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    Summary
    EudraCT Number:2019-001106-23
    Sponsor's Protocol Code Number:APL2-307
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2019-09-30
    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 ConcernedGermany - BfArM
    A.2EudraCT number2019-001106-23
    A.3Full title of the trial
    An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of Pegcetacoplan in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH).
    EINE OFFENE, NICHT RANDOMISIERTE, MULTIZENTRISCHE VERLÄNGERUNGSSTUDIE ZUR BEURTEILUNG DER LANGFRISTIGEN SICHERHEIT UND WIRKSAMKEIT VON PEGCETACOPLAN BEI DER BEHANDLUNG VON PAROXYSMALER NÄCHTLICHER HÄMOGLOBINURIE (PNH)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of Pegcetacoplan in the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH).
    A.4.1Sponsor's protocol code numberAPL2-307
    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 organisationBiorasi GmbH
    B.5.2Functional name of contact pointEuropean Project Operations
    B.5.3 Address:
    B.5.3.1Street AddressNiermannsweg 11-15
    B.5.3.2Town/ cityErkrath
    B.5.3.3Post code40699
    B.5.3.4CountryGermany
    B.5.4Telephone number+492112503180
    B.5.5Fax number+4921125033233
    B.5.6E-mailhschmied@biorasi.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/3/17/1873
    D.3 Description of the IMP
    D.3.1Product namePegcetacoplan (APL-2)
    D.3.2Product code Pegcetacoplan (APL-2)
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    Subcutaneous 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 codePegcetacoplan (APL-2)
    D.3.9.3Other descriptive namePegcetacoplan (APL-2)
    D.3.9.4EV Substance CodeSUB192794
    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 No
    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
    Paroxysmal Nocturnal Hemoglobinuria
    E.1.1.1Medical condition in easily understood language
    PNH is a rare disorder causing red blood cells to break down too early
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10055629
    E.1.2Term Paroxysmal nocturnal hemoglobinuria
    E.1.2System Organ Class 100000004857
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The objectives of this study are to:
    1. Establish the long-term safety of Pegcetacoplan (APL-2) in PNH subjects
    2. Establish the long-term efficacy of Pegcetacoplan (APL-2) in PNH subjects
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Subjects enrolled in Study APL2-307 who experience acute hemolysis (AH) will be offered the opportunity to enter a substudy in which they receive either intensive SC or intravenous pegcetacoplan as an acute treatment for AH. In order to qualify for participation in the substudy, subjects must have LDH >2 × the ULN and the presence of at least one new or worsening sign or symptom of hemolysis (eg, decrease in Hb, hemoglobinuria, fatigue, etc), warranting acute intervention in the opinion of the investigator.
    E.3Principal inclusion criteria
    1. Subjects at least 18 years of age with PNH who have participated in an antecedent Pegcetacoplan (APL-2) clinical trial. Subjects who received treatment with Pegcetacoplan (APL-2) must have experienced clinical benefit and adequate tolerability in the opinion of the Investigator.
    Note: Subjects with PNH who completed an antecedent Pegcetacoplan (APL-2) clinical trial without receiving Pegcetacoplan (APL-2) (or without receiving Pegcetacoplan (APL-2) for long enough to demonstrate clinical benefit) may be enrolled in this study if, in the opinion of the Investigator, the subject is expected to demonstrate clinical benefit upon the initiation or continuation of Pegcetacoplan (APL-2) therapy.
    2.Vaccination against Neisseria meningitidis types A, C, W, Y and B, Streptococcus pneumoniae and Haemophilus influenzae Type B (Hib) either within 2 years prior to Day 1 dosing, or within 14 days after starting treatment with Pegcetacoplan (APL-2). Unless documented evidence exists that subjects are non-responders to vaccination as evidenced by titers or display titer levels within acceptable local limits. Immunization status checks will be performed to determine whether subjects require primary or booster vaccinations.
    3. Willing and able to give written informed consent.
    4. Willing and able to self-administer Pegcetacoplan (APL-2) (administration by caregiver will be allowed).
    5. Women of child-bearing potential, defined as any females who
    have experienced menarche and who are NOT permanently sterile or
    postmenopausal, must have a negative pregnancy test and must agree to continue to use an approved method of contraception for the duration of the study and 90 days after their last dose of study drug. Note: Postmenopausal is defined as 12 consecutive months with no menses without an alternative medical cause.
    6. Males must agree to continue to use an approved method of
    contraception and must agree to refrain from donating sperm for the duration of the study and 90 days after their last dose of study drug.
    E.4Principal exclusion criteria
    1. Subjects who have withdrawn from an Pegcetacoplan (APL-2) clinical study and/or subjects who met study drug discontinuation criteria during a pegcetacoplan clinical study.
    2. Any condition that could increase the subject’s risk by participating in the study
    3. Any comorbidity or condition (such as malignancy) that, in the opinion of the Investigator, could put the subject at increased risk or potentially confound study data.
    4. History or presence of hypersensitivity or idiosyncratic reaction to compounds related to the investigational product or SC administration.
    5. Acute or active infection with hepatitis B, hepatitis C, or HIV infection.
    6. Hereditary complement deficiency.
    7. History of bone marrow transplant.
    8. Concurrent severe aplastic anemia (defined by bone marrow cellularity <25% [or 25% to 50% if less than 30% of residual cells are hematopoietic] and at least 2 of the following values: peripheral blood absolute neutrophil count <500/μL [<0.5 × 109/L], peripheral blood platelet count <20,000/μL, peripheral blood reticulocyte count <20,000/μL).
    9. History of meningococcal disease.
    10. Concomitant treatment with any complement inhibitor (eg, eculizumab, ravulizumab).
    11. Pregnancy, breastfeeding, or positive pregnancy test.
    E.5 End points
    E.5.1Primary end point(s)
    Safety Endpoints
    • Incidence and severity of Treatment Emergent Adverse Events (TEAE)
    • Incidence of thromboembolic events
    • Laboratory parameters
    • ECG parameters

    Efficacy Endpoints
    • Lactate dehydrogenase level
    • Indirect bilirubin level
    • Absolute Reticulocyte count
    • Hemoglobin level
    • Red blood cell (RBC) transfusions
    • Functional Assessment of Chronic Illness Therapy–Fatigue scale score
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety Endpoints
    • Incidence and severity of Treatment Emergent Adverse Events : after dosing begins, or worsen of severity, for up to 30 days after the last dose of Pegcetacoplan (APL-2)
    • Incidence of thromboembolic events:
    after dosing begins for up to 30 days after the last dose of Pegcetacoplan (APL-2)
    • Laboratory parameters: Please refer to Schedule Events in Protocol
    • ECG parameters: Please refer to Schedule Events in Protocol
    Efficacy Endpoints:
    Please Refer to Schedule Events in Protocol
    E.5.2Secondary end point(s)
    Pharmacokinetic Endpoint
    • Pegcetacoplan (APL-2) pharmacokinetic concentrations

    Pharmacodynamic Endpoints
    • Complement (eg, classical pathway hemolytic activity [CH50], alternative pathway hemolytic activity [AH50], and C3) levels
    • C3 deposition on RBC cells
    • Clonal distribution of PNH RBCs
    • Incidence of anti-pegcetacoplan peptide and anti-polyethylene glycol antibodies
    E.5.2.1Timepoint(s) of evaluation of this end point
    Pharmacokinetic Endpoints:
    Blood samples will be collected at the time points delineated in the Schedule of Events in Section 3 of Protocol

    Pharmacodynamic Endpoints: Blood samples will be collected at the time points delineated in the Schedule of Events in Section 3 of Protocol
    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 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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA30
    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
    Australia
    Canada
    Hong Kong
    Japan
    Korea, Republic of
    Malaysia
    Thailand
    United States
    Bulgaria
    Russian Federation
    Serbia
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months0
    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: 128
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 32
    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 state11
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 48
    F.4.2.2In the whole clinical trial 160
    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)
    None. Expected normal treatment of the condition.
    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 Decision2020-01-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
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