A Study to Evaluate the Safety and Effectiveness of Pegcetacoplan to Treat Post-Transplant Recurrence of C3G or IC‑MPGN

Overview

About this study

The purpose of this research study is to is to learn about how the investigational medication works to treat the disease and how safe and acceptable it is to use in adult patients with post-transplant recurrence of C3G or IC-MPGN. The investigational medication being studied is called pegcetacoplan.

The primary objective is to evaluate the effectiveness of pegcetacoplan in improving the underlying pathophysiology of complement 3 glomerulopathy (C3G)/immune complex membranoproliferative glomerulonephritis (IC-MPGN) after 12 weeks of treatment. 

A secondary objective is to evaluate the safety of pegcetacoplan for up to 52 weeks in patients with recurrent C3G/IC-MPGN in a renal allograft; To evaluate the effect of pegcetacoplan on key clinical manifestations of the disease after 52 weeks of treatment.

The exploratory objective is to characterize the additional clinical, laboratory, and histologic findings of C3G/IC-MPGN in response to pegcetacoplan. 

 

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria: 

Individuals must meet all of the following criteria at screening visits to be included in the study: 

  • Patients at least 18 years of age at screening.
  • Patients must have clinical and pathologic evidence of recurrent C3G or IC-MPGN, as evidenced by all of the following:
    • A diagnosis of C3G or IC-MPGN, with at least 2+ staining for C3c in the renal allograft, confirmed by a central pathologist, based on the screening renal allograft biopsy;
    • Clinical evidence of C3G or IC-MPGN recurrence in the form of at least 1 g/day of proteinuria in a 24-hour urine collection;
    • C3G or IC-MPGN must be primary and not secondary to another condition (eg, infection, malignancy, monoclonal gammopathy, autoimmunity, chronic antibody-mediated rejection, chronic thrombotic microangiopathy, or a medication).
  • Stable (not improving) or worsening disease, in the opinion of the investigator, in the 2 months preceding the first dose of pegcetacoplan.
  • eGFR ≥ 30 mL/min/1.73 m^2, calculated by the Chronic Kidney Disease–Epidemiology Collaboration (CKD-EPI) creatinine equation for adults.
  • No more than 50% glomerulosclerosis or interstitial fibrosis on the screening renal allograft biopsy.
  • Stable regimen for recurrent C3G/IC-MPGN for at least 4 weeks prior to the screening renal allograft biopsy and from the time of the screening renal allograft biopsy until randomization.
  • Willing to receive vaccinations against Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae if applicable vaccination records are not available.
  • Women of childbearing potential must each have a negative blood pregnancy test at screening (and negative urine pregnancy at Visit 4) and must agree to use protocol-defined methods of contraception from screening through 12 weeks after receiving last dose of pegcetacoplan.
  • Men must agree to use protocol-defined methods of contraception and agree to refrain from donating semen from screening through 12 weeks after receiving last dose of pegcetacoplan.
  • Willing and able to provide written informed consent.
  • Able to understand and willing to comply with all scheduled procedures and other requirements of the study in the opinion of the investigator.
  • Willing and able to self-administer pegcetacoplan or have an identified caregiver who can perform the administration.

Exclusion Criteria: 

Individuals meeting any of the following criteria at screening or baseline are ineligible to participate in this study: 

  • Absolute neutrophil count < 1000 cells/mm^3 during screening (not including Day 1).
  • Previous treatment with pegcetacoplan.
  • Evidence of rejection on the screening renal allograft biopsy that requires treatment.
  • Diagnosis or history of HIV, hepatitis B, or hepatitis C infection or positive serology at screening indicative of infection.
  • Malignancy, except for the following:
    • Cured basal or squamous cell skin cancer;
    • Curatively treated in situ disease;
    • Malignancy free and off treatment for ≥ 5 years.
  • Significant renal disease in the renal allograft secondary to another condition (eg, infection, malignancy, monoclonal gammopathy, rejection, or a medication) that would confound interpretation of the study results.
  • Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days or 5 half-lives from the last dose of the investigational agent (whichever is longer) prior to screening.
  • Women who are currently breastfeeding.
  • Inability to cooperate or any condition that, in the opinion of the investigator, could increase the patient’s risk by participating in the study or confound the outcome of the study.
  • Evidence of drug or alcohol abuse or dependence, in the opinion of the investigator.

Eligibility last updated 10/18/21. Questions regarding updates should be directed to the study team contact.

 

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Scottsdale/Phoenix, Ariz.

Mayo Clinic principal investigator

Girish Mour, M.B.B.S., M.D.

Contact us for the latest status

Contact information:

Leena Abraham R.N.

(480) 301-6198

Abraham.Leena@mayo.edu

More information

Publications

Publications are currently not available