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The European Union Clinical Trials Register   allows you to search for protocol and results information on:
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    The EU Clinical Trials Register currently displays   43936   clinical trials with a EudraCT protocol, of which   7310   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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    EudraCT Number:2017-000806-39
    Sponsor's Protocol Code Number:BAY2599023/19429
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2018-06-25
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedGermany - PEI
    A.2EudraCT number2017-000806-39
    A.3Full title of the trial
    A phase 1/2 open-label safety and dose-finding study of BAY 2599023 (DTX201), an adeno-associated virus (AAV) hu37-mediated gene transfer of B-domain deleted human factor VIII, in adults with severe hemophilia A
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to test the safety and how well patients with severe hemophilia A respond to treatment with BAY 2599023 (DTX 201), a drug therapy that delivers a healthy version of the defective Factor VIII gene into the nucleus of liver cells using an altered, non-infectious virus (AAV) as a “shuttle”.
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberBAY2599023/19429
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03588299
    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 SponsorBayer AG
    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 supportBayer AG
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBayer AG
    B.5.2Functional name of contact pointBayer Clinical Trials Contact
    B.5.3 Address:
    B.5.3.2Town/ cityBerlin
    B.5.3.3Post code13342
    B.5.4Telephone number+4930300139003
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namerecombinant AAV vector with hu37 serotype capsid (AAVhu37) encoding human FVIII
    D.3.2Product code BAY 2599023 (DTX201)
    D.3.4Pharmaceutical form Concentrate for 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 INNN/A
    D.3.9.1CAS number N/A
    D.3.9.2Current sponsor codeBAY 2599023 (DTX201)
    D.3.9.3Other descriptive nameBAY 2599023 (DTX201)
    D.3.9.4EV Substance CodeSUB192025
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number5e12
    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. cell therapy medicinal product No
    D. therapy medical product Yes
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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 Yes
    D.3.11.10Medicinal product containing genetically modified organisms Yes
    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
    Hemophilia A
    E.1.1.1Medical condition in easily understood language
    Hemophilia A is an X-linked congenital bleeding disorder causing frequent bleedings and recurrent spontaneous bleeds into the soft tissue and joints, leading to joint damage and severe disability.
    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 20.0
    E.1.2Level LLT
    E.1.2Classification code 10060612
    E.1.2Term Hemophilia A
    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
    Investigate the safety and tolerability of single ascending intravenous (IV) doses of BAY 2599023 (DTX201) in adult patients with severe hemophilia A, who have been previously treated with FVIII product
    E.2.2Secondary objectives of the trial
    Identify a dose of BAY 2599023 (DTX201) that will achieve sustained expression of vector-derived B-domain deleted human factor VIII above 5% at 6 months and 12 months following an IV administration
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Males ≥ 18 years of age
    • Subjects with severe hemophilia A (baseline FVIII activity FVIII:C <1%) determined by measurement at the time of Screening (unless there is genetic testing compatible with severe deficiency)
    • Previously treated with FVIII concentrate(s) (plasma derived or recombinant) or cryoprecipates for a minimum of 150 exposure days (ED)
    • Are on one of the following therapies: Prophylaxis, and is willing to stop prophylactic treatment at specified time points throughout the study or On-demand: have had > 4 bleeding events in the last 52 weeks
    • Subjects must agree to use double barrier and effective contraception methods. Vasectomized subjects must agree to use condoms. This is
    applicable from the time of the study drug administration until notified by the investigator. Time until discontinuation of contraception will be at a minimum 6 months, and will progressively increase with increasing dose. Recommendation to investigators is to continue the
    contraception until three consecutive blood and semen samples BLOD of shed virus have been obtained.
    Acceptable methods of contraception include, but are not limited to, (i) condoms with a spermicidal agent (ii) diaphragm or cervical cap with
    spermicide; if an intra-uterine device or hormone-based contraception is used by the patient's partner, an additional barrier method must be used.
    • Male subjects must agree not to donate cells, semen, blood, tissue or organs from the time of study drug administration

    E.4Principal exclusion criteria
    • Current evidence of inhibitor to FVIII with a titer ≥ 0.6 BU/mL
    • History of inhibitor to FVIII with a titer ≥ 0.6 BU, or clinical history requiring modification of treatment, suggestive of inhibitor. Family history of inhibitors will not exclude the subject.
    • Have significant underlying liver disease as evidenced by any of the following: portal hypertension, splenomegaly, ascites, esophageal varices, hepatic encephalopathy, reduction below normal limits of serum albumin or a liver biopsy with evidence of stage 3 fibrosis
    • Any of the following: Hemoglobin <11 g/dL; Platelets <100,000 cells/µL; Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >1.5 × ULN; Alkaline phosphatase (AP) >2.5 × ULN; Total bilirubin >1.5 × ULN; Prothrombin time (PT) or international normalized ratio (INR) >1.0 × ULN; Serum creatinine >1.5 mg/dL
    • Any individual with another bleeding disorder that is different from hemophilia A (e.g., von Willebrand disease, hemophilia B)
    • Have active hepatitis B or C infection, as reflected by HBsAg or HCV-RNA viral load positivity
    • Currently on antiviral therapy for hepatitis B or C.
    • Serological evidence of active HIV-1 or HIV-2 as measured by CD4+ cell count <200 cells/mm3 and a viral load >50 gc/mL
    • Anti-AAVhu37 neutralizing antibody titer ≥1:5
    • Any major and/or orthopedic surgery within screening period prior to trial product administration, and at least 6 months thereafter
    • History of a malignancy for which the subject has received treatment in the past 2 years except for prostate cancer being monitored without medical intervention, or surgically removed non-melanoma skin cancer
    • Known or suspected autoimmune diseases
    • Known prior history of hypersensitivity or anaphylaxis associated with any FVIII or immunoglobulin administration.
    • Known or suspected hypersensitivity or allergic reaction to trial product(s) or related FVIII products or any component of BAY 2599023 (DTX201), or a contraindication to prednisolone (as of amendment 6)
    • Live vaccines and COVID-19 vaccines are not allowed within the last 30 days prior to the study drug administration; live vaccines may be re-introduced after viral shedding has been cleared
    • Subjects on treatment with immunomodulatory agents within the last 3 months prior to study entry or during the study
    • Any individual who requires any pre-medication to tolerate FVIII treatment (e.g., antihistamines)
    • Prior use of emicizumab within 3 months before dosing
    • Clinically relevant findings in the physical examination considered critical by the treating physician, including obesity with BMI > 35 kg/m2
    E.5 End points
    E.5.1Primary end point(s)
    Incidence of adverse events, treatment-emergent adverse events, serious adverse events and Adverse events/serious adverse events of special interest
    E.5.1.1Timepoint(s) of evaluation of this end point
    End points will be assessed over the course of part A and Part B of the study.
    E.5.2Secondary end point(s)
    1. Expression pattern of FVIII activity.
    2. Proportion of patients in the respective dose step, that reached an expression of FVIII above 5% at 6 months and 12 months following the
    IV administration of BAY2599023
    E.5.2.1Timepoint(s) of evaluation of this end point
    at 6 months & 12 months following an IV administration
    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 No
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    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) Yes
    E.7.1.1First administration to humans Yes
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E. 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 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 EEA21
    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
    United States
    United Kingdom
    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
    LVLS (primary completion event for this study will be reached after the last subject completes his last visit).
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    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 years5
    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 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: 28
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    F.2 Gender
    F.2.1Female No
    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 No
    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 state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 25
    F.4.2.2In the whole clinical trial 30
    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)
    After completion of the study, subjects may restart their prior prophylactic or on demand treatment. No post-study therapy is planned.
    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 Decision2019-02-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-11-29
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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