Home Reported Outcomes in C3G Study
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: C3 Glomerulopathy. Basic parameters: 18 years — 100 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
A Mobile App-Based, Prospective, Observational Study to Evaluate Disease Burden and Treatment Patterns in C3 Glomerulopathy (C3G) in the US
Overview
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
Detailed description
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.
Primary outcome measures
- Number of participants reporting the top 10 symptoms most frequently self-reported [Time frame: 6 months]
- Participant average of self-reported severity of each of the top 10 symptoms [Time frame: 6 months]
- Within-participant variability in self-reported severity of each of the top 10 symptoms [Time frame: 6 months]
Secondary outcome measures (7)
- Average temporal frequency of treatment administration [Time frame: 6 months]
- Counts of patient-reported reasons for skipping treatment [Time frame: 6 moths]
- Proportion of participants who report symptom burden [Time frame: 6 months]
- Change in average symptom burden from pre-switch to post-switch, for participants who switched onto iptacopan during the study period [Time frame: 6 months]
- Differences in number of participants by flare burden [Time frame: 6 months]
- Change over time in monthly check-in responses assessing HRQoL [Time frame: 6 months]
- Differences in PRO scores [Time frame: 6 months]
Eligibility criteria
Inclusion criteria
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.
Exclusion criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Other
Study locations
United States · 1 center
- Novartis Investigative Site — East Hanover
Identifiers
NCT: NCT07029542 · CLNP023B1US01