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    Summary
    EudraCT Number:2016-002959-17
    Sponsor's Protocol Code Number:CR-BD-001
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2016-09-07
    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 ConcernedUK - MHRA
    A.2EudraCT number2016-002959-17
    A.3Full title of the trial
    An exploratory, open-label study to evaluate the safety and feasibility of ATIR201, a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells (using photodynamic treatment), as adjuvant treatment to a T-cell depleted haploidentical hematopoietic stem cell transplantation in patients with beta-thalassemia major
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety and feasibility of ATIR201, a T-lymphocyte enriched leukocyte preparation depleted of host alloreactive T-cells, in patients with beta-thalassemia major who received a hematopoietic stem cell transplantation from a haploidentical donor
    A.4.1Sponsor's protocol code numberCR-BD-001
    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 SponsorKiadis Pharma Netherlands B.V.
    B.1.3.4CountryNetherlands
    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 supportKiadis Pharma Netherlands B.V.
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationKiadis Pharma Netherlands B.V.
    B.5.2Functional name of contact pointDirector Clinical Operations
    B.5.3 Address:
    B.5.3.1Street AddressEntrada 231-234
    B.5.3.2Town/ cityAmsterdam-Duivendrecht
    B.5.3.3Post code1114 AA
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+31203140250
    B.5.6E-mailregulatory.affairs@kiadis.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/08/561 and EU/3/16/1678
    D.3 Description of the IMP
    D.3.1Product nameATIR201
    D.3.4Pharmaceutical form 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 INN-
    D.3.9.1CAS number -
    D.3.9.2Current sponsor code-
    D.3.9.3Other descriptive nameATIR
    D.3.9.4EV Substance CodeSUB121624
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number1 to 10
    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) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product Yes
    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
    Patients with beta-thalassemia major who are eligible for a haploidentical HSCT
    E.1.1.1Medical condition in easily understood language
    Patients with beta-thalassemia major who are eligible for a stem cell transplantation from a related haploidentical donor
    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 19.0
    E.1.2Level LLT
    E.1.2Classification code 10054661
    E.1.2Term Thalassemia major
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The objective of the study is to evaluate the safety and feasibility of ATIR201 in patients with beta-thalassemia major who received a T-cell depleted haploidentical hematopoietic stem cell transplantation (HSCT).
    Safety and tolerability will be primarily evaluated by the occurrence of acute graft-versus- host disease (GVHD) grade III/IV within 180 days post HSCT.
    Efficacy will be primarily evaluated by transfusion-free survival (TFS), occurrence of severe infections, and time to T-cell reconstitution, taking into account hematologic and sustained engraftment.
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patient Inclusion Criteria:
    • Confirmed diagnosis of beta-thalassemia major:
    - Hypertransfusion program with eight or more red blood cell (RBC) transfusions a year and a confirmed annual transfusion of ≥ 100 ml/kg/year.
    - Genotype consistent with clinical diagnosis of beta-thalassemia major (including hemoglobin E-beta-thalassemia genotype)
    - Iron chelation therapy initiated, or not yet initiated but at risk of transfusional iron overload
    • Lansky / Karnofsky performance score > 70%
    • Eligible for haploidentical stem cell transplantation according to the investigator taking into account the feasibility of transplanting patients with pre-existing antibodies (e.g. anti-A, anti-B, and/or other anti-donor red cell antibodies or known presence of HLA antibodies against the non-shared donor haplotype)
    • Male or female, age ≥ 2 years and ≤ 25 years

    Donor Inclusion Criteria:

    • Haploidentical family donor with at least 4 out of 8 or 5 out of 10 matches at the HLA- A, -B , -C, -DR, and/or DQ loci as determined by high resolution typing
    • Male or female, age ≥ 16 and ≤ 75 years (If applicable, local legal requirements for
    donors under the age of 18 will be followed)
    • Eligible for donations of human blood and blood components according to local requirements and regulations
    • Fit to receive granulocyte colony-stimulating factor (G-CSF) and to give peripheral blood stem cells (blood counts and blood pressure within standards)
    • Eligible for donation according to the transplantation center
    E.4Principal exclusion criteria
    Exclusion Criteria Patient:

    • Availability of a fully matched related or unrelated donor following a donor search, which might have been initiated before screening.
    • Patient assigned to Pesaro risk class 3
    • Symptomatic cardiac failure: left ventricular ejection fraction at rest < 50% and no improvement with exercise, or shortening fraction < 26% (evaluated by echocardiogram or MUGA)
    • Hepatic failure: > 5 times the upper limit of normal range (ULN) for age of AST or > 2.5 times the ULN for age of total serum bilirubin
    • Renal function (creatinine clearance or GFR) < 50% of the lower limit of normal (LLN) for age
    • Symptomatic pulmonary failure: DLCO, FEV1, or FEC (diffusion capacity) < 50% of predicted (corrected for hemoglobin); if unable to perform a pulmonary function test, oxygen saturation < 85% on room air
    • Positive pregnancy test (females of childbearing age only)
    • Positive test for HIV-1 or HIV-2
    • Active viral, bacterial, fungal, or parasitic infection, including hepatitis B, hepatitis C
    • Prior HSCT for beta-thalassemia major
    • Known allergy to any of the components of ATIR201 (e.g., dimethyl sulfoxide)
    • Any other condition which, in the opinion of the investigator, makes the patient ineligible for the study

    Donor Exclusion Criteria:

    • Positive pregnancy test or nursing (females of childbearing age only)
    • Positive viral test for HIV-1, HIV-2, HBV, HCV, or Treponema pallidum
    • Hemoglobin S ≥ 50%, or presence of beta-thalassemia intermedia
    • History of congestive heart failure, unstable angina, or history of stroke
    E.5 End points
    E.5.1Primary end point(s)
    Incidence of acute graft versus host disease (GvHD) grade III/IV within 180 days post HSCT.
    E.5.1.1Timepoint(s) of evaluation of this end point
    180 days post HSCT
    E.5.2Secondary end point(s)
    Secondary safety endpoints:
    • Occurrence of acute and chronic GVHD (all grades/severities)
    • Occurrence of graft rejection
    • Occurrence of prolonged donor red cell aplasia
    • Donor chimerism

    Secondary efficacy endpoints:
    • Transfusion-free survival (TFS)
    • Occurrence of severe (NCI grade 3-5) infections
    • Time to T-cell reconstitution, defined as the time to CD3+ > 0.2×10E9/l in peripheral blood (at two consecutive measurements; time to first measurement)
    • Overall survival (OS), defined as the time from HSCT until death from any cause
    • Transplant-related mortality (TRM), defined as death due to causes other than disease recurrence
    E.5.2.1Timepoint(s) of evaluation of this end point
    Up to 12 months post HSCT
    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 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 No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans Yes
    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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA3
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    The end of the study is defined as the date at which the last data point from the last patient is received.
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    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: 7
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 3
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 4
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 3
    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 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 10
    F.4.2.2In the whole clinical trial 10
    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)
    Not applicable
    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 Decision2016-12-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-11-30
    P. End of Trial
    P.End of Trial StatusOngoing
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