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    Clinical Trial Results:
    An Open-Label, Exploratory, Multicenter, Extension Study to Evaluate the Long-Term Safety, Tolerability, Pharmacokinetics and Efficacy of UCB5857 in Subjects with Activated Phosphoinositide 3 Kinase (PI3K) Delta Syndrome (APDS)

    Summary
    EudraCT number
    2015-005541-30
    Trial protocol
    DE   IT   ES   FR  
    Global end of trial date
    13 Dec 2018

    Results information
    Results version number
    v1(current)
    This version publication date
    14 Jul 2019
    First version publication date
    14 Jul 2019
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    APD003
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    -
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    UCB BioPharma SPRL
    Sponsor organisation address
    Allée de la Recherche 60, Brussels, Belgium, B-1070
    Public contact
    Clin Trial Reg & Results Disclosure, UCB BIOSCIENCES GmbH, clinicaltrials@ucb.com
    Scientific contact
    Clin Trial Reg & Results Disclosure, UCB BIOSCIENCES GmbH, clinicaltrials@ucb.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    23 Jan 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    13 Dec 2018
    Global end of trial reached?
    Yes
    Global end of trial date
    13 Dec 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the long-term safety and tolerability of UCB5857 in participants with activated PI3K delta syndrome (APDS).
    Protection of trial subjects
    During the conduct of the study all participants were closely monitored.
    Background therapy
    Background therapy as permitted in the protocol.
    Evidence for comparator
    Not applicable.
    Actual start date of recruitment
    11 Jan 2017
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    France: 1
    Country: Number of subjects enrolled
    Germany: 1
    Country: Number of subjects enrolled
    Italy: 2
    Worldwide total number of subjects
    4
    EEA total number of subjects
    4
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    3
    Adults (18-64 years)
    1
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    The study started to enroll participants in January 2017 and concluded in December 2018.

    Pre-assignment
    Screening details
    Participant Flow refers to the Safety Set (SS), which consisted of all participants in the Enrolled Set (ES) who received at least 1 dose of IMP in APD003.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    Not blinded

    Arms
    Arm title
    UCB5857
    Arm description
    Participants were administered the investigational medicinal product (IMP) once daily, in the morning, initially in the same dosage that was given last in the APD001 study. Dose adjustments could have occurred based on the opinion of the Investigator and confirmed by the recommendation of the Safety Monitoring Committee (SMC).
    Arm type
    Experimental

    Investigational medicinal product name
    Seletalisib
    Investigational medicinal product code
    UCB5857
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Capsules of UCB5857 5 mg, 15 mg or 30 mg, administered daily, by oral intake.

    Number of subjects in period 1
    UCB5857
    Started
    4
    Completed
    3
    Not completed
    1
         Consent withdrawn by subject
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    UCB5857
    Reporting group description
    Participants were administered the investigational medicinal product (IMP) once daily, in the morning, initially in the same dosage that was given last in the APD001 study. Dose adjustments could have occurred based on the opinion of the Investigator and confirmed by the recommendation of the Safety Monitoring Committee (SMC).

    Reporting group values
    UCB5857 Total
    Number of subjects
    4 4
    Age categorical
    Units: Subjects
        <=18 years
    3 3
        Between 18 and 65 years
    1 1
        >=65 years
    0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    15.8 ± 5.7 -
    Gender categorical
    Units: Subjects
        Male
    2 2
        Female
    2 2
    Subject analysis sets

    Subject analysis set title
    UCB5857 (SS)
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Participants were administered the IMP once daily, in the morning, initially in the same dosage that was given last in the APD001 study. Dose adjustments could have occurred based on the opinion of the Investigator and confirmed by the recommendation of the SMC, forming the Safety Set.

    Subject analysis sets values
    UCB5857 (SS)
    Number of subjects
    4
    Age categorical
    Units: Subjects
        <=18 years
    3
        Between 18 and 65 years
    1
        >=65 years
    0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    15.8 ± 5.7
    Gender categorical
    Units: Subjects
        Male
    2
        Female
    2

    End points

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    End points reporting groups
    Reporting group title
    UCB5857
    Reporting group description
    Participants were administered the investigational medicinal product (IMP) once daily, in the morning, initially in the same dosage that was given last in the APD001 study. Dose adjustments could have occurred based on the opinion of the Investigator and confirmed by the recommendation of the Safety Monitoring Committee (SMC).

    Subject analysis set title
    UCB5857 (SS)
    Subject analysis set type
    Safety analysis
    Subject analysis set description
    Participants were administered the IMP once daily, in the morning, initially in the same dosage that was given last in the APD001 study. Dose adjustments could have occurred based on the opinion of the Investigator and confirmed by the recommendation of the SMC, forming the Safety Set.

    Primary: The total number of participants experiencing at least one Treatment Emergent Adverse Event during the study

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    End point title
    The total number of participants experiencing at least one Treatment Emergent Adverse Event during the study [1]
    End point description
    Treatment-emergent Adverse Events (TEAEs) were defined as those events which started on or after the date of first dose of APD003 IMP, or events in which severity worsened on or after the date of first dose of APD003 study medication.
    End point type
    Primary
    End point timeframe
    From Baseline, until the study end
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No formal statistical hypothesis testing was planned for this study. Results were summarized as descriptive statistics only.
    End point values
    UCB5857 (SS)
    Number of subjects analysed
    4
    Units: participants
    4
    No statistical analyses for this end point

    Primary: The total number of participants experiencing at least one Serious Adverse Event during the study

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    End point title
    The total number of participants experiencing at least one Serious Adverse Event during the study [2]
    End point description
    A Serious Adverse Event (SAE) must have met 1 or more of the following criteria: •Death, •Life threatening, •Significant or persistent disability/incapacity, •Congenital anomaly/birth defect (including that occurring in a fetus), •Important medical event that, based upon appropriate medical judgment, may have jeopardized the study participant and may have required medical or surgical intervention to prevent 1 of the other outcomes listed in the definition of serious, •Initial inpatient hospitalization or prolongation of hospitalization.
    End point type
    Primary
    End point timeframe
    From Baseline, until the study end
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No formal statistical hypothesis testing was planned for this study. Results were summarized as descriptive statistics only.
    End point values
    UCB5857 (SS)
    Number of subjects analysed
    4
    Units: participants
    1
    No statistical analyses for this end point

    Secondary: Plasma concentration of UCB5857

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    End point title
    Plasma concentration of UCB5857
    End point description
    Plasma concentrations of UCB5857 were measured using a validated high-performance liquid chromatography method with tandem mass spectrometry. Note 1: Due to technical reasons, BLQ (below limit of quantification) could not be entered in the system, thus a placeholder value “999” was used instead.
    End point type
    Secondary
    End point timeframe
    From Baseline, until the Final Treatment Period Visit
    End point values
    UCB5857 (SS)
    Number of subjects analysed
    4
    Units: ng/mL
    number (not applicable)
        Subject 1 - Day 1
    999
        Subject 1 - Week 12
    364
        Subject 1 - Week 24
    352
        Subject 1 - Final Treatment Period Visit
    318
        Subject 2 - Day 1
    6.43
        Subject 2 - Week 12
    245
        Subject 2 - Week 24
    533
        Subject 2 - Week 36
    403
        Subject 2 - Week 48
    231
        Subject 2 - Week 60
    360
        Subject 2 - Week 72
    306
        Subject 2 - Week 84
    391
        Subject 2 - Final Treatment Period Visit
    282
        Subject 3 - Day 1
    999
        Subject 3 - Week 12
    610
        Subject 3 - Week 24
    485
        Subject 3 - Week 36
    298
        Subject 3 - Week 48
    434
        Subject 3 - Week 60
    503
        Subject 3 - Week 72
    756
        Subject 3 - Week 84
    307
        Subject 3 - Final Treatment Period Visit
    587
        Subject 4 - Day 1
    1.26
        Subject 4 - Week 12
    1480
        Subject 4 - Week 24
    1230
        Subject 4 - Week 36
    1210
        Subject 4 - Week 48
    999
        Subject 4 - Week 60
    504
        Subject 4 - Week 72
    449
        Subject 4 - Week 84
    541
        Subject 4 - Final Treatment Period Visit
    677
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From Baseline, until the study end
    Assessment type
    Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    20.1
    Reporting groups
    Reporting group title
    UCB5857 (SS)
    Reporting group description
    Participants were administered the IMP once daily, in the morning, initially in the same dosage that was given last in the APD001 study. Dose adjustments could have occurred based on the opinion of the Investigator and confirmed by the recommendation of the SMC, forming the Safety Set.

    Serious adverse events
    UCB5857 (SS)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 4 (25.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Gastrointestinal disorders
    Stomatitis
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    UCB5857 (SS)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    4 / 4 (100.00%)
    Vascular disorders
    Haematoma
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    5
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Skin papilloma
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    General disorders and administration site conditions
    Adverse drug reaction
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Asthenia
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Influenza like illness
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Pyrexia
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Vessel puncture site haematoma
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Ligament sprain
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Investigations
    C-reactive protein increased
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Haemoglobin decreased
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Weight decreased
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Blood and lymphatic system disorders
    Lymphadenopathy
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Tonsillar hypertrophy
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    3
    Dysphonia
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Catarrh
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Dyspnoea
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Epistaxis
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Snoring
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 4 (50.00%)
         occurrences all number
    8
    Aphonia
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Eye disorders
    Lacrimation increased
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Ear and labyrinth disorders
    Deafness
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    2
    Ear pain
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Otorrhoea
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Gastrointestinal disorders
    Aphthous ulcer
         subjects affected / exposed
    2 / 4 (50.00%)
         occurrences all number
    8
    Vomiting
         subjects affected / exposed
    2 / 4 (50.00%)
         occurrences all number
    2
    Nausea
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    3
    Diarrhoea
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Coating in mouth
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Hepatobiliary disorders
    Hepatomegaly
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Eczema
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Acne
         subjects affected / exposed
    2 / 4 (50.00%)
         occurrences all number
    2
    Night sweats
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Rash
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    2
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    3 / 4 (75.00%)
         occurrences all number
    8
    Sinusitis
         subjects affected / exposed
    3 / 4 (75.00%)
         occurrences all number
    7
    Rhinitis
         subjects affected / exposed
    2 / 4 (50.00%)
         occurrences all number
    3
    Conjunctivitis
         subjects affected / exposed
    2 / 4 (50.00%)
         occurrences all number
    2
    Otitis media acute
         subjects affected / exposed
    2 / 4 (50.00%)
         occurrences all number
    2
    Ear infection
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Gastroenteritis
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1
    Pneumonia
         subjects affected / exposed
    1 / 4 (25.00%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    24 Feb 2016
    Global Protocol Amendment 1, dated 24 Feb 2016, provided the following key changes: •Prohibited the co-administration of known P-glycoprotein (PGP) inhibitors with UCB5857. Since UCB5857 was likely (although not yet confirmed) to be a substrate of PGP, the possibility existed that UCB5857 might be susceptible to pharmacokinetic (PK) interactions as victim drug when co‑administered with a PGP inhibitor. •The local Protocol Amendment 0.1, for Germany only, was included. The local amendment restricted the maximum number of years in the study to 5 years per study participant, since insurance coverage in Germany was limited to 5 years per participant or 8 years for the duration of the complete APD003 study.
    22 Sep 2016
    Global Protocol Amendment 2, an urgent safety measure amendment dated 22 Sep 2016, provided the following key changes: •Included updated and new potential drug-induced liver injury (PDILI) exclusion criteria, withdrawal criteria, and guidance for the management of such cases. Cases of elevated alanine aminotransferase (ALT) and aspartate aminotransferase (AST) had been observed in 3 participants receiving UCB5857 in ongoing studies (2 participants in SS0004 and 1 participant in APD001). All available blinded clinical data for these cases were medically assessed and, in addition, all available data from other participants in the ongoing studies were reviewed to identify any other potential cases of interest but no other clinically relevant elevations in aminotransferases or other hepatobiliary laboratory values were noted. UCB considered that from the currently available information, there was a possible causal association of UCB5857 with increased aminotransferases. Consequently, additional risk minimization and pharmacovigilance measures were implemented in the protocol in order to safeguard participants against any possible liver injury caused by UCB5857. •Provided further guidance on suspected transmission of an infectious agent via a medicinal product in alignment with UCB’s updated procedures.
    05 Apr 2017
    Global Protocol Amendment 3, a substantial amendment dated 05 Apr 2017, provided the following key changes: •Modified the restriction regarding P-glycoprotein (PGP) inhibitors and removed the table of PGP inhibitors from the protocol based on newly available nonclinical data. •Added procedures for assessment and management of tuberculosis (TB) in order to comply with the UCB policy applied to all UCB-sponsored studies (excluding noninterventional studies) that include participants with immunological diseases, who are at increased risk of TB infection either associated with the investigational drug, underlying disease, concomitant treatments, or other medical or sociological factors. •Aligned local versions of the protocol into a single global version. •Added guidance on adverse events of interest (AEOI), management of AEOIs, and immediate reporting of Adverse Events (AEs).
    22 Jan 2018
    Global Protocol Amendment 4, a substantial amendment dated 22 Jan 2018, provided the following key changes: •Changed the duration for safety follow up. UCB5857 has a relative short half-life of 18 hours and therefore 30 days was considered long enough to ascertain any potential late safety events and the 90-day follow-up call was removed from the schedule of assessments. •Adjusted the frequency of an imaging technique scan (high-resolution computer tomography [HRCT] only) for the assessment of global lung disease and hepatosplenomegaly, due to the high level of exposure to radiation. Based on the date of the amendment, 4 participants were enrolled at the time of the amendment. This protocol amendment was not submitted in France because at the time of submission, the single participant enrolled in APD001 in France was withdrawn.

    Interruptions (globally)

    Were there any global interruptions to the trial? No

    Limitations and caveats

    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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