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    Summary
    EudraCT Number:2021-001010-10
    Sponsor's Protocol Code Number:CGT9486-20-201
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-09-23
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2021-001010-10
    A.3Full title of the trial
    A Phase 2 Open-Label, Multicenter Clinical Study of the Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Profiles of CGT9486 as a Single Agent in Patients With Advanced Systemic Mastocytosis
    Een fase 2 open-label, multicenter klinische studie van de veiligheids-, werkzaamheids-, farmacokinetische en farmacodynamische profielen van CGT9486 als enkelvoudig middel bij patiënten met gevorderde systemische mastocytose
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of CGT9486 in Patients with Advanced Systemic Mastocytosis
    Een onderzoek naar CGT9486 bij patiënten met geavanceerde systemische mastocytose
    A.4.1Sponsor's protocol code numberCGT9486-20-201
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04996875
    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 SponsorCogent Biosciences, 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 supportCogent Biosciences, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCogent Biosciences, Inc
    B.5.2Functional name of contact pointRegulatory Affairs
    B.5.3 Address:
    B.5.3.1Street Address275 Wyman Street, 3rd Floor
    B.5.3.2Town/ cityWaltham, MA
    B.5.3.3Post code02451
    B.5.3.4CountryUnited States
    B.5.4Telephone number+16172297380
    B.5.6E-mailsabrina.khalil@cogentbio.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 nameBezuclastinib
    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 INNBezuclastinib
    D.3.9.1CAS number 1616385-51-3
    D.3.9.2Current sponsor codeCGT9486
    D.3.9.3Other descriptive namePLX9486
    D.3.9.4EV Substance CodeSUB197172
    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.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
    Advanced Systemic Mastocytosis (AdvSM)
    Geavanceerde systemische mastocytose
    E.1.1.1Medical condition in easily understood language
    Mastocytosis
    Mastocytose
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 26.1
    E.1.2Level LLT
    E.1.2Classification code 10056453
    E.1.2Term Aggressive systemic mastocytosis
    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
    Part 1: Dose Optimization
    - To identify a clinically active and tolerable systemic exposure range of bezuclastinib in subjects with AdvSM
    Part 2 Stage 1: Dose Confirmation
    - To confirm the optimal dose of bezuclastinib in subjects with AdvSM
    Part 2 Stage 2: Expansion
    - To determine the efficacy of bezuclastinib at the selected optimal dose in subjects with AdvSM
    Deel 1: Dosisoptimalisatie
    - Een klinisch actieve en verdraagbare systemische blootstellingbereik van bezuclastinib vaststellen bij proefpersonen met AdvSM
    Deel 2, fase 1: Dosisbevestiging
    - Bevestigen van de optimale dosis bezuclastinib bij proefpersonen met AdvSM
    Deel 2, fase 2: Uitbreiding
    - Het vaststellen van de werkzaamheid van bezuclastinib in de geselecteerde optimale dosis bij proefpersonen met AdvSM die evalueerbaar zijn op basis van de gemodificeerde IWGMRT-ECNM-respons criteria
    E.2.2Secondary objectives of the trial
    Part 1 and Part 2 :
    - To characterize the safety and tolerability of bezuclastinib in subjects with AdvSM
    - To evaluate additional efficacy parameters with bezuclastinib in subejcts with AdvSM
    - To determine the effects of bezuclastinib on serum tryptase.
    - To determine the effects of bezuclastinib on KIT D816V mutation allele burden.
    - To evaluate histopathologic response in the blood and bone marrow
    - To assess the PK of bezuclastinib in patients with AdvSM.
    - To assess patient-reported outcomes in subjects with AdvSM
    - To explore the effect of bezuclastinib in subjects with AdvSM who are nonevaluable based on the modified IWG-MRT-ECNM response criteria.
    Het karakteriseren van de veiligheid en verdraagbaarheid van bezuclastinib bij proefpersonen met AdvSM
    Het evalueren van aanvullende werkzaamheidsparameters van bezuclastinib bij proefpersonen met AdvSM
    Het beoordelen van de effecten van bezuclastinib op serumtryptase
    Het vaststellen van de effecten van bezuclastinib op KIT D816V mutatie allelbelasting
    Het evalueren van histopathologische respons in bloed en beenmerg
    Het beoordelen van de PK van bezuclastinib bij proefpersonen met AdvSM
    Het beoordelen van de door de patiënt gerapporteerde resultaten bij proefpersonen met AdvSM
    Het verkennen van het effect van bezuclastinib bij proefpersonen met AdvSM die niet evalueerbaar zijn op basis van de aangepaste IWG- MRT-ECNM-responscriteria
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Diagnosed with 1 of the following advanced mastocytosis diagnoses by Eligibility Committee:
    a. Aggressive Systemic Mastocytosis (ASM)
    b. Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN)
    c. Mast Cell Leukemia (MCL)
    2. Measurable disease according to modified IWG-MRT-ECNM criteria
    3. ECOG (0 to 3)
    4. Have clinically acceptable local laboratory screening results (clinical chemistry, hematology) within certain limits
    1. Door de geschiktheidscommissie gediagnosticeerd met 1 van de volgende diagnoses van gevorderde mastocytose:
    a. Agressieve systemische mastocytose (ASM)
    b. Systemische mastocytose met een geassocieerd hematologisch neoplasma (SM-AHN)
    c. Mestcelleukemie (MCL)
    2. Meetbare ziekte volgens gewijzigde IWG-MRT-ECNM-criteria
    3. ECOG (0 tot 3)
    4. Heeft klinisch aanvaardbare lokale laboratoriumscreeningsresultaten (klinische chemie, hematologie) binnen bepaalde limieten
    E.4Principal exclusion criteria
    1. Persistent toxicity from previous therapy for Advanced Systemic Mastocytosis that has not resolved to ≤ Grade 1
    2. Associated hematologic neoplasm requiring immediate antineoplastic therapy
    3. Clinically significant cardiac disease
    4. Known positivity for the FIP1L1 PDGFRA fusion (Patients with eosinophilia without detectable KIT D816V mutation must also lack the PDGFRA fusion mutation prior to enrollment)
    5. Seropositive for human immunodeficiency virus (HIV) 1 or 2, positive for hepatitis B surface antigen, or positive for hepatitis C virus (HCV) antibody
    6. History of clinically significant bleeding event within 30 days before the first dose of study drug or need for therapeutic anticoagulation on study
    7. Diagnosed with or treated for malignancy other than the disease under study within the prior 3 years before enrollment
    8. Received any cytoreductive therapy or any investigational agent less than 14 days, and for cladribine, interferon alpha, pegylated interferon, and any antibody therapy less than 28 days, before screening bone marrow biopsy
    9. Received hematopoietic growth factor support within 14 days before the first dose of study drug
    10. Received strong CYP3A4 inhibitors or inducers before the first dose of study drug
    11. Need for treatment with steroids
    1. Aanhoudende toxiciteit van eerdere therapie voor geavanceerde systemische mastocytose die niet is verdwenen tot ≤ graad 1
    2. Geassocieerd hematologisch neoplasma dat onmiddellijke antineoplastische therapie vereist
    3. Klinisch significante hartziekte
    4. Bekende positiviteit voor de FIP1L1 PDGFRA-fusie (patiënten met eosinofilie zonder detecteerbare KIT D816V-mutatie moeten ook de PDGFRA-fusiemutatie missen voorafgaand aan inschrijving)
    5. Seropositief voor humaan immunodeficiëntievirus (HIV) 1 of 2, positief voor hepatitis B-oppervlakteantigeen of positief voor antilichaam tegen hepatitis C-virus (HCV)
    6. Geschiedenis van klinisch significante bloedingen binnen 30 dagen vóór de eerste dosis van het onderzoeksgeneesmiddel of de noodzaak van therapeutische antistolling tijdens het onderzoek
    7. Gediagnosticeerd met of behandeld voor een andere maligniteit dan de ziekte die wordt onderzocht in de voorafgaande 3 jaar vóór inschrijving
    8. Kreeg een cytoreductieve therapie of een onderzoeksmiddel minder dan 14 dagen, en voor cladribine, interferon-alfa, gepegyleerd interferon en elke antilichaamtherapie minder dan 28 dagen, vóór screening van beenmergbiopsie
    9. Kreeg hematopoëtische groeifactorondersteuning binnen 14 dagen vóór de eerste dosis van het onderzoeksgeneesmiddel
    10. Sterke CYP3A4-remmers of -inductoren gekregen vóór de eerste dosis onderzoeksgeneesmiddel
    11. Behoefte aan behandeling met steroïden
    E.5 End points
    E.5.1Primary end point(s)
    Part 1: Dose Optimization
    - Safety assessments and dose modifications;
    - PK and pharmacodynamic assessments;
    - Overall response rate (ORR)
    Part 2 Stage 1: Dose Confirmation
    - Safety assessments and dose modifications
    - PK and pharmacodynamic assessments
    - Overall response rate (ORR)
    Part 2 Stage 2: Expansion
    - ORR
    Deel 1: Dosisoptimalisatie
    -Veiligheidsbeoordelingen en dosisaanpassingen;
    -PK- en PD-beoordelingen;
    -ORR
    Deel 2 Fase 1: Dosisbevestiging
    - Veiligheidsbeoordelingen en dosisaanpassingen
    - PK en farmacodynamische beoordelingen
    - Totale respons (ORR
    Deel 2 Fase 2: Uitbreiding
    -ORR
    E.5.1.1Timepoint(s) of evaluation of this end point
    Defined in the endpoint or according to the time points listed in the Schedule of Assessments and Procedures (Table 1 of the Protocol) and Schedule of Pharmacokinetics, Pharmacodynamics, and Biomarker Assessments (Table 2 of the Protocol)
    Gedefinieerd in het eindpunt of volgens de tijdstippen vermeld in het schema van beoordelingen en procedures (tabel 1 van het protocol) en het schema van farmacokinetiek, farmacodynamiek en biomarkerbeoordelingen (tabel 2 van het protocol)
    E.5.2Secondary end point(s)
    Part 1 (Dose Optimization) and Part 2 (Expansion):
    -Incidence of AEs, SAEs, and AEs leading to dose modifications, and changes from baseline in laboratory results
    -DOR
    -TTR
    -PFS
    -OS
    -PPR
    -Changes in spleen and liver size
    -Changes in serum tryptase levels
    -Changes in levels of KIT D816V mutation allele burden
    -Change in pathologic findings in the blood and bone marrow
    -Plasma concentration of CGT9486
    -PGIC and change in the following patient-reported outcome measures: PGIS, MC-QoL, and MAS
    Deel 1 (Dosisoptimalisatie) en Deel 2 (Uitbreiding):
    - Incidentie van AE's, SAE's en AE's die leiden tot dosisaanpassingen en veranderingen ten opzichte van baseline in laboratoriumresultaten
    -DOR
    -TTR
    -PFS
    -OS
    -PPR
    -Veranderingen in milt en levergrootte
    -Veranderingen in serumtryptasespiegels
    -Veranderingen in niveaus van KIT D816V-mutatie-allelbelasting
    - Verandering in pathologische bevindingen in het bloed en beenmerg
    -Plasmaconcentratie van CGT9486
    -PGIC en verandering in de volgende door de patiënt gerapporteerde uitkomstmaten: PGIS, MC-QoL en MAS
    E.5.2.1Timepoint(s) of evaluation of this end point
    Defined in the endpoint or according to the time points listed in the Schedule of Assessments and Procedures (Table 1 of the Protocol) and Schedule of Pharmacokinetics, Pharmacodynamics, and Biomarker Assessments (Table 2 of the Protocol)
    Gedefinieerd in het eindpunt of volgens de tijdstippen vermeld in het schema van beoordelingen en procedures (tabel 1 van het protocol) en het schema van farmacokinetiek, farmacodynamiek en biomarkerbeoordelingen (tabel 2 van het 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 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 Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Tolerability of bezuclastinib in patients with AdvSM
    Verdraagbaarheid van bezuclastinib bij patiënten met AdvSM
    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 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
    Parallel groepsontwerp in deel 1 van de studie (4 cohorten: verschillende doses van hetzelfde medici
    Parallel group design in Part 1 of the study (4 cohorts: different doses of the same drug)
    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.8.3 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 EEA24
    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
    Switzerland
    Australia
    Canada
    Israel
    United Kingdom
    United States
    Austria
    Belgium
    France
    Germany
    Italy
    Netherlands
    Norway
    Poland
    Spain
    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 study will continue until all subjects have completed at least 2 years of follow up for overall survival, withdrawn from study participation, been lost to follow-up, or died, whichever occurs first
    De studie zal doorgaan totdat alle proefpersonen ten minste 2 jaar follow-up hebben voltooid voor algehele overleving, zich hebben teruggetrokken uit de studiedeelname, verloren zijn gegaan voor follow-up of zijn overleden, afhankelijk van wat zich het eerst voordoet
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years7
    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 years7
    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: 60
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 60
    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 60
    F.4.2.2In the whole clinical trial 140
    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
    Geen
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-10-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-03-29
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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