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    The EU Clinical Trials Register currently displays   43233   clinical trials with a EudraCT protocol, of which   7153   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2020-001350-21
    Sponsor's Protocol Code Number:APL2-PNH-209
    National Competent Authority:Czechia - SUKL
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2020-12-08
    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 ConcernedCzechia - SUKL
    A.2EudraCT number2020-001350-21
    A.3Full title of the trial
    An Open-Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients with Paroxysmal Nocturnal Hemoglobinuria
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A single arm study to evaluate the safety and biological activity of pegcetacoplan in patients 12- 17 years old with paroxysmal nocturnal hemoglobinuria
    A.4.1Sponsor's protocol code numberAPL2-PNH-209
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/149/2019
    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 organisationArgint International Clinical Research & Development Services Kft.
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street AddressBölöni György u. 22.
    B.5.3.2Town/ cityBudapest
    B.5.3.3Post code1021
    B.5.3.4CountryHungary
    B.5.6E-mailclinicaltrials@argintinternational.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 No
    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
    D.3.2Product code pegcetacoplan
    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 codepegcetacoplan
    D.3.9.3Other descriptive namepegcetacoplan
    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 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
    • To define the pharmacokinetics of pegcetacoplan in adolescents with PNH
    • To evaluate the efficacy of pegcetacoplan based on Hb level, LDH level, and ARC
    • To assess the safety of pegcetacoplan as measured by the incidence and severity of treatment-emergent adverse events (TEAEs), including bacterial infections
    E.2.2Secondary objectives of the trial
    • To assess the pharmacodynamics and biological activity of pegcetacoplan as measured by effects on complement levels, C3 deposition on RBCs, and clonal distribution of PNH RBCs
    • To evaluate the efficacy of pegcetacoplan as assessed by effects on transfusion requirements and episodes of breakthrough hemolysis
    • To assess the effect of pegcetacoplan on health-related quality of life (HRQOL) as measured by PROMIS-Fatigue and PedsQL General Well-Being Scale
    • To assess the long-term safety and efficacy of pegcetacoplan
    • To assess the safety of pegcetacoplan as measured by the occurrence of thromboembolic events
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Between the ages of 12 and 17, inclusive, at time of study entry.
    2. A diagnosis of PNH, confirmed by high-sensitivity flow cytometry (granulocyte or monocyte clone >10%).
    3. Be either a naïve patient or a switch patient, as defined below.
    a. A naïve patient must:
    i. Not be currently receiving an approved complement inhibitor, and must not have received a complement inhibitor within at least 5 half-lives of that drug prior to starting pegcetacoplan
    ii. Have evidence of a hemolytic anemia based on a hemoglobin less than the lower limit of the normal range (LLN), and LDH >1.5 × ULN.
    b. A switch patient must:
    i. Be currently receiving treatment with an approved complement inhibitor, and the dose of that inhibitor must have been stable for at least 5 half-lives of that drug
    ii. Have evidence of anemia based on a hemoglobin less than the LLN.
    iii. Have ARC > ULN.
    4. Platelet count >75,000/mm3.
    5. Absolute neutrophil count >1000/mm3.
    6. Weigh at least 30 kg.
    7. Have a body mass index (BMI) that is less than the 95th percentile for their age.
    8. Either not receiving the following medications, or on a stable regimen for at least the minimum time period indicated below, prior to the first screening visit, with no anticipated changes to the regimen over the course of the study:
    a. Erythropoietin: 8 weeks
    b. Systemic corticosteroids: 4 weeks
    c. Immunosuppressants (other than steroids): 8 weeks
    d. Vitamin K antagonists (eg, warfarin): 4 weeks, with a stable international normalized ratio (INR) over that period
    e. Iron supplements, vitamin B12, or folic acid: 4 weeks
    f. Low-molecular weight heparin or direct oral anticoagulants (DOACs): 4 weeks
    9. Have received vaccinations against Neisseria meningitidis (types A, C, W, Y, and B), Streptococcus pneumoniae, and Haemophilus influenzae (type B) prior to dosing on Day 1, or agree to receive vaccinations within 14 days after starting treatment with pegcetacoplan. Vaccination is mandatory, unless there is documented evidence of titers within acceptable local limits, or documented evidence of nonresponse to vaccination based on titers. Subjects receiving vaccinations after starting pegcetacoplan must be willing to take prophylactic antibiotics from the first day of treatment with pegcetacoplan until at least 2 weeks after vaccination as described in Section 8.2.1.
    10. Female subjects of childbearing potential must have a negative blood pregnancy test at screening (and negative urine pregnancy test on Day 1) and must agree to practice abstinence or to use another protocol-defined method of contraception, as described in Section 10.3.5.1, from screening through at least 90 days after receiving the last dose of pegcetacoplan.
    11. Male subjects who have reached sexual maturity must agree to practice abstinence or to use another protocol-defined method of contraception, as described in Section 10.3.5.1, and agree to refrain from donating semen from screening through at least 90 days after receiving the last dose of pegcetacoplan.
    12. Willing and able to self-administer pegcetacoplan or has a caregiver who is willing and able to do so.
    13. The subject or their legally authorized representative must be willing and able to provide written informed consent as described in Section 12.1.2, including compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. Where appropriate, the subject must also give their assent to participation in the study.
    E.4Principal exclusion criteria
    1. Known or suspected hereditary fructose intolerance (HFI).
    2. Active bacterial infection that has not resolved within at least 1 week before the first dose of pegcetacoplan.
    3. Hereditary complement deficiency.
    4. History of bone marrow transplantation.
    5. History or presence of hypersensitivity or idiosyncratic reaction to compounds related to the formulation or SC administration of pegcetacoplan.
    6. Participation in another investigational drug trial or exposure to another investigational agent, device, or procedure within 30 days or 5 half-lives (whichever is longer) from the last dose of investigational agent prior to screening period.
    7. Planning to become pregnant during study participation, or currently breastfeeding.
    8. History of meningococcal disease.
    9. Inability to cooperate, or any condition that, in the opinion of the investigator makes the subject inappropriate for the study or could confound the outcome of the study.
    E.5 End points
    E.5.1Primary end point(s)
    Pharmacokinetics
    • Pegcetacoplan pharmacokinetic concentrations over the course of the 16-week treatment period, including steady state concentration, Cmax, AUC, Cavg, and Cmin
    Efficacy and Pharmacodynamics
    • Change from baseline to Week 16 in Hb level
    • Change from baseline to Week 16 in LDH level
    • Change from baseline to Week 16 in ARC
    Safety
    • Incidence and severity of TEAEs over the course of the 16-week treatment period, including bacterial infections
    E.5.1.1Timepoint(s) of evaluation of this end point
    Provided in E.5.1
    E.5.2Secondary end point(s)
    Efficacy and Pharmacodynamics
    • Change from baseline to Week 16 and Week 48 in:
    − Complement levels (eg, total complement hemolytic activity [CH50], alternative complement pathway hemolytic activity [AH50], and C3 level)
    − C3 deposition on RBCs
    − Clonal distribution of PNH RBCs
    • Number of transfusions, number of packed RBC units, and total units (mL/kg) transfused over 16 and 48 weeks of treatment with pegcetacoplan
    • Occurrence of breakthrough hemolysis over 16 and 48 weeks of treatment with pegcetacoplan
    • Change from baseline to Week 48, and from Week 16 to Week 48, in hemoglobin, LDH, and ARC
    • Change from baseline to Week 16 and to Week 48 in Health-Related Quality of Life assessments (PROMIS-Fatigue and PedsQL General Well-Being Scale)
    Safety
    • Incidence of thromboembolic events (major adverse vascular events [MAVE]) over the course of the 16-week treatment period and over 48 weeks of treatment with pegcetacoplan
    E.5.2.1Timepoint(s) of evaluation of this end point
    Provided in E.5.2
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 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 EEA20
    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
    Czechia
    France
    Germany
    Hungary
    Ireland
    Italy
    Malaysia
    Philippines
    Poland
    Romania
    Serbia
    Spain
    Thailand
    United Kingdom
    United States
    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
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    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: 12
    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: 12
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2020-12-08. Yes
    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 state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 6
    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)
    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 Decision2020-12-08
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
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