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    The EU Clinical Trials Register currently displays   43801   clinical trials with a EudraCT protocol, of which   7259   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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    EudraCT Number:2019-003830-17
    Sponsor's Protocol Code Number:ACH228-110
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-07-14
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2019-003830-17
    A.3Full title of the trial
    A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of the Oral Factor D (FD) Inhibitor ALXN2050 (ACH-0145228) in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy
    Ensayo abierto fase 2 de prueba de concepto para evaluar la eficacia, seguridad y farmacocinética del inhibidor oral del factor D (FD) ALXN2050 (ACH 0145228) en pacientes con hemoglobinuria paroxística nocturna (HPN) como monoterapia.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of the Oral Factor D (FD) Inhibitor ALXN2050 (ACH-0145228) in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy
    Estudio del inhibidor oral del factor D (FD) ALXN2050 (ACH-0145228) en pacientes con hemoglobinuria paroxística nocturna (HPN) como monoterapia
    A.3.2Name or abbreviated title of the trial where available
    Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy
    A.4.1Sponsor's protocol code numberACH228-110
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04170023
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1241-2441
    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 SponsorAlexion 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 supportAlexion Pharmaceuticals Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAlexion Europe SAS
    B.5.2Functional name of contact pointEuropean Clinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address103-105 Rue Anatole France
    B.5.3.2Town/ cityLevallois-Perret
    B.5.3.3Post code92300
    B.5.4Telephone number+34 93 2723019
    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/3/21/2401
    D.3 Description of the IMP
    D.3.1Product nameALXN2050
    D.3.2Product code ALXN2050
    D.3.4Pharmaceutical form Capsule
    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.3.8INN - Proposed INNN/A
    D.3.9.1CAS number 2086178-00-7
    D.3.9.2Current sponsor codeALXN2050
    D.3.9.3Other descriptive nameACH-0145228
    D.3.9.4EV Substance CodeSUB204167
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number60
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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 (PNH)
    Hemoglobinuria paroxística nocturna (HPN)
    E.1.1.1Medical condition in easily understood language
    Paroxysmal Nocturnal Hemoglobinuria (PNH)
    Hemoglobinuria paroxística nocturna (HPN)
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10034042
    E.1.2Term Paroxysmal nocturnal haemoglobinuria
    E.1.2System Organ Class 10038359 - Renal and urinary disorders
    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 ALXN2050 based on improvement in hemoglobin (Hgb)
    Evaluar la eficacia de ALXN2050 en función de la mejora de la hemoglobina (Hgb)
    E.2.2Secondary objectives of the trial
    • To evaluate the efficacy of ALXN2050 based on reduction in transfusion requirements
    • To evaluate the efficacy of ALXN2050 based on lactate dehydrogenase (LDH)
    • To assess laboratory markers of hemolysis and other markers relevant in patients with paroxysmal nocturnal hemoglobinuria (PNH)
    • Evaluar la eficacia de ALXN2050 en función de la reducción de las necesidades de transfusión
    • Evaluar la eficacia de ALXN2050 en función de la lactato deshidrogenasa (LDH)
    • Evaluar los marcadores analíticos de hemólisis y otros marcadores relevantes en pacientes con hemoglobinuria paroxística nocturna (HPN)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    All patients must meet all of the following conditions:
    1. Diagnosis of PNH.
    2. Male or female, ≥ 18 years of age (or minimum adult age in accordance with local legal requirements).

    Eligibility Criteria Specific for Group 1:
    1. PNH Patients who have no history of treatment with any complement inhibitor at any dose.
    2. PNH Type III erythrocyte or granulocyte clone size ≥10%
    3. Absolute reticulocyte count ≥100×10^9/liter [L].
    4. Anemia (Hgb <10.5 grams/deciliter [g/dL]).
    5. LDH ≥1.5× upper limit of normal.
    6. Platelet count ≥30,000/microliter (µL) without the need for platelet transfusions.
    7. Absolute neutrophil count (ANC) ≥750/ µL.

    Eligibility Criteria Specific for Group 2:
    Patients on stable eculizumab switching to ALXN2050 (Group 2) must meet the following criteria:
    1. Stable background regimen of at least 24 weeks for eculizumab without change in dose or interval for at least the past 8 weeks
    2. Anemia (Hgb <10 g/dL)
    3. Absolute reticulocyte count ≥100×10^9/L
    4. Platelet count ≥30,000/µL without the need for platelet transfusions
    5. Absolute neurophil count (ANC) ≥750/ µL

    Eligibility Criteria Specific for Group 3:
    1. Patient received danicopan during Study ACH-471-103.
    Todos los pacientes deben cumplir todas las condiciones siguientes:
    1. Diagnóstico de HPN.
    2. Hombre o mujer, ≥18 años de edad (o edad adulta mínima de acuerdo con los requisitos legales locales).
    Criterios de elegibilidad específicos para el grupo 1:
    1. Pacientes con HPN que no tienen antecedentes de tratamiento con ningún inhibidor del complemento a ninguna dosis.
    2. Tamaño del clon de granulocitos o eritrocitos tipo III de HPN ≥10 %
    3. Recuento absoluto de reticulocitos ≥100×10^9/litro [l].
    4. Anemia (Hgb <10,5 gramos/decilitro [g/dl]).
    5. ≥1,5 x LSN (límite superior de la normalidad).
    6. Recuento plaquetario ≥30 000/microlitro (μl) sin necesidad de transfusiones de plaquetas.
    7. Cifra absoluta de neutrófilos (ANC) ≥750/μL.

    Criterios de elegibilidad específicos para el grupo 2:
    Los pacientes con eculizumab estable que cambien a ALXN2050 (grupo 2) deben cumplir los siguientes criterios:
    1. Régimen de base estable de al menos 24 semanas para eculizumab sin cambio en la dosis o el intervalo durante al menos las últimas 8 semanas
    2. Anemia (Hgb <10 g/dl)
    3. Recuento absoluto de reticulocitos ≥100×10^9/l
    4. Recuento plaquetario ≥30 000/μl sin necesidad de transfusiones de plaquetas 5. Cifra absoluta de neutrófilos (ANC) ≥750/ μL
    Criterios de elegibilidad específicos para el grupo 3:
    1. El paciente recibió danicopán durante el estudio ACH-471-103.
    E.4Principal exclusion criteria
    1. History of a major organ transplant or hematopoietic stem cell/marrow transplant .
    2. Known aplastic anemia or other bone marrow failure that requires HSCT, or if these patients are on immunosuppressive agents such as (but not limited to) cyclosporine, tacrolimus, mycophenolate, or others for less than 24 weeks prior to enrollment.
    3. Known underlying bleeding disorders (eg, coagulation factor deficiencies, idiopathic thrombocytopenic purpura, Von Willebrand disease) or any other conditions leading to anemia not primarily associated with PNH.
    4. Estimated glomerular filtration rate <30 milliliters/minute/1.73 meters squared and/or are on dialysis.
    1. Antecedentes de trasplante de órganos importantes o trasplante de células madre hematopoyéticas/médula ósea.
    2. Anemia aplásica conocida u otra insuficiencia de la médula ósea que requiera TCMH, o si estos pacientes están recibiendo inmunodepresores como (entre otros) ciclosporina, tacrolimus, micofenolato u otros durante menos de 24 semanas antes de la inscripción.
    3. Trastornos hemorrágicos subyacentes conocidos (p. ej., deficiencias del factor de coagulación, púrpura trombocitopénica idiopática, enfermedad de Von Willebrand) o cualquier otra afección que lleve a anemia no asociada principalmente a la HPN.
    4. Tasa de filtración glomerular estimada <30 mililitros/minuto/1,73 metros cuadrados y/o están en diálisis.
    E.5 End points
    E.5.1Primary end point(s)
    1. Change in Hgb relative to baseline
    1. Cambio en la Hgb en relación con el valor inicial
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Week 12
    1. Semana 12
    E.5.2Secondary end point(s)
    2. Number Of Patients Who Have Transfusion Avoidance
    3. Number Of RBC Units Transfused and Transfusion Instances
    Transfusion requirements during the 12 weeks of treatment will be compared to 12 weeks of historical transfusion requirements.
    4. Change In LDH Relative to Baseline
    5. Change From Baseline In Absolute Reticulocyte Count
    6. Change From Baseline In Direct Bilirubin
    7. Change From Baseline In Total Bilirubin
    8. Change From Baseline In PNH RBC Clone Size
    9. Change From Baseline In C3 Complement Protein Fragment Deposition On PNH RBCs
    10. Incidence of TEAEs, SAEs, and Events Leading To Discontinuation Of Study Medication
    11. Change in HgB Relative To Baseline
    12. Change in LDH Relative To Baseline
    13. Change in FACIT Fatigue Scale (Version 4) Scores Relative To Baseline
    2. Número de pacientes con ahorro de transfusiones
    3. Número de unidades de eritrocitos Transfundidas e Instancias de transfusión Las necesidades de transfusión durante las 12 semanas de tratamiento se compararán con las 12 semanas de necesidades de transfusión históricas.
    4. Cambio en la LDH con respecto al inicio
    5. Cambio desde el inicio en el recuento absoluto de reticulocitos
    6. Cambio desde el inicio en la bilirrubina directa
    7. Cambio con respecto al inicio en la bilirrubina total
    8. Cambio desde el inicio en el tamaño del clon de eritrocitos de HPN
    9. Cambio desde el inicio en el fragmento de proteína del complemento C3
    Depósito en los eritrocitos de HPN
    10. Incidencia de AAST, AAG y acontecimientos que provocaron la interrupción del medicamento del estudio
    11. Cambio en HgB con respecto al inicio
    12. Cambio en la LDH con respecto al inicio
    13. Cambio en las puntuaciones de la escala de fatiga FACIT (versión 4) en relación con el inicio
    E.5.2.1Timepoint(s) of evaluation of this end point
    2 - 3: Up to Week 12
    4 - 9: Week 12
    10: Through Study Completion, An Average Of 121 Weeks
    11 - 12: Long-term Extension Period, Week 16 to Week 108
    13: Week 12, Week 108
    2 - 3: Hasta la semana 12
    4 - 9: Semana 12
    10: Hasta la finalización del estudio, una media de 121 semanas
    11 - 12: Período de extensión a largo plazo, de la semana 16 a la semana 108
    13: Semana 12, Semana 108
    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 Yes
    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
    Effect on biomarkers
    Efecto en los biomarcadores
    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. 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 trial3
    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 EEA7
    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
    Korea, Republic of
    New Zealand
    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 the study is defined as the date of the last visit of the last patient remaining in the study.
    El final del estudio se define como la fecha de la última visita del último paciente que queda en el estudio.
    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 months4
    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 months11
    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.1.1Number of subjects for this age range: 0
    F.1.1.1In Utero No
    F. of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F. of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F. of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F. of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F. of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) No
    F. of subjects for this age range: 0
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 25
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 10
    F.4.2.2In the whole clinical trial 26
    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)
    The Sponsor currently does not intend to provide any treatment or care after the subjects have ended the participation in the trial. However the Sponsor considers to review the decision before the end of the global trial.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-11-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-09-13
    P. End of Trial
    P.End of Trial StatusOngoing
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