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    Summary
    EudraCT Number:2016-000468-41
    Sponsor's Protocol Code Number:CCDZ173X2201E1
    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:2021-09-02
    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 number2016-000468-41
    A.3Full title of the trial
    An open-label, non-randomized extension study to evaluate the long term safety, tolerability, efficacy and pharmacokinetics of CDZ173 (leniolisib) in patients with APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/p110d-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency)
    Studio di estensione in aperto, non randomizzato, per valutare sicurezza, tollerabilità, efficacia e farmacocinetica di CDZ173 (leniolisib) a lungo termine in pazienti con APDS/PASLI (Sindrome da attivazione di PIK3- delta/immunodeficienza, linfadenopatia e cellule T senescenti da mutazione attivante p110-delta)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to assess long-term safety and efficacy of CDZ173 in patients with APDS/PASLI
    Studio per valutare la sicurezza e l'efficacia a lungo termine di CDZ173 nei pazienti con APDS/PASLI
    A.3.2Name or abbreviated title of the trial where available
    Extension to the study of efficacy of CDZ173 in patients with APDS/PASLI
    Estensione allo studio dell'efficacia del CDZ173 nei pazienti con APDS/PASLI
    A.4.1Sponsor's protocol code numberCCDZ173X2201E1
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberISRCTN12345678
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT12345678
    A.5.3WHO Universal Trial Reference Number (UTRN)U1234-1234-1234
    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 SponsorNOVARTIS PHARMA SERVICES AG
    B.1.3.4CountrySwitzerland
    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 supportNovartis Pharma Services AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Farma
    B.5.2Functional name of contact pointRegulatory Affairs
    B.5.3 Address:
    B.5.3.1Street AddressLargo Umberto Boccioni, 1
    B.5.3.2Town/ cityOriggio (VA)
    B.5.3.3Post code21040
    B.5.3.4CountryItaly
    B.5.4Telephone number0296541
    B.5.5Fax number029659066
    B.5.6E-mailinfo.studiclinici@novartis.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 nameLeniolisib
    D.3.2Product code CDZ173
    D.3.4Pharmaceutical form Capsule, hard
    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 INNleniolisib
    D.3.9.2Current sponsor codeCDZ173
    D.3.9.4EV Substance CodeSUB168935
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number70
    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.IMP: 2
    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 nameLeniolisib
    D.3.2Product code [CDZ173]
    D.3.4Pharmaceutical form 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 INNleniolisib
    D.3.9.2Current sponsor codeCDZ173
    D.3.9.4EV Substance CodeSUB168935
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number70
    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
    APDS/PASLI (Activated phosphoinositide 3-kinase delta syndrome/p110d-activating mutation causing senescent T cells, lymphadenopathy and immunodeficiency)
    APDS/PASLI - Sindrome da attivazione di PIK3-delta/immunodeficienza, linfadenopatia e cellule T senescenti da mutazione attivante p110-delta
    E.1.1.1Medical condition in easily understood language
    Gain of function genetic mutation in the PI3Kdelta gene causing immunodeficiency
    Sindrome da attivazione di PIK3-delta/immunodeficienza, linfadenopatia e cellule T senescenti da mutazione attivante p110-delta
    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 10064859
    E.1.2Term Primary immunodeficiency syndrome
    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 long term safety and tolerability of CDZ173 in patients with APDS/PASLI.
    Valutare sicurezza e tollerabilità di CDZ173 a lungo termine su pazienti con APDS/PASLI.
    E.2.2Secondary objectives of the trial
    - To evaluate the long term efficacy of CDZ173 to modify healthrelated quality of life in patients with APDS/PASLI.
    - To evaluate the long term efficacy of CDZ173 by means of biomarkers reflecting the efficacy of CDZ173 to reduce systemic inflammatory components of the disease in patients with APDS/PASLI.
    - To characterize the pharmacokinetics (trough concentrations) of CDZ173 in patients with APDS/PASLI.
    - To evaluate the pharmacokinetics and relative bioavailability of CDZ173 film-coated tablets compared to CDZ173 hard-gelatin capsules.
    - Valutare l’efficacia a lungo termine di CDZ173 nel modificare la qualità della vita in relazione alla salute nei pazienti con APDS/PASLI.
    - Valutare l’efficacia a lungo termine di CDZ173 attraverso la valutazione di biomarcatori che riflettono l’efficacia di CDZ173 nel ridurre le componenti dell’infiammazione sistemica su pazienti con APDS/PASLI.
    - Caratterizzare la farmacocinetica (livelli pre dose) di CDZ173 su pazienti con APDS/PASLI.
    - Valutare la farmacocinetica e la biodisponibilità relativa del CDZ173 in forma di compresse rivestite da film rispetto al CDZ173 in capsule di gelatina dura
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Patients must have participated in study CCDZ173X2201 or were treated previously with PI3Kd inhibitors other than CDZ173.
    - Patients who are deemed by the Investigator to benefit from PI3Kd inhibitor therapy.
    - Patients or their legal representatives (for patients under the age of 18 years) must be able to communicate well with the Investigator, to understand and comply with the requirements of the study.
    - Documented APDS/PASLI-associated genetic PI3K delta mutation. Patients with mutations in either PIK3CD or PIK3R1 can be included.
    - I pazienti devono aver partecipato allo studio CCDZ173X2201 o aver ricevuto precedente terapia con altri inibitori PI3K-delta, diversi dal CDZ173.
    - Pazienti che secondo lo sperimentatore possono trarre beneficio dalla terapia con inibitori del PI3K-delta.
    - Pazienti o loro rappresentanti legali (per pazienti con meno di 18 anni) devono essere in grado di comunicare con lo sperimentatore in modo da capire e rispettare i requisiti dello studio.
    - Diagnosi documentata di APDS/PASLI associata alla mutazione genetica PI3K delta. Possono essere inclusi pazienti con le mutazioniPIK3CD o PIK3R1.
    E.4Principal exclusion criteria
    - Patients who withdrew consent from the study CCDZ173X2201.
    - Use of other investigational drugs, except CDZ173, within 5 half-lives of enrollment, or within 30 days, whichever is longer.
    - Previous or concurrent use of immunosuppressive medication
    - Administration of any live vaccines (including any attenuated live vaccines) starting from 6 weeks before study entry, during the study and up to 7 days
    after the last dose of CDZ173 should be excluded.
    - Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation.
    - Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective
    methods of contraception during dosing and for 2 days after last dose ofstudy medication.
    - Uncontrolled chronic or recurrent infectious disease (with the exception of those that are considered to be characteristic of APDS/PASLI).
    - Pazienti che hanno ritirato il consenso dello studio CCDZ173X2201.
    - Uso di farmaci sperimentali, eccetto CDZ173, entro il periodo definito come 5 volte il tempo di dimezzamento dall’arruolamento o nei 30 giornì precedenti l’arruolamento, qualunque delle due situazioni si verifichi per prima.
    - Uso precedente o concomitante di farmaci immunosoppressori.
    - Dovrebbero essere esclusi pazienti che hanno assunto qualsiasi vaccino vivo (compresi vaccini vivi attenuati) nelle 6 settimane precedenti l’arruolamento. Durante lo studio e fino a 7 giorni dopo l’ultima dose di CDZ173 è vietata la somministrazione di qualsiasi vaccino vivo.
    - Donne in gravidanza o allattamento, con la gravidanza definita come lo stato di una donna dopo il concepimento e fino al termine della gestazione, confermato da un test di laboratorio positivo per hCG.
    - Donne potenzialmente fertili, definite come tutte le donne fisiologicamente in grado di iniziare una gravidanza, a meno che non utilizzino metodi contraccettivi di efficacia elevata (ad esclusione di contraccettivi ormonali orali, cerotti, impianti sottocutanei o iniettabili) durante la somministrazione del trattamento in studio e per i 2 giorni successivi all'interruzione del trattamento.
    - Pazienti con infezioni croniche o ricorrenti non controllate ( con eccezione di quelle che sono considerate essere caratteristiche della patologia APDS/PASLI).
    E.5 End points
    E.5.1Primary end point(s)
    All safety parameters (including AEs, physical exam, vital signs, ECG, safety laboratory (hematology, blood chemistry, urinalysis))
    Tutti i parametri di sicurezza (AEs, visita medica, segni vitali, ECG, esami di sicurezza di laboratorio (ematologia, chimica clinica, ecc).
    E.5.1.1Timepoint(s) of evaluation of this end point
    5 years and 3 months
    5 anni e 3 mesi
    E.5.2Secondary end point(s)
    - SF-36 (Short Form 36) Survey and WPAI-CIQ (Work Productivity Activity Impairment plus Classroom Impairment Questionnaire), Visual analogue scales for Physician's Global Assessment (PGA) and Patient's Global Assessment (PtGA), patient narratives by Investigator
    - High sensitivity C-reactive protein (CRP), lactate dehydrogenase (LDH), frequencies of infections and other disease complications.
    - Steady-state trough concentration of CDZ173.
    - PK parameters (including but not limited to AUC0-12,ss and Cmax,ss)
    - SF-36 (Short Form 36) Survey e WPAI-CIQ (Work Productivity Activity Impairment plus Classroom Impairment Questionnaire), scale visive analogiche per Physician's Global Assessment (PGA) e Patient's Global Assessment (PtGA), racconti dei pazienti per Investigatore
    - Proteina C reattiva (CRP) ad alta sensibilità, lattato deidrogenasi (LDH), frequenze di infezioni e altre complicanze della malattia.
    - Concentrazione minima allo stato stazionario di CDZ173.
    - Parametri PK (inclusi ma non limitati a AUC0-12, ss e Cmax, ss)
    E.5.2.1Timepoint(s) of evaluation of this end point
    5 years and 3 months
    5 anni e 3 mesi
    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 Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    Tollerabilità
    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 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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA8
    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
    Belarus
    Russian Federation
    United States
    Ireland
    Italy
    Netherlands
    United Kingdom
    Czechia
    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 years2
    E.8.9.1In the Member State concerned months7
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months8
    E.8.9.2In all countries concerned by the trial days29
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 15
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 26
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 19
    F.4.2.2In the whole clinical trial 42
    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 Decision2018-06-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-02-20
    P. End of Trial
    P.End of Trial StatusOngoing
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