Complement C5 mAb in the Treatment of Anti-GBM Disease

November 28, 2025 updated by: Zhao CUI, Peking University First Hospital

A Single Center, Open-label, Single Arm Phase II Trial of the Efficacy and Safety of Complement C5 Monoclonal Antibody in the Treatment of Anti-glomerular Basement Membrane Disease

Anti-GBM disease is the most severe form of glomerulonephritis. Despite of the standard treatment including plasmapheresis and immunosuppressant, 70% of the patients still go into end-stage kidney disease. Complement has been shown to participate in the pathogenesis of anti-GBM disease. This study aims to the investigate the therapeutic effects and safety of C5 monoclonal antibody in the treatment of anti-GBM disease.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

Anti-glomerular basement membrane (anti-GBM) disease is the most severe form of autoimmune glomerulonephritis, characterized by the production of autoantibodies targeting the components of basement membrane within the kidney and/or the lung.Patients with anti-GBM disease typically present rapidly progressive glomerulonephritis, and often accompanied by lung hemorrhage. The hallmark of the disease is the linear deposition of IgG along the GBM on kidney biopsy. Complement activation is a pivotal step for kidney injuries during the development of human anti-GBM disease. Our previous study showed that the The levels of plasma SC5b-9 and urinary C5a were positively correlated with the serum creatinine at presentation and the percentage of crescents in glomeruli. Eculizumab is a recombinant humanized monoclonal antibody that specifically binds to a C5 terminal complement and inhibits the cleavage of C5 to C5a and C5b through complement activation. There are a few case reports showing therapeutic effects in anti-GBM disease. This trial will aim to evaluate the efficacy and safety of Eculizumab plus standard treatment in anti-GBM disease.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
16

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • China
      • Beijing, China, 100038
        • Peking University First Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria

  1. Positive circulating anti-GBM antibody, with proteinuria or hematuria or any clinical signs of kidney injuries.
  2. And or kidney biopsy showed typical IgG linear deposition along the GBM
  3. At least 18 years old

Exclusion Criteria

  1. Allergic to eculizumab, mouse protein or the investigational drug and any of its excipients;
  2. Uncontrolled meningococcal infection,or those who have not received meningitis prophylactic antibiotic treatment or meningitis vaccination;
  3. Diagnosis of anti-GBM disease for more than 12 weeks before signing the informed consent form;
  4. Pregnant or lactating
  5. Received investigational drug within 30 days or 4 half-lives (whichever is longer) prior to screening;
  6. Other serious poorly controlled comorbid diseases that affect the compliance of the trial protocol or the interpretation of results within 3 months prior to screening, including cardiovascular and cerebrovascular diseases, lung disease, etc.;
  7. Presence of any medical history or disease that, in the opinion of the investigator, may expose the patient's participation in the study to an unacceptable risk;

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: eculizumab
C5 monoclonal treatment plus standard treatment
Drug: Eculizumab
eculizumab 900mg iv. per week for 4 weeks, then 1200mg every two weeks for 8 weeks

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
kidney prognosis
Time Frame: 6 months after the first infusion of eculizumab
ESKD (dialysis dependent) or kidney translation
6 months after the first infusion of eculizumab

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Changes in glomerular filtration rate (eGFR);
Time Frame: from baseline to study completion, an average of 1 year
Change in level of estimated Glomerular Filtration Rate (eGFR) as measured by laboratory testing, reported in mL/min/1.73m².
from baseline to study completion, an average of 1 year
Changes in serum creatinine
Time Frame: from baseline to study completion, an average of 1 year
Change in concentration of serum creatinine as measured by laboratory testing, reported in µmol/L
from baseline to study completion, an average of 1 year
Changes in blood urine nitrogen (BUN)
Time Frame: from baseline to study completion, an average of 1 year
Change in concentration of Blood Urea Nitrogen (BUN) as measured by laboratory testing, reported in mmol/L
from baseline to study completion, an average of 1 year
side effects of eculizumab
Time Frame: at 6 months
The number of participants experiencing any adverse events determined by the investigator to be related to eculizumab treatment.
at 6 months

Other Outcome Measures

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
kidney function recovery
Time Frame: 6 months after the first eculizumab infusion
Kidney recovery was defined as independence from kidney replacement therapy (KRT, such as dialysis) lasting at least 12 weeks during follow-up
6 months after the first eculizumab infusion

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Peking University First Hospital

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Zhao Cui, PKUFH

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 1, 2024

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
November 3, 2025

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 3, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 16, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 16, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 22, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
December 5, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 28, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • C5mAb in anti-GBM disease

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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