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    Summary
    EudraCT Number:2022-000513-14
    Sponsor's Protocol Code Number:NBK154/2/2021
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-10-11
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2022-000513-14
    A.3Full title of the trial
    Evaluation of the Efficacy of Valsartan in Slowing Down Aortic Root Dilatation in Children and Young Adults with Marfan-type Heritable Thoracic Aortic Diseases
    – Valsar-TAD, a randomised, double-blind, placebo-controlled multicentre trial
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Evaluation of the Efficacy of Valsartan in Slowing Down Aortic Root Dilatation in Children and Young Adults with Marfan
    A.3.2Name or abbreviated title of the trial where available
    Valsar-TAD
    A.4.1Sponsor's protocol code numberNBK154/2/2021
    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 SponsorMedical University of Gdańsk
    B.1.3.4CountryPoland
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportMedical Research Agency
    B.4.2CountryPoland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPiotr Widłak
    B.5.2Functional name of contact pointCentrum Wsparcia Badań Klinicznych
    B.5.3 Address:
    B.5.3.1Street AddressM. Skłodowskiej-Curie 3a
    B.5.3.2Town/ cityGdańsk
    B.5.3.3Post code80-210
    B.5.3.4CountryPoland
    B.5.4Telephone number+4858349 27 67
    B.5.6E-mailpiotr.widlak@gumed.edu.pl
    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 Valsacor
    D.2.1.1.2Name of the Marketing Authorisation holderTAD Pharma GmbH, Heinz-Lohmann-Straße 5, 27472 Cuxhaven
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameValsartanum 40 mg / Placebo 1
    D.3.4Pharmaceutical form Coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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.IMP: 2
    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 Valzek
    D.2.1.1.2Name of the Marketing Authorisation holderCelon Pharma S.A, ul. Ogrodowa 2A, Kiełpin, 05-092 Łomianki
    D.2.1.2Country which granted the Marketing AuthorisationPoland
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameValsartanum 80 mg / Placebo 2
    D.3.4Pharmaceutical form Coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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 placeboCoated tablet
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 2
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCoated tablet
    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
    Marfan Syndrome
    Diseases related to Marfan syndrome (rare diseases with aneurysms of the thoracic aorta and dissection of the aorta), among others:
    Loeys-Dietz syndrome, Vascular type of Ehlers-Danlos syndrome,
    Arterial Tortuosity syndrome, Shprintzen-Goldberg syndrome,
    Neonatal form of Marfan syndrome, Aneurysms-osteoarthritis syndrome, Multi-system smooth muscle dysfunction syndrome,
    Familial thoracic aortic aneurysms and aortic dissections, etc.
    E.1.1.1Medical condition in easily understood language
    Marfan Syndrome and diseases related to Marfan Syndrome
    E.1.1.2Therapeutic area Diseases [C] - Cardiovascular Diseases [C14]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    absolute annual difference in aortic root diameter measured by transthoracic echocardiography and expressed in mm/year
    E.2.2Secondary objectives of the trial
    - annual difference in the aortic root diameter expressed in z-scores, indexed to sex and body surface area (BSA), measured by transthoracic echocardiography and expressed as z-score/year,
    - the absolute annual difference in aortic root diameter expressed in millimetres as assessed by angio- CT,
    - absolute annual difference in the diameter of the aortic annulus, sinotubular junction, distal ascending aorta, aortic arch, thoracic aorta, and abdominal aorta assessed by transesophageal echocardiography and expressed in mm/year,
    - annual difference in the diameter of the aortic annulus, sinotubular junction, distal ascending aorta, aortic arch, thoracic aorta and abdominal aorta expressed in z-score (sex-indexed and BSA), assessed by transesophageal echocardiography and expressed as z-score/year, etc.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Individuals who meet all of the following criteria will be eligible for the study:
    1. Age 1- 39 years.
    2. Diagnosis of a syndrome classified under the HTAD group:
    • Marfan Syndrome
    • Diseases related to Marfan syndrome (rare diseases with aneurysms of the thoracic aorta and dissection of the aorta), among others:
    Loeys-Dietz syndrome,
    Vascular type of Ehlers-Danlos syndrome,
    Arterial Tortuosity syndrome,
    Shprintzen-Goldberg syndrome,
    Neonatal form of Marfan syndrome,
    Aneurysms-osteoarthritis syndrome,
    Multi-system smooth muscle dysfunction syndrome,
    Familial thoracic aortic aneurysms and aortic dissections,
    Bicuspid aortic valve syndrome - familial BAV.
    3. Aortic root dilatation (z-score ≥ 2).
    4. Signed informed consent to participate in the study.
    E.4Principal exclusion criteria
    A person who meets any of the following criteria may not be included in the study:
    1. Past aortic cardiac surgery.
    2. Eligibility for aortic cardiac surgery at the time of examination.
    3. Hemodynamically significant, severe or moderate aortic valve defect assessed by echocardiography.
    4. Inability to obtain diagnostic echocardiographic images necessary for the evaluation of the aorta in a transthoracic echocardiographic examination - absence of the "Acoustic window".
    5. Heart failure; defined as left ventricular ejection fraction <40%.
    6. ARB therapy, unless 3 months have elapsed from the last dose taken before qualification.
    7. Taking ACE inhibitors, unless 3 months have elapsed from the last dose taken before qualification.
    8. Previous adverse reactions to valsartan or other ARB drugs.
    9. Contraindications to valsartan (including hypersensitivity to the active substance or to any of the excipients, severe liver dysfunction, biliary cirrhosis, cholestasis, bilateral renal artery stenosis).
    10. Breastfeeding, pregnancy or planning pregnancy in the next 12 months and for women of childbearing age, not on an effective method of contraception.
    11. Known renal impairment as manifested by estimated creatinine clearance <30 ml/min in children and <10 ml/min in adults.
    E.5 End points
    E.5.1Primary end point(s)
    absolute annual difference in aortic root diameter measured by transthoracic echocardiography and expressed in mm/year
    E.5.2Secondary end point(s)
    - annual difference in the aortic root diameter expressed in z-scores, indexed to sex and body surface area (BSA), measured by transthoracic echocardiography and expressed as z-score/year,
    - the absolute annual difference in aortic root diameter expressed in millimetres as assessed by angio- CT,
    - absolute annual difference in the diameter of the aortic annulus, sinotubular junction, distal ascending aorta, aortic arch, thoracic aorta, and abdominal aorta assessed by transesophageal echocardiography and expressed in mm/year,
    - annual difference in the diameter of the aortic annulus, sinotubular junction, distal ascending aorta, aortic arch, thoracic aorta and abdominal aorta expressed in z-score (sex-indexed and BSA), assessed by transesophageal echocardiography and expressed as z-score/year,
    - difference in aortic root diameter in subgroups of patients with different types of HTAD, identified by genetic testing,
    - acute aortic syndromes: aortic dissection, aortic perforation, intramural hematoma, penetrating ulcer), aortic dilatation requiring surgery, death from cardiovascular causes,
    - adverse events related to / potentially related to the administered pharmacotherapy (serious adverse events - SAE, adverse events – AE),
    - comparison of systolic and diastolic blood pressure values based on Holter blood pressure measurements.
    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 No
    E.6.5Efficacy No
    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) 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 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 concerned4
    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 months52
    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 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: 20
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 20
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 140
    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 No
    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
    Minority
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state180
    F.4.2 For a multinational trial
    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)
    According to the therapeutic standard used in the given disease entity
    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 Decision2023-03-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-10-13
    P. End of Trial
    P.End of Trial StatusOngoing
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