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    Summary
    EudraCT Number:2014-003929-17
    Sponsor's Protocol Code Number:CPDR001X2101
    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:2020-11-10
    Trial results View 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 number2014-003929-17
    A.3Full title of the trial
    Open label multicenter Phase I/II study of the safety and efficacy of PDR001 administered to patients with advanced malignancies
    -
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An interventional study of PDR001 in patients with advanced malignancies
    -
    A.3.2Name or abbreviated title of the trial where available
    An interventional study of PDR001 in patients with advanced malignancies
    Studio interventistico di PDR001 in pazienti con tumori di stadio avanzato
    A.4.1Sponsor's protocol code numberCPDR001X2101
    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 S.p.A.
    B.5.2Functional name of contact pointDrug Regulatory 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 nameIMP1
    D.3.2Product code PDR001
    D.3.4Pharmaceutical form Powder for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPDR001
    D.3.9.2Current sponsor codePDR001
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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
    Solid tumors
    -
    E.1.1.1Medical condition in easily understood language
    Solid tumors
    -
    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 LLT
    E.1.2Classification code 10065252
    E.1.2Term Solid tumor
    E.1.2System Organ Class 100000004864
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Phase I: To estimate the RP2D and/or the MTD for PDR001
    Phase II: To estimate the anti-tumor activity of PDR001
    -
    E.2.2Secondary objectives of the trial
    1- To characterize the safety and tolerability of PDR001
    2- To characterize the pharmacokinetic profile of PDR001
    3- To further investigate the anti-tumor activity of PDR001
    -
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Written informed consent must be obtained prior to any screening procedures
    2. Patient (male or female) = 18 years of age
    3. Phase I part: Patients with advanced/metastatic solid tumors, with measurable or non-measurable disease as determined by RECIST version
    1.1 (refer to Appendix 1), who have progressed despite standard therapy or are intolerant of standard therapy, or for whom no standard therapy exists.
    4. Phase II part: Patients with advanced/metastatic solid tumors, with at least one measurable lesion as determined by RECIST version 1.1,
    who have progressed following their last prior therapy, and fit into one of the following groups:
    • Group 1a and 1b: NSCLC
    • Patients with NSCLC must have had disease recurrence or progression during or after no more than one prior systemic chemotherapy regimen
    (platinum doublet-based) for advanced or metastatic disease. Prior maintenance therapy is allowed (e.g. pemetrexed, erlotinib,
    bevacizumab).
    • Only patients with EGFR mutation-negative tumor are eligible (defined as negative for exon 19 deletions and for the L858R mutation in EGFR at a minimum; however, if more extensive EGFR mutation testing has been performed, the tumor must not harbor any known activating EGFR mutations in Exons 18-21 in order to be considered EGFR mutationnegative). All patients must be tested for EGFR mutational status and, for ALK translocation status if no mutation is detected in EGFR. Patients with ALK translocation-positive NSCLC must have had disease progression following treatment with a corresponding inhibitor and no more than one systemic chemotherapy regimen (platinum doubletbased), in any sequence.
    • Group 2: Melanoma
    • All patients must have been tested for BRAF mutations. Patients with BRAF V600 mutation positive melanoma must have clinical or radiological evidence of disease progression during or after
    • Treatment with a BRAF inhibitor (alone or in combination with other agents)
    • Group 3: Triple negative breast cancer
    • Group 4: Anaplastic thyroid cancer
    • Patients are not required to have received or progressed on a prior therapy.
    • Patients in this indication must not be at short term risk for life threatening complications (such as airway compromise or bleeding from locoregional or metastatic disease).
    • Chemoradiation and/or surgery should be considered prior to study entry for those patients with locally advanced disease if those therapies
    are considered to be in the best interest of the patient.
    5. ECOG Performance Status = 1.
    6. Patients must have a site of disease amenable to biopsy, and be a candidate for tumor biopsy. Patient must be willing to undergo a new tumor biopsy at baseline or at molecular pre-screening if applicable, and during therapy on this study. For patients in the phase II part of the study, exceptions may be granted after documented discussion with Novartis. After a sufficient number of paired biopsies are collected, the decision may be taken to stop the collection of biopsies.
    Other protocol-defined inclusion criteria may apply.
    -
    E.4Principal exclusion criteria
    1. History of severe hypersensitivity reactions to other mAbs
    2. Subjects with active, known or suspected autoimmune disease. Subjects with vitiligo, type I diabetes mellitus, residual hypothyroidism due to autoimmune condition only requiring hormone replacement, psoriasis not requiring systemic treatment, or conditions not expected to recur in the absence of an external trigger are permitted to enroll.
    3. History of drug-induced pneumonitis or current pneumonitis.
    4. HIV infection.
    5. Active HBV or HCV infection.
    6. Patients with ocular melanoma.
    7. Systemic anti-cancer therapy within 2 weeks of the first dose of study treatment. For cytotoxic agents that have major delayed toxicity, e.g. mitomycin C and nitrosoureas, 4 weeks washout period. For patients receiving anticancer immunotherapies such as CTLA-4 antagonists, 6 weeks is indicated as the washout period.
    8. Prior PD-1- or PD-L1-directed therapy.
    9. Patients requiring chronic treatment with systemic steroid therapy, other than replacement-dose steroids in the setting of adrenal insufficiency. Topical, inhaled, nasal and ophthalmic steroids are not prohibited.
    10. Patients receiving systemic treatment with any immunosuppressive medication (other than steroids as described above).
    11. Use of any live vaccines against infectious diseases (e.g. influenza, varicella, pneumococcus) within 4 weeks of initiation of study treatment.
    12. Presence of = CTCAE grade 2 toxicity (except alopecia, peripheral neuropathy and ototoxicity, which are excluded if = CTCAE grade 3) due to prior cancer therapy.

    Other protocol-defined exclusion criteria may apply.
    -
    E.5 End points
    E.5.1Primary end point(s)
    Phase I:
    - The exposure (AUC(0-336h)) after first dose of treatment at cycle 3
    - The incidence of DLTs

    Phase II: Overall response rate (ORR) per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1.
    -
    E.5.1.1Timepoint(s) of evaluation of this end point
    Phase I:
    - The AUC(0-336h) : after first dose of treatment at cycle 3
    - the incidence of DLTs: in first cycle of treatment
    Phase II:
    - Every 8 weeks ± 1 week from 8 weeks post Cycle 1 Day 1 up to 40 weeks, then every 12 weeks ± 1 week until progression of disease per irRC or patient withdrawal.
    -
    E.5.2Secondary end point(s)
    1- Safety: Incidence and severity of adverse events (AEs) and serious adverse events (SAEs), including changes in laboratory parameters, vital signs and electrocardiograms (ECGs)
    Tolerability: Dose interruptions, reductions and dose intensity
    2- Serum PK parameters (e.g., AUC, Cmax, Tmax, half-life); Serum concentration vs. time profiles
    Presence and/or concentration of anti-PDR001 antibodies.
    3- Phase I: ORR, progression free survival (PFS), duration of response (DOR) and disease control rate (DCR)
    Phase II: ORR per immune related Response Criteria (irRC), PFS, DOR,
    DCR
    -
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Physical examination, vital signs etc: Every week until Cycle 1 Day 15.
    Every two weeks from Cycle 1 Day 15 until Cycle 3 Day 1. Every cycle from Cycle 3 Day 1 onwards. Continous assessment of AEs at every visit.
    ECG will be assessed at screening and as clinically indicated.
    2. Cycle 1 and Cycle 3.
    3. Every 8 weeks ± 1 week from 8 weeks post Cycle 1 Day 1 up to 40 weeks, then every 12 weeks ± 1 week until progression of disease per
    irRC or patient withdrawal.
    -
    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 No
    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) Yes
    E.7.1.1First administration to humans Yes
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA25
    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
    France
    Germany
    Hungary
    Italy
    Japan
    Lebanon
    Netherlands
    Norway
    Poland
    Spain
    Taiwan
    Thailand
    Turkey
    United States
    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
    The end of the study will be when 80% of the patients per disease group in the phase II part have completed the follow-up for disease progression or discontinued the study for any reason, and all patients have completed treatment and the 150 day safety follow-up period, or if the study is terminated early.
    -
    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 months0
    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: 182
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 106
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 150
    F.4.2.2In the whole clinical trial 318
    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)
    not applicable
    -
    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 Decision2016-05-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-03-16
    P. End of Trial
    P.End of Trial StatusCompleted
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