date/time of bottle opening and provides participants with a reminder when a dosage is due and/or missed. All participants will be provided with the electronic pill bottle cap to use with their regular HCQ prescription at the start of the study. For the first 2 weeks of the trial, the electronic notifications will be disabled to determine a baseline measure of adherence. Within 1 day after Visit 2, the electronic notifications will be turned on for all participants, resulting in a series of alerts (via electronic pill bottle cap alerts and smartphone push notifications, text message, and/or automated phone calls). Participants will continue to receive notifications via the electronic pill bottle cap through Visit 4. Throughout the study, participants will undergo a series of blood draws and urinalysis collections, and be asked to complete various questionnaires.

Official Title
-----------------
Individual Patient Exposure and Response in Pediatric Lupus

Conditions
-----------------
Systemic Lupus Erythematosus

Intervention / Treatment
-----------------
* Behavioral: Pillsy


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Signed and dated informed consent form and assent, when necessary Age 5-17.5 years at consent Enrolled in the CARRA Registry with a diagnosis of systemic lupus erythematosus as documented in the CARRA Registry Receiving hydroxychloroquine as standard of care for ≥ 3 months Participant and Caregiver primary language of English Willing and able to comply with study procedures, at the discretion of the study principal investigator Access to internet Exclusion Criteria: There are no pre-defined exclusion criterion for this trial.

Ages Eligible for Study
-----------------
Minimum Age: 5 Years
Maximum Age: 17 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Supportive Care
Intervention Model: Single Group Assignment
Interventional Model Description: Open-label, direct-to-family, adherence and exposure-response study of hydroxychloroquine (HCQ) self-administration in pediatric lupus participants with the use of an electronic pill bottle cap that provides automatic dosing reminders
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Device use<br>All participants will be provided with the electronic pill bottle cap called Pillsy to use with their regular HCQ prescription bottles at the start of the study. | Behavioral: Pillsy<br>* The electronic pill bottle cap, Pillsy, and its accompanying software application provides reminders to take a scheduled dosage of medication via bottle cap alerts and smartphone push notifications, text messages, and/or automated phone calls. The Pillsy application records dosing dispense date and time when the user opens the bottle. During the first 2 weeks of the trial, the electronic notifications will be disabled to determine a baseline measure of adherence. Within 1 day after Visit 2, the electronic notifications will be turned on for all participants, resulting in a series of alerts (via electronic pill bottle cap alerts and smartphone push notifications, text message, and/or automated phone calls). The electronic pill bottle cap, Pillsy, is a commercially available device marketed to consumers. The electronic pill bottle cap does not capture or store PHI. Quick tips on how to use Pillsy can be found at https://www.pillsy.com/instructions<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in proportion of dispensed doses from the first two weeks (baseline) to last two weeks of study (device use) | The Pillsy device will record the number of dispensed doses | Days 1-14 compared to days 166-180 (+/- 30) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adherence to hydroxychloroquine using the Medication Adherence Self-Reported Inventory (MASRI) score | The MASRI is a concise, self-administered survey completed by the patient at study visits. The MASRI is a patient-reported numeric estimate of medication adherence rate (0-100%) with non-adherence defined as MASRI <80% and adherence defined as >/=80%. | Up to 6 months | 
| Adherence to hydroxychloroquine using serum drug levels | Serum drug levels will be measured by patient blood collection at study visits | Up to 6 months | 
| Adherence to hydroxychloroquine using electronic pill counts | Electronic pill counts will be obtained by the Pillsy device | Up to 6 months | 
| Adherence to hydroxychloroquine using manual pill counts | Manual pill counts will be conducted by the nurse at study visits | Up to 6 months | 
| Disease activity as measured by Systemic Lupus Activity Questionnaire (SLAQ) score | The SLAQ is a concise, self-administered survey completed by the patient at study visits. The SLAQ measures patient-reported disease activity and scores range from 0-44, with 0 representing no disease activity and 44 representing maximum disease activity. | Up to 6 months | 
| Disease activity as measured by the Systemic Lupus Erythematosus Disease Activity Index (SLEDAI-2K) score collected through a remote teleresearch exam | A physician will conduct a remote SLEDAI-2K assessment of the patient at study visit 1. The SLEDAI-2K measures physician-reported disease activity and scores range from 0-105, with 0 representing no disease activity and 105 representing maximum disease activity. A score of >/=6 represents clinically important disease activity and a reduction of at least 4 represents clinically meaningful improvement in disease activity. | Up to 2 months | 
| Disease activity as measured by the Systemic Lupus Erythematosus Disease Activity Index (SLEDAI-2K) score recorded in the CARRA Registry | SLEDAI-2K score recorded from the patient's closest CARRA Registry visit relative to visit 1 | Up to 6 months | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
hydroxychloroquine, virtual trial