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    Summary
    EudraCT Number:2020-002637-15
    Sponsor's Protocol Code Number:APL2-C3G-204
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-12-23
    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 ConcernedAustria - BASG
    A.2EudraCT number2020-002637-15
    A.3Full title of the trial
    AN OPEN-LABEL, RANDOMIZED, CONTROLLED, PHASE 2 STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PEGCETACOPLAN IN THE TREATMENT OF POST-TRANSPLANT RECURRENCE OF C3G OR IC‑MPGN
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Phase 2, multicenter, open-label, randomized, controlled study is designed to evaluate the safety and efficacy of APL-2 in patients who have post-transplant recurrence of C3G or IC-MPGN
    A.3.2Name or abbreviated title of the trial where available
    NOBLE
    A.4.1Sponsor's protocol code numberAPL2-C3G-204
    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 pointClin. Development and Med. Affairs
    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 AuthorisationSweden
    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 milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1080
    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
    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.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of pegcetacoplan in improving the underlying pathophysiology of complement 3 glomerulopathy (C3G)/immune complex membranoproliferative glomerulonephritis (IC-MPGN) after 12 weeks of treatment.
    E.2.2Secondary objectives of the trial
    Secondary:
    - To evaluate the safety of pegcetacoplan for up to 52 weeks in patients with recurrent C3G/IC-MPGN in a renal allograft.
    - To evaluate the effect of pegcetacoplan on key clinical manifestations of the disease after 52 weeks of treatment.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Individuals must meet all of the following criteria at screening visits to be included in the study:
    1. At least 18 years of age at screening
    2. Must have clinical and pathologic evidence of recurrent C3G or IC-MPGN, as evidenced by all of the following:
    a. A diagnosis of C3G or IC-MPGN, with at least 2+ staining for C3c in the renal allograft, confirmed by a central pathologist, based on the screening renal allograft biopsy
    b. Clinical evidence of C3G or IC-MPGN recurrence in the form of at least 1 g/day of proteinuria in a 24-hour urine collection that is attributable to disease recurrence in the opinion of the investigator
    c. C3G or IC-MPGN must be primary and not secondary to another condition (eg, infection, malignancy, monoclonal gammopathy, autoimmunity, chronic antibody-mediated rejection, chronic thrombotic microangiopathy, or a medication)
    3. Stable (not improving) or worsening disease, in the opinion of the investigator, in the 2 months preceding the first dose of pegcetacoplan
    4. eGFR ≥15 mL/min/1.73 m2, calculated by the Chronic Kidney Disease–Epidemiology Collaboration (CKD-EPI) creatinine equation for adults
    5. No more than 50% glomerulosclerosis or interstitial fibrosis on the screening renal biopsy
    6. Stable regimen for recurrent C3G/IC-MPGN for at least 4 weeks prior to the screening renal allograft biopsy and from the time of the screening renal allograft biopsy until randomization
    7. Have received required vaccinations against N. meningitides, S. pneumoniae, and H. influenzae (type B) or agree to receive vaccinations if applicable vaccination records are not available, Vaccination is mandatory unless documented evidence exist that subjects are nonresponders to vaccination
    8. Women of childbearing potential, defined as any women who have experienced menarche and who are not permanently sterile or postmenopausal, must have a negative blood pregnancy test at screening (and negative urine pregnancy at Visit 4) and must agree to use protocoldefined methods of contraception from screening through 12 weeks after receiving last dose of pegcetacoplan
    9. Men must agree to use protocol-defined methods of contraception and agree to refrain from donating semen from screening through 12 weeks after receiving last dose of pegcetacoplan
    10. Willing and able to provide written informed consent
    11. Able to understand and willing to comply with all scheduled procedures and other requirements of the study in the opinion of the investigator
    12. Willing and able to self-administer pegcetacoplan or have an identified caregiver who can perform the administration
    E.4Principal exclusion criteria
    Individuals meeting any of the following criteria at screening or baseline are ineligible to participate in this study:
    1. Absolute neutrophil count <1000 cells/mm3 during screening (not including Day 1)
    2. Previous treatment with pegcetacoplan
    3. Evidence of rejection on the screening renal allograft biopsy that requires treatment
    4. Diagnosis or history of HIV, hepatitis B, or hepatitis C infection or positive serology at screening indicative of infection with any of these viruses
    5. Weight more than 100 kg at screening
    6. Hypersensitivity to pegcetacoplan or any of the excipients
    7. History of meningococcal disease
    8. 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
    9. Significant renal disease in the renal allograft secondary to another condition (eg, infection, malignancy, monoclonal gammopathy, rejection, or a medication) that would, in the opinion of the investigator, confound interpretation of the study results
    10. 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 the investigational agent (whichever is longer) prior to screening period
    11. Women who are pregnant, or who are currently breastfeeding
    12. Inability to cooperate or any condition that, in the opinion of the investigator, could increase the subject’s risk by participating in the study or confound the outcome of the study
    13. Evidence of drug or alcohol abuse or dependence, in the opinion of the investigator
    14. 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 reduction in C3c staining on renal biopsy after 12 weeks of treatment with pegcetacoplan.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 12 Biopsy
    E.5.2Secondary end point(s)
    The secondary endpoints are as follows:
    • The number and incidence of treatment-emergent TEAEs
    • The proportion of subjects with reduction in C3c staining on renal biopsy after 52 weeks of treatment
    • The proportion of subjects achieving at least a 50% reduction in proteinuria over time
    • The proportion of subjects achieving complete clinical remission of proteinuria after 12 weeks of treatment, defined as normalization of proteinuria
    • The proportion of subjects with stabilization or improvement in estimated glomerular filtration rate (eGFR) over time
    • The proportion of subjects with stabilization or improvement of serum creatinine concentration over time
    • Changes from baseline biopsy in C3c staining over time
    • Changes and percentage changes from baseline in eGFR and serum
    creatinine concentration over time
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 52 Biopsy
    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 Yes
    E.6.4Safety No
    E.6.5Efficacy No
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    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 No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Variation in treatment schedule: Group2 will not receive pegcetacoplan during the Controlled Portion
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA8
    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
    Switzerland
    Australia
    Brazil
    United Kingdom
    United States
    Austria
    France
    Italy
    Netherlands
    Spain
    E.8.7Trial has a data monitoring committee No
    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 patient’s last visit or the last patient’s scheduled visit/assessment as indicated in the Schedule of Events 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 months11
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months11
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 10
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 7
    F.4.2.2In the whole clinical trial 12
    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)
    All patients who complete study treatment and don't enter Part B, the long-term extension, will return to the site for follow-up visits for 8 weeks, where assessments will be performed per the SoE in Protocol, and where the visit window allowances are also provided. Patients who discontinue treatment early and don't elect to continue their participation in the study will complete a follow-up visit 6 weeks after discontinuation of treatment and also the exit visit, 6 weeks thereafter.
    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 Decision2021-04-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-03-18
    P. End of Trial
    P.End of Trial StatusOngoing
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