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    The EU Clinical Trials Register currently displays   43206   clinical trials with a EudraCT protocol, of which   7151   are clinical trials conducted with subjects less than 18 years old.
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    EudraCT Number:2013-002447-29
    Sponsor's Protocol Code Number:CL01-ORY-1001
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-09-20
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2013-002447-29
    A.3Full title of the trial
    A phase I study of Human Pharmacokinetics and Safety of ORY-1001, and LSD1 inhibitor, in relapsed or refractory acute leukaemia (AL)
    Estudio en fase I de seguridad y farmacocinética humanas de ORY-1001, un inhibidor de LSD1, en leucemia aguda (LA) recurrente o refractaria
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to establish the pharmacokinetics (how the human body processes the drug) and safety of a new anti-cancer drug, ORY-1001 in cancer of blood and bone marrow after treatment or remission
    A.4.1Sponsor's protocol code numberCL01-ORY-1001
    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 SponsorOryzon Genomics S.A.
    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 supportOryzon Genomics S.A.
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationOryzon Genomics S.A.
    B.5.2Functional name of contact pointTamara Maes
    B.5.3 Address:
    B.5.3.1Street AddressC/Sant Ferran,74
    B.5.3.2Town/ cityCornellà de Llobregat
    B.5.3.3Post code08940
    B.5.4Telephone number+34935151313
    B.5.5Fax number+34933774028
    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 Yes
    D.2.5.1Orphan drug designation numberEMA/OD/064/13
    D.3 Description of the IMP
    D.3.1Product nameORY-1001
    D.3.2Product code ORY-1001
    D.3.4Pharmaceutical form Concentrate for oral solution
    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 INNORY-1001
    D.3.9.2Current sponsor codeORY-1001
    D.3.9.3Other descriptive nameOG-RR
    D.3.9.4EV Substance CodeSUB124833
    D.3.10 Strength
    D.3.10.1Concentration unit µg/m2 microgram(s)/square meter
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number5 to 141
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    Refractory or Relapsed acute leukaemia (AL)
    Leucemia aguda (LA) recurrente o refractaria
    E.1.1.1Medical condition in easily understood language
    Cancer of blood and bone marrow after treatment or remission
    Cáncer de la sangre y la médula ósea después del tratamiento o remisión
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.1
    E.1.2Level LLT
    E.1.2Classification code 10000835
    E.1.2Term Acute leukemia
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.1
    E.1.2Level LLT
    E.1.2Classification code 10024330
    E.1.2Term Leukemia acute
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.1
    E.1.2Level LLT
    E.1.2Classification code 10024329
    E.1.2Term Leukemia
    E.1.2System Organ Class 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 16.1
    E.1.2Level PT
    E.1.2Classification code 10000830
    E.1.2Term Acute leukaemia
    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
    Evaluate the safety (hematological and non-hematological toxicities) and tolerability of ORY-1001
    Evaluar la seguridad y tolerabilidad de ORY-1001 en humanos
    E.2.2Secondary objectives of the trial
    - Characterize the PK of orally administered ORY-1001 in patients with relapsed / refractory AL
    - Assess responses (CR/CRi/PR) with ORY-1001 in patients with relapsed or refractory AL, particularly in those with rMLL gene
    - Evaluate surrogate PD markers for target engagement
    - Describir la farmacocinética (FC) de ORY-1001 en humanos
    - Valorar las respuestas (RC/RCi/RP) con ORY-1001 en pacientes con leucemia aguda (LA) recurrente o refractaria, especialmente en aquellos que presentan el gen rMLL
    - Evaluar los marcadores de farmacodinámica (PD) indirectos para la unión de fármaco a su diana
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patients aged 16 and above.
    2. Patients must have relapsed or refractory AL (exluding promyelocytic leukaemia) considered by the investigator ineligible for intensive chemotherapy regimen at that time
    3. Patients must have ECOG Performance Status (PS) of 0-2
    4. Women of child-bearing potential must have negative serum or urine pregnancy test within two weeks prior treatment start
    5. Fertile male or female patients must use highly efficient contraception for the duration of the study and 6 months after the last ORY-1001 dose.
    6. Male patients must use condoms to avoid drug exposure of their partner.
    7. Patients must be capable of understanding and complying with protocol requirements, and they must be able and willing to sign a written informed consent
    8. Life expectancy of at least 2 months
    1. Pacientes con edad a partir de 16 años
    2. Leucemia aguda (LA) recurrente o refractaria (sin incluir la leucemia promielocítica) que el investigador considere no apta para el régimen quimioterápico intensivo en ese momento
    3. Escala de estado funcional (EF) ECOG de 0-2
    4. Las mujeres en edad fértil deben presentar una prueba de embarazo en suero u orina negativa dentro de las dos semanas antes del inicio del tratamiento
    5. Los pacientes hombres o mujeres fértiles deben usar un método anticonceptivo altamente eficaz durante el período de estudio y 6 meses después de la última dosis de ORY-1001.
    6. Los pacientes hombres deben utilizar preservativo con el fin de evitar exponer a su pareja al fármaco.
    7. Los pacientes deben poder entender y cumplir con los requisitos que establece el protocolo, y deberán ser capaces de y estar dispuestos a firmar un consentimiento informado por escrito
    8. La esperanza de vida es de al menos 2 meses
    E.4Principal exclusion criteria
    1. Cancer history that according to the investigator might confound the assessment of the study endpoints
    2. Patients with uncontrolled hypertension or diabetes, hepatitis or HIV.
    3. Inter-current illness or social situation that will limit compliance with study requirements
    4. Pregnancy or lactating / breast feeding
    5. Any medical condition which in the opinion of the investigator places the patient at an unacceptably high risk for toxicities if entered into the clinical study
    6. Acute myeloid leukaemia treatment within the previous 14 days. Hydroxyurea or 6-mercaptopurine are allowed until 12 hours prior study treatment start and after the first treatment block (day 1-5) in case of hyperleucocytosis.
    7. Patients medicated with anti-depressants reported to have KDM1A/LSD1 inhibitory activity: Tranylcypromine or Phenelzine.
    8. Radiotherapy less than 2 weeks prior to the start of the study
    1. Antecedentes de cáncer que, según la opinión del investigador, puedan crear confusión en la evaluación de los criterios de valoración del estudio
    2. Pacientes con hipertensión o diabetes insuficientemente controladas, hepatitis o VIH.
    3. Patología intercurrente o situación social que pueda afectar al cumplimiento de los requisitos del estudio
    4. Pacientes embarazadas o en período de lactancia
    5. Cualquier afección médica que, en opinión del investigador, ponga al paciente en una situación inaceptable de alto riesgo de toxicidad al participar en el estudio clínico
    6. Tratamiento de leucemia mieloide aguda recibido en los 14 días anteriores. La hidroxiurea o la 6-mercaptopurina están permitidas hasta 12 horas antes del inicio del tratamiento del estudio y tras la primera parte del tratamiento (días 1-5) en caso de hiperleucocitosis.
    7. Los pacientes tratados con antidepresivos informaron una actividad inhibitoria KDM1A/LSD1: Tranilcipromina o fenelzina.
    8. Radioterapia aplicada menos de 2 semanas antes del inicio del estudio
    E.5 End points
    E.5.1Primary end point(s)
    The PK of ORY-1001 in patients with relapsed /refractory AL
    The PD of ORY-1001 in patients with relapsed / refractory AL
    Assess maximum tolerated dose of ORY-1001 in humans as a monotherapy
    Adverse events assessed by patient reporting
    Clinical observations and vital signs
    Laboratory data
    E.5.1.1Timepoint(s) of evaluation of this end point
    Patients are to attend study centre visits during baseline (within 14 days before the first dose) and at defined days after start of treatment. Assessments will be made at baseline, day 1, 2, 3, 4, 5, 6, 7 of the first treatment block and on the first, third and fifth day of administration for the 3 other treatment blocks in the 28 day cycle. Additional visits may be made for safety reasons. Hematology samples (5ml) will be obtained on the first, third and fifth day of each treatment block. Samples for the analysis of blood chemistry (5ml) will be obtained weekly on the first day of each treatment block. A follow up visit will be performed at a minimum of 30 days after the last ORY-1001 dose.
    E.5.2Secondary end point(s)
    Remission rate (CR + CRi)
    Gene expression changes in blood cells
    E.5.2.1Timepoint(s) of evaluation of this end point
    DAY1: 0h (pre-dosing)2h, 4h, 6h, 8h, 12h, 18h
    DAY2: 24h, 28h,
    DAY3: 48h, 52h,
    DAY4: 72h, 76h,
    DAY5: 96h,100h,
    During the next 3 treatment blocks in the first cycle, samples will be taken only on the first day and the fifth day, just prior to administration
    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 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) Yes
    E.7.1.1First administration to humans Yes
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E. trial type description
    dose escalation followed by an extension cohort at the MTD
    escalado de dosis seguida de un cohorte de extensión a la DMT
    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 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.8.3 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.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA2
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    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 months7
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months7
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 2
    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. of subjects for this age range: 1
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 8
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 20
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 30
    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)
    Additional cycles of therapy may be decided upon on a case by case basis in mutual agreement between the investigator and Oryzon genomics
    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 Decision2013-12-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-07-31
    P. End of Trial
    P.End of Trial StatusCompleted
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