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    Summary
    EudraCT Number:2020-003767-25
    Sponsor's Protocol Code Number:APL2-C3G-310
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-11-09
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2020-003767-25
    A.3Full title of the trial
    A Phase 3, Randomized, Placebo-Controlled, Double-Blinded, Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients with C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3 study to Evaluate the Efficacy and Safety of Pegcetacoplan administered subcutaneously in Patients with C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
    A.3.2Name or abbreviated title of the trial where available
    VALIANT
    A.4.1Sponsor's protocol code numberAPL2-C3G-310
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorApellis Pharmaceuticals, Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportApellis Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationApellis Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointClinical Trial
    B.5.3 Address:
    B.5.3.1Street Address100 5th Avenue, 3rd Floor
    B.5.3.2Town/ cityWaltham
    B.5.3.3Post codeMA 02451
    B.5.3.4CountryUnited States
    B.5.6E-mailclinicaltrials@apellis.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Aspaveli
    D.2.1.1.2Name of the Marketing Authorisation holderSwedish Orphan Biovitrum AB (publ)
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/19/2201
    D.3 Description of the IMP
    D.3.1Product namePegcetacoplan
    D.3.2Product code APL-2
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPegcetacoplan
    D.3.9.1CAS number 2019171-69-6
    D.3.9.2Current sponsor codeAPL-2
    D.3.9.4EV Substance CodeSUB195466
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number54
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Yes
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection/infusion
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    complement 3 glomerulopathy (C3G)/immune complex membranoproliferative glomerulonephritis (IC-MPGN)
    E.1.1.1Medical condition in easily understood language
    Rare kidney disease which can reoccur after a kidney transplant.
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level SOC
    E.1.2Classification code 10038359
    E.1.2Term Renal and urinary disorders
    E.1.2System Organ Class 10038359 - Renal and urinary disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10077827
    E.1.2Term C3 glomerulopathy
    E.1.2System Organ Class 10038359 - Renal and urinary disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10027168
    E.1.2Term Membranoproliferative glomerulonephritis
    E.1.2System Organ Class 10038359 - Renal and urinary disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is to assess the efficacy of twice-weekly SC doses of pegcetacoplan compared with that of placebo in patients with primary C3G or IC-MPGN on the basis of a reduction in proteinuria.
    E.2.2Secondary objectives of the trial
    •To assess the effect of pegcetacoplan on eGFR
    •To assess the effect of pegcetacoplan on additional C3G/IC-MPGN disease–related parameters
    •To evaluate the safety of pegcetacoplan over 52 weeks of treatment
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Aged at least 18 years; where approved, adolescents (aged 12-17 years) weighing at least 30 kg may also be enrolled.
    2. A diagnosis of primary C3G or IC-MPGN (with or without previous renal transplant).
    3. Evidence of active renal disease, based on one or more of the following:
    a. In adults or adolescents with a baseline renal biopsy, at least 2+ C3c staining on the baseline renal biopsy
    b. In adolescents not providing a baseline renal biopsy, at least one of the following:
    − Serum C5b-9 level above the upper limit of normal during screening
    − Serum C3 below the lower limit of normal (LLN) during screening
    − Presence of an active urine sediment during screening, as evidenced by hematuria with at least 5 red blood cells per high-power field and/or red blood cell casts on routine local or central microscopic analysis of urine
    − Presence of C3 nephritic factor within 6 months of screening, based on central laboratory results or medical history
    4. No more than 50% global glomerulosclerosis or interstitial fibrosis on the baseline biopsy for adult subjects or adolescent subjects providing a baseline biopsy.
    5. At least 1 g/d of proteinuria on a screening 24-hour urine collection and a uPCR of at least 1000 mg/g on at least 2 FMU samples collected during screening.
    6. eGFR ≥30 mL/min/1.73 m2 calculated by the CKD-EPI creatinine equation for adults or the Bedside Schwartz equation for adolescents.
    7. Stable regimen for C3G/IC-MPGN treatment, as described below:
    a. Angiotensin-converting enzyme inhibitor/angiotensin receptor blocker therapy that is stable and optimized, in the opinion of the investigator, for at least 12 weeks prior to randomization
    b. Stable doses of other medications that can affect proteinuria (eg, steroids, mycophenolate mofetil, and/or other allowed immunosuppressants that the patient is receiving for treatment of C3G) for at least 8 weeks prior to the baseline renal biopsy and at least 12 weeks prior to randomization.
    c. If a subject is on prednisone (or other systemic corticosteroid) for C3G/IC-MPGN treatment, the dosage is stable and no higher than 20 mg/d (or equivalent dosage of a corticosteroid other than prednisone) for at least 8 weeks prior to the baseline renal biopsy and at least 12 weeks prior to randomization.
    8. Have received vaccinations against S pneumoniae, N meningitidis (types A, C, W, Y, and B), and H influenzae (type B) within 5 years prior to randomization or agree to receive vaccinations during screening. Vaccination series should be initiated at least 14 days prior to randomization. Vaccination is mandatory unless documented evidence exists that subjects are nonresponders to vaccination.
    9. Female subjects of childbearing potential, defined as any women who have experienced menarche and who are not permanently sterile or postmenopausal, must have negative blood pregnancy tests at screening (and negative urine pregnancy tests on Day 1) and must agree to use protocol-defined methods of contraception from screening through at least 90 days after receiving the last dose of pegcetacoplan.
    10. Male subjects must agree to use protocol-defined methods of contraception and agree to refrain from donating semen from screening through at least 90 days after receiving the last dose of pegcetacoplan.
    11. Subjects 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 subjects under the legal age of consent must be willing and able to provide informed consent; where appropriate, subjects under the legal age of consent must also give their assent to participation in the study.
    12. Willing and able to self-administer pegcetacoplan or have an identified caregiver who can perform the administration.
    E.4Principal exclusion criteria
    1. Previous exposure to pegcetacoplan.
    2. Evidence of improving renal disease in the 8 weeks prior to screening or during the screening period according to available data; improving renal disease is defined as >30% increase in eGFR or >50% decrease in proteinuria.
    3. From a renal transplant subject, evidence of rejection that requires treatment in the baseline renal biopsy collected during screening.
    4. C3G/IC-MPGN secondary to another condition (eg, infection, malignancy, monoclonal gammopathy, a systemic autoimmune disease such as systemic lupus erythematosus, chronic antibody-mediated rejection, or a medication), in the opinion of the investigator.
    5. Current or prior diagnosis of HIV, hepatitis B, or hepatitis C infection or positive serology during screening that is indicative of infection with any of these viruses.
    6. Weight more than 100 kg at screening.
    7. Hypersensitivity to pegcetacoplan or to any of the excipients.
    8. History of meningococcal disease.
    9. Malignancy, except for the following:
    a. Cured basal or squamous cell skin cancer
    b. Curatively treated in situ disease
    c. Malignancy-free and off treatment for ≥5 years
    10. An absolute neutrophil count less than 1000 cells/mm3 at screening.
    11. Significant other renal disease that would, in the opinion of the investigator, confound interpretation of study results.
    12. Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days or 5 half-lives from the last dose of investigational agent (whichever is longer) prior to screening period.
    13. Use of rituximab, belimumab, or any approved or investigational anticomplement therapy other than pegcetacoplan within 5 half-lives of that product prior to the screening period.
    14. Female subjects who are 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.
    15. Inability to cooperate or any condition that, in the opinion of the investigator, creates an undue risk for the subject by participating in the study or is likely to confound interpretation of the study results.
    16. Evidence of ongoing drug or alcohol abuse or dependence, in the opinion of the investigator.
    17. Presence or suspicion of severe recurrent or chronic infections that, in the opinion of the investigator, may place the subject at unacceptable risk by study participation.
    18. Known or suspected hereditary fructose intolerance.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is The proportion of subjects with a reduction from baseline in uPCR of at least 50%. The primary and secondary endpoints will be evaluated at Week 26. The primary and secondary endpoints will also be evaluated at Week 52 as exploratory endpoints
    E.5.1.1Timepoint(s) of evaluation of this end point
    Weeks 26 and 52
    E.5.2Secondary end point(s)
    •The proportion of subjects with eGFR values that are stable or improved from baseline
    For subjects with evaluable renal biopsies, the change from baseline in the C3G histologic activity index score (Bomback et al. 2018)
    The proportion of subjects with evaluable renal biopsies showing decreases in C3c staining on renal biopsy from baseline
    The proportion of subjects achieving proteinuria <1 g/d
    Change from baseline in uPCR
    Change from baseline in eGFR
    For subjects with serum albumin levels below the LLN at baseline, the proportion of subjects with normalization of serum albumin levels
    For subjects with serum C3 levels below the LLN at baseline, the proportion of subjects with serum C3 levels above the LLN
    The change from baseline in the FACIT-Fatigue Scale score
    The change from baseline in the KDQOL score
    The primary and secondary endpoints will be evaluated at Week 26. The primary and secondary endpoints will also be evaluated at Week 52 as exploratory endpoints
    E.5.2.1Timepoint(s) of evaluation of this end point
    Weeks 26 and 52
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA44
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Argentina
    Australia
    Brazil
    Canada
    Israel
    Japan
    Korea, Republic of
    Peru
    United States
    Austria
    Finland
    France
    Poland
    Netherlands
    Spain
    Switzerland
    Czechia
    Germany
    Italy
    Belgium
    Ukraine
    United Kingdom
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    The end of the study will be the last subject’s last visit or the last subject’s last scheduled visit/assessment as indicated in the schedule of activities or as requested by the sponsor.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months10
    E.8.9.2In all countries concerned by the trial days22
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 8
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 8
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 91
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 43
    F.4.2.2In the whole clinical trial 100
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    After completion of the open-label period, subjects who would benefit from continuing to receive pegcetacoplan, as per investigator’s opinion, may roll over into a long-term extension study. Subjects who do not enter a long-term extension study will discontinue pegcetacoplan treatment and complete the 8-week follow-up period.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-02-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-11-16
    P. End of Trial
    P.End of Trial StatusCompleted
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