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    Summary
    EudraCT Number:2017-003309-16
    Sponsor's Protocol Code Number:JZP963-201
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-11-28
    Trial results View 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 ConcernedGermany - BfArM
    A.2EudraCT number2017-003309-16
    A.3Full title of the trial
    A Phase 2, Prospective, Randomized, Open-label Study on the Efficacy of Defibrotide Added to Standard of Care Immunoprophylaxis for the Prevention of Acute Graft-versus-Host-Disease in Adult and Pediatric Patients After Allogeneic Hematopoietic Stem Cell Transplant
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study to investigate the efficacy of Defibrotide Added to Standard of Care Immunoprophylaxis for the prevention of Acute Graft-versus-Host-Disease in adult and pediatric patients after allogeneic hematopoietic stem cell transplant
    A.4.1Sponsor's protocol code numberJZP963-201
    A.5.4Other Identifiers
    Name:INDNumber:62,118
    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 SponsorJazz 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 supportJazz Pharmaceuticals Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationJazz Pharmaceuticals Inc.
    B.5.2Functional name of contact pointClinical Dev and Lifecycle Mgmt
    B.5.3 Address:
    B.5.3.1Street Address3170 PorterDrive
    B.5.3.2Town/ cityPalo Alto
    B.5.3.3Post codeCA 94304
    B.5.3.4CountryUnited States
    B.5.4Telephone number1650496-3777
    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.2.1.1.1Trade name Defitelio
    D.2.1.1.2Name of the Marketing Authorisation holderGentium Srl, Italy
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/13/1201
    D.3 Description of the IMP
    D.3.1Product nameDefibrotide
    D.3.4Pharmaceutical form Concentrate 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 INNDEFIBROTIDE
    D.3.9.1CAS number 83712-60-1
    D.3.9.3Other descriptive name"Defibrotide sodium"
    D.3.9.4EV Substance CodeSUB01572MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number80
    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 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
    Prevention of Acute Graft-versus-Host Disease (aGvHD) after allogeneic hematopoietic stem cell transplant
    E.1.1.1Medical condition in easily understood language
    Prevention of Acute Graft-versus-Host Disease (aGvHD)
    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 20.0
    E.1.2Level LLT
    E.1.2Classification code 10068908
    E.1.2Term AGVHD
    E.1.2System Organ Class 100000004870
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the study is to compare the efficacy of defibrotide added to standard of care immunoprophylaxis vs standard of care immunoprophylaxis alone for the prevention of acute graft-versus-host disease (aGvHD) as measured by the cumulative incidence of Grade B-D by Day +100 post-allogeneic hematopoietic stem cell transplant (HSCT) in adult and pediatric patients.
    E.2.2Secondary objectives of the trial
    •To compare the efficacy of defibrotide added to standard of care immunoprophylaxis vs standard of care immunoprophylaxis alone on additional variables:
    -Grade B-D aGvHD-free survival by Days +100 and +180 post-HSCT
    -Cumulative incidence of Grade B-D aGvHD by Day +180 post-HSCT
    -Cumulative incidence of Grade C-D aGvHD by Days +100 and +180 post-HSCT
    -Cumulative incidence of relapse by Days +100 and +180 post-HSCT
    •To evaluate steroid use in the treatment of aGvHD by Day +180 post-HSCT
    •To compare the health-related quality of life (HRQoL) using the following questionnaires:
    -FACT-BMT-TOI (adults only)
    -EuroQoL-5D (EQ-5D; version dependent on age group)
    •To compare the safety of defibrotide added to standard of care immunoprophylaxis vs standard of care immunoprophylaxis alone, including AE profile, SAE profile, laboratory abnormalities, neutrophil and platelet engraftment, graft failure, and infection.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patient must be ≥1 year of age at screening and undergoing allogeneic HSCT.
    2. Patient must be diagnosed with acute leukemia in morphologic complete remission (CR1 or CR2) or with MDS with no circulating blasts and with less than 5% blasts in the bone marrow
    3. Patient must have planned to receive either a myeloablative or reducedintensity conditioning regimen and have an unrelated donor who is HLA matched or single-allele mismatched
    4. Graft must be a CD3+ T-cell replete PBSC graft or non-manipulated BM graft.
    5. Adult patients must be able to understand and sign a written informed consent. For pediatric patients, the parent/legal guardian or representative must be able to understand and sign a written informed
    consent. Assent, when appropriate, will be obtained according to institutional guidelines.
    E.4Principal exclusion criteria
    1. Patient has had a prior autologous or allogeneic HSCT.
    2. Patient is using or plans to use an investigational agent for the prevention of GvHD.
    3. Patient is receiving or plans to receive other investigational therapy
    4. Patient, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study.
    5. Patient has a psychiatric illness that would prevent the patient or legal guardian or representative from giving informed consent and/or assent.
    6. Patient has a serious active disease or co-morbid medical condition, as judged by the investigator, which would interfere with the conduct of this study.
    7. Patient is pregnant or lactating and does not agree to stop breastfeeding.
    8. Any other condition that would cause a risk to the patient if he/she participated in the trial.
    9. Patient has a known history of hypersensitivity to defibrotide or any of its excipients. Patient has a known hypersensitivity to any agent(s) or excipients of agent(s) within patient's planned immunoprophylaxis regimen.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint of this study is cumulative incidence of Grade B-D aGvHD by Day +100 post-HSCT.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day +100 post-HSCT
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints include the following:
    • Grade B-D aGvHD-free survival by Days +100 and +180 post-HSCT
    • Cumulative incidence of Grade B-D aGvHD by Day +180 post-HSCT
    • Cumulative incidence of Grade C-D aGvHD by Days +100 and +180 post-HSCT
    • Cumulative incidence of relapse by Days +100 and +180 post-HSCT
    E.5.2.1Timepoint(s) of evaluation of this end point
    Day +100 and +180 post-HSCT for endpoints except for Cumulative incidence of Grade B-D aGvHD: Day +180 post-HSCT only
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic Yes
    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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    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) Yes
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Standard of Care
    E.8.2.4Number of treatment arms in the trial2
    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 concerned11
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA42
    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
    Austria
    Belgium
    Bulgaria
    Canada
    Croatia
    France
    Germany
    Greece
    Italy
    Poland
    Portugal
    Spain
    Turkey
    United Kingdom
    United States
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months21
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months24
    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 Yes
    F.1.1Number of subjects for this age range: 30
    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.1.1.4.1Number of subjects for this age range: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 10
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 15
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 114
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 8
    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
    For pediatric patients, the parent/legal guardian or representative must be able to understand and sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state33
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 83
    F.4.2.2In the whole clinical trial 152
    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)
    At the end of the study, patients will continue with their normal treatment, medication, and care as their doctor advises.
    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 Decision2018-04-05
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-04-25
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2020-05-12
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