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    Summary
    EudraCT Number:2020-003087-45
    Sponsor's Protocol Code Number:TheMYLOX-1study
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-05-24
    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 number2020-003087-45
    A.3Full title of the trial
    An open-label, phase IIa study of the safety, tolerability, pharmacokinetics and pharmacodynamics of oral GB2064 (a LOXL2 inhibitor) in participants with myelofibrosis (The MYLOX-1 study).
    Studio in aperto, di fase IIa, sulla sicurezza, tollerabilità, farmacocinetica e farmacodinamica di GB2064 (un inibitore LOXL2) per via orale in partecipanti con mielofibrosi (Studio MYLOX-1).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of the safety, tolerability, pharmacokinetics and pharmacodynamics of oral GB2064 in participants with myelofibrosis.
    Studio della sicurezza, tollerabilità, farmacocinetica e farmacodinamica di GB2064 per somministrazione orale in partecipanti con mielofibrosi.
    A.3.2Name or abbreviated title of the trial where available
    The MYLOX-1 study
    Studio MYLOX-1
    A.4.1Sponsor's protocol code numberTheMYLOX-1study
    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 SponsorGalecto Biotech AB
    B.1.3.4CountryDenmark
    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 supportGalecto Biotech AB
    B.4.2CountryDenmark
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationOPIS s.r.l.
    B.5.2Functional name of contact pointDipartimento Medico
    B.5.3 Address:
    B.5.3.1Street AddressPalazzo Aliprandi - Via Matteotti, 10
    B.5.3.2Town/ cityDesio (MB)
    B.5.3.3Post code20832
    B.5.3.4CountryItaly
    B.5.4Telephone number003903626331
    B.5.5Fax number00390362633633
    B.5.6E-mailinfo.studiclinici@opis.it
    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 nameGB2064
    D.3.2Product code [PAT-1251]
    D.3.4Pharmaceutical form Tablet
    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 INNGB2064
    D.3.9.1CAS number 2205037-03-0
    D.3.9.2Current sponsor codeGB2064
    D.3.9.3Other descriptive namePAT-1251, PTC1568
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Primary (PMF) or secondary (SMF) myelofibrosis.
    Mielofibrosi primaria (PMF) o secondaria (SMF).
    E.1.1.1Medical condition in easily understood language
    Primary (PMF) or secondary (SMF) myelofibrosis.
    Mielofibrosi primaria (PMF) o secondaria (SMF).
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10077161
    E.1.2Term Primary myelofibrosis
    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 10074691
    E.1.2Term Post polycythaemia vera myelofibrosis
    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 21.0
    E.1.2Level LLT
    E.1.2Classification code 10074690
    E.1.2Term Post essential thrombocythemia myelofibrosis
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the safety, tolerability, pharmacokinetics, pharmacodynamics and clinical effects of 1000 mg BID GB2064 in participants with PMF or SMF.
    Valutare la sicurezza, tollerabilità, farmacocinetica, farmacodinamica ed effetti clinici di 1000 mg BID di GB2064 in partecipanti affetti da PMF o SMF.
    E.2.2Secondary objectives of the trial
    - To assess the pharmacokinetics (PK) in blood of GB2064 in participants with PMF or SMF;
    - To assess the effect of GB2064 on clinical parameters of disease activity in participants with PMF or SMF;
    - To assess the effect of GB2064 on MFrelated symptoms and quality of life (QoL);
    - To assess the effect of GB2064 on the bone marrow.
    - Valutare la farmacocinetica (PK) nel sangue di GB2064 in partecipanti con PMF o SMF;
    - Valutare l'effetto di GB2064 sui parametri clinici dell'attività della malattia in partecipanti con PMF o SMF;
    - Valutare l'effetto di GB2064 sui sintomi correlati alla MF e sulla qualità della vita (QoL);
    - Valutare l'effetto di GB2064 sul midollo osseo.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    The main inclusion criteria include a diagnosis of PMF or SMF, Eastern Cooperative Oncology Group (ECOG) performance status 0-2, and clinical laboratory parameters within appropriate limits for participants with MF.
    I principali criteri di inclusione comprendono una diagnosi di PMF o SMF, performance status ECOG (Eastern Cooperative Oncology Group) 0-2, e parametri di laboratorio clinico entro limiti appropriati per partecipanti con MF.
    E.4Principal exclusion criteria
    Participants are excluded if they are taking or have taken a JAK inhibitor (ruxolitinib or fedratinib) within two weeks of enrolment or chronic (> 14 days) treatment with corticosteroids at a dose more than 10mg of prednisone (or its glucocorticoid equivalent) per day.
    I partecipanti sono esclusi se assumono o hanno assunto un inibitore JAK (ruxolitinib o fedratinib) entro le due settimane precedenti l’arruolamento o trattamento cronico (> 14 giorni) con corticosteroidi ad un dosaggio superiore a 10 mg di prednisone (o il suo glucocorticoide equivalente) al giorno.
    E.5 End points
    E.5.1Primary end point(s)
    Adverse events (AE), serious adverse events (SAE), clinical laboratory assessments, vital signs, ECG, physical examination, body weight.
    Eventi avversi (Adverse Events - AE), eventi avversi seri (Serious Adverse Events - SAE), valutazioni di laboratorio clinico, segni vitali, ECG, esame obiettivo, peso corporeo.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At screening and during the study.
    Allo screening e durante lo studio.
    E.5.2Secondary end point(s)
    1. Pre-dose and post-dose plasma levels of GB2064
    2. Clinical parameters including, but not limited to changes in anaemia, platelets and transfusion dependence, splenic volume.
    3. Clinical Benefit Rate (CBR) and Overall Survival (OS) (Tefferi et al. 2013)
    4. Patient-reported assessments of symptoms and quality of life, as measured by MPN10 and EQ-5D-5L
    5. Histopathological examination of bone marrow biopsy tissue including the extent of fibrosis
    1. Livelli plasmatici pre-dose e post-dose di GB2064
    2. Parametri clinici compresi, a titolo esemplificativo ma non esaustivo, variazioni dell’anemia, delle piastrine e della dipendenza da trasfusione, del volume splenico
    3. Tasso di beneficio clinico (Clinical Benefit Rate - CBR) e sopravvivenza globale (Overall Survival - OS) (Tefferi et al. 2013)
    4. Valutazioni riportate dai pazienti di sintomi e qualità della vita, misurati tramite MPN10 e EQ-5D-5L
    5. Esame istopatologico del tessuto ottenuto dalla biopsia del midollo osseo, compresa l’estensione della fibrosi
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Each month during the study.
    2. At screening and during the study.
    3. During the study.
    4. On Day 1 and Month, 3, 6 and 9.
    5. On Day 1 and Month, 3, 6 and 9.
    1. Ad ogni mese durante lo studio.
    2. Allo screening e durante lo studio.
    3. Durante lo studio.
    4. Al Giorno 1 e al Mese, 3, 6 e 9.
    5. Al Giorno 1 e al Mese, 3, 6 e 9.
    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 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) 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 No
    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 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 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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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
    United States
    Germany
    Italy
    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 years2
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months2
    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: 16
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 8
    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 12
    F.4.2.2In the whole clinical trial 24
    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)
    Participants who derive benefit may continue therapy for up to 3 years.
    I partecipanti che trarranno beneficio dal trattamento potranno continuare la terapia fino a 3 anni.
    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 Decision2021-02-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-03-12
    P. End of Trial
    P.End of Trial StatusOngoing
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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