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    The EU Clinical Trials Register currently displays   43851   clinical trials with a EudraCT protocol, of which   7283   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:2020-005987-67
    Sponsor's Protocol Code Number:AK802
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-07-16
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedGermany - BfArM
    A.2EudraCT number2020-005987-67
    A.3Full title of the trial
    A randomized, part A partial blinded and part B double blinded, placebo-controlled 24-week clinical study to evaluate the efficacy and safety of nomacopan therapy in adult patients with bullous pemphigoid receiving adjunct oral corticosteroid therapy (ARREST-BP)
    Eine randomisierte, in Teil A teilweise verblindete und in Teil B doppelt verblindete, placebokontrollierte 24-wöchige klinische Studie zur Bewertung der Wirksamkeit und Sicherheit der Nomacopan-Therapie bei erwachsenen Patienten mit bullösem Pemphigoid, die eine begleitende orale Kortikosteroidtherapie (ARREST-BP) erhalten
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to see if 24-weeks of treatment with nomacopan works and is safe in adult patients with bullous pemphigoid who are also being treated with steroid tablets
    Eine Studie, um zu sehen, ob eine 24-wöchige Behandlung mit Nomacopan bei erwachsenen Patienten mit bullösem Pemphigoid, die auch mit Steroidtabletten behandelt werden, funktioniert und sicher ist
    A.4.1Sponsor's protocol code numberAK802
    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 SponsorAkari Therapeutics Plc
    B.1.3.4CountryUnited Kingdom
    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 supportAkari Therapeutics Plc
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAkari Therapeutics Plc
    B.5.2Functional name of contact pointDr Miles Nunn
    B.5.3 Address:
    B.5.3.1Street Address75-76 Wimpole Street
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeW1G 9RT
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+44208 004 0261
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/20/2289
    D.3 Description of the IMP
    D.3.1Product namenomacopan
    D.3.2Product code rVA576
    D.3.4Pharmaceutical form Powder 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 INNNOMACOPAN
    D.3.9.3Other descriptive namerVA576
    D.3.9.4EV Substance CodeSUB194416
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number18
    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. cell therapy medicinal product No
    D. therapy medical product No
    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 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 placeboPowder for solution 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
    moderate to severe bullous pemphigoid
    E.1.1.1Medical condition in easily understood language
    (moderate to severe) disease that causes an itch, red rash and blisters of the skin and sometimes the mouth and eyes
    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
    Confirm the dose of nomacopan and sample size for Part B and evaluate the rank order of secondary endpoints
    E.2.2Secondary objectives of the trial
    Compare nomacopan with adjunct OCS to placebo with adjunct OCS in achievement of Complete Remission of disease on minimal OCS (CRon)

    Further objectives are listed in section of the protocol based on different assessments inculding safety assessments (assess the safety and tolerability of nomacopan with adjunct OCS compared to placebo and adjunct OCS)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female between 18 and 89 years of age inclusive at the time of consent with Karnofsky score of 50% or more at screening.
    2. Male or female ≥90 years of age at the time of consent with Karnofsky score of 70% or more at screening.
    3. Diagnosis of Bullous Pemphigoid (either newly diagnosed or relapsing); if relapsing they may have previously been shown to satisfy these diagnostic criteria for BP:
    a. blisters alone, or blisters and/or urticaria or papules or erythema with or without itch,
    b. direct immunofluorescence of perilesional skin collected near a fresh blister, erosion or papule showing linear deposition of immunoglobulin G (IgG) and/or C3 along the epidermal basement membrane zone.
    c. indirect immunofluorescence studies performed with patient serum on 1.0M NaCl human salt split skin, showing anti-basement membrane zone antibodies binding the epidermal side of the salt split skin OR anti-BP180 or anti-BP230 antibody positive by enzyme linked immunosorbent assay. Note, if the serum binds both the epidermal and dermal side of the salt split skin the serum must also be anti-BP180/BP230 antibody positive by ELISA for inclusion of the subject.
    4. Patients with atypical Bullous Pemphigoid (ie without blisters) if they have positive direct immunofluorescence AND positive indirect immunofluorescence AND are anti-BP180 antibody positive.
    5. Bullous Pemphigoid classified as either moderate or severe on the basis of the Investigator Global Assessment (IGA) at randomisation. Moderate BP classified as an IGA score of 3 and severe BP classified as an IGA score of 4.
    6. Willing to receive immunisation against Neisseria meningitidis and/or antibiotic prophylaxis in accordance with applicable guidelines and local standard of care (SOC).
    7. Ability to travel to site for scheduled clinic visits and be available for scheduled assessments to be performed in their place of residence by trained healthcare practitioners.
    8. Provision of voluntary written informed consent. Note: Consent must be obtained prior to any study-related procedures.
    9. Women of childbearing potential must agree to use two methods of contraception, one highly effective and one effective (barrier), consistently throughout the study and have a negative serum pregnancy test at screening and a negative urine pregnancy test per the schedule of events. Women are considered post-menopausal and not of childbearing potential if they have had 12 months of amenorrhea, without an alternative medical cause, or have had permanent sterilisation methods (including hysterectomy, bilateral salpingectomy and bilateral oophorectomy) at least six weeks before randomisation.
    10. Males with a childbearing potential partner must agree to use two methods of contraception, one highly effective and one effective, consistently throughout the study including a barrier method.
    11. Patients who have recovered from a proven SARS-CoV2 infection within the 6-months prior to screening or who have received SARS-CoV2 full vaccination according to local guidelines.
    E.4Principal exclusion criteria
    1. Patients with recalcitrant BP that have never achieved CDA or who have never been in complete disease remission with persistent disease after a period of 12 months from the start of super potent steroid or OCS treatment.
    2. Epidermolysis bullosa acquisita, mucous membrane pemphigoid, or anti p200 pemphigoid.
    3. Mucosal lesion BPDAI score accounts for ≥30% of total BPDAI activity score at randomisation
    4. BP considered to be drug induced, in particular diagnosis of BP made within two months of starting a drug well known to induce BP – notably including, but not limited to, dipeptidyl peptidase-4 (DDP-4) inhibitors (eg, sitagliptin, vildagliptin, linagliptin), anti-PD1 inhibitors (eg, pembrolizumab, nivolumab), and certain diuretics (eg, furosemide, spironolactone) and neuroleptics (eg, doxepin, risperidone).
    5. Participation in a clinical trial of an investigational product within 6 weeks of screening.
    6. Treatment with BP-directed biologics as follows:
    a. Any cell-depleting agents including, but not limited to, rituximab within 12 months prior to baseline.
    b. Other biologics within five half-lives (if known) or 16 weeks prior to the baseline, whichever is longer.
    c. Intravenous immunoglobulin within 16 weeks prior to the baseline.
    7. Taking > 0.3 mg/kg/day OCS at screening
    8. Treatment with systemic immunomodulators such as dapsone or doxycycline within four half-lives of the drugs prior to baseline Day 1 (5 days for dapsone, 3 days for doxycycline).
    9. Treatment with immunosuppressants (such as methotrexate, azathioprine, mycophenolate, calcineurin inhibitors) within the last two weeks prior to baseline (Day 1).
    10. Treatment with an anti-complement therapy or with Zileuton (Zyflo) within the last three months prior to baseline (Day 1).
    11. OCS dose no more than 0.3mg/kg/day in the 7 days before screening visit.
    12. Taking super-potent topical corticosteroids (such as clobetasol propionate, augmented betamethasone dipropionate, diflucortolone valerate, fluocinonide, flurandrenolide and halobetasol propionate) and unable to discontinue them at or before the screening assessment.
    13. Medical or surgical conditions at screening or Day 1, that in the Investigator's opinion would make the patient inappropriate for study entry; this might include severe medical conditions such as stroke, heart failure, or serious neoplasia with a very high risk of mortality. In patients considered at high risk (e.g., HIV, silicosis, organ transplantation, etc.) of reactivation of latent tuberculosis (TB) infection, a recent (within 6-months) negative TB investigation (e.g., chest X-ray (CXR), negative interferon gamma release assay) should be available or undertaken at screening.
    14. Impaired neurological function which, in the Investigator’s opinion, will prevent participation in the study.
    15. Active systemic or organ system bacterial or fungal infection or progressive severe infection (including unresolved or untreated N. meningitidis infection and Escherichia coli Shiga toxin).
    16. Known congenital immunodeficiency or a history of acquired immunodeficiency including a positive human immunodeficiency virus (HIV) test.
    17. Active infection with hepatitis B or C.
    18. Positive nasal throat swab for Neisseria species.
    19. Planned major surgical procedures during the conduct of the study.
    20. Known hypersensitivity to nomacopan and any of its excipients.
    21. Contraindication to OCS including uncontrolled diabetes mellitus, uncontrolled hypertension, cardiac insufficiency, recent serious infection and allergy to OCS.
    22. Receipt of live attenuated vaccines for example yellow fever vaccine or some influenza vaccines within 2 weeks of Day 1 (Baseline).
    23. Pregnant or breast feeding or planning to become pregnant during the study.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of patients in Complete Disease Remission on (CRon) minimal OCS therapy (0.1 mg/kg/day) for 8 weeks or more at week 24 [Time Frame: weeks 16 – 24].
    E.5.1.1Timepoint(s) of evaluation of this end point
    week 24
    E.5.2Secondary end point(s)
    Other Endpoints (Exploratory for Part A, Secondary for Part B)
    1. Cumulative OCS dose during treatment [Time Frame: randomization to 24 weeks].
    2. Proportion of patients requiring rescue therapy during the 24 weeks of treatment [Time Frame: randomization to 24 weeks].
    3. Proportion of patients in Partial Disease Remission on (PRon) minimal OCS therapy (0.1 mg/kg/day) for 8 weeks or more at week 24 [Time Frame: weeks 16 – 24].
    4. Time (weeks) to onset of Complete Remission on (CRon) Minimal OCS and Partial Remission on (PRon) Minimal OCS [Time Frame: from week 6 to end of study].
    5. Duration (weeks) of Complete Remission on (CRon) Minimal OCS and Partial Remission on (PRon) Minimal OCS [Time Frame: from week 6 to end of study].
    6. Proportion of patients with Investigator Global Assessment (IGA) score of 0 or 1 (clear or almost clear) at 6 weeks, 12 weeks and end of treatment [Time Frame: weeks 6 – 24].
    7. Frequency, type and relationship of AEs to treatment [Time Frame: randomisation to end of study].
    8. Incidence of steroid-related AEs [Time frame randomisation to end of study].
    9. Change from baseline in Dermatology Life Quality Index (DLQI) [Time Frame: randomisation to week 24].
    10. Incidence of treatment-emergent anti-drug antibody (ADA) responses and titre and neutralising potential assessed in vitro at baseline and every 4 weeks [Time Frame: randomisation to end of study].
    E.5.2.1Timepoint(s) of evaluation of this end point
    randomization to end of study or intermediate depending on the endpoint (details given in brackets "[]" with the respective end point)
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    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 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. 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 No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E. trial design description
    Partial-blind design: Blind for placebo/active but unblind for low/high dose
    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 trial4
    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 concerned9
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA17
    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
    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
    When the last subject completes their last follow-up 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 months3
    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: 14
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 34
    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 state13
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 25
    F.4.2.2In the whole clinical trial 48
    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)
    Patients will be referred to their dermatologists or practitioner for SOC.
    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 Decision2021-12-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-12-22
    P. End of Trial
    P.End of Trial StatusOngoing
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