A Mobile App-Based, Prospective, Observational Study to Evaluate Disease Burden and Treatment Patterns in C3 Glomerulopathy (C3G) in the US
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 100
- 试验地点
- 1
- 主要终点
- Number of participants reporting the top 10 symptoms most frequently self-reported
研究概览
简要总结
The study aims to longitudinally capture the full spectrum of symptoms, treatment utilization, and overall Health-related Quality of Life (HRQoL) experienced by C3 glomerulopathy (C3G) patients and their caregivers. By primarily utilizing home reported outcomes (HRO) data on symptom burden and treatment usage, supplemented with patient-reported outcome (PRO) measures (collected at baseline and monthly), the study seeks to establish a new real-world data (RWD) source to understand symptom variability and HRQoL as reported by C3G patients and caregivers, including those taking iptacopan
详细描述
The study will be prospective and observational, conducted over an initial period of six months per individual from the point of study enrollment, with the potential option for participants to extend data collection beyond this period through reconsenting procedures. Participants will utilize the Folia mobile app to enroll, consent, and complete all study activities. A hybrid referral-based recruitment method will be used to identify eligible participants such as through the Novartis APPRISE and MPGN data platforms, clinician referrals, specialty pharmacy inserts, and community and advocacy groups. Participants will be asked to track routine treatment, symptoms, changes in treatment plans, and HRQoL using the Folia Health mobile app. Monthly survey check-ins will be conducted to capture additional data inputs such as patient-reported outcomes (PROs) data, which may be tokenized and integrated into the relevant Novartis APPRISE or MPGN data platform during and after their data collection period. Additional real-world evidence (RWE) datasets such as electronic health record (EHR), claims, or wearable/device data may also be linked to this prospective dataset. Data from integration sources would serve as a complement to the primary study dataset; integration does not affect study objectives or endpoints, which are achieved through HRO tracking data. Participants will be consented for tokenization and linkage through language built into the informed consent form.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 100 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Eligible participants will meet the following basic criteria:
- •Clinical diagnosis of C3G, regardless of symptom, treatment, or transplant history
- •Adult aged 18 or older; adult caregiver to an adult patient aged 18 or older; or adult caregiver to a pediatric patient under 18 years of age
- •Able to provide informed consent
- •Has access to technology (i.e. mobile phone, tablet) that facilitates their participation in the app-based study
- •US-based with a proficient understanding of and ability to read the English language
- •The study team will aim to enroll and collect data on participants who are taking any form of treatment. Participants may be asked to recall the start date of taking their current therapy.
- •Diagnosis of C3G will be confirmed through self-reported screening procedures, patient-supplied documentation, and/or successful linkage of the patient's data with their record in a Novartis data platform. Confirmation of diagnosis for each participant will be reviewed by the Folia Health study team as part of standard validation procedures.
排除标准
- •Study participants who do not fit all inclusion criteria listed above are unable to participate in this study. Outside of the stated inclusion criteria, there is currently no other exclusion criteria in order to meet the exploratory nature of the primary endpoint. This includes treatment regimen; there is no exclusion criteria on the basis of treatment prescription or use.
结局指标
主要结局
Number of participants reporting the top 10 symptoms most frequently self-reported
时间窗: 6 months
Identification of the top 10 symptoms most frequently self-reported by participants, as well as the total number of participants reporting each of those symptoms
Participant average of self-reported severity of each of the top 10 symptoms
时间窗: 6 months
Across-participant average (and standard deviation) of self-reported severity of each of the top 10 symptoms over the course of the 6-month study
Within-participant variability in self-reported severity of each of the top 10 symptoms
时间窗: 6 months
Within-participant variability in self-reported severity of each of the top 10 symptoms over the course of the 6-month study
次要结局
- Average temporal frequency of treatment administration(6 months)
- Counts of patient-reported reasons for skipping treatment(6 moths)
- Proportion of participants who report symptom burden(6 months)
- Change in average symptom burden from pre-switch to post-switch, for participants who switched onto iptacopan during the study period(6 months)
- Differences in number of participants by flare burden(6 months)
- Change over time in monthly check-in responses assessing HRQoL(6 months)
- Differences in PRO scores(6 months)