Individual Patient Exposure and Response in Pediatric Lupus

Not Applicable

Completed

• Conditions: Systemic Lupus Erythematosus
• Interventions: Behavioral: Pillsy

• Registration Number: NCT04358302
• Lead Sponsor: Duke University

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.

Study Timeline

• Study First Submitted: 2020-04-16
• Study First Submitted QC: 2020-04-20
• Study First Posted: 2020-04-24
• Study Start: 2020-09-28
• Primary Completion: 2021-06-16
• Study Completion: 2021-06-16
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-11
• Last Update Posted: 2024-07-15

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 26

Inclusion Criteria

1. Signed and dated informed consent form and assent, when necessary
2. Age 5-17.5 years at consent
3. Enrolled in the CARRA Registry with a diagnosis of systemic lupus erythematosus as documented in the CARRA Registry
4. Receiving hydroxychloroquine as standard of care for ≥ 3 months
5. Participant and Caregiver primary language of English
6. Willing and able to comply with study procedures, at the discretion of the study principal investigator
7. Access to internet

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Exclusion Criteria

There are no pre-defined exclusion criterion for this trial.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Device use	Pillsy	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.

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
Adherence to hydroxychloroquine using manual pill counts	Up to 6 months	Manual pill counts will be conducted by the nurse at study visits
Adherence to hydroxychloroquine using electronic pill counts	Up to 6 months	Electronic pill counts will be obtained by the Pillsy device
Adherence to hydroxychloroquine using the Medication Adherence Self-Reported Inventory (MASRI) score	Up to 6 months	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%.
Adherence to hydroxychloroquine using plasma drug levels	Up to 6 months	Plasma drug levels will be measured by patient blood collection at study visits
Disease activity as measured by the Systemic Lupus Erythematosus Disease Activity Index (SLEDAI-2K) score collected through a remote teleresearch exam	Up to 2 months	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.
Disease activity as measured by the Systemic Lupus Erythematosus Disease Activity Index (SLEDAI-2K) score recorded in the CARRA Registry	Up to 6 months	SLEDAI-2K score recorded from the patient's closest CARRA Registry visit relative to visit 1
Disease activity as measured by Systemic Lupus Activity Questionnaire (SLAQ) score	Up to 6 months	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.

Trial Locations

Locations (1)

Duke Clinical Research Institute; 🇺🇸 Durham, North Carolina, United States