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    Summary
    EudraCT Number:2014-001936-12
    Sponsor's Protocol Code Number:MD2014.01
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-11-28
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2014-001936-12
    A.3Full title of the trial
    Efficacy and Safety of human apotransferrin in patients with β-thalassemia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    The effect and safety of human apotransferrin in patients with β-thalassemia
    A.3.2Name or abbreviated title of the trial where available
    Apotransferrin in patients with β-thalassemia
    A.4.1Sponsor's protocol code numberMD2014.01
    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 SponsorSanquin Plasma Products BV
    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 supportSanquin Plasma Products BV
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSanquin Plasma Products BV
    B.5.2Functional name of contact pointIlona Kleine Budde
    B.5.3 Address:
    B.5.3.1Street AddressPlesmanlaan 125
    B.5.3.2Town/ cityAmsterdam
    B.5.3.3Post code1066CX
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+31205123537
    B.5.6E-maili.kleinebudde@sanquin.nl
    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/3/18/2093
    D.3 Description of the IMP
    D.3.1Product namehuman apotransferrin
    D.3.2Product code apotransferrin
    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 INNHuman Apotransferrin
    D.3.9.3Other descriptive nameAPOTRANSFERRIN
    D.3.9.4EV Substance CodeSUB96094
    D.3.10 Strength
    D.3.10.1Concentration unit g/l gram(s)/litre
    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.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 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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Beta thalassemia intermedia
    E.1.1.1Medical condition in easily understood language
    Beta thalassemia
    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 20.1
    E.1.2Level LLT
    E.1.2Classification code 10054660
    E.1.2Term Thalassemia beta
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10062923
    E.1.2Term Thalassemia intermedia
    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
    To study the effect of apotransferrin administration in patients with β thalassemia intermedia on erythropoiesis as reflected by hemoglobin level or transfusion dependency.
    E.2.2Secondary objectives of the trial
    Secondary objectives are the effect of apotransferrin on transferrin plasma levels, iron metabolism, oxidative stress, and erythropoiesis. Moreover the safety of apotransferrin transfusion will be studied.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Non-transfusion dependent β-thalassemia intermedia, defined as patients with microcytic anemia in combination with an elevated HbA2 (>2.5%) and a hemoglobin of <6.2 mmol/L, or transfusion dependent β-thalassemia treated with a regular transfusion schedule.
    2. Age >=18 years
    3. Adequate renal and hepatic function tests as indicated by the following laboratory values:
    •Serum creatinine ≤1.0 mg/dl (≤ 88.7 μmol/L); if serum creatinine >1.0 mg/dl (>88.7 μmol/L), then the glomerular filtration rate (GFR) must be >60 ml/min/1.73 m2 as calculated by the Modification of Diet in Renal Disease equation where the predicted GFR (ml/min/1.73 m2) = 186 x (Serum Creatinine in mg/dl) -1.154 x (age in years) -0.203 x (0.742 if patient is female) x (1.212 if patient is black)
    NOTE: if serum creatinine is measured in μmol/L, recalculate it in mg/dl according to the equation: 1 mg/dl = 88.7 μmol/L) and used above mentioned formula.
    • Aspartate transaminase (AST)/alanine transaminase (ALT) ≤2.5 × ULN
    • Alkaline phosphatase (AP) ≤ 2.5 × ULN
    4. WHO performance 0, 1 or 2.
    5. Signed informed consent.
    E.4Principal exclusion criteria
    1.Known with allergic reactions against human plasma or plasma products.
    2.Concurrent severe and/or uncontrolled medical condition (e.g. uncontrolled diabetes, infection, hypertension, pulmonary disease).
    3.Cardiac dysfunction as defined by: myocardial infarction within the last 6 months of study entry, unstable angina, or unstable cardiac arrhythmias.
    4.Pregnant or lactating females.
    5.Known with IgA deficiency with anti-IgA antibodies
    E.5 End points
    E.5.1Primary end point(s)
    Hematological response defined as the change from baseline of Hb level, expressed as mmol/L and %, in NTDβTI and TDβTI patients or change from baseline of number of RBC in TDβTI patients
    E.5.1.1Timepoint(s) of evaluation of this end point
    Ad 1: Day 0, week 2, 4, 6, 8, 10, 12 , 14, 16 in NTDβTI and TDβTI patients, and in TDβTI patients week 18 and 20 as well
    Ad 2: study period of 20 weeks
    E.5.2Secondary end point(s)
    1. Increase of > 0.9 mmol/l (1.5 g/dl) in NTDβTI and TDβTI (before transfusion)
    2. The reduction of at least 50% of the number of RBC units transfused/week from baseline in TDβTI
    3. Pharmacokinetics of transferrin
    4. Increase iron metabolism as reflected by serum iron, hepcidin, NTBI levels and iron saturation.
    5. Oxidative stress as assessed by plasma levels of advanced glycation end products (AGEs)
    6. The effect on markers of erythropoiesis like reticulocyte count, erythropoietin levels and red cell indices
    7. All adverse events (number, type) will be analysed regarding to causality, seriousness, outcome and expectedness.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Ad 1: Day 0, week 2, 4, 6, 8, 10, 12 , 14, 16 and for TDβTI week 18, 20 as well
    Ad 2: study period of 20 weeks
    Ad 3: before every infusion, and 5 minutes, 2 hours and 1, 4, 7, and 14 days after the stop of infusion no.4, week 16 (NTDβTI) and week 20 (TDβTI) and subsequently till transferrin levels are back to normal
    Add 4-6: Day 0, week 2, 4, 6, 8, 10, 12 , 14, 16 and for TDβTI week 18, 20 as well
    Ad 7: study period of 16 weeks (NTDβTI) or 20 weeks (TDβTI) or till transferrin levels are back to normal.
    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 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 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.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    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 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 months9
    E.8.9.1In the Member State concerned days0
    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: 12
    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 For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2018-11-28. Yes
    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 state12
    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, common treatment
    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 Decision2018-11-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-02-11
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2022-03-31
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