search
Back to results

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)

Primary Purpose

C3G, IC-MPGN, C3 Glomerulopathy

Status
Not yet recruiting
Phase
Phase 3
Locations
Study Type
Interventional
Intervention
Pegcetacoplan
Sponsored by
Apellis Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for C3G

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    Pegcetacoplan administered subcutaneously

    Arm Description

    Pegcetacoplan administered subcutaneously twice weekly according to protocol defined dosing regimen

    Outcomes

    Primary Outcome Measures

    Proportion of participants with a reduction in urine protein-to-creatinine ratio (uPCR) of at least 50% from the pretreatment value over time.

    Secondary Outcome Measures

    Full Information

    First Posted
    March 30, 2023
    Last Updated
    April 13, 2023
    Sponsor
    Apellis Pharmaceuticals, Inc.
    search

    1. Study Identification

    Unique Protocol Identification Number
    NCT05809531
    Brief Title
    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
    Acronym
    VALE
    Official Title
    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
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    April 2023
    Overall Recruitment Status
    Not yet recruiting
    Study Start Date
    May 2023 (Anticipated)
    Primary Completion Date
    July 2027 (Anticipated)
    Study Completion Date
    July 2027 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Apellis Pharmaceuticals, Inc.

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    Yes
    Studies a U.S. FDA-regulated Device Product
    No
    Data Monitoring Committee
    Yes

    5. Study Description

    Brief Summary
    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).

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    C3G, IC-MPGN, C3 Glomerulopathy, C3 Glomerulonephritis, Complement 3 Glomerulopathy, Complement 3 Glomerulopathy (C3G), Complement 3 Glomerulonephritis, Dense Deposit Disease, DDD, Membranoproliferative Glomerulonephritis, Membranoproliferative Glomerulonephritis (MPGN), Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 3
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    100 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    Pegcetacoplan administered subcutaneously
    Arm Type
    Experimental
    Arm Description
    Pegcetacoplan administered subcutaneously twice weekly according to protocol defined dosing regimen
    Intervention Type
    Drug
    Intervention Name(s)
    Pegcetacoplan
    Other Intervention Name(s)
    APL-2
    Intervention Description
    Complement (C3) Inhibitor
    Primary Outcome Measure Information:
    Title
    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

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    12 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    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
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Apellis Clinical Trial Information Line
    Phone
    617-977-5700
    Email
    clinicaltrials@apellis.com

    12. IPD Sharing Statement

    Learn more about this trial

    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

    We'll reach out to this number within 24 hrs