Currently Enrolling
Interventional

NOBLE

Phase II Study Assessing the Safety and Efficacy of Pegcetacoplan in Post-Transplant Recurrence of C3G (Complement 3 glomerulopathy) or IC-MPGN (immune-complex membranoproliferative glomerulonephritis)

Brief Description

Phase II Study Assessing the Safety and Efficacy of Pegcetacoplan in Post-Transplant Recurrence of C3G (Complement 3 glomerulopathy) or IC-MPGN (immune-complex membranoproliferative glomerulonephritis)

Trial Physician / Study Coordinator

Elizabeth Carfioli

Email
Site Name

Botucatu, Sao Paulo, Brazil

Sponsor

APELLIS Pharmaceuticals, Inc.

Study Drug

Pegcetacoplan

Estimated enrollment

12 patients

Estimated end date

End of enrollment – December 2021; End of study – September 2022

If there is not a site for a clinical trial nearby, you can ask the study team about the possibility of travel reimbursements (i.e., paying you back for your travel costs). Alternatively, you can ask about the possibility of participating from home.
Currently Enrolling
Interventional

NOBLE

Phase II Study Assessing the Safety and Efficacy of Pegcetacoplan in Post-Transplant Recurrence of C3G (Complement 3 glomerulopathy) or IC-MPGN (immune-complex membranoproliferative glomerulonephritis)

Brief Description

Phase II Study Assessing the Safety and Efficacy of Pegcetacoplan in Post-Transplant Recurrence of C3G (Complement 3 glomerulopathy) or IC-MPGN (immune-complex membranoproliferative glomerulonephritis)

Trial is for people with

Diagnosis of C3G or IC-MPGN that has recurred after a kidney transplant. In addition, participants must be:
- at least 18 years of age
- have significant proteinurea (protein in urine)
- have normal to moderately impaired kidney function
- willing to use contraception during the study, and for 90 days after the last dose of the investigational medication

Study Goal

The NOBLE study is exploring whether pegcetacoplan has an effect on the build up of the complement protein C3 in the kidney, that causes kidney damage.

What is involved for the Patient?

After completing a screening period, study participants will be randomly assigned (randomized) to one of two treatment groups:
o Group 1: Will receive pegcetacoplan, for the entire duration of the study (up to 1 year). There is a 3 in 4 (75%) chance of being assigned to this group.
o Group 2: Will continue their current treatment for the first 12 weeks (3 months) of the study and then transition to receive pegcetacoplan for the remaining 40 weeks (9 months) of the study. There is a 1 in 4 (25%) chance of being assigned to this group.
o While study participants will know their treatment group assignment, they will not have the option of choosing their group.
• Study participants who complete the 1-year study treatment period and are experiencing a benefit may enter a long-term extension study where they will continue receiving pegcetacoplan. Otherwise, they will enter a 6-month follow-up period after their last dose of investigational medication.
• There are approximately 14 clinic visits during the 1-year study treatment period and 6 visits during the 6-month follow-up period. Reimbursement or pre-paid travel support may be available to study participants.
• In addition to having study procedures at study visits, participants will regularly complete urine collections at home during the study.

About the drug or intervention

Pegcetacoplan is an investigational, targeted C3 inhibitor. It is administered via a needle, under the skin (subcutaneous), twice weekly at home. Investigational means that pegcetacoplan is not approved by any health authority for the treatment of C3G or MPGN.

Botucatu, Sao Paulo, Brazil
Frequently Asked Questions

Nephrotic Syndrome is not a disease itself, but rather a group of signs and symptoms that result from damage in the part of the kidney that filters blood (glomeruli).

Common symptoms include:

  • Foamy urine (called proteinuria) caused by protein “spilling” into the urine
  • Severe swelling in parts of the body, most noticeably around the eyes, hands, feet, and abdomen (called edema)
  • Weight gain due to a buildup of extra fluid
  • Fatigue
  • Loss of appetite
  • Low levels of protein in the blood (hypoalbuminemia)
  • Higher than normal fat and cholesterol levels in the blood (hyperlipidemia)

Nephrotic Syndrome can typically be diagnosed with a urine test.

Nephrotic Syndrome can be “primary” or “secondary” in nature.

Diseases that affect only the kidneys are called primary causes of Nephrotic Syndrome. Doctors often call these diseases “idiopathic,” which means that they arise from an unknown cause. Some of these diseases include:

  • Minimal Change Disease (MCD) – most common in children
  • Focal Segmental Glomerulosclerosis (FSGS)
  • Membranous Nephropathy (MN) – most common in adults
  • IgA Nephropathy (IgAN)

Secondary Nephrotic Syndrome is caused by an underlying, systemic condition like diabetes, lupus, HIV, and others.

The Kidney Health Gateway is a website owned and operated by NephCure Kidney International. The purpose of this website is to help patients with rare forms of primary Nephrotic Syndrome get connected to expert care and cutting-edge treatment options. By answering a few questions about you or your loved one’s condition, we can provide you with a list of clinical trials and/or expert doctors in your area.

If you have additional questions, please visit NephCure.org or email Info@NephCure.org.

 

See other frequently asked questions
Did you know that some forms of kidney disease can be genetic?Researchers are continually discovering genetic causes of Nephrotic Syndrome.

Learn more about genetic causes of kidney disease and find out if you may be affected.