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    Summary
    EudraCT Number:2015-002900-10
    Sponsor's Protocol Code Number:APD001
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2016-04-27
    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 ConcernedFrance - ANSM
    A.2EudraCT number2015-002900-10
    A.3Full title of the trial
    AN OPEN-LABEL EXPLORATORY STUDY OF UCB5857 IN SUBJECTS WITH ACTIVATED PHOSPHOINOSITIDE 3 KINASE (PI3K) DELTA SYNDROME (APDS)
    ÉTUDE EXPLORATOIRE OUVERTE ÉVALUANT L’UCB5857 CHEZ DES PATIENTS PRÉSENTANT UN SYNDROME DE PHOSPHO-INOSITIDE-3-KINASE (PI3K) DELTA ACTIVÉE (SYNDROME APDS)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of UCB5857 in subjects with APDS.
    Étude évaluant l´UCB5857 chez des patients présentant un sydrome APDS.
    A.4.1Sponsor's protocol code numberAPD001
    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 SponsorUCB Celltech, UK Registered Branch of UCB Pharma SA
    B.1.3.4CountryUnited Kingdom
    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 supportUCB Celltech
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUCB BIOSCIENCES GmbH
    B.5.2Functional name of contact pointClin Trial Reg & Results Disclosure
    B.5.3 Address:
    B.5.3.1Street AddressAlfred-Nobel-Strasse 10
    B.5.3.2Town/ cityMonheim
    B.5.3.3Post code40789
    B.5.3.4CountryGermany
    B.5.4Telephone number+492173481515
    B.5.5Fax number+492173481573
    B.5.6E-mailclinicaltrials@ucb.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 nameUCB5857
    D.3.2Product code UCB5857
    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 INNUCB5857
    D.3.9.1CAS number 1362850-20-1
    D.3.9.2Current sponsor codeUCB5857
    D.3.9.4EV Substance CodeSUB122669
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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 nameUCB5857
    D.3.2Product code UCB5857
    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 INNUCB5857
    D.3.9.1CAS number 1362850-20-1
    D.3.9.2Current sponsor codeUCB5857
    D.3.9.4EV Substance CodeSUB122669
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number15
    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: 3
    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 nameUCB5857
    D.3.2Product code UCB5857
    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 INNUCB5857
    D.3.9.1CAS number 1362850-20-1
    D.3.9.2Current sponsor codeUCB5857
    D.3.9.4EV Substance CodeSUB122669
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number30
    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
    Activated PI3K delta Syndrome (APDS)
    Syndrome de PI3K delta activée (syndrome APDS)
    E.1.1.1Medical condition in easily understood language
    Activated PI3K delta Syndrome (APDS)
    Syndrome de PI3K delta activée (syndrome APDS)
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective is to evaluate the safety and tolerability of UCB5857 in subjects with Activated phosphoinositide-3 kinase (PI3K) delta Syndrome (APDS).
    L’objectif principal est d’évaluer la tolérance et la sécurité d’emploi de l’UCB5857 chez des patients présentant un syndrome APDS.
    E.2.2Secondary objectives of the trial
    The secondary objective is to assess the pharmacokinetic (PK) profile of UCB5857 in subjects with Activated phosphoinositide-3 kinase (PI3K) delta Syndrome (APDS).
    L’objectif secondaire est d’évaluer le profil pharmacocinétique (PK) de l’UCB5857 chez des patients présentant un syndrome APDS.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Subject/legal representative is considered reliable and capable of adhering to the protocol (e.g. able to understand and complete diaries), visit schedule, or medication intake.

    - Subject must have a confirmed genotypic diagnosis of APDS (i.e. proven pathogenic mutation in either the PIK3R1 or PIK3CD gene).

    - Subjects must have had clinical findings and manifestations compatible with APDS such as nodal and/or extranodal lymphoproliferation, history of repeated oto-sinopulmonary infections and/or organ dysfunction (e.g., lung, liver)
    - Le patient/représentant légal est considéré comme fiable et capable de respecter le protocole (par exemple, a la capacité de comprendre et de remplir les carnets journaliers), le calendrier des visites ou à la prise du médicament.

    - Le patient doit avoir un diagnostic génotypique confirmé de syndrome APDS (c’est-à-dire une mutation pathogène avérée dans le gène PIK3R1 ou PIK3CD).

    - Les patients doivent avoir eu des anomalies et manifestations cliniques compatibles avec un syndrome APDS, telles qu’une lymphoprolifération ganglionnaire et/ou extraganglionnaire, des antécédents d’infections oto-sino-pulmonaires répétées et/ou un dysfonctionnement d’un organe (par exemple, poumon, foie).
    E.4Principal exclusion criteria
    1. Subject has participated in another study of an investigational medicinal product (IMP) (or a medical device) within the previous 30 days or is currently participating in another study of an IMP (or a medical device).
    2. Subject has a history of allogeneic bone marrow transplantation.
    3. Subject has no history of any clinical manifestations in last 12 months.
    4. Subject tests positive for human immunodeficiency virus Type 1 or Type 2, hepatitis B surface antigen, or hepatitis C virus antibody.
    5. Subject whose only clinical manifestations in the last 12 months are due to advanced bronchiectasis (declining lung function, 3 or more recurrent exacerbations, lung abscess, pneumothorax for repeating cough).
    6. Subject who has a severe (life-threatening) infection during the screening period.
    7. Subject who have lymphoma at the time of the screening or have been treated for lymphoma in the previous 5 years prior Screening (Visit 1).
    8. Subject is female and is breast-feeding, pregnant, or plans to become pregnant or to start breastfeeding during the study or within 3 months following last dose of IMP.
    9. Subject has WBC<2000/mm3, or absolute neutrophil count <1000/mm3 at Screening (Visit 1).
    10. Subject has a history of chronic alcohol or drug abuse within the previous 6 months.
    11. Subject has any relevant medical or psychiatric condition that could jeopardize or would compromise the subject’s ability to participate in this study.
    12. Subject has a known hypersensitivity to any components of the IMP or comparative drugs as stated in this protocol.
    13. Subject has been treated with any mTOR inhibitors (unless washed out, approximately 6 weeks).
    14. Subject has a history of inflammatory bowel disease, peptic ulcers, or recurrent colitis including rectal bleeding (within 1 year prior to Screening [Visit 1]).
    15. Subject has a body weight of less than 20 kg at Screening (Visit 1).
    1. Participation à une autre étude évaluant un médicament expérimental (ME) (ou un dispositif médical) au cours des 30 jours précédents ou participe actuellement à une autre étude évaluant un ME (ou un dispositif médical).
    2. Antécédents de greffe allogénique de moelle osseuse.
    3. Absence d’antécédents de manifestations cliniques au cours des 12 derniers mois.
    4. Résultats positifs aux tests de dépistage du virus de l’immunodéficience humaine de type 1 ou de type 2, de l’antigène de surface du virus de l’hépatite B ou des anticorps dirigés contre le virus de l’hépatite C.
    5. Uniques manifestations cliniques au cours des 12 derniers mois dues à une bronchiectasie avancée (diminution de la fonction pulmonaire, 3 exacerbations récurrentes ou plus, abcès pulmonaire, pneumothorax pour toux répétée).
    6. Infection sévère (pouvant mettre en jeu le pronostic vital) au cours de la période de sélection.
    7. Présence d’un lymphome au moment de la sélection ou traitement pour un lymphome dans les 5 ans précédant la Sélection (Visite 1).
    8. Allaitement ou grossesse en cours ou prévu(e) pendant l’étude dans les 3 mois suivant la dernière administration du ME.
    9. Numération des globules blancs < 2 000/mm3 ou numération absolue des polynucléaires neutrophiles < 1 000/mm3 lors de la Sélection (Visite 1).
    10. Antécédents de consommation chronique d’alcool ou de toxicomanie au cours des 6 mois précédents.
    11. Pathologie médicale ou psychiatrique significative, susceptible de mettre en péril ou compromettre sa capacité à participer à cette étude.
    12. Hypersensibilité connue à l’un des composants du ME ou aux médicaments comparatifs indiqués dans ce protocole.
    13. Traitement antérieur par un inhibiteur de mTOR (sauf après une période de sevrage d’environ 6 semaines).
    14. Antécédents de maladie inflammatoire de l’intestin, ulcères gastro-duodénaux ou colite récurrente, y compris saignement rectal (dans l’année précédent la Sélection [Visite 1]).
    15. Le poids du patient est inférieur à 20 kg lors de la Sélection (Visite 1).
    E.5 End points
    E.5.1Primary end point(s)
    Safety variables
    The safety variables will include:
    • Incidence of AEs
    • Incidence of SAEs
    • Changes from Baseline in safety laboratory tests (serum chemistry, haematology, and urinalysis) at the time points measured
    • Change from Baseline in vital sign parameters (systolic and diastolic blood pressure, temperature, pulse rate and respiratory rate) and body weight at the time points measured
    • Change from Baseline in ECG parameters at the time points measured
    • Physical examination findings.

    Pharmacokinetic variable
    The PK variable will be the plasma concentration of UCB5857.
    Variables de sécurité d’emploi
    Les variables de sécurité d’emploi inclueront:
    • Incidence des évènements indésirables (EI)
    • Incidence des évènements indésirables graves (EIG)
    • Variation par rapport aux analyses biologiques de tolérance (biochimie sanguine, hématologie et analyse d’urine) en cours d´étude
    • Variation par rapport aux paramètres de signes vitaux (pression artérielle systolique et diastolique, température, pouls et fréquence respiratoire) et du poids en cours d´étude
    • Variation par rapport aux paramètres de l´ECG en cours d´étude
    • Anomalies des examens cliniques.

    Variable pharmacocinétique
    La variable PK sera la concentration plasmatique de l’UCB5857.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At the time points measured.
    E.5.2Secondary end point(s)
    Please refer to the study variables of the Clinical Trial Protocol.
    Se référer aux variables étudiées du protocole de l´essai clinique
    E.5.2.1Timepoint(s) of evaluation of this end point
    Please refer to the study variables of the Clinical Trial Protocol.
    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 No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    A phase 1b study to evaluate the safety and tolerability of UCB5857 in subjects with APDS
    E.7.2Therapeutic exploratory (Phase II) No
    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.8.3 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 EEA4
    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
    The end of the study is defined as the date of the last visit (or follow-up telephone call) of the last subject in the study.
    La fin de l´étude est définie comme la date de la dernière visite (ou appel téléphonique de suivi) du dernier patient dans l´étude.
    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 months10
    E.8.9.1In the Member State concerned days14
    E.8.9.2In all countries concerned by the trial years2
    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 Yes
    F.1.1Number of subjects for this age range: 3
    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: 3
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 3
    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 state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 6
    F.4.2.2In the whole clinical trial 6
    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)
    Subjects who complete the treatment period per protocol, and benefit from UCB5857 may have the option to receive UCB5857, as part of an open-label extension study, contingent on receipt of all required ethics and regulatory approvals.
    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 Decision2016-04-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-03-15
    P. End of Trial
    P.End of Trial StatusCompleted
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