Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register allows you to search for protocol and results information on:
  • interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA);
  • clinical trials conducted outside the EU / EEA that are linked to European paediatric-medicine development.
  • Learn   more about the EU Clinical Trials Register   including the source of the information and the legal basis.


    The EU Clinical Trials Register currently displays   40165   clinical trials with a EudraCT protocol, of which   6574   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).
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2016-002652-25
    Sponsor's Protocol Code Number:ACH471-100
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-02-08
    Trial results View 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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2016-002652-25
    A.3Full title of the trial
    A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Studio proof-of-concept, di fase 2, in aperto per valutare l'efficacia, la sicurezza e la farmacocinetica di ACH-0144471 in pazienti non trattati con emoglobinuria parossistica notturna (EPN)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A treatment study of ACH-0144471 in Untreated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Studio sul trattamento con ACH-0144471 in pazienti non trattati con Emoglobinuria Parossistica Notturna (PNH).
    A.3.2Name or abbreviated title of the trial where available
    A treatment study of ACH-0144471 in Untreated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH
    Studio sul trattamento con ACH-0144471 in pazienti non trattati con Emoglobinuria Parossistica Nottu
    A.4.1Sponsor's protocol code numberACH471-100
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03053102
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1190-3490
    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 SponsorACHILLION 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 supportAchillion Pharmaceuticals INC.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAchillion Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointClinical Operations
    B.5.3 Address:
    B.5.3.1Street Address300 George Street
    B.5.3.2Town/ cityNew Heaven
    B.5.3.3Post codeCT 06511
    B.5.3.4CountryUnited States
    B.5.4Telephone number0012037525577
    B.5.5Fax number0012037525577
    B.5.6E-mailPNHTrialInquiries@achillion.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/3/17/1946
    D.3 Description of the IMP
    D.3.2Product code ACH-0144471
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 1903768-17-1
    D.3.9.2Current sponsor codeACH-0144471
    D.3.9.4EV Substance CodeSUB189885
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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.IMP: 2
    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/3/17/1946
    D.3 Description of the IMP
    D.3.2Product code ACH-0144471
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 1903768-17-1
    D.3.9.2Current sponsor codeACH-0144471
    D.3.9.3Other descriptive nameACH-0144471
    D.3.9.4EV Substance CodeSUB189885
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number75
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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.IMP: 3
    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/3/17/1946
    D.3 Description of the IMP
    D.3.2Product code ACH-0144471
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 1903768-17-1
    D.3.9.2Current sponsor codeACH-0144471
    D.3.9.3Other descriptive nameACH-0144471
    D.3.9.4EV Substance CodeSUB189885
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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
    Untreated Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Emoglobinuria parossistica notturna non trattata (EPN)
    E.1.1.1Medical condition in easily understood language
    Rare blood disorder that that causes red blood cells to break down earlier than they should
    Disturbo raro del sangue che causa una rottura precoce dei globuli rossi
    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 evaluate the efficacy of 28 days of oral dosing with ACH-0144471 in currently untreated PNH patients, based on decreases in lactate dehydrogenase (LDH)
    Valutare l¿efficacia di 28 giorni di somministrazione orale di ACH-0144471 in pazienti con EPN attualmente non trattati, sulla base delle riduzioni della lattato deidrogenasi (LDH)
    E.2.2Secondary objectives of the trial
    ¿ To evaluate the efficacy of 28 and 84 days of oral dosing with ACH-0144471 in currently untreated PNH patients, based on increases in hemoglobin (Hgb) levels
    ¿ To evaluate the safety and tolerability of ACH-0144471 in currently untreated PNH patients receiving daily oral dosing by assessing SAEs, AEs = Grade 3, and laboratory abnormalities = Grade 3, and events leading to discontinuation of study drug
    ¿ To evaluate the pharmacokinetic (PK) and pharmacodynamics (PD) profile of ACH-0144471 in currently untreated PNH patients receiving daily oral dosing for 28 days
    ¿ Valutare l¿efficacia di 28 e 84 giorni di somministrazione orale di ACH-0144471 in pazienti con EPN attualmente non trattati, sulla base degli aumenti dei livelli di emoglobina (Hb)
    ¿ Valutare la sicurezza e la tollerabilit¿ di ACH-0144471 nei pazienti con EPN attualmente non trattati che ricevono la somministrazione orale giornaliera, mediante la valutazione degli eventi avversi gravi (SAE), degli eventi avversi (AE) di grado = 3, delle anomalie di laboratorio di grado = 3 e degli eventi che determinano l¿interruzione del farmaco dello studio
    ¿ Valutare il profilo farmacocinetico (PK) e farmacodinamico (PD) di ACH-0144471 nei pazienti con EPN attualmente non trattati che ricevono la somministrazione orale giornaliera per 28 giorni
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Currently untreated PNH patients with PNH Type III erythrocyte and/or granulocyte clone size =10% and anemia (Hgb <12 g/dL) with adequate reticulocytosis (as determined by the investigator)
    2. LDH =1.5× upper limit of normal (ULN)
    3. Platelet count =50,000/µL without the need for platelet transfusions
    4. Documentation of vaccination for N. meningitidis, H. influenzae, and S. pneumoniae, or willingness to receive vaccinations as described in Section 6.3
    5. Age =18 years (or = minimum adult age in accordance with local legal requirements)
    6. Female participants of childbearing potential must agree to use an acceptable method of contraception (as defined in Section 5.5.5) from the date of signing the informed consent to the first day of dosing (Day 1), and must agree to use a highly effective method of contraception (as defined in Section 5.5.5) from the first day of dosing to 30 days after their last dose of study drug. Female participants of childbearing potential must also have a negative serum pregnancy test during Screening and negative urine pregnancy test on Day 1. Female participants of non-childbearing potential need not employ a method of contraception.
    7. Non-sterile male participants must agree to use a highly effective method of contraception (as defined in Section 5.5.5) with their partner(s) of childbearing potential from the first day of dosing to 90 days after their last dose of study drug. Males who are surgically sterile need not employ additional contraception. Males must agree not to donate sperm while enrolled in this study and for 90 days after their last dose of study drug.
    8. Must agree to provide written informed consent
    9. Must be willing, at all times, to have transportation and telephone access, and to be within one hour of an emergency medical center
    1. Pazienti con EPN attualmente non trattati che presentano dimensioni dei cloni eritrocitari e/o granulocitari EPN di tipo III = 10% e anemia (Hb < 12 g/dl) con reticolocitosi adeguata (come determinato dallo sperimentatore)
    2. LDH = 1,5 volte il limite superiore della norma (LSN)
    3. Conta piastrinica = 50.000/µl senza necessità di trasfusioni di piastrine
    4. Documentazione della vaccinazione contro N. meningitidis, H. influenzae e S. pneumoniae o disponibilità a ricevere le vaccinazioni, come riportato al Paragrafo 6.3
    5. Età = 18 anni (o = età adulta minima in conformità ai requisiti legali locali)
    6. Le partecipanti di sesso femminile in età fertile devono acconsentire ad adottare un metodo contraccettivo accettabile (come indicato al Paragrafo 5.5.5) dalla data di firma del consenso informato fino al primo giorno di somministrazione (Giorno 1) nonché acconsentire a usare un metodo contraccettivo altamente efficace (come indicato al Paragrafo 5.5.5) dal primo giorno di somministrazione fino a 30 giorni dopo l’ultima dose del farmaco dello studio. Le partecipanti di sesso femminile in età fertile devono inoltre risultare negative a un test di gravidanza sul siero durante lo screening e a un test di gravidanza sulle urine il Giorno 1. Le partecipanti di sesso femminile che non sono in età fertile non devono adottare un metodo contraccettivo
    7. I partecipanti di sesso maschile non sterili devono acconsentire ad adottare un metodo contraccettivo altamente efficace (come indicato al Paragrafo 5.5.5) con la/e propria/e compagna/e in età fertile dal primo giorno di somministrazione fino a 90 giorni dopo l’ultima dose del farmaco dello studio. I soggetti di sesso maschile chirurgicamente sterili non devono adottare metodi contraccettivi supplementari. I soggetti di sesso maschile devono acconsentire a non donare il seme mentre sono arruolati in questo studio e fino a 90 giorni dopo l’ultima dose del farmaco dello studio.
    8. Devono acconsentire a fornire un consenso informato scritto
    9. Devono essere disponibili ad avere accesso costante ai trasporti e al telefono, e risiedere in un’area in cui un pronto soccorso sia raggiungibile entro un’ora
    E.4Principal exclusion criteria
    1. History of a major organ transplant (e.g., heart, lung, kidney, liver) or hematopoietic stem cell/marrow transplant
    2. History of dosing with another investigational agent within 30 days or 5 half-lives of the investigational agent prior to study drug administration, whichever is greater
    3. History of dosing with eculizumab at any dose or interval within the past 75 days before study drug administration
    4. Known or suspected complement deficiency
    5. Contraindication to one or more of the required vaccinations
    6. Active bacterial infection or clinically significant active viral infection, a body temperature >38°C, or other evidence of infection on Day 1, or history of febrile illness within 14 days prior to first study drug administration
    7. History of meningococcal infection, or a first-degree relative or household contact with a history of meningococcal infection
    8. History of hypersensitivity reactions to commonly used antibacterial agents, including beta-lactams, penicillin, aminopenicillins, fluoroquinolones (specifically including ciprofloxacin), cephalosporins, and carbapenems, which in the opinion of the investigator would make it difficult to properly provide either empiric antibiotic therapy or treat an active infection
    9. History or presence of any clinically relevant co-morbidities that would make the patient inappropriate for the study (for example, is likely to result in deterioration of the patient's condition, affect the patient's safety during the study, or confound the results of the study)
    10. Laboratory abnormalities at screening, including: • Alkaline phosphatase (ALP) >1.5× upper limit of normal (ULN) • Absolute neutrophil count (ANC) <1,000/µL • Alanine aminotransferase (ALT) > ULN • Any other clinically significant laboratory abnormality that, in the opinion of the Primary Investigator (PI), would make the patient inappropriate for the study or put the patient at undue risk
    11. Females who are pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration or patients with a female partner who is pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration
    12. Prior history or current evidence of biliary cholestasis
    13. Gilbert's syndrome Patients with history or family history suggestive of Gilbert's syndrome should be tested and excluded from study if positive for UGT1A1 genotyping polymorphism or missense change
    14. Evidence of human immunodeficiency virus (HIV), hepatitis B or hepatitis C infection (positive serology for HIV-1 antibody (HIV Ab), positive hepatitis B surface antigen (HbsAg), or positive anti-HCV antibody (HCV Ab) at Screening or historically)
    1. Anamnesi di trapianto d’organo maggiore (per es. cuore, polmone, rene, fegato) o trapianto di cellule staminali emopoietiche/midollo osseo
    2. Anamnesi di somministrazione di un altro agente sperimentale entro 30 giorni o 5 emivite dell’agente stesso, a seconda di quale dei due periodi abbia durata maggiore, prima della somministrazione del farmaco dello studio
    3. Anamnesi di somministrazione di eculizumab, a qualsiasi dosaggio o intervallo, entro i 75 giorni precedenti la somministrazione del farmaco dello studio
    4. Deficit di complemento, noto o sospetto
    5. Controindicazione a una o più delle vaccinazioni richieste
    6. Infezione batterica attiva o infezione virale attiva clinicamente significativa, temperatura corporea > 38 °C o altra evidenza di infezione il Giorno 1, oppure anamnesi di malattia febbrile nei 14 giorni precedenti la prima somministrazione del farmaco dello studio
    7. Anamnesi di infezione meningococcica oppure contatto con un parente di primo grado o convivente con anamnesi di infezione meningococcica
    8. Anamnesi di reazioni da ipersensibilità agli agenti antibatterici comunemente impiegati, tra cui i beta-lattamici, la penicillina, le amminopenicilline, i fluorochinoloni (segnatamente la ciprofloxacina), le cefalosporine e i carbapenemi, che secondo l’opinione dello sperimentatore renderebbero difficoltoso somministrare una terapia antibiotica empirica o un trattamento per un’infezione attiva adeguati
    9. Anamnesi o presenza di qualsiasi comorbilità clinicamente rilevante che renderebbe il paziente inadatto allo studio (ad esempio, verosimilmente in grado di determinare un deterioramento delle condizioni del paziente, influire sulla sicurezza del paziente durante lo studio o confondere i risultati dello studio)
    10. Valori di laboratorio non normali allo screening, tra cui: • Fosfatasi alcalina (ALP) > 1,5 volte il limite superiore della norma (LSN) • Conta assoluta dei neutrofili (CAN) < 1.000/µl • Alanina transaminasi (ALT) > LSN • Qualsiasi altro valore di laboratorio non normale clinicamente significativo che, secondo l’opinione dello sperimentatore principale (PI), renderebbe il paziente inadatto allo studio o lo esporrebbe a rischi inutili
    11. Donne in gravidanza, in allattamento o che pianificano una gravidanza durante lo studio o entro 90 giorni dalla somministrazione del farmaco dello studio oppure pazienti con una compagna in gravidanza, in allattamento o che pianifica una gravidanza durante lo studio o entro 90 giorni dalla somministrazione del farmaco dello studio
    12. Anamnesi remota o attuale evidenza di colestasi biliare
    13. Sindrome di Gilbert I pazienti con anamnesi personale o familiare indicativa di sindrome di Gilbert devono essere sottoposti ad analisi ed esclusi dallo studio nel caso alla genotipizzazione risultassero positivi per polimorfismo o mutazione missenso di UGT1A1
    14. Evidenza di infezione da virus dell’immunodeficienza umana (HIV), infezione da virus dell’epatite B o C (test sierologico positivo per anticorpi anti-HIV-1 [HIV-Ab], positivo per antigene di superficie dell’epatite B [HbsAg] o positivo per anticorpi anti-HCV [HCVAb] allo screening o all’anamnesi)
    E.5 End points
    E.5.1Primary end point(s)
    Relative decrease in LDH from baseline
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 28
    E.5.2Secondary end point(s)
    ¿ To evaluate the efficacy of oral dosing with ACH-0144471 in currently untreated PNH patients, based on increases in hemoglobin (Hgb) levels
    ¿ To evaluate the safety and tolerability of ACH-0144471 in currently untreated PNH patients receiving daily oral dosing by assessing SAEs, AEs = Grade 3, and laboratory abnormalities = Grade 3, and events leading to discontinuation of study drug
    ¿ To evaluate the pharmacokinetic (PK) and pharmacodynamics (PD) profile of ACH-0144471 in currently untreated PNH patients
    E.5.2.1Timepoint(s) of evaluation of this end point
    ¿ To evaluate the efficacy of oral dosing with ACH-0144471 in currently untreated PNH patients, based on increases in hemoglobin (Hgb) levels (Day 28 and Day 84)
    ¿ To evaluate the safety and tolerability of ACH-0144471 in currently untreated PNH patients receiving daily oral dosing by assessing SAEs, AEs = Grade 3, and laboratory abnormalities = Grade 3, and events leading to discontinuation of study drug (up to 15 weeks)
    ¿ To evaluate the pharmacokinetic (PK) and pharmacodynamics (PD) profile of ACH-0144471 in currently untreated PNH patients (up to Day 28)
    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 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 Yes
    E.6.13.1Other scope of the trial description
    Assessment of Biomarkers
    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 No
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA3
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Italy
    Korea, Republic of
    New Zealand
    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 Day 84 to Day 104 (6-day taper + 14 day follow up)
    The sponsor reserves the right to close any study site or terminate the study at any time for any reason at the sole discretion of the sponsor.
    LVLS da Giorno 84 a Giorno 104 (6-giorni di riduzione della dose + 14 day follow up)
    Lo sponsor si riserva il diretto di chiudere qualsiasi sito o di terminare lo studio in ogni momento per ogni ragione a sola discrezione dello 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 months1
    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 months1
    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: 12
    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 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 state4
    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)
    After the patient has finished the trial, they will have the option to participate in a long-term-treatment study with ACH-0144471 (Patients may remain in that study until ACH-0144471 is commercially available in their country or the development of ACH-0144471 as a potential therapy for PNH is terminated, or until the therapy is no longer tolerated or effective).
    Sponsor reserves the right to close any study site or terminate the study at any time for any reason (sole discretion of the sponsor).
    Alla fine del trial i pazienti avranno l'opzione di partecipare in un trattamento di lungo periodo con ACH-0144471 (I pazienti potranno rimanere in questo studio fino a quando ACH-0144471 non sarà disponibile commercialmente nel loro paese o fino a quando lo sviluppo come ACH-0144471 come potenziale terapia sarà terminato o fino a quando la terapia non sarà più efficace o tollerata.
    Lo sponsor si riserva il diritto di chiudere qualsiasi centro o di terminare lo studio in ogni momento per ogni ra
    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 Decision2018-03-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-12-20
    P. End of Trial
    P.End of Trial StatusCompleted
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
    European Medicines Agency © 1995-2021 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    Legal notice
    EMA HMA