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    Summary
    EudraCT Number:2021-003087-27
    Sponsor's Protocol Code Number:ARGX-113-2009
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-03-11
    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 ConcernedFrance - ANSM
    A.2EudraCT number2021-003087-27
    A.3Full title of the trial
    A Phase 2/3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to assess the efficacy and safety of a subcutaneous formulation of efgartigimod PH20 in adults with bullous pemphigoid.
    A.3.2Name or abbreviated title of the trial where available
    A phase 2/3 study of efgartigimod PH20 SC in adult participants with bullous pemphigoid
    A.4.1Sponsor's protocol code numberARGX-113-2009
    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 Sponsorargenx BV
    B.1.3.4CountryBelgium
    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 supportargenx BV
    B.4.2CountryBelgium
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationargenx BV
    B.5.2Functional name of contact pointRegulatory
    B.5.3 Address:
    B.5.3.1Street AddressIndustriepark Zwijnaarde 7
    B.5.3.2Town/ cityZwijnaarde (Ghent)
    B.5.3.3Post codeB-9052
    B.5.3.4CountryBelgium
    B.5.4Telephone number003293103400
    B.5.6E-mailregulatory@argenx.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameEfgartigimod PH20 SC
    D.3.4Pharmaceutical form 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 INNEFGARTIGIMOD ALFA
    D.3.9.1CAS number 1821402-21-4
    D.3.9.2Current sponsor codeARGX-113
    D.3.9.4EV Substance CodeSUB198780
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number180
    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
    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 placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Bullous Pemphigoid
    E.1.1.1Medical condition in easily understood language
    Autoimmune disease that causes painful or itchy blisters on the skin.
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10006567
    E.1.2Term Bullous pemphigoid
    E.1.2System Organ Class 100000004858
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Part A and B: To evaluate the efficacy of EFG PH20 SC on achieving sustained remission in the treatment
    of participants with bullous pemphigoid (BP).
    E.2.2Secondary objectives of the trial
    Parts A and B:
    • To evaluate the corticosteroid-sparing effects of EFG PH20 SC in participants with BP
    • To characterize the overall efficacy of EFG PH20 SC in the treatment of participants with BP
    • To evaluate the efficacy of EFG PH20 SC in preventing relapse of BP
    • To evaluate the effect of EFG PH20 SC on pruritus in participants with BP
    • To assess the safety and tolerability of EFG PH20 SC administered to participants with BP
    • To assess glucocorticoid-associated morbidity and evaluate the impact of EFG PH20 SC on reducing
    glucocorticoid toxicity
    • To evaluate the effects of EFG PH20 SC on the quality of life (QoL) of participants with BP
    • To evaluate the PK of EFG PH20 SC in participants with BP
    • To evaluate the PD of EFG PH20 SC in participants with BP
    • To evaluate the immunogenicity of EFG PH20 SC in participants with BP
    • To evaluate the competency of participants or caregivers to (self-)administer EFG PH20 SC.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    1. Vaccination Response Sub-study :
    Sites will collect all available vaccination history from all study participants.
    Selected sites may collect additional blood samples for additional/optional/future vaccination research, such as: (1) measurement of historical protective antibody titers in serum; (2) humoral (serum) and/or cellular (peripheral blood mononuclear cells [PBMCs]) responses to vaccinations received during the study. These samples will only be collected from participants who have provided additional consent for the procedure. Such samples will be collected at the visits identified in the SoA.
    Data obtained from this substudy will not be part of the clinical database; instead, they may be described in a separate report.

    2. Imaging Substudy:
    A photography substudy will be performed at selected study sites. In this substudy, site staff will take photographs of bullous lesions located at various anatomical regions of participants per the discretion of the investigator. As a guidance, timepoints of baseline, CDA, CR, and relapse are indicated; however, photographs may also be taken at intermediate timepoints.
    Photography is generally accepted as a routine practice for documenting dermatological conditions in medicine. Participants will be requested to provide consent for any use that will be made of the image and for sharing anonymized pictures with the sponsor.
    E.3Principal inclusion criteria
    * The participant is willing and able to do the following:
    a)understand the requirements of the study
    b)provide written informed consent
    c)comply with the study protocol procedures.
    * The participant is male or female, and aged ≥18 years at the time of signing the informed consent form (ICF).
    * Participants have clinical signs of BP.
    * Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies and:
    a) Male participants:
    - Must agree to use an acceptable method of contraception and not donate sperm from the time that the ICF is signed until they have received their last dose of IMP.
    b) Female participants:
    - Women of childbearing potential (WOCBP) must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before study intervention can be administered.
    - WOCBP must agree to use a highly effective or acceptable contraception method until at least 90 days after they receive their last dose of IMP.

    The full list of inclusion criteria can be found in the protocol.
    E.4Principal exclusion criteria
    • Other forms of pemphigoid or other autoimmune bullous diseases (AIBDs).
    • Received unstable dose of treatments known to cause or exacerbate BP for at least 4 weeks prior to the baseline visit
    • Use of BP treatments other than oral corticosteroids (OCS), topical corticosteroids, conventional immunosuppressants or dapsone.
    • Known contraindication to OCS therapy.
    • Clinically significant uncontrolled active or chronic, bacterial, viral, or fungal infection at screening.
    • Positive nasopharyngeal swab test for SARS-CoV-2 at screening.
    • History of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of the IMP. Participants with the following cancers can be included at any time, provided they are adequately treated prior to their participation in the study:
    o Basal cell or squamous cell skin cancer
    o Carcinoma in situ of the cervix
    o Carcinoma in situ of the breast
    o Incidental histological finding of prostate cancer
    • Clinical evidence of other significant serious diseases, have had a recent surgery, or who have any other condition that, in the opinion of the investigator, could confound the results of the study or put the patient at undue risk
    • Use of an investigational product within 3 months before the first dose of IMP
    • Previously participated in a clinical study with EFG
    • Known hypersensitivity to any of the components of the administered treatments
    • Positive serum test at screening for an active infection:
    o HBV
    o HCV
    o HIV
    • Current or history (ie, within 12 months of screening) of alcohol, drug, or medication abuse
    • Pregnant or lactating females and those who intend to become pregnant during the study or within 90 days after last dose of the IMP

    The full list of exclusion criteria can be found in the protocol.
    E.5 End points
    E.5.1Primary end point(s)
    The proportion of participants who are in complete remission (CR) while receiving minimal OCS therapy for ≥8 weeks at week 26. Minimal OCS therapy is defined as ≤0.1 mg/kg/day of prednisone (or equivalent).
    E.5.1.1Timepoint(s) of evaluation of this end point
    At week 26.
    E.5.2Secondary end point(s)
    1. Cumulative dose of oral corticosteroid (OCS) from baseline to week 36
    2. The proportion of participants who achieve an Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) score of 0 or 1 while being off OCS therapy for ≥8 weeks at week 36
    3. Proportion of participants who achieve control of disease activity (CDA) and remain free of relapse through week 36
    4. The proportion of participants who are in CR while being off OCS therapy for ≥8 weeks at week 36
    5. Changes from baseline to week 36 in the 24-hour average itch score from the Itch Numerical Rating Scale (Itch NRS)
    6. Proportion of participants who achieve an IGA-BP score of 0 or 1 at any time through week 36
    7. Change from baseline to week 36 in the Bullous Pemphigoid Disease Area Index (BPDAI) activity score
    8. Time to achieve the following:
    -Control of disease activity (CDA)
    -CR
    -CR while being on minimal OCS therapy for ≥8 weeks
    -CR/PR while being off OCS therapy for ≥8 weeks
    -CR while being off OCS therapy for ≥8 weeks
    -Relapse
    9. Cumulative OCS dose for the participant at the timepoints where they exhibit the following:
    -CDA
    -CR
    -CR while being on minimal OCS therapy for ≥8 weeks
    -CR/PR while being off OCS therapy for ≥8 weeks
    -CR while being off OCS therapy for ≥8 weeks
    -Relapse
    10. Changes from baseline to week 36 in the 24-hour worst itch score from the Itch NRS
    11. Proportion of participants who are off OCS therapy and have received no rescue therapy at week 36
    12. Proportion of participants who received rescue therapy before week 36
    13 Incidence and severity of TEAEs, AESIs, and SAEs
    14. The Aggregate Improvement Score (AIS) from the Glucocorticoid Toxicity Index (GTI)
    15. The Cumulative Worsening Score (CWS) from the GTI
    16. The Glucocorticoid Toxicity Index Specific List (GTI-SL)
    17. The EuroQol 5-Dimension 5-Level (EQ-5D-5L)
    18. The Dermatology Life Quality Index (DLQI)
    19. The Autoimmune Bullous Disease Quality of Life (ABQoL) score
    20. EFG serum concentrations
    21. Total IgG serum levels
    22. Anti-BP180 and anti-BP230 antibodies
    23. Antidrug antibodies (ADA) to EFG (in serum) and antibodies produced against rHuPH20 (in plasma)
    24. Number and percentage of participants (or their caregivers) who complete the (self-)administration training at study sites
    25. Number and percentage of participants (or their caregivers) who are determined by site staff to be sufficiently competent in (self-)administering EFG PH20 SC
    26. Number and percentage of participants (or their caregivers) who successfully (self-)administer EFG PH20 SC under site staff supervision
    Part B
    1. Cumulative dose of oral corticosteroid (OCS) from baseline to week 36
    2. The proportion of participants who achieve an Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) score of 0 or 1 while being off OCS therapy for ≥8 weeks at week 36
    3. Proportion of participants who achieve an IGA-BP score of 0 or 1 at any time through week 36
    4. Change from baseline to week 36 in the Bullous Pemphigoid Disease Area Index (BPDAI) activity score
    5. The proportion of participants who are in CR while being off OCS therapy for ≥8 weeks at week 36
    6. Time to achieve the following:
    -Control of disease activity (CDA)
    -CR
    -CR while being on minimal OCS therapy for ≥8 weeks
    -CR/PR while being off OCS therapy for ≥8 weeks
    -CR while being off OCS therapy for ≥8 weeks
    -Relapse
    7. Cumulative OCS dose for the participant at the timepoints where they exhibit the following:
    -CDA
    -CR
    -CR while being on minimal OCS therapy for ≥8 weeks
    -CR/PR while being off OCS therapy for ≥8 weeks
    -CR while being off OCS therapy for ≥8 weeks
    -Relapse
    8. Proportion of participants who are off OCS therapy and have received no rescue therapy at week 36
    9. Proportion of participants who received rescue therapy before week 36
    10. Proportion of participants who achieve CDA and remain free of relapse through week 36
    11. Changes from baseline to week 36 in the 24-hour average itch score from the Itch Numerical Rating Scale (Itch NRS)
    12. Changes from baseline to week 36 in the 24-hour worst itch score from the Itch NRS
    13. Incidence and severity of TEAEs, AESIs, and SAEs
    14. The Aggregate Improvement Score (AIS) from the Glucocorticoid Toxicity Index (GTI)
    15. The CWS from the GTI
    16. The GTI-SL
    17. The EQ-5D-5L
    18. The DLQI
    19. The ABQoL score
    20. EFG serum concentrations
    21. Total IgG serum levels
    22. Anti-BP180 and anti-BP230 antibodies
    23. ADA to EFG (in serum) and antibodies produced against rHuPH20 (in plasma)
    24. Number and percentage of participants (or their caregivers) who complete the (self-)administration training at study sites
    25. Number and percentage of participants (or their caregivers) who are determined by site staff to be sufficiently competent in (self-)administering EFG PH20 SC
    26. Number and percentage of participants (or their caregivers) who successfully (self-)administer EFG PH20 SC under site staff supervision
    E.5.2.1Timepoint(s) of evaluation of this end point
    Part A:
    - Endpoint 24 and 25 up to week 32
    - Endpoint 26 up to week 35
    - Endpoints 2, 4 and 11 at week 36
    - Endpoints 1, 3, 5-10, 12 and 14-19, up to week 36
    - Endpoint 20 up to week 43
    - Endpoints 21-23 up to week 46
    - Endpoint 13 until end of study

    Part B:
    - Endpoint 24 and 25 up to week 32
    - Endpoint 26 up to week 35
    - Endpoints 2, 5 and 8 at week 36
    - Endpoints 1, 3, 4, 6, 7, 9, 10-12 and 14-19 up to week 36
    - Endpoint 20 up to week 43
    - Endpoints 21-23 up to week 46
    - Endpoint 13 until end of study
    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 Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Immunogenicity, Tolerability
    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) Yes
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA70
    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
    China
    Israel
    Japan
    Russian Federation
    Serbia
    Ukraine
    United States
    Bulgaria
    Croatia
    France
    Germany
    Greece
    Hungary
    Italy
    Poland
    Spain
    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
    - The end of the study is defined as the date of the last visit of the last participant in part B of the study. Alternatively, it is defined as the date of the last participant visit following the sponsor’s decision to terminate the study for any reason.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    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 years2
    E.8.9.2In all countries concerned by the trial months1
    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: 40
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 120
    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 state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 97
    F.4.2.2In the whole clinical trial 160
    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 reach the EoTP visit (week 36) may be offered the option to roll over into a long-term, OLE study (ARGX-113-2010).

    Participants who are eligible but choose not to roll over into the OLE study ARGX-113-2010 will complete a 7-week treatment-free follow-up period.

    If a participant permanently withdraws from the study or is permanently withdrawn from the study by the investigator, standard-of-care BP treatment may be administered by the participant’s primary care physician.
    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 Decision2022-04-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-05-11
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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