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    Summary
    EudraCT Number:2020-000086-17
    Sponsor's Protocol Code Number:AK901
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-05-21
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2020-000086-17
    A.3Full title of the trial
    Multicentre Study of nomacopan in Paediatric Haematopoietic
    Stem-Cell Transplant Associated Thrombotic Microangiopathy
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical study to confirm the dose, effectiveness and safety of nomacopan in patients with Thrombotic Microangiopathy (clotting of the blood vessels) which can be a complication of bone marrow transplants.
    A.4.1Sponsor's protocol code numberAK901
    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 pointWynne Weston-Davies
    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+44 20 8004 0261
    B.5.6E-mailwynne.weston-davies@akaritx.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 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.3.9.2Current sponsor coderVA576
    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.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
    E.1.1.1Medical condition in easily understood language
    Haematopoietic stem cell transplantation thrombotic microangiopathy
    E.1.1.2Therapeutic area Body processes [G] - Immune system processes [G12]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10043645
    E.1.2Term Thrombotic microangiopathy
    E.1.2System Organ Class 10005329 - Blood and lymphatic system disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10063581
    E.1.2Term Stem cell transplant
    E.1.2System Organ Class 10042613 - Surgical and medical procedures
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 22.0
    E.1.2Level PT
    E.1.2Classification code 10067859
    E.1.2Term Allogenic stem cell transplantation
    E.1.2System Organ Class 10042613 - Surgical and medical procedures
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 22.0
    E.1.2Level PT
    E.1.2Classification code 10081347
    E.1.2Term Autologous haematopoietic stem cell transplant
    E.1.2System Organ Class 10042613 - Surgical and medical procedures
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Part A (7 patients aged ≥ 0.5 to < 18 years, in three age range cohorts)
    Dose algorithm confirmation:
    ▪ To confirm the effective dose of nomacopan for the ablation of terminal complement activity. The data derived from Part A of the trial will be used together with existing data for PK/PD simulation modelling to define an age-based dosing regimen to completely control terminal complement activity in paediatric patients treated with nomacopan in Part B
    E.2.2Secondary objectives of the trial
    Efficacy:
    To determine that the age-based dosing regimen defined in Part A can completely control complement activity in paediatric patients treated with nomacopan.
    Safety:
    To evaluate safety and tolerability of nomacopan
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Aged ≥ 0.5 and < 18 years at the time of diagnosis of TMA.
    2. Undergone allogeneic or autologous HSCT.
    3. TMA diagnosis within a year of their first allogeneic or autologous HSCT.
    4. Clinical diagnosis of TMA with all of the following diagnostic criteria:
    ▪ elevated plasma serum C5b-9 (80% or more of ULN for age)
    ▪ Urine protein creatinine ratio > 2 mg/mg (demonstrated on two separate morning samples, at least one day apart)
    ▪ elevated LDH (> ULN)
    ▪ thrombocytopaenia (< 50,000 per mm3)
    ▪ low haemoglobin concentration (< LLN) or Histological diagnosis of TMA with evidence of complement deposition:
    ▪ Urine protein creatinine ratio > 2 mg/mg (demonstrated on two separate morning samples, at least one day apart), and
    ▪ elevated serum C5b-9 (80% or more of ULN for age)
    5. Provision of written informed consent.
    6. Provision of informed assent (where appropriate)
    E.4Principal exclusion criteria
    1. Patients weighing less than 5 kg.
    2. Patients with a positive direct Coomb's test.
    3. Patients who do not receive nomacopan within 21 days of the initial diagnosis of TMA.
    4. Patients having an active systemic or organ system bacterial or fungal infection or progressive severe infection (including unresolved or untreated Neisseria meningitidis infection and E. coli Shiga toxin) at the time of diagnosis of the TMA.
    5. Grade 4 Acute GVHD (as per the Glucksberg grading system, see section 18.9).
    6. Received eculizumab or any other complement blocker therapy at any time.
    7. Known hypersensitivity to the active ingredient or excipients
    8. Patients who are pregnant and/or breastfeeding. All females of childbearing potential require a negative pregnancy test at screening.
    E.5 End points
    E.5.1Primary end point(s)
    Composite Primary Endpoint
    ▪ RBC transfusion independence for ≥ 28 days immediately prior to any scheduled clinical visit up to Week 24 or Urine protein creatinine ratio ≤ 2 mg/mg for ≥ 28 days immediately prior to any scheduled clinical visit up to Week 24
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 24
    E.5.2Secondary end point(s)
    -Secondary Efficacy Endpoints
    ▪Percentage of patients who achieve the primary endpoint of urine protein creatinine ratio ≤ 2 mg/mg (the nephrotic threshold) for ≥ 28 days
    ▪ Platelet transfusion independence† for ≥ 28 days.
    ▪ Plasma sC5b-9 ≤ ULN
    ▪ Lactate dehydrogenase (LDH) ≤ULN
    ▪Normalization of haptoglobin

    -Secondary Safety Endpoints
    ▪ Safety and tolerability of nomacopan

    E.5.2.1Timepoint(s) of evaluation of this end point
    At 24 weeks and the sC5b-9, LDH and haptoglobin will be measured at the last efficacy assessment in the trial before nomacopan treatment is stopped.
    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 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
    Dose algorithm
    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 Yes
    E.8.1.7.1Other trial design description
    Open-label, multi-centre two-part study
    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 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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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
    Italy
    Poland
    United Kingdom
    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
    The End of the Study will occur when all surviving
    patients have attended the long-term follow-up visit at two years after diagnosis of HSCT
    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 months3
    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 months6
    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 Yes
    F.1.1Number of subjects for this age range: 72
    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: 18
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 36
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 18
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children/minors
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state7
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 12
    F.4.2.2In the whole clinical trial 72
    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)
    None, as per country standard of care
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation PrimeVigilance Limited
    G.4.3.4Network Country Czechia
    G.4 Investigator Network to be involved in the Trial: 2
    G.4.1Name of Organisation PPD CRO Services
    G.4.3.4Network Country United Kingdom
    G.4 Investigator Network to be involved in the Trial: 3
    G.4.1Name of Organisation Yourway Transport Biopharma Services
    G.4.3.4Network Country United States
    G.4 Investigator Network to be involved in the Trial: 4
    G.4.1Name of Organisation Colpitts Clinical
    G.4.3.4Network Country United Kingdom
    G.4 Investigator Network to be involved in the Trial: 5
    G.4.1Name of Organisation SciLucent, Inc
    G.4.3.4Network Country United States
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2020-07-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-06-18
    P. End of Trial
    P.End of Trial StatusOngoing
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