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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);
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    The EU Clinical Trials Register currently displays   42782   clinical trials with a EudraCT protocol, of which   7047   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).

    Phase 1 trials conducted solely in adults and that are not part of an agreed PIP are not public in the EU CTR (refer to European Guidance 2008/C 168/02   Art. 3 par. 2 and   Commission Guideline 2012/C 302/03,   Art. 5) .

    Clinical Trials marked as "Trial now transitioned" were transitioned to the Clinical Trial Regulation 536/2014 and can be further followed in the Clinical Trial Information System  
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
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    EudraCT Number:2018-000329-29
    Sponsor's Protocol Code Number:CSL964_2001
    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:2018-10-09
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2018-000329-29
    A.3Full title of the trial
    A Phase 2/3, Multicenter, randOmized, Double-blind, placebo-controlled, stUdy to evaLuate the safety and efficacy of Alpha-1 AntiTrypsin for the prEvention of graft-versus-host disease in patients receiving hematopoietic cell transplant (MODULAATE Study)
    Estudio de fase 2/3, multicéntrico, aleatorizado, con doble enmascaramiento, controlado con placebo para evaluar la seguridad y la eficacia de la alfa 1-antitripsina en la prevención de la enfermedad de injerto-contra-huésped en pacientes que reciben un trasplante de células hematopoyéticas (Estudio MODULAATE).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    The safety and efficacy of Alpha-1 Antitrypsin (AAT) for the prevention of graft-versus-host disease (GVHD) in patients receiving hematopoietic cell transplant
    La seguridad y la eficacia de la alfa 1-antitripsina en la prevención de la enfermedad de injerto-contra-huésped en pacientes que reciben un trasplante de células hematopoyéticas
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberCSL964_2001
    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 SponsorCSL Behring LLC
    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 supportCommercial/Industry
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCSL Behring LLC
    B.5.2Functional name of contact pointTrial Registration Coordinator
    B.5.3 Address:
    B.5.3.1Street Address1020 First Avenue
    B.5.3.2Town/ cityKing of Prussia
    B.5.3.3Post code19406
    B.5.3.4CountryUnited States
    B.5.4Telephone number6108784000
    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 Yes
    D. name Respreeza
    D. of the Marketing Authorisation holderCSL Behring GmbH
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameAlpha-1 antitrypsin
    D.3.2Product code Alpha-1 proteinase inhibitor
    D.3.4Pharmaceutical form Powder and solvent for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAlpha-1 antitrypsin
    D.3.9.1CAS number 9041-92-3
    D.3.9.2Current sponsor codeCSL964
    D.3.9.3Other descriptive nameAlpha-1 antitrypsin
    D.3.9.4EV Substance CodeSUB12796MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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 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. 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 Yes
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for 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
    Graft versus host disease
    la enfermedad de injerto-contra-huésped
    E.1.1.1Medical condition in easily understood language
    Graft-versus-host disease is a complication that frequently occurs following hematopoietic cell transplantation when the transplanted cells attack the patients’ organs leading to organ damage.
    la enfermedad de injerto-contra-huésped es una complicación que ocurre frecuentemente despues de un transplante de celulas hamatopoyéticas estas dañan los organos del paciente
    E.1.1.2Therapeutic area Body processes [G] - Immune system processes [G12]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10018651
    E.1.2Term Graft versus host disease
    E.1.2System Organ Class 10021428 - Immune system 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 AAT at the selected dose for the prevention of acute GVHD following HCT.
    Evaluar la eficacia de la AAT a la dosis seleccionada para la prevención de la EICH aguda tras un THB.
    E.2.2Secondary objectives of the trial
    1. To evaluate the efficacy of AAT, including the prevention of post-hematopoietic cell transplant complications.
    2. To evaluate the safety of AAT, based on incidence of systemic infections and related adverse events.
    3. To evaluate the pharmacokinetics of AAT in HCT recipients.
    1.Evaluar la eficacia de la AAT, incluida la prevención de las complicaciones posteriores al THB.
    2.Evaluar la seguridad de la AAT tomando como base la incidencia de infecciones sistémicas y acontecimientos adversos (AA) relacionados.
    3.Evaluar la FC de la AAT en los receptores de THB.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female subjects, ≥12 years of age, undergoing HCT for hematological malignancies, including leukemia, lymphoma and multiple myeloma
    2. Planned myeloablative conditioning regimen
    1.Sujetos de sexo masculino o femenino, ≥12 años y sometidos a un THB para el tratamiento de neoplasias hematológicas como la leucemia, el linfoma y el mieloma múltiple.
    2.Pauta de acondicionamiento mieloablativo prevista
    E.4Principal exclusion criteria
    1. Prior autologous or allogeneic HCT
    2. T-cell depleted transplant or planned use of anti-T cell antibody therapy either ex vivo or in vivo
    3. Planned umbilical cord blood (UCB) transplant
    1.THB autólogo o alógeno anterior.
    2.Trasplante con reducción de linfocitos T o uso previsto de un tratamiento con anticuerpos antilinfocitos T ex vivo o in vivo (p. ej., globulina antitimocítica [GAT], alemtuzumab) durante el estudio.
    3.Trasplante de sangre del cordón umbilical previsto.
    E.5 End points
    E.5.1Primary end point(s)
    Percent of subjects with acute graft-versus-host-disease-free survival
    la incidencia de la supervivencia sin enfermedad injerto contra huésped aguda
    E.5.1.1Timepoint(s) of evaluation of this end point
    180 days post-hematopoietic cell transplantation
    a los 180 días del THB.
    E.5.2Secondary end point(s)
    -Percent of subjects with Grade II-IV acute GVHD
    -Percent of subjects with Grade III-IV acute GVHD
    -Number of subjects with all-cause mortality
    -Percent of subjects with Grade II aGVHD
    -Percent of subjects with Grade III aGVHD
    -Percent of subjects with Grade IV aGVHD
    -Time to all-cause mortality
    -Time to non-relapse mortality
    -Percent of subjects with moderate-to-severe chronic GVHD
    -Percent of subjects with discontinuation of immune suppression
    -Time to neutrophil engraftmen
    -Time to GVHD relapse-free survival
    -Percent of subjects with relapse of primary malignancies
    -Percent of subjects with systemic infections
    -Percent of subjects with study drug related adverse events
    -Maximum concentration (Cmax) of AAT
    -Area under the concentration curve (AUC) for AAT
    -Clearance (CL) of AAT
    -Volume of distribution (V) for AAT
    •180 y en los 365 días posteriores al Incidencia de EICHa de grado II-IV en los 100 y en los 180 días posteriores al THB
    •Incidencia de EICHa de grado III-IV en los 100 y en los 180 días posteriores al THB
    •Mortalidad por cualquier causa a los 180 y a los 365 días del THB
    •Incidencia de EICH de grados II, III o IV en los 100 y en los 180 días posteriores al THB
    •Tiempo transcurrido hasta la muerte por cualquier causa
    •Tiempo transcurrido hasta la muerte sin recaída
    •Incidencia de EICH crónica de moderada a grave en los 180 y en los 365 días posteriores al THB
    •Incidencia de la interrupción de la inmunodepresión en los 180 y en los 365 días posteriores al THB
    •Tiempo hasta la aceptación del injerto de neutrófilos
    •Supervivencia sin recaída de la EICH tras más de 365 días desde el THB
    •Incidencia de recaídas de las neoplasias malignas principales en los THB
    •Incidencia de infecciones sistémicas a los 60 y a los 180 días del THB
    •Incidencia de AA relacionados con el fármaco del estudio
    •Parámetros de FC, incluidos la concentración máxima (Cmáx), el área bajo la curva (ABC)0-t, las concentraciones mínimas (Cmín), el aclaramiento, el volumen de distribución (V) y otros, según resulte necesario
    E.5.2.1Timepoint(s) of evaluation of this end point
    -Within 100 and 180 days post-HCT
    -Within 100 and 180 days post-hematopoietic cell transplant (HCT)
    -Within 180 and 365 days post-HCT
    -Within 100 and 180 days post-HCT
    -Within 100 and 180 days post-HCT
    -Within 100 and 180 days post-HCT
    -Up to 365 days post-HCT
    -Up to 365 days post-HCT
    -Within 180 and 365 days post-HCT
    -Within 180 and 365 days post-HCT
    -Up to 365 days post-HCT
    -Up to 365 days post-HCT
    -Within 180 and 365 days post-HCT
    -At Days 60 and 180 post-HCT
    -Up to 365 days post-HCT
    -Before and up to 72 after infusion of AAT
    -Before and up to 72 after infusion of AAT
    -Before and up to 72 after infusion of AAT
    -Entre 100 y 180 dias post-THB
    -Entre 100 y 180 días post-THB
    -Entre 180 y 365 días post-THB
    -Entre 100 y 180 días post-THB
    -Entre100 y 180 días post- THB
    -Entre 100 y 180 días post-THB
    -Hasta 365 días post-THB
    -Hasta 365 días post-THB
    -Entre 180 y 365 días post-THB
    -Entre 180 y 365 días post-THB
    -Hasta 365 días post-THB
    -Up to 365 days post-HCT
    -entre 180 y 365 días post-THB
    -A los 60 and 180 días post-THB
    -Hasta 365 días post-THB
    -Antes y hasta 72 días despues de la infusion AAT
    -Antes y hasta 72 días despues de la infusion AAT
    -Antes y hasta 72 días despues de la infusion AAT
    -Antes y hasta 72 días despues de la infusion AAT
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis Yes
    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 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. trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 Yes
    E. trial design description
    Sequential: Subjects receive interventions after reaching prior milestones, eg dose escalation stud.
    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 trial5
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA27
    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
    United Kingdom
    United States
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    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 years4
    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: 10
    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. of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 290
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 179
    F.4.2.2In the whole clinical trial 310
    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)
    Standard of care
    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 Decision2019-01-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-11-22
    P. End of Trial
    P.End of Trial StatusOngoing
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