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    Summary
    EudraCT Number:2021-006776-17
    Sponsor's Protocol Code Number:BCX9930-205
    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:2022-11-22
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2021-006776-17
    A.3Full title of the trial
    An Open-label Study to Evaluate the Long-term Safety of BCX9930 Monotherapy in Subjects with Paroxysmal Nocturnal Hemoglobinuria Who Previously Received BCX9930 in a BioCryst sponsored Study
    Estudio abierto para evaluar la seguridad a largo plazo de BCX9930 en monoterapia en sujetos con hemoglobinuria paroxística nocturna que habían recibido previamente BCX9930 en un estudio promovido por BioCryst
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    REDEEM - access to BCX9930
    REDEEM
    A.4.1Sponsor's protocol code numberBCX9930-205
    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 SponsorBioCryst 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 supportBioCryst Pharmaceuticals Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAMS Advanced Medical Services
    B.5.2Functional name of contact pointProject Management
    B.5.3 Address:
    B.5.3.1Street Address26 -28 Hammersmith Grove
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeW6 7BA
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+44208834 1144
    B.5.6E-mailoperations@ams-europe.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameBCX9930
    D.3.2Product code BCX9930
    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.3.8INN - Proposed INNBCX9930
    D.3.9.1CAS number BCx9930
    D.3.9.2Current sponsor codeBCX9930
    D.3.9.3Other descriptive nameBCX9930
    D.3.9.4EV Substance CodeSUB215798
    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 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
    Hemoglobinuria Paroxística Nocturna
    E.1.1.1Medical condition in easily understood language
    PNH
    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 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 assess the safety and tolerability of long-term monotherapy with BCX9930 in subjects with paroxysmal nocturnal hemoglobinuria (PNH) who previously received BCX9930 in a BioCryst-sponsored study
    Evaluar la seguridad y tolerabilidad de la monoterapia a largo plazo con BCX9930 en sujetos con hemoglobinuria paroxística nocturna (HPN) que recibieron previamente BCX9930 en un estudio patrocinado por BioCryst
    E.2.2Secondary objectives of the trial
    • To assess the continued effectiveness of BCX9930 in treatment of PNH during long-term administration
    • To evaluate the effects of long-term monotherapy with BCX9930 on the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale and Quality of Life Questionnaire for Patients with Aplastic Anemia/PNH (QLQ-AA/PNH)
    • To characterize plasma concentrations of BCX9930 and its metabolite BCX13559 (M1a) throughout the treatment period
    • To characterize the effects of long-term BCX9930 monotherapy in subjects with PNH on pharmacodynamic (PD) and complement biomarkers
    • Analizar la efectividad continua de BCX9930 en el tratamiento de la HPN durante la administración a largo plazo
    • Evaluar los efectos de la monoterapia a largo plazo con BCX9930 en la Evaluación Funcional de la Terapia de Enfermedades Crónicas (FACIT)- Escala de fatiga y Cuestionario de Calidad de Vida para Pacientes con Anemia Aplásica/HPN (QLQ-AA/HPN)
    • Caracterizar las concentraciones plasmáticas de BCX9930 y su metabolito BCX13559 (M1a) durante todo el período de tratamiento
    • Caracterizar los efectos de la monoterapia BCX9930 a largo plazo en sujetos con HPN en biomarcadores farmacodinámicos (PD) y del complemento
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Able to provide written informed consent.
    2. Non-pregnant, non-lactating female subjects.
    3. Subjects with PNH who have completed treatment in another BCX9930 clinical study and, in the opinion of the investigator, would benefit from continued treatment with BCX9930.
    4. Female participants will continue to meet at least one of the following requirements per the prior study:
    a. Be a woman of nonchildbearing potential.
    b. Be a woman of childbearing potential who agrees to use a highly effective contraceptive method throughout the study and for a duration of 30 days after the last dose of BCX9930.
    c. Alternatively, true abstinence is acceptable for women of childbearing potential when it is in line with the subject’s preferred and usual lifestyle.
    5. Male participants will continue to meet at least one of the following requirements per the prior study:
    a. Males with a female partner of childbearing potential (including a pregnant partner) must use condoms throughout the study and for a duration of 90 days after the dose of BCX9930 unless their partner is using a highly effective contraceptive method independent of the study.
    b. Alternatively, true abstinence is acceptable when it is in line with the subject’s preferred and usual lifestyle.
    6. In the opinion of the investigator, the subject is expected to adequately comply with all required study procedures and restrictions for the study
    1. Ser capaz de proporcionar un consentimiento informado por escrito.
    2. Mujeres no embarazadas ni lactantes.
    3. Los sujetos con HPN que hayan completado el tratamiento en otro estudio clínico BCX9930 y, en opinión del investigador, se beneficiarían de un tratamiento continuo con BCX9930.
    4. Las participantes de sexo femenino continuarán cumpliendo al menos uno de los siguientes requisitos según el estudio anterior:
    a. Ser una mujer no fértil.
    b. Ser una mujer en edad fértil que acepte utilizar un método anticonceptivo altamente efectivo durante todo el estudio y durante 30 días después de la última dosis de BCX9930.
    c. Como alternativa, la verdadera abstinencia es aceptable para las mujeres en edad fértil, siempre que esto pueda llevarse a cabo, teniendo en cuenta el estilo de vida preferido y habitual de la paciente.
    5. Los participantes de sexo masculino continuarán cumpliendo al menos uno de los siguientes requisitos según el estudio anterior:
    a. Los hombres con una pareja femenina en edad fértil (incluida una pareja embarazada) deben usar preservativos durante todo el estudio y por un período de 90 días después de la dosis de BCX9930, a menos que su pareja esté utilizando un método anticonceptivo altamente eficaz independiente del estudio.
    b. Como alternativa, la verdadera abstinencia es aceptable, siempre que esto pueda llevarse a cabo, teniendo en cuenta el estilo de vida preferido y habitual del paciente.
    6. En opinión del investigador, se espera que el sujeto cumpla adecuadamente con todos los procedimientos y restricciones de estudio requeridos para el estudio.
    E.4Principal exclusion criteria
    1. Any clinically significant medical or psychiatric condition including alcohol or drug dependency that, in the opinion of the investigator or sponsor, would interfere with the subject’s ability to participate in the study or increase the risk of participation for that subject.
    2. An ongoing adverse event (AE), including a laboratory abnormality, or other unacceptable toxicity that, in the judgment of the investigator, compromises the ability of the subject to continue study-specific procedures or it is considered not to be in the subject’s best interest to continue or benefit-risk assessment is no longer in favor of the subject’s continued treatment.
    3. Daily use of medications listed in the currently applicable prohibited medications list.
    4. Known or suspected hypersensitivity to BCX9930 or any of its formulation excipients (Note: prior drug rash is not exclusionary).
    1. Cualquier afección médica o psiquiátrica clínicamente significativa, incluyendo dependencia al alcohol o las drogas, que en opinión del investigador o promotor pueda interferir con la capacidad del sujeto para participar en el ensayo, o aumentar el riesgo de participación para este sujeto.
    2. Un acontecimiento adverso (AA) continuo, que incluye una anormalidad de laboratorio u otra toxicidad inaceptable que, a juicio del investigador, compromete la capacidad del sujeto para continuar los procedimientos específicos del estudio o se considera que no es lo mejor para el sujeto continuar o la evaluación de riesgo-beneficio ya no está a favor del tratamiento continuo del sujeto.
    3. Uso diario de fármacos actualmente incluidos en la lista de medicamentos prohibidos en vigor en ese momento.
    4. Hipersensibilidad conocida o sospechada a BCX9930 o cualquiera de sus excipientes de formulación (Nota: la erupción previa del fármaco no es excluyente).
    E.5 End points
    E.5.1Primary end point(s)
    • Subject incidence of graded treatment-emergent adverse events (TEAEs), laboratory abnormalities, changes to vital signs, electrocardiogram (ECG) results, and physical examination findings
    • Incidencia en las personas de eventos adversos relacionados con el tratamiento (EART), anomalías de laboratorio, cambios en los signos vitales, resultados del electrocardiograma (ECG) y hallazgos del examen físico
    E.5.1.1Timepoint(s) of evaluation of this end point
    week 48
    Semana 48
    E.5.2Secondary end point(s)
    • Change from baseline (CFB) in hemoglobin (Hb)
    • Proportion of subjects with Hb ≥ 12 g/dL
    • Proportion of subjects with Hb stabilization, defined as avoidance of a > 2 g/dL decrease in the absence of transfusion
    • Percent CFB in lactate dehydrogenase (LDH)
    • Number of units of packed red blood cells (pRBCs) transfused
    • Proportion of subjects who are transfusion-free
    • CFB in FACIT-Fatigue scale score through 48 weeks of treatment in Study 205
    • CFB in QLQ-AA/PNH scores through 48 weeks of treatment in Study 205
    • Plasma concentrations of BCX9930 and BCX13559 at steady state in subjects with PNH
    • Cambio desde el inicio (CFB) en la hemoglobina (Hb)
    • Proporción de sujetos con Hb ≥ 12 g/dl
    • Proporción de sujetos con estabilización de Hb, definida como evitar de una disminución > 2 g/dL en ausencia de transfusión
    • Porcentaje de CFB en lactato deshidrogenasa (LDH)
    • Número de unidades de concentrado de glóbulos rojos (pRBC) transfundidos
    • Proporción de sujetos que están libres de transfusiones
    • CFB en la puntuación de la escala de fatiga FACIT hasta las 48 semanas de tratamiento en el estudio 205
    • CFB en las puntuaciones QLQ-AA/PNH hasta las 48 semanas de tratamiento en el Estudio 205
    • Concentraciones plasmáticas de BCX9930 y BCX13559 en estado estacionario en sujetos con HPN
    E.5.2.1Timepoint(s) of evaluation of this end point
    week 48
    Semana 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 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 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.8.3 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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA25
    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
    Brazil
    Canada
    China
    Hong Kong
    Japan
    Malaysia
    South Africa
    United States
    Austria
    France
    Lithuania
    Netherlands
    Romania
    Spain
    Czechia
    Italy
    Albania
    Azerbaijan
    Hungary
    Slovakia
    Turkey
    United Kingdom
    Serbia
    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
    LPLV
    LPLV
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    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: 20
    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 80
    F.4.2.2In the whole clinical trial 200
    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)
    BCX9930 will continue to be supplied, either through an extension of this study or a locally approved access program, until available commercially or development is discontinued.
    Subjects who do not wish to continue BCX9930 will return to the Standard of Care offered locally
    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 Decision2023-02-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-02-14
    P. End of Trial
    P.End of Trial StatusOngoing
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