Home Reported Outcomes in C3G Study

A Mobile App-Based, Prospective, Observational Study to Evaluate Disease Burden and Treatment Patterns in C3 Glomerulopathy (C3G) in the US

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

Study Overview

• Status: Recruiting
• Conditions: C3 Glomerulopathy

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.

• Study Type: Observational
• Enrollment (Estimated): 100

Contacts and Locations

• Study Contact: Name: Novartis Pharmaceuticals; Phone Number: 1-888-669-6682; Email: novartis.email@novartis.com
• Study Contact Backup: Name: Novartis Pharmaceuticals; Phone Number: +41613241111; Email: novartis.email@novartis.com

Study Locations

• United States
  • New Jersey
    • East hanover, New Jersey, United States, 07936
      • Recruiting
      • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

A sample of 100 adults, caregivers of adults, or caregivers of children diagnosed with C3G across an initial 8-month enrollment period. The study team will aim to enroll approximately 50 participants who report taking iptacopan and 50 participants who are not taking a primary therapy of interest.

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
C3G; patients with confirmed diagnosis of C3 glomerulopathy (C3G)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants reporting the top 10 symptoms most frequently self-reported	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	6 months
Participant average of self-reported severity of each of the top 10 symptoms	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	6 months
Within-participant variability in self-reported severity of each of the top 10 symptoms	Within-participant variability in self-reported severity of each of the top 10 symptoms over the course of the 6-month study	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Average temporal frequency of treatment administration	Average temporal frequency of treatment administration, for each primary treatment	6 months
Counts of patient-reported reasons for skipping treatment	Identification and counts of patient-reported reasons for skipping treatment	6 moths
Proportion of participants who report symptom burden	Differences in symptom burden for participants in each treatment group, including symptom occurrence (proportion of participants who report it), severity (when reported), and frequency (when reported).	6 months
Change in average symptom burden from pre-switch to post-switch, for participants who switched onto iptacopan during the study period	Change in average symptom burden (including occurrence, severity, and frequency) from pre-switch to post-switch, for participants who switched onto iptacopan during the study period, if there are enough participants who switch	6 months
Differences in number of participants by flare burden	Differences in number of participants in each treatment group who are classified as having a high vs moderate vs low flare burden	6 months
Change over time in monthly check-in responses assessing HRQoL	Change over time in monthly check-in responses assessing HRQoL, for participants in each treatment group (and in the transplant vs no transplant groups, if possible)	6 months
Differences in PRO scores	Differences in PRO scores, for participants in each treatment group (and in the transplant vs no transplant groups, if possible)	6 months

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): April 7, 2025
• Primary Completion (Estimated): April 30, 2026
• Study Completion (Estimated): April 30, 2026

Study Registration Dates

• First Submitted: June 11, 2025
• First Submitted That Met QC Criteria: June 11, 2025
• First Posted (Actual): June 19, 2025

Study Record Updates

• Last Update Posted (Actual): June 19, 2025
• Last Update Submitted That Met QC Criteria: June 11, 2025
• Last Verified: June 1, 2025

More Information

Terms related to this study

• Keywords: Treatments; Symptoms,; Home reported outcomes (HRO),; NIS,; FACIT-F,; EQ-5D,
• Other Study ID Numbers: CLNP023B1US01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO