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    Summary
    EudraCT Number:2014-005384-33
    Sponsor's Protocol Code Number:PQR309-002
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-06-08
    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 ConcernedGermany - BfArM
    A.2EudraCT number2014-005384-33
    A.3Full title of the trial
    Open-Label, Non-randomized Phase 2 study with Safety Run-in Evaluating Efficacy and Safety of PQR309 in Patients with Relapsed or Refractory Lymphoma
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Phase 2 study to evaluate efficacy and safety of PQR309 in patients with relapsed or refractory lymphoma
    A.4.1Sponsor's protocol code numberPQR309-002
    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 SponsorPIQUR Therapeutics 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 supportPIQUR Therapeutics AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPIQUR Therapeutics AG
    B.5.2Functional name of contact pointChief Development Officer
    B.5.3 Address:
    B.5.3.1Street AddressHochbergerstrasse 60C
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4057
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number+4161633 29 35
    B.5.6E-mailsasa.dimitrijevic@piqur.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 namePQR309
    D.3.2Product code PQR309
    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 INNNot available yet
    D.3.9.1CAS number 1225037-39-7
    D.3.9.2Current sponsor codePQR309
    D.3.9.3Other descriptive name5-(4,6-di-4-morpholinyl-1,3,5-triazin-2-yl)-4-(trifluoromethyl)-2-pyridinamine
    D.3.9.4EV Substance CodeSUB172213
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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.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 namePQR309
    D.3.2Product code PQR309
    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 INNNot available yet
    D.3.9.1CAS number 1225037-39-7
    D.3.9.2Current sponsor codePQR309
    D.3.9.3Other descriptive name5-(4,6-di-4-morpholinyl-1,3,5-triazin-2-yl)-4-(trifluoromethyl)-2-pyridinamine
    D.3.9.4EV Substance CodeSUB172213
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number80
    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
    Relapsed or refractory lymphoma
    E.1.1.1Medical condition in easily understood language
    Relapsed or refractory lymphoma
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.1
    E.1.2Level PT
    E.1.2Classification code 10025310
    E.1.2Term Lymphoma
    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 evaluate the clinical efficacy of PQR309 in patients with relapsed or refractory lymphoma.
    E.2.2Secondary objectives of the trial
    To evaluate safety and pharmacokinetics of PQR309 in patients with relapsed or refractory lymphoma.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Histologically confirmed diagnosis* of relapsed or refractory lymphoma, received at least two prior lines of therapy including immuno-chemotherapy. Patients with relapsed Chronic Lymphoid Leukemia (CLL) are eligible if they have received one or more prior lines of any standard therapy.
    * archival biopsies may be used if obtained up to a year prior to enrollment; re-biopsy is strongly recommended if last biopsy was obtained more than a year ago.
    2. Only for patients in the phase 2 part: At least one measurable nodal or extra-nodal lesion defined as follows: Clearly measurable (i.e., well-defined boundaries) in at least two perpendicular dimensions on imaging scan, or lymph node or nodal mass bi-dimensional measurement with > 1.5 cm in longest transverse diameter.
    3. Age ≥ 18 years.
    4. Eastern Cooperative Oncology Group (ECOG) Performance Score of 0-1.
    5. Adequate organ system functions defined as follows:
    a. Absolute neutrophil count (ANC) ≥1.0x10^9/l
    b. Platelets ≥ 75x10^9/l
    c. Haemoglobin ≥ 85g/L
    d. Adequate hepatic function, defined as total bilirubin ≤ 1.5 times the upper limit of normal (ULN) and alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5 times ULN (or ALT/AST ≤ 5 times ULN in patients with liver involvement)
    e. Adequate renal function, defined as serum creatinine ≤ 1.5 times ULN
    f. Fasting glucose <7.0 mmol/l; Glycated haemoglobin (HbA1c) <6.4%.
    6. Ability and willingness to swallow and retain oral medication.
    7. Willingness and ability to comply with the trial procedures.
    8. Female and male patients with reproductive potential must agree to use effective contraception from screening until 90 days after discontinuation of PQR309.
    9. Signed informed consent.
    E.4Principal exclusion criteria
    1. Immunosupression due to:
    -Allogeneic hematopoietic stem cell transplant (HSCT)
    -Any immune-supressive therapy within 4 weeks prior to trial treatment start
    -Known HIV infection.
    2. Autologous stem cell transplant within 3 months prior to trial treatment start.
    3. Concomitant anticancer therapy (e.g. chemotherapy, radiotherapy, hormonal therapy, immunotherapy, biological response modifier, signal transduction inhibitors).
    4. Concomitant treatment with medicinal products that increase the pH (reduce acidity) of the upper gastrointestinal tract, including, but not limited to, proton-pump inhibitors (e.g. omeprazole), H2-antagonists (e.g. ranitidine) and antacids. Patients may be enrolled in the study after a wash-out period sufficient to terminate their effect.
    5. Use of any investigational drug within 21 days prior to trial treatment start.
    6. Patients who experienced National Cancer Institute (NCI) Common Terminology Criteria For Adverse Events (CTCAE) ≥ Grade 3 on PI3K/mTOR inhibitors.
    7. Any major surgery, chemotherapy or immunotherapy within 21 days prior to trial treatment start.
    8. Symptomatic or progressing Central Nervous System (CNS) involvement. Exception: Patients with meningeal involvement can be included upon discussion between the sponsor and the investigator.
    9. Persisting toxicities NCI CTCAE ≥2 related to prior anticancer therapy.
    10. Presence of gastrointestinal disease or any other condition that could interfere significantly with the absorption of the study drug.
    11. Severe/unstable angina, myocardial infarction or coronary artery bypass within the last 3 years prior to trial treatment start, symptomatic congestive heart failure New York Heart Association (NYHA) Class 3 or 4, hypertension BP>150/100mmHg.
    12. A serious active infection at the time of treatment, or another serious underlying medical condition that could impair the ability of the patient to receive treatment.
    13. Lack of appropriate contraceptive measures (male and female).
    14. Pregnant or lactating women.
    15. Known HIV infection.
    16. Significant medical conditions which could jeopardize compliance with the protocol.
    17. Uncontrolled diabetes mellitus; patients with controlled diabetes may be enrolled (see fasting glucose and HbA1c levels in inclusion criteria).
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy of PQR309 in patients with relapsed or refractory lymphoma according to Cheson Criteria.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Radiological lymphoma evaluation will be performed at baseline (within 28 days prior to first PQR309 intake) and during treatment at the following time points: every 8 weeks during the first 6 months of PQR309 treatment, subsequently every 2 months up to 2 years and every 6 months afterwards.
    E.5.2Secondary end point(s)
    Safety parameters:
    • Incidence and severity of AEs including SAEs
    • Changes in vital signs (pulse rate, blood pressure, body temperature, ECOG performance status, physical examination, body weight and ECG)
    • Changes of routine laboratory assessments (haematology, blood chemistry, urinalysis).

    Additional Clinical Efficacy:
    • Time to Response (TTR), defined as the time from the date of enrollment to the first documentation of response (complete or partial)
    • Duration of response (DOR) defined as the time from the date of the first confirmed response to the first documentation of relapse or progressive disease, whichever occurs first
    • Time to disease progression (TTP) defined as the time from the date of the first dose of study treatment to the first documentation of progressive disease or death as a result of lymphoma.

    Pharmacokinetics:
    •PQR309 plasma concentration and PK parameters: Cmax, tmax, AUC0-24, AUClast, AUC0–∞, t1/2 and RAC.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Safety parameters:
    AEs and SAEs: continuous monitoring from Day 1 to 30 days after last dose.
    Vital signs and physical examination: all visits.
    Physical examination and ECOG: all visits except Day 2.
    Haematology and blood chemistry: all visits except Day 2.
    ECG and urinalysis: Screening, Day 1, Day 22, Day 50 , end of treatment.

    Pharmacokinetics:
    PK Sampling Run In: Day 1, Day 2, Day 8, Day 15, Day 22 and Day 50.
    PK Sampling Step 1+2: Day 1, Day 15, Day 22 and Day 50.
    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 Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    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 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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA12
    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
    Bosnia and Herzegovina
    France
    Germany
    Israel
    Serbia
    Switzerland
    United Kingdom
    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
    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 months2
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    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: 36
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 36
    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 state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 38
    F.4.2.2In the whole clinical trial 72
    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)
    The patients will continue in the study until unacceptable AE, disease progression, patient's request for withdrawal, investigator judgement or death – whichever comes first. No further treatment with PQR309 will be offered after the patient has completed the study.
    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 Decision2015-08-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-07-24
    P. End of Trial
    P.End of Trial StatusCompleted
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