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    Summary
    EudraCT Number:2020-004839-25
    Sponsor's Protocol Code Number:BO42451
    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:2021-09-16
    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 number2020-004839-25
    A.3Full title of the trial
    A RANDOMIZED DOUBLE-BLIND PHASE IIA STUDY EVALUATING THE EFFICACY, SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF CROVALIMAB AS ADJUNCT TREATMENT IN PREVENTION OF VASO-OCCLUSIVE EPISODES (VOE) IN SICKLE CELL DISEASE (SCD)
    ESTUDIO DE FASE IIA ALEATORIZADO Y DOBLE CIEGO PARA EVALUAR LA EFICACIA, LA SEGURIDAD, LA FARMACOCINÉTICA Y LA FARMACODINÁMICA DE CROVALIMAB COMO TRATAMIENTO COMPLEMENTARIO EN LA PREVENCIÓN DE EPISODIOS VASOOCLUSIVOS (EVO) EN LA ANEMIA DE CÉLULAS FALCIFORMES (ACF)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Crovalimab as Adjunct Treatment in Prevention of Vaso-Occlusive Episodes (VOE) in Sickle Cell Disease
    ESTUDIO PARA EVALUAR LA EFICACIA, LA SEGURIDAD, LA FARMACOCINÉTICA Y LA FARMACODINÁMICA DE CROVALIMAB COMO TRATAMIENTO COMPLEMENTARIO EN LA PREVENCIÓN DE EPISODIOS VASOOCLUSIVOS (EVO) EN LA ANEMIA DE CÉLULAS FALCIFORMES (ACF)
    A.4.1Sponsor's protocol code numberBO42451
    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 SponsorRoche Farma S. A. que realiza el ensayo en España y que actúa como representante F. Hoffmann-La Roche Ltd
    B.1.3.4CountrySwitzerland
    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 supportF. Hoffman-La Roche Ltd.
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRoche Farma S.A
    B.5.2Functional name of contact pointTrial Information Support Line-TISL
    B.5.3 Address:
    B.5.3.1Street AddressGrenzacherstrasse 124
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4070
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number+349132557300
    B.5.5Fax number+34913248196
    B.5.6E-mailspain.start_up_unit@roche.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 nameCrovalimab
    D.3.2Product code RO7112689/F03
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    Subcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCROVALIMAB
    D.3.9.2Current sponsor codeRO7112689
    D.3.9.3Other descriptive nameC5 inh MAb, SKY59, RO/CH7092230
    D.3.9.4EV Substance CodeSUB197998
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number170
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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 placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Sickle Cell Disease (SCD); vaso-occlusive episodes in SCD
    ANEMIA DE CÉLULAS FALCIFORMES (ACF);episodios vasooclusivos (EVO)
    E.1.1.1Medical condition in easily understood language
    Sickle cell disease is an inherited red blood cell disorder that affects hemoglobin and leads to painful episodes (also known as vaso-occlusive episodes [VOE]).
    La anemia de células falciformes es un trastorno hereditario de los glóbulos rojos que afecta la hemoglobina y conduce a episodios dolorosos (también conocidos como episodios vasooclusivos [VOE]
    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 21.0
    E.1.2Level PT
    E.1.2Classification code 10040644
    E.1.2Term Sickle cell disease
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10072397
    E.1.2Term Vaso-occlusive crisis
    E.1.2System Organ Class 100000004851
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    - To evaluate the efficacy of crovalimab compared with placebo
    -evaluar la eficacia de crovalimab en comparación con un placebo
    E.2.2Secondary objectives of the trial
    - To evaluate the efficacy of crovalimab compared with placebo
    - To evaluate the safety and tolerability of crovalimab compared with placebo
    - To evaluate the pharmacokinetics of crovalimab
    - To evaluate the immune response to crovalimab
    -Evaluar la eficacia de crovalimab en comparación con un placebo
    - Evaluar la seguridad y tolerabilidad de crovalimab en comparación con placebo.
    - Evaluar la farmacocinética de crovalimab.
    - Evaluar la respuesta inmune al crovalimab.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Signed ICF or Assent Form (as determined by patient’s age and individual site and country standards)
    - Age >=12 to <=55 years
    - Body weight >=40 kg
    - Male or female with confirmed diagnosis of HbSS (SCD genotype of sickle cell anemia) or HbSβ0 (SCD genotype of sickle cell beta zero thalassemia)
    - Two or more (>=2) to <=10 documented VOEs in the 12 months prior to randomization
    - If receiving concurrent SCD-directed therapy, the patient must have been on a stable dose for a minimum of 3 months prior to study enrollment. There should be no plans to modify the patients’ dosing throughout the study duration, other than for safety reasons.
    - If receiving erythropoietin, the patient must have been prescribed this medication for the preceding 3 months and be dose-stabilized for at least 3 months prior to study enrollment
    - Vaccination against N. meningitides, Vaccinations against H. influenza type B and S. pneumonia
    - Patients who have been vaccinated (partially or in full) against SARS-CoV-2 with a locally approved vaccine are eligible to be enrolled in the study, 3 days or longer after inoculation
    - Adequate hepatic and renal function
    - For women of childbearing potential, agreement to remain abstinent or use contraception during the treatment period and for 6 months after the final dose of study treatment
    -Firma del documento de consentimiento informado (DCI) o asentimiento firmado cuando proceda, según lo determinado por la edad del paciente y las normas de cada centro y país.
    -Edad >= 12 y <=55 años
    -Peso>= 40 kg
    -Varones o mujeres con diagnóstico confirmado de anemia de células falciformes (genotipo homocigótico falciforme de la globina beta [HbSS] o falciforme de la globina beta y talasemia beta cero [HbSβ0]).
    -Dos o más (>=2) a <=10 EVO documentados en los 12 meses previos a la aleatorización

    -En caso de recibir tratamiento dirigido contra la ACF concomitante,el paciente debe estar recibiendo una dosis estable, exceptuando los ajustes basados en el peso, con un buen cumplimiento terapéutico según la evaluación del investigador, durante un mínimo de 3 meses antes de su inclusión en el estudio (día 1). No está previsto modificar la dosis de los pacientes a lo largo del estudio, salvo por motivos de seguridad.
    -En caso de recibir eritropoyetina, el paciente deberá haber recibido este medicamento durante los 3 meses anteriores y haber mantenido una dosis estable durante al menos 3 meses antes de su inclusión.
    -Vacunación contra Neisseria meningitidis,H. influenzae de tipo B y S. pneumoniae
    -pacientes que hayan sido vacunados (parcial o totalmente) contra (SARS-CoV-2) con una vacuna aprobada localmente 3 días o más después de la inoculación.
    -Función hepática y renal adecuada,
    -Las mujeres con capacidad de quedarse embarazadas deben comprometerse a practicar abstinencia sexual (no mantener relaciones sexuales) o a usar métodos anticonceptivos durante el período de tratamiento y durante 6 meses después de la última dosis del tratamiento del estudio con crovalimab
    E.4Principal exclusion criteria
    - History of hematopoietic stem cell transplant
    - Participating in a chronic transfusion program and/or planning on undergoing an exchange transfusion during the duration of the study
    - History of hypersensitivity, allergic, or anaphylactic reactions to any ingredient contained in the study treatment
    - Received active treatment on another investigational trial within 28 days (or within five half-lives of that agent, whichever is greater) prior to screening visit, or plans to participate in another investigational drug trial
    - Hemoglobin <6 g/dL
    - Known or suspected hereditary complement deficiency
    - Active systemic bacterial, viral, or fungal infection within 14 days before first drug administration
    - Presence of fever (>=38 degrees Celsius) within 7 days before the first drug administration
    - Immunized with a live attenuated vaccine within 1 month before first drug administration
    - Pregnant or breastfeeding, or intending to become pregnant during the study or within 6 months after the final dose of study treatment
    - Known HIV infection with documented CD4 count <200 cells/microliter within 24 weeks prior to screening
    - History of N. meningitidis infection within the prior 6 months
    -Antecedentes de trasplante de células madre.
    -Participación en un programa de transfusiones crónicas y/o previsión de someterse a una transfusión de intercambio durante el estudio. Se permite una transfusión simple episódica
    -Antecedentes de reacciones de hipersensibilidad, alérgicas o anafilácticas a cualquiera de los componentes contenidos en el tratamiento del estudio
    -Haber recibido tratamiento activo en otro ensayo de investigación en los 28 días previos a la visita de selección o tener previsto participar en otro ensayo con un fármaco en investigación
    -Hemoglobina <6 g/dL
    -Sospecha o certeza de deficiencia hereditaria del complemento.
    -Infección bacteriana, vírica o micótica sistémica activa en los 14 días previos a la primera administración del fármaco
    - Presencia de fiebre (38 C) en los 7 días previos a la primera administración del fármaco
    - Vacunación con una vacuna de microorganismos vivos atenuados en el mes previo a la primera administración del fármaco

    - Embarazo o lactancia, o intención de quedarse embarazada durante el estudio o en los 6 meses siguientes a la última dosis del tratamiento del estudio
    - Infección conocida por el VIH con un recuento documentado de CD4 <200 cells/microliter en las 24 semanas previas a la selección
    - Antecedentes de infección por N. meningitidis en los 6 meses anteriores
    E.5 End points
    E.5.1Primary end point(s)
    1. Annualized rate of medical facility VOEs (AVR)
    1.Tasa anualizada de EVO de atención en centro médico (TEA).
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Up to 48 weeks
    1. Hasta 48 semanas
    E.5.2Secondary end point(s)
    1. Annualized rate of home VOE captured by patient report
    2. Annualized rate of uncomplicated medical facility VOE
    3. Annualized rate of acute chest syndrome (ACS)
    4. Annualized rate of days hospitalized for medical facility VOE
    5. Annualized rate of days hospitalized for treatment of non-VOE complications of SCD
    6. Change in hematologic measures from baseline to Week 49
    7. Time to first medical facility VOE from randomization
    8. Change in urinary albumin-creatinine ratio from baseline to Week 49
    9. Change from baseline to Week 49 in tricuspid regurgitant jet velocity (TRV)
    10. Proportion of patients with TRV >2.5 m/s at Week 49
    11. Change from baseline to Week 49 in Patient-Reported Outcomes Measurement Information System (PROMIS)-Fatigue score in adults
    1.Tasa anualizada de EVO de atención a domicilio registrados en el informe del paciente
    2.Tasa anualizada de EVO de atención en centro médico (TEA).
    3-Síndrome torácico agudo (STA)
    4-Tasa anualizada de días de hospitalización por EVO de atención en centro médico
    5-Tasa anualizada de días de hospitalización para el tratamiento de complicaciones de la ACF distintas de un EVO.
    6-Variación de los parámetros hematológicos entre el momento basal y la semana 49.
    7-Tiempo transcurrido hasta el primer EVO de atención en centro médico desde el momento de la aleatorización
    8-Variación del cociente albúmina/creatinina en orina entre el momento basal y la semana 49.
    9-Variación entre el momento basal y la semana 49 de la velocidad del jet de regurgitación tricuspídea (VRT).
    10-Proporción de pacientes con VRT >2,5 m/s en la semana 49.
    11-Variación entre el momento basal y la semana 49 de la puntuación del Sistema de información de medición de resultados comunicados por los pacientes (PROMIS)-Cansancio en adultos.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1-5. Baseline to Week 49
    6. Baseline to Week 49
    7. Up to Week 49
    8-9. Baseline to Week 49
    10. At Week 49
    11. Baseline to Week 49
    1-5. Visita basal a la semana 49
    6.Visita basal a la semana 49
    7. Hasta la semana 49
    8-9. Visita basal a la semana 49
    10. En la semana 49
    11. Visita basal hasta la semana 49
    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) 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 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 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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA11
    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
    Brazil
    Turkey
    France
    Italy
    Spain
    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 this study is defined as the date when the last patient, last visit, occurs, which is defined as completion of 48 weeks of the study treatment, followed by the end of safety follow-up period of 24 weeks. In addition, the study treatment may be discontinued, the patient may discontinue from the study, or the Sponsor may decide to terminate the study or discontinue a site at any time.
    El final de este estudio se define como la fecha en que se produce el último paciente, la última visita, que se define como la finalización de 48 semanas del tratamiento del estudio, seguida del final del período de seguimiento de seguridad de 24 semanas. Además, el tratamiento del estudio puede interrumpirse, el paciente puede interrumpir el estudio o el Patrocinador puede decidir finalizar el estudio o interrumpir un sitio en cualquier momento.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months11
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months11
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 23
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 23
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 67
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Minors
    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 28
    F.4.2.2In the whole clinical trial 90
    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)
    Currently, the Sponsor does not have any plans to provide Roche IMP (crovalimab) or any other study treatments to patients who have completed the study. The Sponsor may evaluate whether to
    continue providing crovalimab in accordance with the Roche Global Policy on Continued Access to Investigational Medicinal Product, available at the following website:
    http://www.roche.com/policy_continued_access_to_investigational_medicines.pdf
    Actualmente, el Promotor no tiene planes de proporcionar Roche IMP (crovalimab) o cualquier otro tratamiento del estudio a los pacientes que hayan completado el estudio. El Patrocinador puede evaluar si
    continuar proporcionando crovalimab de acuerdo con la Política global de Roche sobre acceso continuo a medicamentos en investigación, disponible en el siguiente sitio web:
    http://www.roche.com/policy_continued_access_to_investigational_medicines.pdf
    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 Decision2021-09-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-09-16
    P. End of Trial
    P.End of Trial StatusOngoing
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