Clinical trial NCT04358302: Individual Patient Exposure and Response in Pediatric Lupus
Official title: Individual Patient Exposure and Response in Pediatric Lupus
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Systemic Lupus Erythematosus
Keywords: hydroxychloroquine
virtual trial
Lead sponsor: Duke University
Sponsor class: OTHER
Collaborators: name: Food and Drug Administration (FDA)
First posted: 2020-04-24
Enrollment: 26
Sex: ALL
Minimum age (years): 5
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention 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
Primary purpose: SUPPORTIVE_CARE

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.

Eligibility criteria: 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

Exclusion Criteria:

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

Primary outcomes:
measure: Change in proportion of dispensed doses from the first two weeks (baseline) to last two weeks of study (device use)
timeFrame: Days 1-14 compared to days 166-180 (+/- 30)
Secondary outcomes:
measure: Adherence to hydroxychloroquine using the Medication Adherence Self-Reported Inventory (MASRI) score
timeFrame: Up to 6 months
measure: Adherence to hydroxychloroquine using plasma drug levels
timeFrame: Up to 6 months
measure: Adherence to hydroxychloroquine using electronic pill counts
timeFrame: Up to 6 months
measure: Adherence to hydroxychloroquine using manual pill counts
timeFrame: Up to 6 months
measure: Disease activity as measured by Systemic Lupus Activity Questionnaire (SLAQ) score
timeFrame: Up to 6 months
measure: Disease activity as measured by the Systemic Lupus Erythematosus Disease Activity Index (SLEDAI-2K) score collected through a remote teleresearch exam
timeFrame: Up to 2 months
measure: Disease activity as measured by the Systemic Lupus Erythematosus Disease Activity Index (SLEDAI-2K) score recorded in the CARRA Registry
timeFrame: Up to 6 months
Other outcomes:
Locations:
city: Durham
country: United States
facility: Duke Clinical Research Institute
state: North Carolina
Study officials:
affiliation: Duke Clinical Research Institute
name: Stephen J Balevic, MD, MHS
role: PRINCIPAL_INVESTIGATOR
References:
citation: Balevic SJ, Randell R, Weiner D, Beard C, Schanberg LE, Hornik CP, Cohen-Wolkowiez M, Gonzalez D; with the CARRA Registry investigators. Pharmacokinetics of hydroxychloroquine in paediatric lupus: data from a novel, direct-to-family clinical trial. Lupus Sci Med. 2022 Nov;9(1):e000811. doi: 10.1136/lupus-2022-000811.
citation: Randell RL, Singler L, Cunningham A, Schanberg LE, Cohen-Wolkowiez M, Hornik CP, Balevic SJ; with the CARRA Registry investigators. Delivering clinical trials at home: protocol, design and implementation of a direct-to-family paediatric lupus trial. Lupus Sci Med. 2021 May;8(1):e000494. doi: 10.1136/lupus-2021-000494.