Phase II Study Assessing Safety and Efficacy of APL-2 in Glomerulopathies

January 24, 2025 updated by: Apellis Pharmaceuticals, Inc.

A Phase 2 Study to Evaluate the Safety and Biologic Activity of APL- 2 in Patients With IgA Nephropathy, Lupus Nephritis, Primary Membranous Nephropathy, or C3 Glomerulopathy (C3 Glomerulonephritis and Dense Deposit Disease)

This is a Phase II trial assessing the safety and preliminary efficacy of daily APL-2 subcutaneous infusion administered for 16 weeks with a 6 month safety follow up, in patients with glomerulopathies

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.

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.
21

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 Locations

  • United States
    • California
      • Stanford, California, United States, 94305
        • Stanford University
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Children's Hospital Colorado
      • Denver, Colorado, United States, 80205
        • HealthONE Physician Care, Rocky Mountain Hospital for Children
    • District of Columbia
      • Washington, District of Columbia, United States, 20037
        • Washington Nephrology Associates
    • Florida
      • Coral Gables, Florida, United States, 33134
        • Horizon Research Group
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Emory University
    • Indiana
      • Jeffersonville, Indiana, United States, 47130
        • American Research LLC
    • Louisiana
      • Shreveport, Louisiana, United States, 71101
        • Northwest Louisiana Nephrology LLC
    • Maryland
      • Takoma Park, Maryland, United States, 20912
        • Washington Nephrology Associates
    • Missouri
      • Kansas City, Missouri, United States, 64111
        • Clinical Research Consultants
    • New York
      • Bronx, New York, United States, 10461
        • Davita Clinical Research
      • Valhalla, New York, United States, 10595
        • Westchester Medical Center
    • North Carolina
      • Wilmington, North Carolina, United States, 28401
        • Southeastern Nephrology Associates
    • Tennessee
      • Memphis, Tennessee, United States, 38103
        • University Clinical Health
    • Virginia
      • Alexandria, Virginia, United States, 22304
        • Washington Nephrology Associates
      • Chesapeake, Virginia, United States, 23320
        • Davita Clinical Research
    • Wisconsin
      • Wauwatosa, Wisconsin, United States, 53226
        • Milwaukee Nephrologists

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

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Patients of at least 18 years of age at screening (16 years of age for C3G), able to provide written informed consent, and able to understand and comply with all scheduled procedures and other requirements of the study by the opinion of Principal Investigator (PI)

  • Patients must have a diagnosis of IgAN, LN, Primary MN, or C3G confirmed by renal biopsy and required measurements performed prior to study participation

    • IgAN: Prior biopsy results for C3 and C4d staining should be made available
    • LN: Diagnostic biopsy showing proliferative focal, diffuse, or membranous lesions (Class III, IV or V, respectively) by renal biopsy. Subject should have either a biopsy in the last 6 months, or evidence of disease activity (nephritic changes on urinalysis or nephrotic changes)
    • Primary MN: PLA2R positive titer plus nephrotic range proteinuria (defined as uPCR >2350 mg/g)
    • C3G plus one of the following: Low serum C3 level or historical renal biopsy within the last 3 years
  • Have proteinuria >750 mg/g (calculated by uPCR on 24 hour urine collection) collected during the first screening visit (Visit 3a).
  • eGFR≥30mL/min/1.73 m2 calculated by CKD-EPI creatinine equation at screening visit 3a and currently not on dialysis
  • Must have stable or worsening renal disease, on stable and optimized treatment, in the opinion of the PI, for at least 2 months prior to the first dose of APL-2 (Visit 4); treatments may include, but are not limited to, immunosuppressive agents, anti-hypertensives and/or anti-proteinurics.
  • Willing to receive vaccinations against Neisseria meningitidis at least 2 weeks prior to dosing on Day 1 with a booster on Day 56 (for both vaccinations) and Pneumococcal and Hib vaccines at least 2 weeks prior to dosing on Day 1.

Exclusion Criteria:

  • Absolute neutrophil count <1000 cells/mm3 at screening Visits 3a and 3b
  • ALT or AST >3.0 x the upper limit of normal at screening Visits 3a and 3b
  • Previous treatment with APL-2
  • History of solid organ transplant
  • Diagnosis of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection, or positive serology at screening Visits 3a and 3b (previous HBV or HCV diagnosis cleared by treatment is allowed)
  • Renal disease secondary to another condition (e.g. infection, malignancy, monoclonal gammopathy, or a medication)
  • Presence or suspicion of active bacterial or viral infection or severe recurrent bacterial infections
  • Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days prior to screening period
  • Unwillingness to receive or intolerant of SC infusions of study medication or known allergy to ingredients in APL-2.

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: APL-2
Open Label, Study Drug, APL-2
Drug: APL-2
APL-2 administered as a daily subcutaneous infusion for 48 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
Part A: Change From Baseline in Proteinuria at Week 48
Time Frame: Baseline (Day 1) and Week 48
Change from baseline in proteinuria was assessed based on urinary protein-to-creatinine ratio (uPCR). Baseline was defined as the most recent non-missing measurement prior to or on the first administration of study drug.
Baseline (Day 1) and Week 48
Part B: Change From Baseline in Proteinuria at Week 168
Time Frame: Baseline (Part A, Week 48) and Week 168
Change from baseline in proteinuria was assessed based on uPCR. Baseline was defined as the most recent non-missing measurement prior to or on the first administration of study drug during Part B.
Baseline (Part A, Week 48) and Week 168

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
Parts A and B: Change From Baseline in Serum Complement 3 (C3) Levels at Week 48 of Part A and Week 168 of Part B
Time Frame: Part A: Baseline (Day 1) and Week 48; Part B: Baseline (Part A, Week 48) and Week 168
Blood samples were collected to measure serum C3 levels. Part A baseline was defined as the most recent non-missing measurement prior to or on the first administration of study drug. Part B baseline was defined as the most recent non-missing measurement prior to or on the first administration of study drug during Part B.
Part A: Baseline (Day 1) and Week 48; Part B: Baseline (Part A, Week 48) and Week 168
Parts A and B: Change From Baseline in Alternative Pathway Hemolytic Assay (AH50) Activity at Week 48 of Part A and Week 168 of Part B
Time Frame: Part A: Baseline (Day 1) and Week 48; Part B: Baseline (Part A, Week 48) and Week 168
Blood samples were collected to measure AH50 activity. Part A baseline was defined as the most recent non-missing measurement prior to or on the first administration of study drug. Part B baseline was defined as the most recent non-missing measurement prior to or on the first administration of study drug during Part B.
Part A: Baseline (Day 1) and Week 48; Part B: Baseline (Part A, Week 48) and Week 168
Parts A and B: Change From Baseline in C3a Concentrations at Week 48 of Part A and Week 168 of Part B
Time Frame: Part A: Baseline (Day 1) and Week 48; Part B: Baseline (Part A, Week 48) and Week 168
Blood samples were collected to measure C3a concentrations. Part A baseline was defined as the most recent non-missing measurement prior to or on the first administration of study drug. Part B baseline was defined as the most recent non-missing measurement prior to or on the first administration of study drug during Part B.
Part A: Baseline (Day 1) and Week 48; Part B: Baseline (Part A, Week 48) and Week 168
Parts A and B: Change From Baseline in Serum Albumin Levels at Week 48 of Part A and Week 168 of Part B
Time Frame: Part A: Baseline (Day 1) and Week 48; Part B: Baseline (Part A, Week 48) and Week 168
Blood samples were collected to measure serum albumin levels. Part A baseline was defined as the most recent non-missing measurement prior to or on the first administration of study drug. Part B baseline was defined as the most recent non-missing measurement prior to or on the first administration of study drug during Part B.
Part A: Baseline (Day 1) and Week 48; Part B: Baseline (Part A, Week 48) and Week 168
Parts A and B: Number of Subjects With Complete Clinical Remission at Week 48 of Part A and Week 168 of Part B
Time Frame: Part A: Week 48; Part B: Week 168
The complete clinical remission was defined as normalization of proteinuria as defined by <200 mg/g uPCR.
Part A: Week 48; Part B: Week 168
Parts A and B: Number of Subjects With Stabilization or Improvement in Estimated Glomerular Filtration Rate (eGFR) From Baseline at Week 48 of Part A and Week 168 of Part B
Time Frame: Part A: Baseline (Day 1) and Week 48; Part B: Baseline (Part A, Week 48) and Week 168
The eGFR stabilization or improvement was defined as an eGFR value that was no more than a 25% decrease relative to baseline. Part A baseline was defined as the most recent non-missing measurement prior to or on the first administration of study drug. Part B baseline was defined as the most recent non-missing measurement prior to or on the first administration of study drug during Part B.
Part A: Baseline (Day 1) and Week 48; Part B: Baseline (Part A, Week 48) and Week 168

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.
Apellis Pharmaceuticals, Inc.

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.
February 26, 2018

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.
April 16, 2020

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.
August 26, 2023

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).
February 27, 2018

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.
February 27, 2018

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.
March 5, 2018

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.
March 25, 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.
January 24, 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.
July 1, 2024

More Information

Terms related to this study

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.
  • APL2-201

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

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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