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    The EU Clinical Trials Register currently displays   43851   clinical trials with a EudraCT protocol, of which   7283   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).

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    Summary
    EudraCT Number:2015-005241-31
    Sponsor's Protocol Code Number:IEDAT-02-2015
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2016-07-01
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2015-005241-31
    A.3Full title of the trial
    A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients with Ataxia Telangiectasia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    n/a
    A.3.2Name or abbreviated title of the trial where available
    EDS in Ataxia Telangiectasia Patients - IEDAT-02
    A.4.1Sponsor's protocol code numberIEDAT-02-2015
    A.5.4Other Identifiers
    Name:US IND Number:115929
    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 SponsorEryDel S.p.A.
    B.1.3.4CountryItaly
    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 supportEryDel S.p.A.
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIrene Maccabruni at EryDel S.p.A.
    B.5.2Functional name of contact pointClinical Study Manager
    B.5.3 Address:
    B.5.3.1Street AddressVia A. Meucci, 3
    B.5.3.2Town/ cityBresso (MI)
    B.5.3.3Post code20091
    B.5.3.4CountryItaly
    B.5.4Telephone number+390236504470
    B.5.5Fax number+390236504474
    B.5.6E-mailirene.maccabruni@erydel.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 numberEMA/COMP/306983/2013
    D.3 Description of the IMP
    D.3.1Product nameDexamethasone sodium phosphate
    D.3.4Pharmaceutical form Solution for blood fraction modification
    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 INNDexamethasone sodium phosphate
    D.3.9.2Current sponsor codeDSP
    D.3.9.3Other descriptive nameDEXAMETHASONE SODIUM PHOSPHATE PH. EUR.
    D.3.9.4EV Substance CodeSUB174329
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    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 Yes
    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 Yes
    D.3.11.13.1Other medicinal product typeThe IMP refers to dexamethasone sodium phosphate (DSP) for encapsulation into human autologous erythrocytes. DSP is formulated at 25 mg/mL in WFI to produce the drug product, DSP Solution.
    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 injection
    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
    Patient with neurological symptoms of Ataxia Telangiectasia
    E.1.1.1Medical condition in easily understood language
    Patients with neurological symptoms of Ataxia Telangiectasia
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level PT
    E.1.2Classification code 10003594
    E.1.2Term Ataxia telangiectasia
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Initial Treatment Period (6 months)
    To evaluate the effect of two dose ranges (~5-10 and ~14-22 mg DSP/infusion) of EryDex System end product [EDS-EP; the EDS is a combination product that is used to load dexamethasone sodium phosphate (DSP) into autologous erythrocytes, creating the EDS end product, which is infused into the patient], compared to placebo, on central nervous system (CNS) symptoms measured by the ‘Modified’ International Cooperative Ataxia Rating Scale (mICARS) in patients with AT.

    Extension Treatment Period (6 months)
    To evaluate the efficacy of two dose ranges (~5-10 and ~14-22 mg DSP/infusion) of EDS-EP compared to placebo in treating CNS symptoms in AT patients during long-term treatment (up to 12 months), as measured by the ‘Modified’ ICARS

    E.2.2Secondary objectives of the trial
    Initial Treatment Period (6 months)
    To evaluate the effect of EDS-EP, compared to placebo, in this population on the Clinical Global Impression of Change from baseline (CGI-C) and on the following efficacy measures:
    - Clinical Global Impression of Severity (CGI-S) of neurological symptoms of AT;
    - Adaptive behavior measured by the Vineland Adaptive Behavior Scales (VABS);

    Extension Treatment Period (6 months)
    To evaluate the long-term (up to 12 months) safety and tolerability of EDS-EP in AT patients;
    To compare the effects of the two dose ranges of EDS-EP on the clinician’s global impression (CGI-C and CGI-S), adaptive behavior (VABS), and QoL (EQ-5D-5L scale).

    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patient meets clinical criteria for diagnosis of AT. The neurological signs of AT (incoordination of the head and eyes in lateral gaze deflection, gait ataxia associated with an inappropriately narrow base) must be documented. Such signs of AT illustrate the body systems in
    which changes shall be confirmed but the listed changes are examples and other changes in those systems may be observed and documented to
    confirm the diagnosis of AT.
    2. Patient is in autonomous gait or is helped by periodic use of a support. (i.e. local ICARS score for Item 1 – Walking Capacities between 0 and 4,
    included).
    3. Patient will be investigated for the proven genetic diagnosis of AT (prior documentation or by central laboratory test report).
    4. Patient is at least 6 years of age, of either sex
    5. Body weight > 15 kg.
    6. The patient and his/her parent/caregiver (if below the age of consent), or a legal representative, has provided written informed consent to participate. If consent is provided solely by the caregiver in accordance with local regulations, the patient must provide assent to participate in the study.
    E.4Principal exclusion criteria
    General
    General
    1. Females that are
    a. pregnant or are breast-feeding (for EU countries only),
    b. of childbearing potential, pregnant, or are breast -feeding (for US and Rest of Worls countries).
    Females of childbearing potencial using adequate birth control, as determined by their Health Care Provider, will be eligible.
    2. A disability that may prevent the patient from completing all study requirements.
    3. Current participation in another clinical study.

    Medical History and Current Status
    4. CD4+ lymphocytes count <400/mm3 (for patients 6 years of age) or <150/mm3 (for patients > 6 years). In presence of oral infections, like oral candidiasis, documented at the screening or recurrent as per medical history documentation, the limit increases to <200/mm3 (for patients >6 years).
    5. Loss/removal of 250 mL or more of blood within the past 4 weeks prior to screening.
    6. Current neoplastic disease or previous neoplastic disease not in remission for at least 2 years.
    7. History of severe impairment of the immunological system.
    8. Severe or unstable pulmonary disease.
    9. Uncontrolled diabetes.
    Patients with diabetes that has been stabilized (i.e. no hypoglycemic or hyperglycemic episodes in the past 3 months) will be eligible.
    10. Any other severe, unstable, or serious disease or condition that in the Investigator’s opinion would put the patient at risk for imminent life-threatening morbidity, need for hospitalization, or mortality.
    11. Any clinically significant abnormality on standard laboratory examinations (hematology, biochemistry, urinalysis) at screening that remains abnormal on repeat testing. Eligibility of patients with abnormal laboratory test values will be determined by the Investigator in consultation with the Medical Monitor.
    12. Confirmed hemoglobinopathies, e.g. hemoglobin C disease, sickle cell anemia, or thalassemia.
    13. Moderate or severe renal and/or hepatic impairment.

    Prior/Concomitant Medication
    14. Any previous oral or parenteral steroid use within 4 weeks before Baseline. Treatment with inhaled or intranasal steroids for asthma or allergies, as well as use of topical steroids will be permitted.
    15. Chronic condition or prior allergic reaction representing a contraindication to the use of dexamethasone or other steroid drugs.
    16. Has participated in any other trial with an investigational drug and received a dose within 30 days or 10 half-lives (whichever is greater) from the start of the 30-day Screening Period.
    17. Has participated in a previous trial with EDS.
    18. Requires any concomitant medication prohibited by the protocol.
    19. Has taken a drug or treatment known to cause major organ system toxicity during the past year.
    20. Use of any drug that is a strong inducer/inhibitor of CYP3A4 within 4 weeks before baseline.

    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint will be the change from baseline to the follow-up assessments at Days 90 and 180, in the total score on the ‘Modified’ International Cooperative Ataxia Rating Scale (ICARS).
    E.5.1.1Timepoint(s) of evaluation of this end point
    The primary efficacy endpoint will be the change from baseline to the follow-up assessments at Days 90 and 180, in the total score on the ‘Modified’ International Cooperative Ataxia Rating Scale (ICARS). The analysis will be done once all subjects have completed day 180 assessments (month 6)
    E.5.2Secondary end point(s)
    Key Secondary Efficacy Endpoint
    The key secondary efficacy end point is the CGI-C rating of change from baseline at the follow-up assessments at Days 90 and 180.

    Other Secondary Efficacy Endpoints
    The following secondary efficacy endpoints will be assessed in the study:
    - The change of CGI-S score from baseline to follow-up based on repeated measures at Days 90 and 180
    - The change of VABS score from baseline to follow-up based on repeated measures at Days 90 and 180
    E.5.2.1Timepoint(s) of evaluation of this end point
    Days 90 and 180
    For more details, please refer to study protocol.
    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 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) No
    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 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA9
    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
    Australia
    Belgium
    Costa Rica
    Germany
    India
    Israel
    Italy
    Norway
    Poland
    Spain
    Tunisia
    Turkey
    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
    A patient will be considered to have ‘completed’ the Initial Treatment Period of the study when he/she returns for the final evaluations on Month 6. Patients who continue in the Extension Treatment Period will be considered to have ‘completed’ this period of the study when he/she returns for the final evaluations on Month 12.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months6
    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: 180
    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: 117
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 30
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 9
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 64
    F.4.2.2In the whole clinical trial 180
    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 care
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2016-06-30
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2021-05-13
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