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    Summary
    EudraCT Number:2022-002959-18
    Sponsor's Protocol Code Number:PRN1008-017/ACT17125
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2023-01-04
    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 number2022-002959-18
    A.3Full title of the trial
    An open label, two-arm, Phase 2a study to evaluate the effect of rilzabrutinib (PRN1008/SAR444671) on safety and disease activity in patients with IgG4-related disease
    Studio di fase 2a, in aperto, a due bracci per valutare l’effetto di rilzabrutinib (PRN1008/SAR444671) sulla sicurezza e sull’attività della malattia in pazienti con malattia IgG4-correlata
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Open label two-arm study to evaluate rilzabrutinib in IgG4-related disease patients
    Studio in aperto a due bracci per valutare rilzabrutinib in pazienti con malattia IgG4-correlata
    A.3.2Name or abbreviated title of the trial where available
    .
    .
    A.4.1Sponsor's protocol code numberPRN1008-017/ACT17125
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04520451
    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 SponsorPrincipia Biopharma 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 supportPrincipia Biopharma Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSANOFI S.r.l.
    B.5.2Functional name of contact pointContact Point
    B.5.3 Address:
    B.5.3.1Street AddressVIALE LUIGI BODIO 37/B
    B.5.3.2Town/ cityMILANO
    B.5.3.3Post code20158
    B.5.3.4CountryItaly
    B.5.4Telephone number800536389
    B.5.5Fax number000000
    B.5.6E-mailinformazioni.medicoscientifiche@sanofi.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 namerilzabrutinib
    D.3.2Product code [SAR444671]
    D.3.4Pharmaceutical form Film-coated 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 INNRilzabrutinib
    D.3.9.2Current sponsor codeSAR444671
    D.3.9.3Other descriptive namePRN1008
    D.3.9.4EV Substance CodeSUB192772
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number400
    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
    IgG4-related disease
    malattia IgG4-correlata
    E.1.1.1Medical condition in easily understood language
    Immunoglobulin G4 related disease
    Malattia correlata all'immunoglobulina G4
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10077271
    E.1.2Term Immunoglobulin G4 related disease
    E.1.2System Organ Class 10028395 - Musculoskeletal and connective tissue 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 and the ability of daily oral administration of rilzabrutinib to maintain glucocorticoid-free remission in participants with IgG4-RD for at least 24 weeks
    Valutare la sicurezza e l’efficacia della somministrazione orale giornaliera di rilzabrutinib nel mantenimento della remissione senza glucocorticoidi in pazienti con malattia IgG4-correlata (IgG4-RD) per almeno 24 settimane
    E.2.2Secondary objectives of the trial
    - To evaluate the effect of rilzabrutinib on IgG4-RD disease activity over time
    - To evaluate the effect of rilzabrutinib on specific protein biomarkers over time
    - Valutare l’effetto di rilzabrutinib sull’attività della malattia IgG4-RD nel tempo
    - Valutare l’effetto di rilzabrutinib su specifici biomarcatori proteici nel tempo
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Be male or female with age >=18 years.
    - Have a clinical diagnosis of IgG4-RD.
    - Be willing to taper off an equivalent prednisone dose of between 20-40 mg/day in 2 weeks.
    - Essere di sesso maschile o femminile con età >=18 anni.
    - Presentare una diagnosi clinica di IgG4-RD.
    - Essere disposti/e a ridurre gradualmente una dose equivalente di prednisone compresa tra 20-40 mg/die in 2 settimane
    E.4Principal exclusion criteria
    - Currently or within 6 months of screening taking rituximab, other B-cell depleting agents, or alkylating agents unless B cell concentrations have been demonstrated by flow cytometry to return to normal values (defined as 5 cells per cubic mm).
    - History of solid organ transplant
    - Positive at Screening for HIV, hepatitis B, hepatitis C, or TB
    - Female patients who are pregnant or nursing.
    - Avere assunto rituximab, altri agenti di deplezione dei linfociti B o agenti alchilanti, attualmente o entro 6 mesi dallo screening, a meno che le concentrazioni dei linfociti B non siano state dimostrate mediante citometria a flusso per tornare ai valori normali (definita come 5 cellule per mm3).
    - Anamnesi di trapianto di organi solidi.
    - Positività allo screening per HIV, epatite B, epatite C, o tubercolosi.
    - Pazienti di sesso femminile in gravidanza o in allattamento.
    E.5 End points
    E.5.1Primary end point(s)
    1. Proportion of participants who are without disease flare for at least 24 consecutive weeks following the first dose of rilzabrutinib. Disease flare is defined as an increase from screening IgG4-RD responder index (RI) >2 or initiation of rescue treatment.
    2. Incidence of SAE, AE leading to discontinuation and possible glucocorticoid-related AE.
    3. Number of participants with Potentially clinically significant abnormalities (PCSAs) for clinical laboratory tests, vital signs and ECG
    1.Percentuale di partecipanti che sono senza riacutizzazione della malattia per almeno 24 settimane consecutive dopo la prima dose di rilzabrutinib. La riacutizzazione della malattia è definita come un aumento di IgG4-RD indice di risposta (RI) > 2 dallo screening o l’inizio del trattamento di salvataggio.
    2.Incidenza di SAE, AE che portano all'interruzione e possibili effetti avversi correlati ai glucocorticoidi.
    3. Numero di partecipanti con anomalie potenzialmente significative dal punto di vista clinico (PCSA), nei parametri vitali e nei risultati degli esami clinici di laboratorio ed ECG.
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. At Week 52
    2, 3. Up to 68 weeks
    1. Alla settimana 52
    2, 3. Fino a 68 settimane
    E.5.2Secondary end point(s)
    1. Percentuale di pazienti con riduzione rispetto al basale del punteggio di attività IgG4-RD RI >=2 punti alla Settimana 12
    2. Percentuale di pazienti con punteggio di attività IgG4-RD RI = 0 alla Settimana 12
    3. Livello e variazione rispetto al basale di ciascuna sottoclasse dei marcatori sierologici
    4. Percentuale di pazienti che raggiunge una riduzione del livello sierico basale di IgG4 del 10% a 12 settimane
    5. Variazione rispetto al basale di IgG4-RD RI nel tempo
    6. Percentuale di pazienti senza riacutizzazioni della malattia tra la Settimana 4 e la Settimana 12 e tra la Settimana 12 e la Settimana 52 (o la fine del periodo di trattamento di mantenimento) tra i/le pazienti che hanno un’estensione del trattamento
    7. Variazione rispetto al basale nel tempo del danno da IgG4-RD, come registrato sulla parte del IgG4-RD RI relativa al danno
    1. Proportion of participants with reduction from baseline IgG4-RD RI activity score >=2 points at Week 12
    2. Proportion of patients with an IgG4-RD RI activity score = 0 at Week 12
    3. Level and change from baseline of each subclass of the serological markers
    4. Proportion of participants achieving reduction in baseline serum IgG4 level of 10% at 12 weeks
    5. Change from baseline in IgG4-RD RI over time
    6. Proportion of participants with no disease flares between Week 4 and Week 12, and between Week 12 and Week 52 (or the end of the treatment extension period) among the participants who have treatment extension
    7. Change from baseline over time in IgG4-RD damage, as recorded on the damage portion of the IgG4-RD RI
    E.5.2.1Timepoint(s) of evaluation of this end point
    1, 2, 3. At Week 52
    4. At Week 12
    5, 7. From baseline to Week 52
    6. Until Week 52
    1, 2, 3. Alla settimana 52
    4. Alla settimana 12
    5, 7. Dal basale alla settimana 52
    6. Fino alla settimana 52
    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 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 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.8.4.1Number 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 EEA3
    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
    Canada
    United States
    France
    Spain
    Italy
    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
    LVLS
    LVLS
    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 months6
    E.8.9.1In the Member State concerned days10
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days27
    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: 25
    F.1.3Elderly (>=65 years) No
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 5
    F.4.2.2In the whole clinical trial 25
    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 Decision2023-03-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-04-05
    P. End of Trial
    P.End of Trial StatusCompleted
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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
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