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    Summary
    EudraCT Number:2019-003797-10
    Sponsor's Protocol Code Number:APL2-ALS-206
    National Competent Authority:Ireland - HPRA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2020-09-03
    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 ConcernedIreland - HPRA
    A.2EudraCT number2019-003797-10
    A.3Full title of the trial
    A Phase 2, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of pegcetacoplan in subjects with amyotrophic lateral sclerosis (ALS)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate the Safety and Efficacy of Pegcetacoplan in Subjects with ALS
    A.4.1Sponsor's protocol code numberAPL2-ALS-206
    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 AuthorisationSweden
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    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
    Amyotrophic Lateral Sclerosis
    E.1.1.1Medical condition in easily understood language
    Amyotrophic Lateral Sclerosis
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10002026
    E.1.2Term Amyotrophic lateral sclerosis
    E.1.2System Organ Class 10029205 - Nervous system 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 per week subcutaneous (SC) doses of pegcetacoplan 1080 mg compared to placebo in subjects with sporadic ALS as measured by the Combined Assessment of Function and Survival (CAFS) rank score (joint-rank score)
    E.2.2Secondary objectives of the trial
    •To assess the effect of pegcetacoplan compared to placebo as measured by the Revised ALSFRS-R score
    •To assess the effect of pegcetacoplan compared to placebo on disease progression as measured by respiratory function through percentage of slow vital capacity (%SVC)
    •To determine the effect of pegcetacoplan compared to placebo on muscle strength as measured by handheld dynamometry (HHD)
    •To determine the effect of pegcetacoplan compared to placebo on survival or specified state of disease progression
    •To assess the effect of pegcetacoplan compared to placebo on health-related quality of life as measured by ALSAQ-40
    •To assess the safety of pegcetacoplan during the randomized and open-label treatment periods through incidence and severity of TEAEs, clinical laboratory tests (hematology, chemistry), vital signs, and physical examinations
    •To assess the long-term efficacy of pegcetacoplan using ALSFRS-R, %SVC, HHD, and ALSAQ-40 during the open-label treatment period
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Sporadic ALS diagnosed as definite, probable, or laboratory-supported probable as defined by the revised El Escorial criteria (Brooks et al. 2000)
    2.At least 18 years of age
    3.Slow vital capacity ≥60% of the predicted value at screening
    4.Onset of ALS symptoms within 72 weeks prior to screening
    5.Total ALSFRS-R score of ≥30 at screening
    6.Women of childbearing potential defined as any woman who has experienced menarche and who is NOT permanently sterile or postmenopausal
    a.must have a negative pregnancy test at screening and
    b.must agree to use protocol defined methods of contraception for the duration of the study and 90 days after their last dose of investigational product.
    i.Postmenopausal is defined as 12 consecutive months with no menses without an alternative medical cause.
    7.Males must agree to
    a.use protocol defined methods of contraception and
    b.refrain from donating sperm for the duration of the study and 90 days after their last dose of investigational product.
    8.Have vaccination against Streptococcus pneumoniae, N meningitidis (types A, C, W, Y, and B), and H influenzae (type B) either within 5 years prior to Baseline Visit 2b, or agree to receive vaccination at least 7 days prior to Baseline Visit 2b. Vaccination is mandatory, unless documented evidence exists that subjects are nonresponders to vaccination (as evidenced by titers or display titer levels within acceptable local limits).
    9.Willing and able to give informed consent and comply with study procedure and assessments (including at-home assessments)
    E.4Principal exclusion criteria
    1.Confirmed or suspected other causes of neuromuscular weakness
    2.Diagnosis of another neurodegenerative disease(s)
    3.Subject with significant cognitive impairment, clinical dementia, or psychiatric illness that in the opinion of the investigator may increase subject’s risk by participating in the study or confound the outcome of the study
    4.Subjects with a significant pulmonary disorder not attributed to ALS or who require treatments that might complicate the evaluation of the effect of ALS on respiratory function (eg, chronic obstructive pulmonary disease, pulmonary fibrosis, cystic fibrosis, pulmonary arterial hypertension)
    5.Current use or anticipated need, in the opinion of the investigator, of a diaphragm pacing system during the randomized treatment period
    6.Riluzole initiation or change in dose within 30 days prior to the start of the screening period or planned initiation during study participation. If using riluzole, the subject should remain on the drug throughout Part 2 of study participation, but the dosage may be altered or the drug discontinued at any time by the investigator for any safety concern. Riluzole-naïve subjects are allowed in the study.
    7.Edaravone initiation or change in dose within 60 days prior to the start of the screening period or planned initiation during study participation. If using edaravone, the subject should remain on the drug throughout Part 2 of study participation, but the dosage may be altered or the drug discontinued at any time by the investigator for any safety concern. Edaravone-naïve subjects are allowed in the study.
    8.Positive response to Item 4 or 5 of the Columbia Suicide Severity Rating Scale
    9.Subjects with detectable hepatitis C by polymerase chain reaction at screening
    10.Subjects with chronic inactive hepatitis B with viral loads >1000 IU/mL (>5000 copies/mL) at screening. Eligible subjects who are chronic active carriers (≤1000 IU/mL) must receive prophylactic antiviral treatment according to local country guidelines (eg, entecavir, tenofovir, lamivudine)
    11.History of an aggressive lymphoma or presence of a lymphoma requiring therapy by itself
    12.Active or overt malignant disease other than basal cell carcinoma or cutaneous squamous cell carcinoma
    13.Received organ transplant
    14.Presence or suspicion of liver dysfunction as indicated by elevated alanine aminotransferase, aspartate aminotransferase, or bilirubin levels >2 × the upper limit of normal
    15.Presence or suspicion of severe recurrent or chronic infections that, in the opinion of the investigator, increase the subject’s risk by participating in the study
    16.Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days or within 5-half lives of the treatment (whichever is longer) prior to the start of the screening period or during study participation
    17.Use of any other complement inhibitor within 30 days or within 5-half lives of the treatment (whichever is longer) prior to the start of the screening period or during study participation
    18.If breastfeeding, unwilling to discontinue for the duration of the study and for at least 6 months after final dose of drug
    19.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
    20. Subjects with known allergy or hypersensitivity to pegcetacoplan or to any of the components
    21. Known or suspected hereditary fructose intolerance
    E.5 End points
    E.5.1Primary end point(s)
    the difference in CAFS rank score (joint-rank score) at Week 52
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 52
    E.5.2Secondary end point(s)
    •Change from baseline in ALSFRS-R at Week 52
    •Change from baseline in %SVC (at clinic visits) at Week 52
    •Change from first assessment of %SVC (home spirometry) at Week 52
    •Change from baseline in HHD megascore at Week 52
    •Time to death, permanent tracheostomy, or permanent assisted ventilation up to Week 52
    •Time to death up to Week 52
    •Change from baseline in ALSAQ-40 at Week 52
    •Change from baseline of the randomized treatment period (Visit 2) and of the open-label treatment period (Visit 15) to Week 104 for ALSFRS-R, %SVC, HHD, and ALSAQ-40
    •Time to death, permanent tracheostomy, or permanent assisted ventilation up to Week 104
    •Time to death up to Week 104
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 52 and week 104
    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 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) 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 No
    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 Yes
    E.8.1.7.1Other trial design description
    double-blind, placebo-controlled period followed by open-label (pegcetacoplan) treatment
    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 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 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
    Argentina
    Australia
    Brazil
    Canada
    Japan
    United States
    Ukraine
    Belgium
    Czechia
    France
    Germany
    Ireland
    Italy
    Netherlands
    Poland
    Spain
    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
    LVLS
    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 months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months9
    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: 180
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 48
    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 160
    F.4.2.2In the whole clinical trial 228
    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 Decision2020-10-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-04-06
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2023-07-13
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