C3 Glomerulopathy (C3G), Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)
Conditions
Keywords
C3 Glomerulopathy, Primary Immune Complex Membranoproliferative, Glomerulonephritis, Pegcetacoplan, Glomerulopathy, Nephrology, Rare disease
Brief summary
The purpose of this study is to evaluate the effectiveness and safety of Pegcetacoplan in patients with C3G and primary IC-MPGN in the real-world setting. This study will also assess biomarkers not routinely measured in clinical practice. Results will support the long-term evaluation of the benefit-risk profile of pegcetacoplan in a broad patient population, informing clinical decision-making.
Detailed description
The purpose of this study is to evaluate the effectiveness and safety of pegcetacoplan in patients with C3G and primary IC-MPGN in the real-world setting post-approval, as well as to characterise relevant biomarkers and their association with pegcetacoplan treatment. This will provide new information that was not evaluated in the clinical programme and help with understanding the long-term benefit-risk of pegcetacoplan in a broad patient population. This study will evaluate patient data prior to, during and after the use of pegcetacoplan to assess how the real-world use of pegcetacoplan impacts clinical outcomes and health care resource utilisation. This knowledge will contribute to informed clinical decision-making and provide guidance for effective disease management of patients with C3G or primary IC-MPGN.
Interventions
DRUGPegcetacoplan
Solution for infusion for subcutaneous use according to drug use in the real world.
Sponsors
Swedish Orphan Biovitrum
IQVIA Pty Ltd
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Have received or plan to receive pegcetacoplan for the treatment of C3G or primary IC-MPGN. * Provided signed and dated informed consent. For participants under the legal age signed and dated informed consent is provided by the participant's legally authorised representative. Assent will also be obtained from paediatric participants as required by local regulations.
Exclusion criteria
* Receiving an investigational treatment for C3G or primary IC-MPGN at the time of pegcetacoplan initiation. * Initiated treatment with pegcetacoplan in an interventional study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Achieving urine protein-to-creatinine ratio (UPCR) of <1 g/g | at 6 months after starting pegcetacoplan treatment | To describe the real-world clinical effectiveness of pegcetacoplan in participants with C3G or primary IC-MPGN |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Receipt of new kidney transplant | At occurrence, prior to and during pegcetacoplan treatment, up to 5.5 years. | Occurrence of receipt of new kidney transplant from baseline. |
| New onset of maintenance dialysis (i.e., for at least 4 weeks) | At occurrence, prior to and during pegcetacoplan treatment, up to 5.5 years. | Occurrence of new onset of maintenance dialysis (i.e., for at least 4 weeks) |
| Death from kidney failure | At occurrence, prior to and during pegcetacoplan treatment, up to 5.5 years. | Occurrence of death from kidney failure. |
| Sustained doubling of serum creatinine | Whenever the event occurs for the first time during pegcetacoplan treatment, up to 5.5 years. | Occurrence of sustained doubling of serum creatinine from baseline. |
| Participant demographics (age, sex, race) | Day 1 of treatment/Baseline. | To describe the demographics of participants initiating pegcetacoplan treatment. |
| Clinical characteristics of renal disease (UPCR) | Prior to and - Day 1 of treatment/Baseline. | To describe clinical characteristics (UPCR), of renal disease in participants initiating pegcetacoplan treatment. |
| Clinical characteristics of renal disease (eGFR) | Prior to and - Day 1 of treatment/Baseline. | To describe clinical characteristics (eGFR), of renal disease in participants initiating pegcetacoplan treatment. |
| Prior and concomitant C3G and primary IC-MPGN treatment information | Day 1 of treatment/Baseline. | To describe the medication use of participants initiating pegcetacoplan treatment. |
| ≥50% proteinuria reduction | At 1, 3, 6, 12 months and then every 6 months through the last dose received during the study, up to 5.5 years. | Cumulative incidence function will be used to describe the time from the start of pegcetacoplan treatment to the first occurrence of ≥50% proteinuria reduction. |
| <1 g/g UPCR | At 1, 3, 6, 12 months and then every 6 months through the last dose received during the study, up to 5.5 years. | Cumulative incidence function will be used to describe the time from the start of pegcetacoplan treatment to the first occurrence of \<1 g/g UPCR. |
| <0.5 g/g UPCR among participants aged 18 years or older | At 1, 3, 6, 12 months and then every 6 months through the last dose received during the study, up to 5.5 years. | Cumulative incidence function will be used to describe the time from the start of pegcetacoplan treatment to the first occurrence of \<0.5 g/g UPCR among participants aged 18 years or older. |
| <0.2 g/g UPCR among participants less than 18 years of age | Whenever the event occurs for the first time during pegcetacoplan treatment, up to 5.5 years. | Cumulative incidence function will be used to describe the time from the start of pegcetacoplan treatment to the first occurrence of \<0.2 g/g UPCR among participants less than 18 years of age. |
| Resolution of nephrotic syndrome in those with nephrotic syndrome | Whenever the event occurs for the first time during pegcetacoplan treatment, up to 5.5 years. | Cumulative incidence function will be used to describe the time from the start of pegcetacoplan treatment to the first occurrence of resolution of nephrotic syndrome in participants with nephrotic syndrome. |
| Change in urine protein-to-creatinine ratio (UPCR) from the most recent diagnostic test results | Prior to start of pegcetacoplan treatment, at 1, 3, 6, 12 months, and then every 6 months through the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant. | Occurrence of change in UPCR from the most recent diagnostic test results. |
| Achieving UPCR of <1 g/g | At 1, 3, 6, 12 months and then every 6 months through the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant. | Occurrence of UPCR of \<1 g/g |
| Change in eGFR derived from the most recent diagnostic test results of creatinine and height (if applicable) | Prior to start of pegcetacoplan treatment to eGFR derived at 1, 3, 6, 12 months and then until the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant. | Change in eGFR derived at 1, 3, 6, 12 months and then yearly from the most recent diagnostic test results of creatinine prior to start of pegcetacoplan. |
| Proportion of participants with annualised eGFR decline ≤5 mL/min/1.73 m2 | Yearly during pegcetacoplan treatment up to the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant. | Proportion of participants with annualised eGFR decline ≤5 mL/min/1.73 m2 yearly during pegcetacoplan treatment in the study. |
| Change in kidney biopsy findings | At occurrence, during pegcetacoplan treatment, through the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant. | Kidney biopsy findings through the last dose of pegcetacoplan received during the study. |
| Summary of pegcetacoplan treatment information | Throughout the whole study duration whenever pegcetacoplan is administered per standard of care, up to 5.5 years. | To describe pegcetacoplan dosing schedule and treatment patterns. |
| Exposure-adjusted incidence rate of SAEs | Ongoing from the start of pegcetacoplan treatment to study end, or 8 weeks post last dose of pegcetacoplan, whichever occurs earlier. | Exposure-adjusted incidence rate of SAEs. |
| Exposure-adjusted incidence rate of AESIs | Ongoing from the start of pegcetacoplan treatment to study end, or 8 weeks post last dose of pegcetacoplan, whichever occurs earlier. | Exposure-adjusted incidence rate of AESIs |
| Summary scores of FACIT-Fatigue and WPAI | At the start of pegcetacoplan treatment, as available; at study enrolment; and every 6 months during treatment, through the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant. | Summary scores of FACIT-Fatigue and WPAI |
| Summary of TSQM among adult participants | At the start of pegcetacoplan treatment, as available, at study enrolment, and every 6 months during treatment through the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant. | Summary of TSQM among adult participants |
| Annualised number of C3G and primary IC-MPGN-related inpatient or outpatient hospital visits | Prior to, during treatment and after the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant. | Annualised C3G and primary IC-MPGN-related inpatient or outpatient hospital visits before, during and after treatment with pegectacoplan. |
| Annualised C3G-related length of hospital stays | Prior to, during treatment and after the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant. | Annualised C3G-related length of hospital stays before, during and after treatment with pegectacoplan. |
| Annualised primary IC-MPGN-related length of hospital stays | Prior to, during treatment and after the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant. | Annualised primary IC-MPGN-related length of hospital stays before, during and after pegcetacoplan treatment. |
| Annualised number of C3G-related length of ICU admissions | Prior to, during treatment and after the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant. | Annualised number of C3G-related ICU admissions before, during and after pegcetacoplan treatment. |
| Annualised number of primary IC-MPGN-related ICU admissions | Prior to, during treatment and after the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant. | Annualised number of primary IC-MPGN-related ICU admissions before, during and after pegcetacoplan treatment. |
| Annualised C3G-related length of ICU stay | Prior to, during treatment and after the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant. | Annualised C3G-related length of ICU stay before, during and after pegcetacoplan treatment. |
| Annualised primary IC-MPGN-related length of ICU stay | Prior to, during treatment and after the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant. | Annualised primary IC-MPGN-related length of ICU stay before, during and after pegcetacoplan treatment. |
| Annualised number of C3G-related emergency department visits | Prior to, during treatment and after the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant. | Annualised number of C3G-related emergency department visits before, during and after pegcetacoplan treatment. |
| Progression to Chronic Kidney Disease Stage 5 or ESRD | At occurrence, prior to and during pegcetacoplan treatment, up to 5.5 years. | Occurrence of Progression to Chronic Kidney Disease Stage 5 or ESRD from baseline. |
| Annualised number of primary IC-MPGN-related emergency department visits | Prior to, during treatment and after the last pegcetacoplan dose or up to 2 years after the enrolment date of the last participant. | Annualised number of primary IC-MPGN-related emergency department visits before, during and after pegcetacoplan treatment. |
Countries
Australia, Austria, Belgium, Canada, Czechia, France, Germany, Italy, Saudi Arabia, South Korea, Spain, Switzerland, Taiwan, United Kingdom
Contacts
CONTACTStudy Physician
Outcome results
None listed