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    Summary
    EudraCT Number:2021-006906-58
    Sponsor's Protocol Code Number:PHA022121-C303
    National Competent Authority:Hungary - National Institute of Pharmacy
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-05-13
    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 ConcernedHungary - National Institute of Pharmacy
    A.2EudraCT number2021-006906-58
    A.3Full title of the trial
    A Phase II/III, Extension Study of Orally Administered PHA-022121 for Acute Treatment of Angioedema Attacks in Patients with Hereditary Angioedema due to C1-Inhibitor Deficiency (Type I or Type II)
    II/III. fázisú, kiterjesztett vizsgálat az angioödémás rohamok akut kezelésére szájon át alkalmazott PHA-022121 értékelésére C1-inhibitor hiány következtében kialakult örökletes angioödémában (I-es vagy II-es típus) szenvedő betegeknél - RAPIDe-2
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An extension phase 2/3 study to test the safety of long term administration of oral PHA-022121 for acute treatment of angioedema attacks in patients with hereditary angioedema
    Egy 2/3 fázisú kiterjesztett vizsgálat a PHA-022121 hosszú távú orális adagolásának a biztonságosságának tesztelésére, örökletes angioödémában szenvedő betegek, angioödémás rohamainak akut kezelésére.
    A.3.2Name or abbreviated title of the trial where available
    RAPIDe-2
    A.4.1Sponsor's protocol code numberPHA022121-C303
    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 SponsorPharvaris Netherlands BV
    B.1.3.4CountryNetherlands
    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 supportPharvaris Netherlands BV
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPharvaris Netherlands BV
    B.5.2Functional name of contact pointPharvaris Clinical
    B.5.3 Address:
    B.5.3.1Street AddressJ.H. Oortweg 21
    B.5.3.2Town/ cityLeiden
    B.5.3.3Post code2333 CH
    B.5.3.4CountryNetherlands
    B.5.5Fax number +31 (0)712036410
    B.5.6E-mailclinical@pharvaris.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 namePHVS416
    D.3.2Product code PHA-022121
    D.3.4Pharmaceutical form Capsule, soft
    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.3.9.1CAS number 2340111-58-0
    D.3.9.2Current sponsor codePHA-022121
    D.3.9.3Other descriptive namePHA-022121
    D.3.9.4EV Substance CodeSUB206489
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule, soft
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Hereditary angioedema attacks caused by Type 1 and 2 C1-Inhibitor
    Deficiency
    E.1.1.1Medical condition in easily understood language
    Hereditary angioedema (HAE) is a disorder that results in recurrent
    attacks of severe swelling
    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 23.1
    E.1.2Level PT
    E.1.2Classification code 10019860
    E.1.2Term Hereditary angioedema
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10080956
    E.1.2Term Hereditary angioedema type I
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10080957
    E.1.2Term Hereditary angioedema C1 inhibitor deficiency
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 26.0
    E.1.2Level LLT
    E.1.2Classification code 10080960
    E.1.2Term Hereditary angioedema type II
    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 safety of long-term on-demand treatment with PHA-022121 for acute hereditary angioedema (HAE) attacks including laryngeal attacks.
    E.2.2Secondary objectives of the trial
    • To evaluate the efficacy of PHA-022121 in achieving symptom relief in patients with acute HAE attacks including laryngeal attacks,
    • To evaluate the maintenance of PHA-022121 efficacy in achieving symptom relief for any subsequent acute HAE attacks including laryngeal attacks,
    • To evaluate the proportion of PHA-022121-treated attacks requiring a second dose of PHA-022121 to achieve symptom relief.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Provision of written informed consent.
    2. Male or female, aged ≥ 18 years at the time of providing written informed consent.
    3. Patients must have received at least 1 dose of study drug (including the non-attack visit) in Study PHA022121-C201.
    4. Female patients of childbearing potential must agree to the protocol specified pregnancy testing and to be abstinent from heterosexual intercourse or to use an acceptable form of contraception methods from enrollment until 30 days after the last study drug administration. Methods acceptable for this study include male condom with or without spermicide, cervical cap, diaphragm or sponge with spermicide, a combination of male condom with cap, diaphragm or sponge with spermicide (double-barrier methods), progestin-only hormonal methods (oral, injectable, or implantable), intrauterine device (IUD, all types), intrauterine hormone releasing systems (IUS).
    Females of non-childbearing potential, defined as surgically sterile (status post hysterectomy, bilateral oophorectomy, or bilateral tubal ligation) or post-menopausal (defined as no menses for at least 12 months without an alternative medical cause and a follicle-stimulating hormone (FSH) test result indicative of post-menopausal status) do not require contraception during the study. There are no contraceptive requirements for male patients.
    5. In the opinion of the investigator is willing and able to comply with the protocol.
    E.4Principal exclusion criteria
    1. Any female who is pregnant or is breast-feeding.
    2. Presence of any medical condition that could interfere with the assessment of safety or efficacy or negatively affect the patient’s safety, including clinically significant abnormal ECG, most notably a QTcF > 470 ms (for females) or > 450 ms (for males), cardiovascular disease, abnormal liver function and abnormal kidney function.
    3. Any other systemic disease (e.g., gastrointestinal, renal, respiratory, neurological) or significant disease or disorder that would interfere with the patient’s safety or ability to participate in the study.
    4. Use of lanadelumab for long-term HAE prophylactic therapy within 12 weeks prior to study enrollment.
    Patients who have recently used short or long-term HAE prophylaxis or on-demand HAE treatment will not be excluded from the study provided the following washout period is observed (i.e., study screening or enrollment should be delayed allowing for washout):
    • 2-week washout period before enrollment should be respected for patients who have used any C1-INH product, oral kallikrein inhibitors, attenuated androgens, or anti-fibrinolytics for long-term prophylactic HAE therapy.
    • 1-week washout period before enrollment should be respected for patients who have used plasma derived C1-INH concentrates (Berinert, Cinryze, Haegarda) for on-demand treatment or short-term prophylaxis.
    • 24-hour washout period before enrollment should be respected for patients who have used recombinant C1-INH (Ruconest) for on-demand treatment or short-term prophylaxis.
    6. History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse.
    7. Discontinued from Study PHA022121-C201 after enrollment for any study drug-related safety reason.
    8. Participation in any other investigational drug study (except with PHA-022121) currently, within the last 30 days prior to the first PHA-022121 dose or within 5 half-lives of study drug at enrollment, whichever is longer.
    9. Use of concomitant medications that are potent CYP3A4 inhibitors (e.g., clarithromycin, erythromycin, itraconazole, ketoconazole, ritonavir, grapefruit) or potent CYP3A4 inducers (e.g., phenytoin, rifampicin, St. John's Wort).
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoints of the study are
    •TEAEs, treatment-related TEAEs, treatment-emergent serious adverse events (TESAEs), and treatment-related TESAEs
    • Clinical laboratory tests
    • Vital signs
    E.5.1.1Timepoint(s) of evaluation of this end point
    • TEAES will be evaluated when taking place
    • Clinical laboratory tests will be evaluated as follows:
    Part A: at Screening visit and every 3 months
    Part B: when patients transition to Part B, then every 3 months for the first 6 months, and every 6 months thereafter and 14 to 28 days after the last treatment administration
    • Vital signs
    Part A: at Screening visit and every 3 months
    Part B: when patients transition to Part B, then every 3 months for the first 6 months, and every 6 months thereafte
    E.5.2Secondary end point(s)
    •Time to onset of symptom relief (TOSR) defined as ≥ 50% reduction in VAS-3 or VAS-5
    • Time to almost complete (defined as all individual VAS scores are ≤10) or complete (defined as all individual VAS scores = 0) symptom relief (TACSR and TCSR) assessed by VAS-3 or VAS-5
    • Time to symptom improvement based on PGI-S
    • Time to symptom improvement based on PGI-C
    • Change of VAS-3 score and individual VAS score from pre-treatment to 4 h post-treatment for non-laryngeal attacks
    • Change in MSCS score at 4 h post-treatment
    • TOS at 4 h post-treatment
    • Proportion of PHA-022121-treated attacks requiring a second dose of PHA-022121
    • TSQM scores at 48 h post-treatment
    E.5.2.1Timepoint(s) of evaluation of this end point
    -VAS-3 *; VAS-5**, PGI-S and PGI-C: every hour (±30 min) up to 6 h from initiation of PHA-022121 treatment and then at 8 (±1.5 h), 12 h (±2.5 h), 24 h (±9.5 h), and 48 h (±14.5 h) from initiation of treatment
    -MSCS-TOS: at 4 h (±30 min), 12 h (±2.5 h), 24 h (±9.5 h), and 48 h (±14.5 h) from initiation of the PHA-022121 treatment
    -TSQM score: at 48 h (±14.5 h) from initiation of the PHA-022121 treatment

    * Non-laryngeal Attacks only
    ** Laryngeal attacks only
    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 No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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) 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 No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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 Yes
    E.8.2.3.1Comparator description
    Different dosage of the same product
    E.8.2.4Number of treatment arms in the trial3
    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.8.4.1Number 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 EEA13
    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
    Canada
    Israel
    United States
    Bulgaria
    Czechia
    France
    Germany
    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-Study visit is to occur between 14 to 28 days after the last treatment administration and if possible in case of withdrawal from the study.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    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: 54
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 18
    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 Yes
    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 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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 48
    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)
    The study is planned to continue until the availability of commercial supply, or another means of continued treatment can be provided.
    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 Decision2022-07-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-06-03
    P. End of Trial
    P.End of Trial StatusOngoing
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