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    Summary
    EudraCT Number:2019-002221-29
    Sponsor's Protocol Code Number:VIT-2763-THAL-201
    National Competent Authority:Greece - EOF
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-02-25
    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 ConcernedGreece - EOF
    A.2EudraCT number2019-002221-29
    A.3Full title of the trial
    A Phase 2a, Double-blind, Randomised, Placebo-controlled, Parallel Group, Multicentre Study on Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of Multiple Doses of VIT-2763 in Subjects with Non-transfusion Dependent Beta-thalassaemia.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to the assess safety, tolerability and the effect of VIT-2763 on blood markers, symptoms and quality of life in patients with non-transfusion dependent beta-thalassemia
    A.4.1Sponsor's protocol code numberVIT-2763-THAL-201
    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 SponsorVifor (International) Inc.
    B.1.3.4CountrySwitzerland
    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 supportVifor (International) Inc.
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationVifor Pharma Management Ltd.
    B.5.2Functional name of contact pointCommunations
    B.5.3 Address:
    B.5.3.1Street AddressFlughofstrasse 61
    B.5.3.2Town/ cityGlattbrugg
    B.5.3.3Post codeCH-8152
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number+41588 518 000
    B.5.5Fax number+41588 518 001
    B.5.6E-mailinfo@viforpharma.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/3/19/2170
    D.3 Description of the IMP
    D.3.2Product code VIT-2763
    D.3.4Pharmaceutical form Capsule, hard
    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 INNunavailable yet
    D.3.9.1CAS number unavailable
    D.3.9.2Current sponsor codeVIT-2763
    D.3.9.3Other descriptive nameVIT-2763-PM1
    D.3.9.4EV Substance CodeSUB190537
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number60
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule, hard
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Non-transfusion Dependent Beta-thalassaemia.
    E.1.1.1Medical condition in easily understood language
    Iron overload in the blood.
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10074355
    E.1.2Term Non-transfusion dependent thalassaemia
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the safety and tolerability of VIT-2763 versus placebo in adult and adolescent NTDT subjects over a 12-week treatment period.
    E.2.2Secondary objectives of the trial
    - To assess the preliminary efficacy of VIT-2763 versus placebo on iron markers in adult and adolescent NTDT subjects over a 12-week treatment period.
    - To evaluate the PK of VIT-2763 in adult and adolescent NTDT subjects over a 12-week treatment period (using a population PK approach).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Documented diagnosis of NTDT, including a β-thalassaemia intermedia-phenotype.
    2. NTDT is defined as subjects having received <5 units of red blood cells (RBCs) during the 24-week period prior to randomisation/first drug administration of VIT-2763 or placebo (Day 1; 1 unit is defined as 200 to 350 ml of transfused packedRBCs and last RBC transfusion must have been received ≥14 days prior to randomisation).
    Note: Subjects who are supposed to receive RBC transfusions after randomization in the Investigator’s opinion, and according to local practise, and having received at least 1 dose of VIT-2763, may be considered to stay on study treatment for safety reasons, and in case there are no tolerability concerns. Subjects will be censored for secondary efficacy.
    3. Male and female adult NTDT subjects, 18-65 years of age inclusive (Cohort I only) at time of screening.
    4. Male and female adolescent NTDT subjects, 12-17 years of age inclusive (Cohort II only) at time of screening.
    5. Subjects must have a mean baseline Hb ≤11 g/dl, based on 2 consecutive measurements ≥1 week apart within 6 weeks prior to randomisation/baseline, and obtained Hb values show less than 10% relative difference (and equal or less than 1.0 g/dl absolute change between the highest and lowest value) between at least 2 measurements.
    Note: If there is 1 retrospective Hb value available for the subject at maximum of 2 weeks prior to screening (Day -28), the Hb value can be taken into consideration. A subject not meeting this criterion would be excluded but can be rescreened at maximum 2 times at a later time point.
    E.4Principal exclusion criteria
    1. Documented diagnosis of TDT, including a beta-thalassaemia major phenotype (including β0/β0, β+/β+, β0/β+ genotype), and mixed compound heterozygous for sickling phenotype variants such as Hb S/β-thalassaemia, or transfusion dependent non-deletional Hb H disease (i.e., Hb constant spring) or Hb C disease.
    2. Subjects on concomitant ICT or subjects on prior ICT when discontinued less than 4 weeks prior randomisation. If ICT was discontinued ≥4 weeks prior randomization the subject is eligible.
    3. ICT naïve subjects with serum ferritin <150 ng/ml and/or documented LIC ≤1 mg/g liver dry weight assessed through MRI, or subjects on prior ICT with serum ferritin <300 ng/ml and/or documented LIC <3 mg/g liver dry weight assessed through MRI.
    Note: If documented LIC MRI scans retrieved within 24 months prior to randomization are not available per local practice, serum ferritin will be used only to document iron overload status.
    4. Subjects with TSAT <30%.
    5. Subjects with documented LIC >15 mg/g liver dry weight assessed through MRI, or a documented myocardial T2* <20 ms, if available per local practice and retrieved within 24 months prior to randomization.
    6. Adult or adolescent subjects with body weight <40.0 kg or >100 kg at screening.
    7. Chronic liver disease and/or ALT, AST or GGT above 3-fold the ULN range at screening.
    Note: A subject fulfilling this criterion will be excluded but can be rescreened at a later time point (in order to fulfil eligibility, ≥2 values within ≥1 week should be assessed and be within eligibility limits).
    8. eGFR <30 ml/min/1.73 m2 (according to chronic kidney disease classification Stage 4 or higher), and/or significant albuminuria >30 mg/mmol. eGFR should be estimated according to Chronic Kidney Disease Epidemiology Collaboration formula (CKI-EPI) in adults, and Schwartz formula in adolescents.
    9. Newly diagnosed folate deficiency anaemia and/or Vitamin B12 megaloblastic anaemia. Subjects with known folate deficiency anaemia and/or Vitamin B12 megaloblastic anaemia who are on ≥12 weeks stable replacement therapy are eligible.
    Note: A subject fulfilling this criterion will be excluded but can be rescreened at a later time point.
    10. Any history or clinically important finding of cardiac disorders, such as clinically relevant cardiac arrhythmia, cardiomyopathy, coronary disease, valve disorder, or heart failure according to New York Heart Association classification 3-4.
    11. Subjects with history of partial or total splenectomy within 6 months prior to screening.
    E.5 End points
    E.5.1Primary end point(s)
    • Reported or observed AEs: by SOC and PT (MedDRA coded term), by severity and relation to study product in each treatment group.
    • Reported or observed SAEs: by SOC and PT (MedDRA coded term), by severity and relation to study product in each treatment group.
    • Changes in vital signs (blood pressure, pulse rate, and respiratory rate), clinical laboratory safety tests (haematology, serum biochemistry, coagulation, and urinalysis), 12-lead ECG, and physical examination findings.
    E.5.1.1Timepoint(s) of evaluation of this end point
    For AEs: The AE reporting period ends at the last study contact Visit 9/Week 16.
    For SAEs: The SAE reporting period lasts until 4 weeks (28+-4 days) following the last study drug administration.
    For Changes in vital signs: during the study and until Visit 8/Week 12
    E.5.2Secondary end point(s)
    • Assessment of iron parameters (total serum iron, serum ferritin, serum transferrin, unsaturated iron binding capacity, calculated TSAT, from baseline over a 12-week period (absolute and change from baseline)).
    • PK parameters: Individual estimates of Cmax, clearance, distribution volume, AUC will be obtained using a population PK approach in adult and adolescent subjects combined with suitable mathematical/statistical analysis, using nonlinear mixed-effects modelling. Sparse sampling for determination of VIT-2763 plasma concentration following multiple dosing will be obtained from pre-dose trough to 3 or 4 hours post-dose at selected study visits.
    E.5.2.1Timepoint(s) of evaluation of this end point
    • Iron parameters: prior to first administration and over a 12-week period
    • PK parameters: Blood samples for the determination of VIT-2763 plasma concentrations (sparse sampling) are collected on Visit 3 and Visit 7 at pre-dose trough and at approximately 1 hour and 4 hours post-dose, and on Visits 6 and 8 at pre-dose trough and approximately at 1 hour and 3 hours post-dose.
    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 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.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 No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial3
    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 EEA10
    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
    Greece
    Israel
    Italy
    Lebanon
    Thailand
    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 years1
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months1
    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: 12
    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.1.1.6.1Number of subjects for this age range: 12
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 23
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 18
    F.4.2.2In the whole clinical trial 36
    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 Decision2020-02-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-02-14
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2021-11-03
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