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    Summary
    EudraCT Number:2018-002776-40
    Sponsor's Protocol Code Number:SubQ8-01
    National Competent Authority:Bulgarian Drug Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-12-06
    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 ConcernedBulgarian Drug Agency
    A.2EudraCT number2018-002776-40
    A.3Full title of the trial
    Phase 1/2 study to assess the safety and pharmacokinetic of subcutaneous injection of OCTA101 in previously treated adult patients with severe hemophilia A
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to assess the safety and pharmacokinetic of subcutaneous injection of OCTA101 in previously treated adult patients suffering from severe hemophilia A
    A.4.1Sponsor's protocol code numberSubQ8-01
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04046848
    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 SponsorOctapharma AG
    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 supportOctapharma AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationOctapharma AG
    B.5.2Functional name of contact pointJohann Bichler
    B.5.3 Address:
    B.5.3.1Street AddressSeidenstrasse 2
    B.5.3.2Town/ cityLachen
    B.5.3.3Post code8853
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number+ 4155451 21 77
    B.5.5Fax number+ 4155 451 21 51
    B.5.6E-mailjohann.bichler@octapharma.ch
    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 nameOCTA101 (human-cl rhFVIII and recombinant human von Willebrand Factor fragment dimer)
    D.3.4Pharmaceutical form Powder and solution for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNB-domain deleted human coagulation factor VIII (simoctocog alfa)
    D.3.9.3Other descriptive nameOCTA101
    D.3.9.4EV Substance CodeSUB13813MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number500 to 5000
    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 No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    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 RoleComparator
    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 Nuwiq
    D.2.1.1.2Name of the Marketing Authorisation holderOctapharma AB
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameNuwiq
    D.3.4Pharmaceutical form Powder and solution for solution for injection
    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 INNCoagulation Factor VIII (Simoctocog Alfa)
    D.3.9.3Other descriptive nameHUMAN COAGULATION FACTOR VIII (RDNA)
    D.3.9.4EV Substance CodeSUB13815MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number200 to 1200
    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 No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    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
    Severe hemophilia A
    E.1.1.1Medical condition in easily understood language
    Haemophilia A patients have insufficient levels of an important factor (factor VIII) in their blood. Factor VIII is important to stop bleeding.
    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 10060612
    E.1.2Term Hemophilia A
    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
    The primary objective is to assess the safety of various doses of OCTA101 after subcutaneous (sc) injection.
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are to assess
    • the pharmacokinetics of FVIII:C after single sc injection of OCTA101
    • the dose proportionality of FVIII:C after sc injections of different doses of OCTA101
    • the bioavailability of sc OCTA101 compared to iv injection of Nuwiq
    • the pharmacokinetics of OCTA12 (recombinant human von Willebrand Factor fragment dimer) after single injection of OCTA101
    • plasma trough and peak levels of FVIII:C after daily injections of OCTA101
    • plasma trough and peak levels of OCTA12 after daily injections of OCTA101
    • the efficacy of sc injections of OCTA101 in preventing bleeding episodes
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients who meet all of the following criteria are eligible for the study:
    1. Severe hemophilia A (<1% FVIII:C) as documented in medical records
    2. Males ≥18 years of age
    3. Subjects who have had ≥150 exposure days (EDs) with a FVIII product
    4. Written informed consent for study participation obtained before undergoing any study specific procedures
    E.4Principal exclusion criteria
    Patients who meet any of the following criteria are not eligible for the study:
    1. Previous participation in this trial
    2. Use of an Investigational Medicinal Product within 30 days prior to the first OCTA101 injection
    3. History of FVIII inhibitors titre ≥0.6 BU/mL defined by medical records
    4. Inhibitors to FVIII (≥0.6 BU/mL) at screening measured by Nijmegen modified Bethesda method at central laboratory
    5. Human immunodeficiency virus (HIV) positive subjects with a CD4+ count <200/mL
    6. Clinically significant anemia at screening (hemoglobin <8 g/dL)
    7. Presence of any significant comorbidity (at the discretion of the investigator) that might confound the interpretation of the study data and/or that might put the patient at undue risk by participating in the trial
    8. Any coagulation disorder other than hemophilia A
    9. AST or ALT levels >3 times the upper limit of normal
    10. Creatinine >120 μmol/L
    11. Platelet count <100,000 μL
    12. BMI ≥30 kg/m²
    13. For Cohort 6, patients with a positive LumiTope test at screening will be excluded.
    E.5 End points
    E.5.1Primary end point(s)
    - Adverse events
    - Dose Limiting Toxicity
    - Thromboembolic events
    - Local injection site reactions
    - Inhibitor formation to FVIII
    E.5.1.1Timepoint(s) of evaluation of this end point
    Adverse events, Dose Limiting Toxicity, and thromboembolic events and local injection site reactions will be evaluated throughout the study. Patient diaries are reviewed by the investigator at every hospital visit.

    Local injection site reactions will be evaluated after each injection.

    At the screening visit all patients will be tested for presence of FVIII inhibitors, and patients testing positive will be excluded from the trial. During the study, factor VIII inhibitor formation will be evaluated at defined time points, 2 to 9 times, depending on the cohort the patient is assigned to.
    E.5.2Secondary end point(s)
    - Antibody formation to OCTA12
    - LumiTope assay results
    - OCTA12 plasma levels during daily dosing
    - Routine lab tests compared to baseline
    - Vital signs compared to baseline
    - Physical examination results compared to baseline
    E.5.2.1Timepoint(s) of evaluation of this end point
    Physical examinations will be performed for patients in cohort 1 to 3 at the beginning of the study and at the end of the 3-month prophylactic treatment period. For cohort 6, physical examination will be performed at the beginning of the study and at the end of the 6-month prophylactic treatment period.

    Routine lab parameters and vital signs will be evaluated at defined time points, 8 to 24 times, depending on the cohort the patient is assigned to.

    OCTA12 levels during 3 months will be evaluated 10 times.

    Antibody formation to OCTA12 will be evaluated at defined time points, 2 to 9 times, depending on the cohort the patient is assigned to.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis Yes
    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 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.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.8.2.4Number of treatment arms in the trial5
    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 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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months46
    E.8.9.1In the Member State concerned days
    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: 38
    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 state40
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 40
    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 the end of the study, patients will be advised by the investigator to continue treatment with a Factor VIII concentrate that is available in the country.
    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 Decision2019-01-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-01-16
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-02-18
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