Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register allows you to search for protocol and results information on:
  • interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA);
  • clinical trials conducted outside the EU / EEA that are linked to European paediatric-medicine development.
  • Learn   more about the EU Clinical Trials Register   including the source of the information and the legal basis.


    The EU Clinical Trials Register currently displays   41008   clinical trials with a EudraCT protocol, of which   6704   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).


    Phase 1 trials conducted solely in adults and that are not part of an agreed PIP are not public in the EU CTR (refer to European Guidance 2008/C 168/02   Art. 3 par. 2 and   Commission Guideline 2012/C 302/03,   Art. 5) .
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2020-000489-40
    Sponsor's Protocol Code Number:IZD174-002
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2020-04-03
    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 ConcernedUK - MHRA
    A.2EudraCT number2020-000489-40
    A.3Full title of the trial
    A Multi-Centre, Randomised, Open-Label, Phase IIb Study to Evaluate the Safety, Tolerability and Efficacy of IZD174 in Patients with Cryopyrin Associated Periodic Syndromes
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase IIb study to Evaluate the Safety, Tolerability and Efficacy of IZD174 in Patients with Cryopyrin-Associated Periodic Syndromes
    A.4.1Sponsor's protocol code numberIZD174-002
    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 SponsorInflazome (Australia) Pty Ltd.
    B.1.3.4CountryAustralia
    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 supportInflazome (Australia) Pty Ltd.
    B.4.2CountryAustralia
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationInflazome (Australia) Pty Ltd.
    B.5.2Functional name of contact pointClinical Trial Lead
    B.5.3 Address:
    B.5.3.1Street Address71 Eagle Street
    B.5.3.2Town/ cityBrisbane
    B.5.3.3Post codeQLD 4000
    B.5.3.4CountryAustralia
    B.5.4Telephone number+61(0)421 585 707
    B.5.6E-mailf.roodt@inflazome.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 nameIZD174
    D.3.2Product code C-028380
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNN/A
    D.3.9.1CAS number 1995067-59-8
    D.3.9.2Current sponsor codeIZD174
    D.3.9.3Other descriptive nameInzomelid
    D.3.9.4EV Substance CodeSUB198782
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    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 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 nameIZD174
    D.3.2Product code C-028380
    D.3.4Pharmaceutical form Capsule
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNN/A
    D.3.9.1CAS number 1995067-59-8
    D.3.9.2Current sponsor codeIZD174
    D.3.9.3Other descriptive nameInzomelid
    D.3.9.4EV Substance CodeSUB198782
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 No
    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
    Cryopyrin-associated periodic syndrome (CAPS)
    E.1.1.1Medical condition in easily understood language
    Autoinflammatory disease of skin, joints, central nervous system, and eyes
    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 PT
    E.1.2Classification code 10068850
    E.1.2Term Cryopyrin associated periodic syndrome
    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 investigate the safety and tolerability of different dose levels of IZD174 in participants with CAPS
    •To explore the efficacy of IZD174 treatment to induce complete remission in participants with CAPS
    E.2.2Secondary objectives of the trial
    •To determine the PK and PD of different dose levels of IZD174 in participants with CAPS
    •To explore the efficacy of different dose levels of IZD174 to induce complete remission in participants with CAPS
    •To explore the efficacy of different dose levels of IZD174 to induce partial remission in participants with CAPS
    •To determine CRP and SAA levels in participants with CAPS following the treatment of IZD174
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Participants must be willing and able to provide written informed consent as described in Section 10.1, Appendix 1 (includes compliance with the requirements and restrictions listed in the ICF and in this protocol) after the nature of the study has been explained and prior to the commencement of any study procedures.
    2. Male or female adult participants with a confirmed diagnosis of CAPS aged between 18 to 75 years (inclusive at the time of signing the informed consent).
    A confirmed diagnosis of CAPS comprises the following:
    a. Documented verification of a genetic mutation in NLRP3; and
    b. Participant has previously experienced at least 2 typical clinical symptoms of CAPS (may include urticarial skin rash, myalgia, arthralgia, recurrent fever, fatigue/malaise, headache, conjunctivitis, and any other autoinflammatory symptom usual for a given participant); and
    c. Participant previously had elevated levels of CRP or SAA (> 2 × ULN).
    3. Positive response to IZD174 in ex vivo whole blood lipopolysaccharide (LPS)+Nigericin induced IL-1β release (IC50 < 50 μM).
    4. Participants must be willing to discontinue current anti-IL-1 inhibitor treatment prior to dosing if applicable.
    5. Participants must demonstrate flaring of CAPS following discontinuation of anti-IL-1 inhibitor treatment or if newly diagnosed. Flaring is defined as IGA score specific at the time of consultation > 5 with an elevation of CRP (> 2 × ULN).
    6. Participants must have a body mass index (BMI) between ≥18.0 and ≤38.0 kg/m2 at Screening.
    7. Female participants of reproductive age must be non-pregnant and non-lactating, and must use an acceptable, highly effective double contraception as detailed in Section 10.4, Appendix 4 from Screening until 1 month after the last treatment day. Double contraception is defined as a condom and one other form of the following:
    - A vaginal ring or an intrauterine device (IUD).
    - Documented evidence of surgical sterilization at least 6 months prior to Screening (e.g., tubal occlusion, hysterectomy, bilateral salpingectomy, or bilateral oophorectomy for women or vasectomy for men [with appropriate post-vasectomy documentation of the absence of sperm in semen] provided the male partner is a sole partner).
    Women not of childbearing potential must be post menopausal for ≥ 12 months. Post menopausal status will be confirmed through testing of follicle-stimulating hormone (FSH) levels ≥ 40 IU/mL at Screening for amenorrhoeic female participants. Females who are abstinent from heterosexual intercourse will also be eligible.
    True abstinence: When this is in line with the preferred and usual lifestyle of the subject. [Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods), declaration of abstinence for the duration of a trial, and withdrawal are not acceptable methods of contraception].
    Participant complete abstinence for the duration of the study and for 1 month after the last study treatment is acceptable.
    Female participants who are in same sex relationships are not required to use contraception.
    Male participants must be abstinent, or if engaged in sexual relations with a woman of childbearing potential (WOCBP), the participant and his partner must be using an acceptable, highly effective double contraceptive method as detailed in Section 10.4, Appendix 4 from Screening until Follow up Visit.
    Participants with same sex partners (abstinence from penile-vaginal intercourse) are eligible when this is their preferred and usual lifestyle.
    WOCBP must have a negative pregnancy test at Screening and Baseline and be willing to have additional pregnancy tests as required throughout the study.
    8. Male participants must not donate sperm for at least 90 days after the last dose of study drug.
    9. Participants must have the ability and willingness to attend the necessary visits to the study centre.
    E.4Principal exclusion criteria
    1. Use of any investigational drug or investigational device or investigational medical device or participation in a clinical study within 4 weeks prior to Screening or 5 half lives of the product (whichever is longer).
    2. Live vaccinations within 3 months prior to Screening, for the duration of the study and for up to 3 months following the last dose of study drug.
    3. Positive QuantiFERON test at the Screening Visit or within 2 months prior to Screening. Participants who have a positive QuantiFERON test with documentation of Bacillus of Calmette and Guerin vaccination, who are at low environmental risk for tuberculosis infection or reactivation and have a negative chest X-ray can be included.
    4. Any severe, progressive, or uncontrolled medical condition at Baseline that in the judgment of the Investigator prevents the participant from participating in the study.
    5. Prior or ongoing medical history, physical findings, or laboratory abnormality at Screening that, in the Investigator’s (or delegate’s) opinion, may affect full participation or compliance with the protocol, or could adversely affect the safety of the participant.
    6. Presence of any underlying physical or psychological medical condition that, in the opinion of the Investigator, would make it unlikely that the participant will complete the study per protocol.
    7. Blood donation or significant blood loss within 60 days prior to the first study drug administration.
    8. Plasma donation within 7 days prior to the first study drug administration.
    9. Active systemic infections (other than common cold) within 2 weeks prior to Baseline.
    10. History of severe allergic or anaphylactic reactions.
    11. History of (or) is currently immunocompromised.
    12. Abnormal laboratory tests at Screening that are considered by the Investigator to be clinically significant.
    13. Alkaline phosphatase (ALP), aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) >1.5 × ULN at Screening. Repeat testing at Screening is acceptable for out of range values following approval by the Investigator or delegate.
    14. Positive test for hepatitis B virus surface antigen (HBsAg), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) antibody at Screening.
    15. Participants with a positive toxicology screening panel (urine test including qualitative identification of barbiturates, tetrahydrocannabinol [THC], amphetamines, benzodiazepines, opiates and cocaine.
    16. Participants with a history of substance abuse or dependency or history of recreational IV drug use over the last 5 years (by self-declaration).
    17. Use of any prescription drugs, over-the-counter (OTC) medication, herbal remedies, supplements or vitamins 1 week prior to dosing. 1 2 therapeutic doses per week of simple analgesia (aspirin, paracetamol, and other nonsteroidal anti-inflammatory drug [NSAID]) may be permitted at the discretion of the Investigator.
    18. Inability or unwillingness to undergo repeated visits and venepunctures (e.g., due to poor tolerability or lack of access to veins).
    19. Participant is unwilling to refrain from strenuous exercise from 2 days prior to Baseline until the Follow-up Visit.
    20. Pregnant or lactating at Screening or planning to become pregnant (self or partner) at any time during the study.
    E.5 End points
    E.5.1Primary end point(s)
    Safety and tolerability:
    •Vital signs
    •12-lead ECG
    •Clinical laboratory tests
    •Physical examination
    •AEs
    Efficacy endpoints:
    •Number of participants with no disease at Week 12 during individual IZD174 treatment (pooled).
    No disease is complete clinical remission, defined as complete clinical response with normal CRP or SAA levels.
    Complete clinical response is defined as Investigator global assessment of IGA score specific at the time of consultation of < 3 and IGA general assessment of absent or minimal
    E.5.1.1Timepoint(s) of evaluation of this end point
    At different time points throughout the study; refer to schedule of assessments within the protocol
    E.5.2Secondary end point(s)
    •Plasma PK: plasma IZD174 concentrations
    •PD: NLRP3 inhibition in ex vivo stimulated whole blood as measured by IL-1β secretion
    •CRP (as part of safety assessments)
    •SAA
    •Efficacy endpoints:
    - Number of participants with no disease at Week 12 for participants receiving single daily dose of IZD174 and participants receiving twice daily dose of IZD174
    - Number of participants with partially active disease at Week 12 for participants receiving single daily dose of IZD174 and participants receiving twice daily dose of IZD174. Partially active disease is incomplete clinical remission, defined as incomplete clinical response with CRP or SAA levels improved by > 50% over Baseline, but still elevated. Incomplete clinical response is defined as IGA score specific at the time of consultation of  3 and > 50% improvement over Baseline and IGA general assessment improvement by at least one category (e.g., moderate to mild)
    - Number of participants with active disease at Week 12 for participants receiving single daily dose of IZD174 and participants receiving twice daily dose of IZD174
    Active disease is no clinical remission, defined as no clinical response with CRP or SAA elevated. No clinical response is defined as no improvement or worsening of the clinical symptoms and IGA general assessment unchanged or worsened.
    - Duration of clinical remission
    Time from onset of remission until loss of complete clinical remission. Loss of Complete clinical remission is defined as: IGA score specific at the time of consultation > 3 and IGA general assessment worse than minimal with CRP or SAA > ULN or IGA score specific at the time of consultation > 3 and IGA general assessment worse than minimal or CRP or SAA > ULN (with no evidence of other cause of CRP/SAA rise such as infection or trauma)
    - Time to flare
    The time from stopping of study drug until flare. For the purpose of this endpoint, flare is defined as loss of complete clinical remission with CRP or SAA levels to be rising on 2 successive samples at the stopping of study drug.
    E.5.2.1Timepoint(s) of evaluation of this end point
    At different time points throughout the study; refer to schedule of assessments within the protocol
    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 Yes
    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
    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) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised Yes
    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
    Dose-escalating
    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 trial2
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA2
    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
    Ireland
    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
    Last visit of the last subject
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    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 years1
    E.8.9.2In all countries concerned by the trial months0
    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: 10
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 4
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 10
    F.4.2.2In the whole clinical trial 14
    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)
    Not planned yet.
    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 Decision2020-05-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-09-22
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2020-10-13
    For support, visit the EMA Service Desk , log in using your EMA account and open a ticket specifying "EU CTR" in your request.
    If you do not have an account, please visit the EMA Account management page page click on "Create an EMA account" and follow the instructions.
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
    European Medicines Agency © 1995-2021 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    Legal notice
    EMA HMA