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    Summary
    EudraCT Number:2021-004732-29
    Sponsor's Protocol Code Number:PTG-300-11
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-05-10
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2021-004732-29
    A.3Full title of the trial
    A Phase 3 Study of the Hepcidin Mimetic Rusfertide (PTG-300) in Patients with Polycythemia Vera
    Studio di fase 3 per valutare il mimetico dell’epcidina rusfertide (PTG-300) in pazienti affetti da policitemia vera
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3 Study of Rusfertide (PTG-300) in Patients with Polycythemia Vera
    Studio di fase 3 di Rusfertide (PTG-300) in pazienti affetti da policitemia vera
    A.3.2Name or abbreviated title of the trial where available
    NA
    NA
    A.4.1Sponsor's protocol code numberPTG-300-11
    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 SponsorProtagonist Therapeutics, Inc.
    B.1.3.4CountryUnited States
    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 supportProtagonist Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMedpace Finland OY
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street AddressKarjalankatu 2, 4 Krs
    B.5.3.2Town/ cityHelsinki
    B.5.3.3Post code00520
    B.5.3.4CountryFinland
    B.5.4Telephone number46702524055
    B.5.5Fax number00000000
    B.5.6E-mailregsubmissions@medpace.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/20/2330
    D.3 Description of the IMP
    D.3.1Product nameRusfertide
    D.3.2Product code [PTG-300]
    D.3.4Pharmaceutical form Powder and solvent 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 INNRusfertide
    D.3.9.1CAS number 1628323-80-7
    D.3.9.2Current sponsor codePTG-300
    D.3.9.3Other descriptive name1-(3-METHYLBUTANOYL)-L-ASPARTYL-L-THREONYL-L-HISTIDYL-L-PHENYLALANYL-LPROLYL-(L-CYSTINYL-L-ISOLEUCYL-[(N6-(S)-4-CARBOXY-4- PALMITAMIDOBUTANOYL)-L-LYSINYL]-L-PHENYLALANYL-L-GLUTAMYL-L-PROLYL-L ARGINYL-L-SERINYL-L-LYSINYL-L-GLYCINYL-L-CYSTINYL)-L-LYSINAMIDE, DISULFIDE, ACETATE
    D.3.9.4EV Substance CodeSUB194130
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number10 to 60
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 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
    Polycythemia Vera
    Policitemia Vera
    E.1.1.1Medical condition in easily understood language
    A type of blood cancer
    Tumore del sangue
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10036057
    E.1.2Term Polycythaemia vera
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10036061
    E.1.2Term Polycythemia vera
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    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 and efficacy of rusfertide in subjects with polycythemia vera in maintaining hematocrit control.
    Valutare la sicurezza e l'efficacia di Rusfertide in soggetti affetti da policitemia vera con ematocrito di controllo
    E.2.2Secondary objectives of the trial
    NA
    NA
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Male and female subjects aged 18 (or the minimum country specific age of consent if >18) years or older.
    -Meet revised 2016 World Health Organization (WHO) criteria for the diagnosis of polycythemia vera and have either JAK2 V617F mutation or JAK2 exon 12 mutation
    -Phlebotomy requiring defined as: At least 3 phlebotomies due to inadequate hematocrit control in 6 months before randomization or at least 5 phlebotomies due to inadequate hematocrit control in 1 year before randomization, and
    -CBC values immediately prior to randomization:
    a. Hematocrit <45%,
    b. WBC 4000/µL to 20,000/µL (inclusive), and
    c. Platelets 100,000/µL to 1,000,000/µL (inclusive).
    -Subjects receiving cytoreductive therapy at randomization must be on a stable PV therapy regimen
    -Subjects treated with phlebotomy alone at randomization must havestopped cytoreductive therapy 2 to 6 months before screening.
    -Soggetti maschi e femmine di età pari o superiore a 18 anni (o il minimo specifico per paese età del consenso se >18) anni o più.
    -Soddisfare i criteri dell'Organizzazione Mondiale della Sanità (OMS) rivisti del 2016 per la diagnosi di policitemia vera: mutazione JAK2 V617F o Mutazione dell'esone 12 di JAK2
    -Requisito di flebotomia definito come: Almeno 3 flebotomie dovute a un inadeguato controllo dell'ematocrito 6 mesi prima della randomizzazione o almeno 5 flebotomie dovute a un ineguato controllo dell'ematocrito 1 anno prima della randomizzazione
    -Valori CBC immediatamente prima della randomizzazione:
    a.ematocrito <45%,
    b.WBC da 4000/µL a 20.000/µL (incluso)
    c.Piastrine da 100.000/µL a 1.000.000/µL (incluse).
    -I soggetti che ricevono la terapia citoriduttiva alla randomizzazione devono essere in un regime di terapia PV stabile
    -I soggetti trattati con la sola flebotomia alla randomizzazione devono avere interrotto la terapia citoriduttiva da 2 a 6 mesi prima dello screening.
    E.4Principal exclusion criteria
    -Clinically meaningful laboratory abnormalities at Screening
    -Subjects who require phlebotomy at hematocrit levels lower than 45%.
    -Clinically significant thrombosis (e.g., deep vein thrombosis or splenic vein thrombosis) within 2 months prior to randomization.
    -Active or chronic bleeding within 2 months prior to randomization.
    -History of invasive malignancies within the last 5 years, except localized cured prostate cancer and cervical cancer.
    -Subjects with non-invasive non-melanomatous (e.g., squamous cell or basal cell carcinoma) skin cancer during screening unless adequately treated before randomization.
    -Received busulfan, pipobroman or 32Phosphorus within 7 months prior to screening.
    -Anomalie di laboratorio clinicamente significative allo screening
    -Soggetti che necessitano di flebotomia a livelli di ematocrito inferiori a 45%.
    -Trombosi clinicamente significativa (ad es. trombosi venosa profonda o splenica trombosi venosa) entro 2 mesi prima della randomizzazione.
    -Sanguinamento attivo o cronico entro 2 mesi prima della randomizzazione.
    -neoplasie maligne invasive negli ultimi 5 anni, eccetto carcinoma prostatico curato localizzato e carcinoma cervicale.
    -Soggetti con non melanomatosi non invasivi (es. cellule squamose o carcinoma basocellulare) cancro della pelle durante lo screening se non adeguatamente trattati prima della randomizzazione.
    -Ricevuto busulfano, pipobroman o 32fosforo entro 7 mesi prima dello screening.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of subjects achieving a response starting at Week 20 through Week 32 (inclusive) who receive rusfertide compared to placebo. A response is defined as absence of phlebotomy eligibility. Phlebotomy eligibility is defined as either:
    • a confirmed hematocrit =45% and that is at least 3% higher than the
    baseline hematocrit (value immediately prior to randomization at Week
    0); or
    • a hematocrit =48%.
    Percentuale di soggetti che ottengono una risposta dalla settimana 20 alla settimana 32 (inclusa) che ricevono rusfertide rispetto al placebo. Una risposta è definita come assenza di ammissibilità alla flebotomia. L'idoneità alla flebotomia è definita come:
    • un ematocrito confermato =45% e che sia almeno il 3% superiore all' ematocrito basale (valore immediatamente prima della randomizzazione alla settimana0); o
    • un ematocrito =48%.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 20 through Week 32 (inclusive)
    Dalla settimana 20 alla settimana 32 (incluse)
    E.5.2Secondary end point(s)
    1. Mean number of phlebotomies between Week 0 through Week 32
    (inclusive).
    2. Proportion of subjects with all hematocrit values <45% between
    Week 0 through Week 32 (inclusive).
    3. Mean change from baseline in total fatigue score based on PROMIS®
    Short Form 8a at Week 32.
    4. Mean change from baseline in total score based on MFSAF v4.0 at
    Week 32.
    1. Numero medio di flebotomie tra la settimana 0 e la settimana 32 (compreso).
    2. Proporzione di soggetti con tutti i valori di ematocrito <45% tra la settimana 0 e la settimana 32 (inclusa).
    3. Variazione media rispetto al basale del punteggio di fatica totale basato su PROMIS® Modulo abbreviato 8a alla settimana 32.
    4. Cambiamento medio dal basale nel punteggio totale basato su MFSAF v4.0 alla Settimana 32.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 0 through Week 32 (inclusive)
    Dalla settimana 0 alla settimana 32 (incluse)
    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 Yes
    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.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 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 Yes
    E.8.1.7.1Other trial design description
    Studio in 3 parti. Parte 1a: come sopra. Parti 1b e 2: in aperto; i soggetti riceveranno il trattam
    3-part study. Part 1a: as above. Parts 1b and 2: open-label; subjects receive active treatment.
    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 concerned10
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA55
    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 Information not present in EudraCT
    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
    Canada
    Hong Kong
    Israel
    United States
    Austria
    France
    Poland
    Netherlands
    Spain
    Czechia
    Germany
    Italy
    Belgium
    Hungary
    Turkey
    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
    LVLS
    LVLS
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months5
    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: 200
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 50
    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 state18
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 200
    F.4.2.2In the whole clinical trial 250
    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
    nessuno
    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 Decision2022-08-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-05-31
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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