An Open-Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in Participants With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis (VALE)

March 7, 2024 updated by: Apellis Pharmaceuticals, Inc.

An Open-Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis

This study is designed as a long-term extension to Study APL2-C3G-310, and is being conducted to establish the long-term safety and efficacy of pegcetacoplan in patients with C3 glomerulopathy (C3G) or immune-complex membranoproliferative glomerulonephritis (IC-MPGN).

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

100

Phase

  • Phase 3

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

  • Australia
      • Fitzroy, Australia, 3065
        • St. Vincent's Hospital Melbourne (61003)
  • Brazil
      • Botucatu, Brazil, 18618-687
        • HC UNESP Botucatu (55010)
      • Porto Alegre, Brazil, 90035-903
        • Hospital de Clinicas de Porto Alegre (55018)
      • Rio De Janeiro, Brazil, 22270-060
        • Ruschel Medicina E Pesquisa Clinica (55012)
      • São Paulo, Brazil, 04038-002
        • Hospital do Rim - Fundacao Oswaldo Ramos (55005)
    • Minas Gerais
      • Belo Horizonte, Minas Gerais, Brazil, 30150-221
        • Santa Casa de Misericordia de Belo Horizonte (55017)
    • RS
      • Porto Alegre, RS, Brazil, 90020-090
        • Irmandade da Santa Casa Misericordia Porto Alegre (55016)
  • Czechia
      • Prague, Czechia, 14021
        • Institute for Clinical and Experimental Medicine (42001)
  • France
      • Bordeaux, France, 33076
        • CHU de Bordeaux - Hopital Pellegrin (33010)
  • Italy
      • Milano, Italy, 20122
        • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (39004)
      • Ranica, Italy, 24020
        • Istituto di Ricerche Farmacologiche Mario Negri IRCCS (39003)
  • Korea, Republic of
      • Seoul, Korea, Republic of, 3722
        • Yonsei University College of Medicine, Sinchon Severance Hospital (82002)
      • Soeul, Korea, Republic of, 3080
        • Seoul National University Hospital (82002)
  • Netherlands
      • Nijmegen, Netherlands, 6500 HB
        • Radboud University Medical Center (31003)
  • Spain
      • Barcelona, Spain, 8035
        • Hosp. Universit. Materno-Infantil Vall d' Hebron (34006)
      • Madrid, Spain, 28041
        • Hospital Universitario 12 de Octubre (34008)
      • Valencia, Spain, 46017
        • Hospital Universitario Dr Peset (34009)
  • Switzerland
      • Lausanne, Switzerland, CH-1011
        • CHUV Lausanne (41002)
  • United Kingdom
      • Manchester, United Kingdom, M13 9WL
        • Royal Manchester Children's Hospital (44011)
      • Nottingham, United Kingdom, NG7 2UH
        • Nottingham University Hospitals NHS Trust (44012)
  • United States
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Emory Investigational Drug Service (01021)
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Hospital and Clinics (01030)
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • University of Michigan Hospital (01008)
    • New Jersey
      • Hackensack, New Jersey, United States, 07601
        • Hackensack Meridian Health (01036)
    • New York
      • New York, New York, United States, 10032
        • Columbia Nephrology (01039)
    • Pennsylvania
      • Bethlehem, Pennsylvania, United States, 18017
        • Northeast Clinical Research Center LLC (01009)

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

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Completed participation in Study APL2-C3G-310 through the week 52 visit requirements
  • Experienced clinical benefit from pegcetacoplan while participating in the previous trial, in the opinion of the investigator
  • Must remain on a stable regimen for C3G or IC-MPGN treatment according to the requirements of Study APL2-C3G-310
  • Received vaccinations against S pneumoniae, N meningitidis (types A, C, W, Y, and B), and H influenzae (type B) according to the requirements of Study APL2-C3G-310 and agree to receive any additional vaccinations recommended according to ACIP recommendations for adults or children with complement deficiencies and/or immunocompromising conditions or other similar local applicable guidelines
  • Female participants of childbearing potential, defined as any woman who has experienced menarche and who is not permanently sterile or postmenopausal, must have a negative urine pregnancy test at visit 1 and must agree to use protocol-defined methods of contraception for the duration of the study through at least 90 days after receiving the last dose of pegcetacoplan
  • Male participants must agree to use protocol-defined methods of contraception and agree to refrain from donating semen for the duration of the study through at least 90 days after receiving the last dose of pegcetacoplan
  • Participants above the legal age of consent, in accordance with local regulations, must be willing and able to provide informed consent. The legally authorized representative of participants under the legal age of consent must be willing and able to provide informed consent; where appropriate, participants under the legal age of consent must also give their assent to participation in the study
  • Willing and able to self-administer pegcetacoplan or have an identified caregiver who can perform the administration

Exclusion Criteria:

  • Female participants who are or are planning to become pregnant or who are currently breastfeeding and are unwilling to discontinue for the duration of the study and for at least 90 days after the final dose of study drug
  • Inability or unwillingness to cooperate with the requirements of the protocol
  • Any condition that, in the opinion of the investigator, creates an undue risk for the participant by participating in the study or is likely to confound interpretation of the study results
  • Evidence of ongoing drug or alcohol abuse or dependence, in the opinion of the investigator

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
Intervention / Treatment
Experimental: Pegcetacoplan administered subcutaneously
Pegcetacoplan administered subcutaneously twice weekly according to protocol defined dosing regimen
Drug: Pegcetacoplan
Complement (C3) Inhibitor
Other Names:
  • APL-2

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Proportion of participants with a reduction in urine protein-to-creatinine ratio (uPCR) of at least 50% from the pretreatment value over time.
Time Frame: 2.5 years
2.5 years

Collaborators and Investigators

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

Sponsor

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)

May 29, 2023

Primary Completion (Estimated)

July 1, 2027

Study Completion (Estimated)

July 1, 2027

Study Registration Dates

First Submitted

March 30, 2023

First Submitted That Met QC Criteria

March 30, 2023

First Posted (Actual)

April 12, 2023

Study Record Updates

Last Update Posted (Actual)

March 12, 2024

Last Update Submitted That Met QC Criteria

March 7, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • APL2-C3G-314
  • 2023-504625-39-00 (Other Identifier: CTIS)
  • U1111-1290-7826 (Other Identifier: Universal Trial Number (UTN))
  • 2022-002833-33 (EudraCT Number)

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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