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    Summary
    EudraCT Number:2021-003160-27
    Sponsor's Protocol Code Number:Sobi.PEGCET-101
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2022-10-19
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2021-003160-27
    A.3Full title of the trial
    A Phase 3, Randomized, Double-blind, Placebo-controlled Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients with Cold Agglutinin Disease (CAD)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    “A Randomized, Double-blind, Placebo-controlled Study to Evaluate Pegcetacoplan in Patients with Cold Agglutinin Disease (CAD)”
    A.4.1Sponsor's protocol code numberSobi.PEGCET-101
    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 SponsorSwedish Orphan Biovitrum AB
    B.1.3.4CountrySweden
    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 supportSwedish Orphan Biovitrum AB
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSwedish Orphan Biovitrum AB
    B.5.2Functional name of contact pointSumaiya Patel
    B.5.3 Address:
    B.5.3.1Street AddressTomtebodavägen 23A
    B.5.3.2Town/ citySolna
    B.5.3.3Post codeSE-112-76
    B.5.3.4CountrySweden
    B.5.4Telephone number00447553895502
    B.5.6E-mailSumaiya.Patel@sobi.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
    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 (APL‑2)
    D.3.2Product code 2019171-69-6
    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.3.9.4EV 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
    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
    Patients with Cold Agglutinin Disease (CAD)
    E.1.1.1Medical condition in easily understood language
    Patients with Cold Agglutinin Disease (CAD)
    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 demonstrate the efficacy of twice-weekly subcutaneous (s.c.) 1080-mg infusions of pegcetacoplan compared with that of placebo in patients with CAD
    E.2.2Secondary objectives of the trial
    • To demonstrate the effect of pegcetacoplan on the number of packed red blood cell (PRBC) transfusions in patients with CAD.
    • To demonstrate the effect of pegcetacoplan on health-related quality of life in patients with CAD.
    • To assess the effect of pegcetacoplan on clinical laboratory markers of hemolysis and transfusion dependence in patients with CAD.
    • To determine the durability of response in patients with CAD receiving pegcetacoplan.
    • To assess tolerability, safety and immunogenicity of pegcetacoplan in patients with CAD.
    • To describe long-term effect of pegcetacoplan in patients with CAD.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age18 years or older.
    2. Diagnosis of primary CAD on the basis of the presence of all the following criteria at screening:
    a Signs of hemolysis with abnormal values by at least 2 of the following hemolytic markers:
    i Reduced haptoglobin level (< LLN).
    ii Elevated LDH level (> ULN).
    iii Elevated indirect bilirubin level (> ULN; > 3 x ULN for patients with Gilbert-Meulengracht Syndrome).
    iv Increased ARC (above the ULN).
    b Monospecific direct antiglobulin test strongly positive for C3d.
    c Cold agglutinin titer ≥ 64 at 4 °C.
    3. Hb level ≤ 9 g/dL at screening.
    4. An absolute neutrophil count ≥ 1500 cells/mm3 at screening.
    5. Documented results from bone marrow biopsy within 1 year of screening with lymphoproliferative infiltration ≤ 20 %. Patients who have not received a bone marrow biopsy within 1 year of their screening visit or those patients for whom bone marrow biopsy reports are incomplete or unavailable will be required to receive a bone marrow biopsy to determine eligibility.
    6. Body weight ≤ 100 kg.
    7. Either have vaccination against Streptococcus pneumoniae, Neisseria meningitidis (Types A, C, W, Y, and B), and Haemophilus influenzae (Type B) within 2 years prior to screening or agree to receive vaccination during screening as follows:
    First dose of vaccine against N. meningitidis Types A, C, W, and Y at least 2 weeks prior to start of study drug with second dose 2 months late (Study Day 57), and then boosters every 5 years.
    First dose of the vaccine against N. meningitidis Type B at least 2 weeks prior to start of study drug with a second dose after at least 1 month (Study Day 29). First booster dose 1 year later, and then additional booster doses every 2 to 3 years.
    S. pneumoniae: pneumococcal conjugate vaccine 23 (PCV13) and/or pneumococcal polysaccharide vaccine 23 (PPSV23) as per Advisory Committee on Immunization Practices (ACIP) guidelines for adults or children with immunocompromising conditions.
    H. influenzae Type B: 1 dose at least 2 weeks prior to start of study drug.
    Vaccination is mandatory, unless documented evidence exists that patients are non responders to vaccination. Vaccinations should be administered following the ACIP recommendations for adults or children with complement deficiencies and/or immunocompromising conditions.
    8. Women of childbearing potential (WOCBP), defined as any women who have experienced menarche and who are NOT permanently sterile or postmenopausal, must have a negative pregnancy test at screening and agree to use protocol-defined methods of contraception for the duration of the study and 8 weeks after their last IMP dose.
    Note: Postmenopausal is defined as having had 12 consecutive months with no menses without an alternative medical cause.
    9.Men must agree to the following for the duration of the study and 8 weeks after their last IMP dose:
    a Avoid fathering a child.
    b Use protocol-defined methods of contraception.
    c Refrain from donating sperm.
    10. Willing and able to give written informed consent.
    E.4Principal exclusion criteria
    1. Have received other anticomplement therapies (approved or investigational) within 5 half-lives of the agent prior to randomization (e.g., eculizumab within 10 weeks, ravulizumab within 36 weeks or sutimlimab within 15 weeks) and are not able or willing to refrain from using them during the study. Patients previously treated with > 1 dose of sutimlimab will be excluded if they have not experienced a documented increase in Hb ≥ 1.0 g/dL during sutimlimab treatment.
    2. Treatment with belimumab, rituximab or other anti-CD20 antibody (such as obinutuzumab or ocrelizumab, with bendamustine, fludarabine, other cytotoxic drugs or Bruton tyrosine kinase inhibitors such as ibrutinib acalabrutinib and zanubrutinib, alone or in combination with rituximab) within 16 weeks prior to randomization.
    3. Use of prohibited medications as described in the protocol. The list of acceptable medications and required stable regimen periods are outlined in the study protocol.
    4. Diagnosis of systemic lupus erythematosus or other autoimmune diseases with antinuclear antibodies (antinuclear antibodies of long standing duration without associated clinical symptoms will be adjudicated on a case-by-case basis by the investigator after discussion with the medical monitor).
    5. History of an aggressive lymphoma or presence of a lymphoma requiring therapy.
    6. Have received an organ transplant.
    7. Cold agglutinin syndrome secondary to Mycoplasma pneumoniae, Epstein-Barr virus or other specific causative infection In patients with long history of CAD, positive IgM titer and IgG titer without associated clinical symptoms will be adjudicated on a case-by-case basis by the investigator after discussion with the medical monitor.
    8. HIV or hepatitis C virus detectable by polymerase chain reaction at screening or documented in the patient’s medical record.
    9. Chronic hepatitis B virus carriers with viral loads > 1000 IU/mL (> 5000 copies/mL) at screening or documented in the patient’s medical record. Eligible patients who are chronic inactive carriers (≤ 1000 IU/mL) must receive prophylactic antiviral treatment (e.g., entecavir, tenofovir, lamivudine) according to local country guidelines.
    10. Presence of an active malignant disease within the last 12 months other than skin basal cell carcinoma or in situ carcinoma of the cervix. A low-grade lymphoproliferative bone marrow disorder not requiring therapy by itself is not defined as a malignant disease in this context.
    11. A monospecific direct antiglobulin test result of IgG > 1+.
    12. Presence or suspicion of liver dysfunction as indicated by elevated alanine aminotransferase (ALT) > 2.5 x ULN, or direct bilirubin levels > 2 x ULN For any patient with increased direct bilirubin, the investigator should exercise medical judgement to ensure that the increased direct bilirubin value is due to hemolysis and discuss inclusion with the medical monitor. If the direct bilirubin is higher than the indirect bilirubin, in addition a thorough search for exclusion of underlying liver or cholestatic disease, including but not necessarily limited to abdominal ultrasound, is warranted to exclude liver and/or cholestatic disorders.
    13. Hypersensitivity to pegcetacoplan or to any of the excipients or placebo compounds.
    14. Known or suspected hereditary fructose intolerance.
    15. Unresolved infection caused by encapsulated bacteria including N. meningitidis, S. pneumoniae and H. influenzae.
    16. Presence or suspicion of severe recurrent or chronic infections that, in the opinion of the investigator, increase the patient’s risk by participating in the study.
    17. Participation in any other investigational drug trial or exposure to other investigational agent, device or procedure within 30 days prior to screening period.
    18. If breastfeeding, is unwilling to discontinue for the duration of study and for at least 8 weeks after the final IMP dose.
    19. Inability to cooperate with study procedures.
    20. Any disease(s), psychiatric condition, metabolic dysfunction, or findings from a physical examination or clinical laboratory test result that would cause reasonable suspicion of a disease or condition that may jeopardize the patient’s wellbeing, that may increase the risk associated with study participation, that may affect the interpretation of the results, or that would make the patient unsuitable for this study.
    21.Protected adults (guardianship, trusteeship) who are unable to express their consent and persons under court protection.
    22. Any infection (including COVID-19) requiring hospitalization or treatment with intravenous anti-infectives not resolved within 2 weeks prior to the first dose of the IMP.
    E.5 End points
    E.5.1Primary end point(s)
    Response to treatment at Week 24.
    Response is defined as:
    • An increase in hemoglobin (Hb) of ≥ 1.5 g/dL from Baseline or Hb
    normalization at Week 16; AND
    • Maintenance of this effect from Week 16 to Week 24; AND
    • The absence of PRBC transfusions (between Week 5 and Week 24).
    Note: Hb normalization is defined as within normal range (between the defined
    upper and lower limits of normal [ULN and LLN]), as set by the testing
    laboratory.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 24.
    E.5.2Secondary end point(s)
    Part A:
    • Number of PRBC transfusions from Week 5 to Week 24.
    • Change from Baseline to Week 24 in the following:
    • Lactate dehydrogenase (LDH) level.
    • Haptoglobin level.
    • Indirect bilirubin level.
    • Absolute reticulocyte counts (ARC).
    • D-dimer level.
    • Normalization of markers of hemolysis at Week 24, specifically:
    • LDH level.
    • Indirect bilirubin level.
    • ARC.
    • Time to first normalization from Baseline to Week 24 for the following:
    • Hb level.
    • LDH level.
    • Indirect bilirubin level.
    • ARC.
    • Number of PRBC units transfused from Week 5 to Week 24.
    • Change from Baseline to Week 24 in the following:
    •Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) subscale score of the FACT-An scale.
    • 12-Item Short Form Survey (SF-12) score.
    • 5-Level EuroQol 5-Dimension (EQ-5D-5L) score.
    Part B:
    • Change from Baseline to Week 48 in the following:
    • Hb level.
    • LDH level.
    • Haptoglobin level.
    • Indirect bilirubin level.
    • ARC.
    • D-dimer level.
    • Normalization of markers of hemolysis at Week 48, specifically:
    • LDH level.
    • Indirect bilirubin level.
    • ARC.
    • Durability of response for patients randomized to pegcetacoplan who achieve the primary endpoint at Week 24.
    • Change from Baseline to Week 48 in the following:
    • FACT-An score.
    • FACIT-F subscale score of the FACT-An scale.
    • SF-12 score.
    • EQ-5D-5L score.
    E.5.2.1Timepoint(s) of evaluation of this end point
    From Week 5 to Week 24. From Baseline to Week 48
    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 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 concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA50
    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
    Canada
    Japan
    United States
    Georgia
    Austria
    Belgium
    Bulgaria
    Finland
    France
    Germany
    Hungary
    Italy
    Netherlands
    Norway
    Spain
    United Kingdom
    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 study is when all randomized patients have terminated the 8 week safety follow-up period and performed the EOS visit or the Early termination visit in case of discontinuation for any reason from the study before completion.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days13
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days10
    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: 60
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 50
    F.4.2.2In the whole clinical trial 57
    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
    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 Decision2022-11-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-01-19
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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