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    Summary
    EudraCT Number:2021-001671-16
    Sponsor's Protocol Code Number:ACT17209
    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:2021-06-23
    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 number2021-001671-16
    A.3Full title of the trial
    A multicenter, open-label, Phase IIb study to evaluate the efficacy, safety and pharmacokinetics of rilzabrutinib in patients with warm autoimmune hemolytic anemia
    Estudio de fase 2b, multicéntrico, abierto, para evaluar la eficacia, seguridad y farmacocinética de rilzabrutinib en pacientes con anemia hemolítica autoinmune por anticuerpos calientes
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Efficacy, safety and pharmacokinetics of rilzabrutinib in patients with warm autoimmune hemolytic anemia
    Eficacia, seguridad y farmacocinética de rilzabrutinib en pacientes con anemia hemolítica autoinmune por anticuerpos calientes
    A.4.1Sponsor's protocol code numberACT17209
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1262-2929
    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 SponsorSanofi aventis recherche et developpement
    B.1.3.4CountryFrance
    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 supportSanofi Aventis Recherche et Developpement
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSanofi-Aventis, S.A
    B.5.2Functional name of contact pointUnidad de Estudios Clínicos
    B.5.3 Address:
    B.5.3.1Street AddressC/ Josep Pla 2, 4ª planta
    B.5.3.2Town/ cityBarcelona
    B.5.3.3Post code08019
    B.5.3.4CountrySpain
    B.5.4Telephone number+3493485 94 00
    B.5.6E-mailes-unidadestudiosclinicos@sanofi.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 nameRilzabrutinib
    D.3.2Product code SAR444671
    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 INNRilzabrutinib
    D.3.9.2Current sponsor codeSAR444671
    D.3.9.3Other descriptive namePRN1008
    D.3.9.4EV Substance CodeSUB192772
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number400
    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
    Warm autoimmune hemolytic anemia
    Anemia hemolítica autoinmune por anticuerpos calientes
    E.1.1.1Medical condition in easily understood language
    Warm autoimmune hemolytic anemia
    Anemia hemolítica autoinmune por anticuerpos calientes
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10073785
    E.1.2Term Autoimmune haemolytic anaemia
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Part A: To evaluate the efficacy of rilzabrutinib in adult patients with wAIHA
    Part B: To evaluate the long-term efficacy of rilzabrutinib in patients with wAIHA
    Parte A: Evaluar la eficacia de rilzabrutinib en pacientes adultos con AHAIc
    Parte B: Evaluar la eficacia a largo plazo de rilzabrutinib en pacientes con AHAIc
    E.2.2Secondary objectives of the trial
    Part A:
    • To evaluate the durable hemoglobin response in adult patients with wAIHA
    • To assess time to response (TTR)

    Parts A and B:
    • To assess the effect of treatment with rilzabrutinib on rescue medication requirement in patients with wAIHA
    • To evaluate the impact of rilzabrutinib treatment on fatigue
    • To evaluate the safety and tolerability of rilzabrutinib in patients with wAIHA
    Parte A:
    • Evaluar la respuesta hemoglobínica duradera en pacientes adultos con AHAIc
    • Evaluar el tiempo hasta la respuesta (THR)
    Parte A y B:
    • Evaluar el efecto del tratamiento con rilzabrutinib sobre la necesidad de medicación de rescate en los pacientes con AHAIc
    • Evaluar el efecto del tratamiento con rilzabrutinib sobre la fatiga
    • Evaluar la seguridad y la tolerabilidad de rilzabrutinib en pacientes con AHAIc
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Male and female patients with a confirmed diagnosis of primary wAIHA or systemic lupus erythematosus (SLE)-associated wAIHA (without other SLE-related manifestations apart from cutaneous and musculoskeletal manifestations)
    - Participants who have previously failed to maintain a sustained response after treatment with corticosteroids.
    - Eastern Cooperative Oncology Group (ECOG) performance status grade 2 or lower.
    - Up-to-date vaccination status as per local guidelines.
    - Body mass index (BMI) >17.5 and <40 kg/m2
    - All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

    Part B only
    - Evidence of treatment efficacy to rilzabrutinib as defined by achieving overall response during Part A.
    - Completion of Part A treatment period (24 weeks).
    -Pacientes de ambos sexos con un diagnóstico confirmado de AHAIc primaria o AHAIc asociada a lupus eritematoso sistémico (LES) (sin otras manifestaciones relacionadas con el LES, aparte de manifestaciones cutáneas y musculoesqueléticas)
    - Participantes que previamente no hayan mantenido una respuesta prolongada después de un tratamiento con corticoesteroides
    - Estado funcional de grado 2 o inferior según el Grupo Oncológico Cooperativo del Este (Eastern Cooperative Oncology Group, ECOG).
    - Calendario de vacunación al día según las directrices locales.
    - Índice de masa corporal (IMC) >17,5 y <40 kg/m2.
    - Todo uso de anticonceptivos por parte de varones y mujeres debe estar en consonancia con la regulación local relativa a métodos anticonceptivos para los participantes en estudios clínicos
    Parte B solamente:
    - Evidencia de eficacia del tratamiento con rilzabrutinib definida por la consecución de una respuesta global durante la parte A.
    - Finalización del periodo de tratamiento de la parte A (24 semanas).
    E.4Principal exclusion criteria
    - Clinically significant medical history or ongoing chronic illness that would jeopardize the safety of the participant or compromise the quality of the data derived from his or her participation in the study as determined by the Investigator.
    - Participants with medical history of lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for the past 3 years.
    - Secondary wAIHA from any cause including drugs, lymphoproliferative disorders (low-count monoclonal B-cell lymphocytosis is allowed), infectious or autoimmune disease, or active hematologic malignancies. Participants with positive antinuclear antibodies but without a definitive diagnosis of an autoimmune disease are allowed.
    - Myelodysplastic syndrome.
    - Uncontrolled or active HBV infection: Patients with positive HBsAg and/or HBV DNA.
    - HIV infection.
    - Concurrent treatment with other experimental drugs or participation in another clinical trial with any investigational drug within 30 days or 5 half-lives, whichever is greater, prior to treatment start.
    - Known allergy to any of the study medications, their analogues, or excipients in the various formulations of any agent.

    Part B only
    - Participants who receive any therapy during Part A known to be active in wAIHA.
    - Presence of unacceptable side effect(s) or toxicity associated with rilzabrutinib such that there is an unfavorable risk-benefit assessment for continued treatment with rilzabrutinib in the opinion of the Investigator and/or Sponsor.
    - Antecedentes médicos clínicamente significativos o enfermedad crónica en curso que ponga en peligro la seguridad del participante o la calidad de los datos derivados de su participación en el estudio, según el criterio del Investigador.
    -Participantes con antecedentes médicos de linfoma, leucemia o cualquier neoplasia maligna en los 5 años anteriores, excepto epiteliomas basocelulares o escamosos de piel que se hayan resecado sin evidencia de metástasis durante los 3 años anteriores.
    - AHAIc secundaria por cualquier causa, lo que incluye fármacos, trastornos linfoproliferativos (se permite linfocitosis B monoclonal con recuento bajo), enfermedad autoinmunitaria o infecciosa (se permite lupus eritematoso sistémico [LES] sin otras manifestaciones relacionadas con él, aparte de las manifestaciones cutáneas y osteomusculares) o neoplasias malignas hematológicas activas. Se permite la inclusión de participantes con anticuerpos antinucleares positivos, pero sin un diagnóstico definitivo de enfermedad autoinmunitaria.
    - Síndrome mielodisplásico.
    - Infección por el VHB activa o no controlada: pacientes con AgsHB positivo y/o ADN del VHB.
    - Infección por VIH.
    - Tratamiento simultáneo con otros medicamentos experimentales o participación en otro ensayo clínico con algún medicamento en investigación en un plazo de 30 días o 5 semividas, lo que sea mayor, antes del inicio del tratamiento.
    - Alergia conocida a cualquiera de los medicamentos del estudio, sus análogos o los excipientes en las distintas formulaciones de cualquier compuesto.
    Parte B solamente:
    -Participantes que reciban cualquier tratamiento durante la parte A que se sepa que actúan contra la AHAIc.
    - Presencia de uno o varios efectos secundarios o toxicidades inaceptables asociados a rilzabrutinib, de modo que, en opinión del Investigador y/o del Promotor, exista una evaluación de riesgo-beneficio desfavorable para el tratamiento continuado con rilzabrutinib
    E.5 End points
    E.5.1Primary end point(s)
    1 - Proportion of participants with overall hemoglobin response ; Response is defined as an increase in hemoglobin (Hb) by ≥2 g/dL from baseline and an absence of transfusion in the last 7 days, without biochemical resolution of hemolysis at the time response is achieved and an absence of rescue medications during the last 4 weeks.
    Complete Response is defined as hemoglobin ≥11 g/dL (women) or ≥12 g/dL (men), no evidence of hemolysis (normal bilirubin, LDH, haptoglobin, and reticulocytes), and absence of transfusion in the last 7 days and an absence of rescue medications during the last 4 weeks.
    2 - Proportion of participants who maintain durable response achieved during Part A or achieve a durable response during Part B and have a hemoglobin response ; Durable response (Part B) is defined as Hb level ≥10 g/dL with an increase from baseline (Part A) of ≥2 g/dL on three consecutive scheduled visits during Week 24 to Week 50; with absence of transfusion and no rescue medication during the period of 3 consecutive visits and for at least 7 days (transfusions) and 4 weeks (rescue medication) prior to the first consecutive visit.
    1- Proporción de participantes con respuesta hemoglobínica global; Respuesta se define como un aumento de la hemoglobina (Hb) ≥2 g/dl con respecto al inicio y ausencia de transfusiones en los 7 días anteriores, sin resolución bioquímica de la hemólisis en el momento de alcanzar la respuesta y ausencia de medicamentos de rescate durante las 4 semanas anteriores.
    Respuesta Completa se define como un nivel de hemoglobina ≥11 g/dl (mujeres) o ≥12 g/dl (varones), sin evidencia de hemólisis (valores normales de bilirrubina, lactato deshidrogenasa (LDH), haptoglobina y reticulocitos) y ausencia de transfusión en los 7 días anteriores y ausencia de medicamentos de rescate durante las 4 semanas anteriores.
    2- Proporción de participantes que mantengan una respuesta duradera lograda durante la parte A o que muestren una respuesta duradera durante la parte B y tengan una respuesta hemoglobínica; Respuesta duradera (parte B) se define como un nivel de Hb ≥10 g/dl con un aumento respecto al inicio (parte A) ≥2 g/dl en tres visitas programadas consecutivas entre la semana 24 y la semana 50; con ausencia de transfusión y de medicación de rescate durante el periodo de 3 visitas consecutivas y durante al menos 7 días (transfusiones) y 4 semanas (medicación de rescate) antes de la primera visita de la serie consecutiva
    E.5.1.1Timepoint(s) of evaluation of this end point
    1 - By Week 24 in Part A
    2 - At Week 50 in Part B
    1-En la semana 24 de la parte A
    2-En la semana 50 de la parte B
    E.5.2Secondary end point(s)
    1 - Proportion of participants with durable hemoglobin response ; Durable response (Part A) is defined as Hb level ≥10 g/dL with an increase from baseline of ≥2 g/dL on three consecutive evaluable visits during the 24-week treatment period; with absence of transfusion and no rescue medication during the period of 3 consecutive visits and for at least 7 days (transfusions) and 4 weeks (rescue medication) prior to the first consecutive visit.
    2 - Median time from baseline to first hemoglobin response
    3 - Frequency of rescue therapy (any wAIHA-directed therapy other than predniso[lo]ne or transfusion) received
    4 - Change from baseline in FACIT-Fatigue scale score
    5 - Safety assessments
    1- Proporción de participantes con respuesta hemoglobínica duradera; Respuesta duradera (Parte A) se define como un nivel de Hb ≥10 g/dl con un aumento respecto al inicio ≥2 g/dl en tres visitas consecutivas evaluables durante el periodo de tratamiento de 24 semanas; con ausencia de transfusión y de medicación de rescate durante el periodo de 3 visitas consecutivas y durante al menos 7 días (transfusiones) y 4 semanas (medicación de rescate) antes de la primera visita de la serie consecutiva.
    2- Mediana del tiempo transcurrido desde el inicio (día 1) hasta la primera respuesta hemoglobínica
    3- Frecuencia de uso de tratamientos de rescate (cualquier tratamiento para la AHAIc que no sean predniso[lo]na o transfusión).
    4- Cambio con respecto al momento inicial en la puntuación de la escala de Evaluación funcional del tratamiento de enfermedades crónicas relativa a la fatiga (FACIT-Fatiga).
    5- Evaluaciones de la seguridad
    E.5.2.1Timepoint(s) of evaluation of this end point
    1 - By Week 24 in Part A
    2 - From Day 1 to Week 24 in Part A
    3 - After Week 1 of treatment to Week 24 in Part A and Week 75 in Part B
    4, 5 - Until Week 24 in Part A and Week 75 in Part B
    1-En la semana 24 de la parte A
    2-Desde día 1 hasta semana 24 en la parte A
    3-Después de la primera semana de tratamiento hasta semana 24 en la parte A y semana 75 en la parte B.
    4,5- Hasta la semana 24 en la parte A y semana 75 en la parte B
    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.8.2.4Number 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 EEA6
    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
    China
    United States
    Denmark
    Italy
    Spain
    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
    LVLS
    La última visita del último paciente
    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 months8
    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 months8
    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: 15
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    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 state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 10
    F.4.2.2In the whole clinical trial 25
    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 Decision2021-10-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-10-01
    P. End of Trial
    P.End of Trial StatusOngoing
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