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    Summary
    EudraCT Number:2023-001026-34
    Sponsor's Protocol Code Number:CSL627_3003
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2024-12-19
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2023-001026-34
    A.3Full title of the trial
    A Phase 3, Open-label, Multicenter, Pharmacokinetics, Efficacy, and Safety Study of a Recombinant Single-chain Factor VIII (rVIII-SingleChain) in Chinese Previously Treated Patients (PTPs) with Hemophilia A
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Recombinant single-chain factor VIII (rVIII-SingleChain) in Chinese participants with hemophilia A previously treated with FVIII products
    A.4.1Sponsor's protocol code numberCSL627_3003
    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 SponsorCSL Behring
    B.1.3.4CountryGermany
    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 supportCSL Behring
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCSL Behring GmbH
    B.5.2Functional name of contact pointTrial Registration Coordinator
    B.5.3 Address:
    B.5.3.1Street AddressEmil-von-Behring-Strasse 76
    B.5.3.2Town/ cityMarburg
    B.5.3.3Post code35041
    B.5.3.4CountryGermany
    B.5.4Telephone number16108784697
    B.5.6E-mailclinicaltrials@cslbehring.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 Yes
    D.2.1.1.1Trade name Afstyla®
    D.2.1.1.2Name of the Marketing Authorisation holderCSL Behring GmbH
    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 nameRecombinant single-chain factor VIII (rVIII-SingleChain)
    D.3.2Product code CSL627
    D.3.4Pharmaceutical form Powder and solvent 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 INNLonoctocog alfa
    D.3.9.1CAS number 1388129-63-2
    D.3.9.2Current sponsor codeCSL627
    D.3.9.3Other descriptive namerVIII-SingleChain
    D.3.9.4EV Substance CodeSUB177134
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number250 to 3000
    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
    Congenital Hemophilia A
    E.1.1.1Medical condition in easily understood language
    Hemophilia A is a rare but serious bleeding disorder which affects males and is characterized by a deficiency in the plasma protein known as coagulation Factor VIII
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    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 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
    To evaluate the pharmacokinetics (PK) of plasma FVIII activity in Chinese PTPs with severe hemophilia A (FVIII activity < 1%) after a single dose of 50 IU/kg rVIII-SingleChain, to evaluate the efficacy of routine prophylaxis dosing with rVIII-SingleChain in preventing spontaneous bleeding episodes in Chinese PTPs with severe hemophilia A (FVIII activity < 1%), and to evaluate the safety of routine prophylaxis dosing with rVIII-SingleChain in Chinese PTPs with severe hemophilia A (FVIII activity < 1%) with respect to development of FVIII inhibitors.
    E.2.2Secondary objectives of the trial
    To further evaluate the PK of plasma FVIII activity in Chinese PTPs with severe hemophilia A (FVIII activity < 1%) after a single dose of 50 IU/kg rVIII-SingleChain, (if data allow), to evaluate the PK of plasma FVIII activity in Chinese PTPs ≥ 12 years of age with severe hemophilia A (FVIII activity < 1%) after a dose of 50 IU/kg rVIII-SingleChain during the Repeat PK Period, to further evaluate the efficacy of rVIII-SingleChain in Chinese PTPs with severe hemophilia A (FVIII activity < 1%), and to further evaluate the safety of rVIII-SingleChain in Chinese PTPs with severe hemophilia A (FVIII activity < 1%).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Male Chinese participants ≤ 65 years of age
    • Participants with severe hemophilia A (FVIII activity < 1%)
    • Participants who have received FVIII products for ≥ 150 EDs (≥ 6 years of age) or ≥ 50 EDs (< 6 years of age)
    E.4Principal exclusion criteria
    • Known hypersensitivity (allergic reaction or anaphylaxis) to any FVIII product or hamster protein.
    • Known congenital or acquired coagulation disorder other than congenital FVIII deficiency.
    • Currently receiving intravenous (IV) immunomodulating agents such as immunoglobulin or chronic systemic corticosteroid treatment.
    • Use of traditional or herbal Chinese medicine(s) with an impact on hemophilia, including coagulation, within 28 days before Day 1 and / or refusal to abstain from these during the study until the end of the participant’s participation in the study.
    E.5 End points
    E.5.1Primary end point(s)
    1. Incremental Recovery (IR) of rVIII-SingleChain
    2. Maximum Concentration (Cmax) of rVIII-SingleChain
    3. Area Under the Plasma Concentration Time Curve from Time Zero to the Last Measurable Concentration (AUC0-last) of rVIII-SingleChain
    4. Area Under the Plasma Concentration Time Curve from Time Zero to Infinity (AUC0-inf) of rVIII-SingleChain
    5. Half-life (t1/2) of rVIII-SingleChain
    6. Clearance (Cl) of rVIII-SingleChain
    7. Annualized Spontaneous Bleeding Rate (AsBR)
    8. Number of participants who develop FVIII inhibitors
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Before, and at 30 minutes after the end of, rVIII-SingleChain administration on Day 1
    2 to 6: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
    7. Up to 29 weeks after rVIII-SingleChain administration
    8. During routine rVIII-SingleChain prophylaxis dosing, up to 29 weeks after rVIII-SingleChain administration.
    E.5.2Secondary end point(s)
    1. Time to reach maximum concentration (Tmax) of rVIII-SingleChain
    2. Last Concentration (Clast) of rVIII-SingleChain
    3. IR (participants ≥ 12 years of age) of rVIII-SingleChain (Repeat pharmacokinetic [PK])
    4. Cmax (participants ≥ 12 years of age) of rVIII-SingleChain (Repeat PK)
    5. AUC0-last of rVIII-SingleChain (Repeat PK)
    6. AUC0-inf of rVIII-SingleChain (Repeat PK)
    7. t1/2 of rVIII-SingleChain (participants ≥ 12 years of age) (Repeat PK)
    8. Clearance (Cl) of rVIII-SingleChain (participants ≥ 12 years of age) (Repeat PK)
    9. Annualized Bleeding Rate (ABR)
    10. Hemostatic Efficacy for Major and Nonmajor Bleeding Episodes
    11. Consumption of rVIII-SingleChain - number of infusions (doses)
    12. Consumption of rVIII-SingleChain - IU/kg per participant per month
    13. Consumption of rVIII-SingleChain - IU/kg per participant per year
    14. Number of bleeding episodes requiring rVIII-SingleChain to achieve hemostasis
    15. Percentage of bleeding episodes requiring rVIII-SingleChain to achieve hemostasis
    16. Number of participants who develop noninhibitory antibodies against rVIII-SingleChain
    17. Number of participants who develop antibodies against Chinese hamster ovary host cell protein
    18. Number of participants with Treatment-emergent Adverse Events (TEAEs), including related TEAEs, and serious adverse events (SAEs)
    19. Percentage of participants with TEAEs, including related TEAEs, and SAEs
    20. Number of TEAEs (Events)
    E.5.2.1Timepoint(s) of evaluation of this end point
    1 and 2: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
    3 to 8: Before, and up to 72h after the end of, rVIII-SingleChain infusion at Week 28
    9 to 15: Up to 29 weeks after rVIII-SingleChain administration
    16 and 17: Before, and up to 29 weeks after, rVIII-SingleChain administration
    18 to 20: Up to 33 weeks after rVIII-SingleChain administration
    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 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 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 trial1
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    China
    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.8.9.2In all countries concerned by the trial months13
    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: 30
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 1
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 19
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 30
    F.1.3Elderly (>=65 years) No
    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 Yes
    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 Yes
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    This clinical trial will be conducted in adults and also in children in different age groups including children younger than 12 years of age.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 60
    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)
    Participants who are withdrawn from the study because they develop a confirmed FVIII inhibitor to FVIII during the study may be treated with immune tolerance induction according to the participant’s needs.
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: China
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