Real-world study to evaluate how effective and safe Pegcetacoplan is in patients with rare kidney diseases called C3G or IC-MPGN in multiple countries (PRISMC3)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Swedish Orphan Biovitrum AB (publ)

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Decision date (initial)

2026-06-17

Transition trial

No

Low-intervention

Yes

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Swedish Orphan Biovitrum AB (Sobi)

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacogenetic, Others, Therapy, Safety, Pharmacodynamic

To describe the real-world clinical effectiveness of pegcetacoplan in participants with C3G or primary IC-MPGN.

Secondary objectives 6

1. To describe the demographics, clinical characteristics (including biomarkers), and medication use of participants initiating pegcetacoplan treatment.
2. To describe renal function, disease progression, and change in biomarkers among participants prior to and during pegcetacoplan treatment.
3. To describe renal function, disease progression, and change in biomarkers among participants with changes in pegcetacoplan treatment patterns.
4. To describe incidence rates of important safety events during treatment with pegcetacoplan.
5. To describe patient-reported outcomes (PROs) during treatment with pegcetacoplan.
6. To describe C3G and primary IC-MPGN-related healthcare resource utilisation (HCRU) prior to and during treatment with pegcetacoplan.

Conditions and MedDRA coding

Primary Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

1. Have received or plan to receive pegcetacoplan for the treatment of C3G or primary IC-MPGN.
2. 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 2

1. Receiving an investigational treatment for C3G or primary IC-MPGN at the time of pegcetacoplan initiation.
2. Initiated treatment with pegcetacoplan in an interventional study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Achieving UPCR of <1 g/g at 6 months after starting pegcetacoplan treatment.

Secondary endpoints 11

1. • Participant demographics at time of starting pegcetacoplan (age, sex, race) • Clinical characteristics (including biomarkers) of renal disease prior to and at the time of starting pegcetacoplan • Prior and concomitant C3G and primary IC MPGN treatment information at the start of pegcetacoplan
2. Occurrence of, and time to the following outcomes: - ≥50% proteinuria reduction - <1 g/g UPCR - <0.5 g/g UPCR among participants aged 18 years or older, or <0.2 g/g UPCR among participants less than 18 years of age at start of pegcetacoplan treatment - Resolution of nephrotic syndrome in those with nephrotic syndrome prior to the start of pegcetacoplan treatment
3. Occurrence of, and time to the following clinical outcomes: - Sustained doubling of serum creatinine - Progression to CKD Stage 5 or ESRD - Receipt of new kidney transplant - New onset of maintenance dialysis (i.e., for at least 4 weeks) - Death from kidney failure
4. • Change in UPCR from the most recent diagnostic test results prior to the start of pegcetacoplan treatment, to test results at 1, 3, 6, 12 months, and then every 6 months after starting pegcetacoplan treatment • Achieving UPCR of <1 g/g at 1, 3, 6, 12 months and then every 6 months after starting pegcetacoplan treatment
5. • Change in eGFR derived from the most recent diagnostic test results of creatinine and height (if applicable) before the start of pegcetacoplan treatment to eGFR derived at 1, 3, 6, 12 months and then yearly after starting pegcetacoplan treatment • Proportion of participants with annualised eGFR decline ≤5 mL/min/1.73 m² yearly after starting pegcetacoplan treatment
6. • Change in laboratory parameters at 1, 3, 6, 12 months and then yearly after starting pegcetacoplan treatment • Change in kidney biopsy findings after starting pegcetacoplan treatment • Change in each biomarker, that is assessed longitudinally, from start of pegcetacoplan at 1, 3, 6, 12 months and then yearly
7. • Summary of pegcetacoplan treatment information (reason for use, start/stop dates, dose regimen, dates and reasons for dose changes) • Changes in UPCR, eGFR, and C3c staining on kidney biopsy, over time including after pegcetacoplan discontinuation, pegcetacoplan restart, or switch to another complement inhibitor (e.g., iptacopan), compared to the most recent measurement before the change in anti-complement therapy
8. For participants who switched from pegcetacoplan to iptacopan or other complement inhibitor and vice versa, or for participants who discontinued or restarted pegcetacoplan : Change in each biomarker, that is assessed longitudinally, from the treatment change, at 2 weeks, 1, 3, 6, 12 months and every 6 months
9. • Exposure-adjusted incidence rate of SAEs during pegcetacoplan treatment • Exposure-adjusted incidence rate of AESIs during pegcetacoplan treatment - Severe or serious infections - Malignancies - Acute kidney injury (AKI) Stage 1–3 (KDIGO classification)
10. • Summary scores of FACIT‑Fatigue and WPAI at the start of pegcetacoplan treatment (as available), at study enrolment, and every 6 months during treatment (by caregivers for minors) • Summary of TSQM among adult participants at the start of pegcetacoplan treatment (as available), at study enrolment, and every 6 months during treatment
11. • Annualised C3G and primary IC‑MPGN‑related length of hospital stays and ICU stay before, during, and after pegcetacoplan treatment • Annualised number of C3G and primary IC‑MPGN‑related inpatient or outpatient hospital visits, ICU admissions, emergency department visits before, during, and after pegcetacoplan treatment

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Swedish Orphan Biovitrum AB (publ)

Sponsor organisation

Swedish Orphan Biovitrum AB (publ)

Address

Norra Stationsgatan 93

City

Stockholm

Postcode

113 64

Country

Sweden

Scientific contact point

Organisation

Swedish Orphan Biovitrum AB (publ)

Contact name

Study Physician

Public contact point

Organisation

Swedish Orphan Biovitrum AB (publ)

Contact name

Study Physician

Third parties 6

Locations

4 EU/EEA countries · 29 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 76 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications