Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43871   clinical trials with a EudraCT protocol, of which   7290   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    A Multi-center, Randomized, Open-Label, Parallel-Group Study with LJPC-401 for the Treatment of Myocardial Iron Overload in Patients with Transfusion-Dependent Beta Thalassemia

    Summary
    EudraCT number
    		 2017-003372-31
    Trial protocol
    		 GB   GR   CY   IT  
    Global end of trial date
    14 Jan 2020

    Results information
    Results version number
    		 v1(current)
    This version publication date
    08 May 2022
    First version publication date
    08 May 2022
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    LJ401-BT01
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03381833
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    La Jolla Pharmaceutical Company
    Sponsor organisation address
    4747 Executive Drive, Suite 240, San Diego, United States, 92121
    Public contact
    Regulatory Affairs, La Jolla Pharmaceutical Company, 1 831-421-1450, meddleman@ljpc.com
    Scientific contact
    Regulatory Affairs, La Jolla Pharmaceutical Company, 1 831-421-1450, meddleman@ljpc.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
    15 Oct 2020
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    15 Nov 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    14 Jan 2020
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    Efficacy on cardiac iron as measured by cardiac T2* MRI in patients with transfusion-dependent beta thalassemia
    Protection of trial subjects
    The study was conducted in accordance with the ethical principles founded in the Declaration of Helsinki and in compliance with the protocol, the International Council on Harmonisation (ICH) for Good Clinical Practice (GCP) and the appropriate regulatory requirements. The study was conducted in accordance with the ICH E6 GCP for obtaining informed consent. Each patient provided written informed consent after the study was fully explained and before any study-specific procedures (including study-specific screening procedures) were performed.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    30 Nov 2017
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United Kingdom: 8
    Country: Number of subjects enrolled
    Greece: 9
    Country: Number of subjects enrolled
    Italy: 12
    Country: Number of subjects enrolled
    Australia: 5
    Country: Number of subjects enrolled
    Lebanon: 13
    Country: Number of subjects enrolled
    Thailand: 4
    Country: Number of subjects enrolled
    Turkey: 29
    Country: Number of subjects enrolled
    United States: 4
    Worldwide total number of subjects
    84
    EEA total number of subjects
    21
    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)
    0
    Adults (18-64 years)
    84
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Patients were screened to ensure they were clinically diagnosed with beta thalassemia for which they were transfusion-dependent and had myocardial iron overload. Patients were to be 14 years or older with a cardiac T2* MRI of 6 to 35 msec.

    Period 1
    Period 1 title
    Period 1
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Group A
    Arm description
    		 Group A did not receive treatment other than Standard of Care during Period 1. During Period 2, Group A received 10 mg BIW LJPC-401 plus Standard of Care.
    Arm type
    		 Standard of Care

    Investigational medicinal product name
    LJPC-401
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intramuscular use
    Dosage and administration details
    Week 1 through 26 (Period 1): SOC Only Week 27 through Week 52 (Period 2): LJPC-401 10 mg Bi-weekly plus SOC. Doses were given as one or more intramuscular injections. If more than one injection was needed, injections were spread over different parts of the body.

    Arm title
    		 Group B
    Arm description
    		 Group B received 5-20 mg LJPC-401 QW plus Standard of Care.
    Arm type
    		 Experimental

    Investigational medicinal product name
    LJPC-401
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intramuscular use
    Dosage and administration details
    The dose level in Group B was escalated the first 3 weeks of Period 1 per the following schedule: Week 1 (Period 1): LJPC-401 5 mg QW and SOC Week 2 (Period 1): LJPC-401 10 mg QW and SOC Week 3 through Week 52 (Period 1 through Period 2): LJPC-401 20 mg QW and SOC Doses were given as one or more intramuscular injections. If more than one injection was needed, injections were spread over different parts of the body.

    Number of subjects in period 1
    		Group A Group B
    Started
    42
    42
    Completed
    26
    26
    Not completed
    16
    16
         Physician decision
    -
    1
         Consent withdrawn by subject
    -
    3
         Adverse event, non-fatal
    -
    1
         Pregnancy
    1
    -
         Sponsor Decision
    15
    11
    Period 2
    Period 2 title
    Period 2
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Group A
    Arm description
    		 Group A did not receive treatment other than Standard of Care during Period 1. During Period 2, Group A received 10 mg BIW LJPC-401 plus Standard of Care.
    Arm type
    		 Standard of Care

    Investigational medicinal product name
    LJPC-401
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intramuscular use
    Dosage and administration details
    Week 1 through 26 (Period 1): SOC Only Week 27 through Week 52 (Period 2): LJPC-401 10 mg Bi-weekly plus SOC. Doses were given as one or more intramuscular injections. If more than one injection was needed, injections were spread over different parts of the body.

    Arm title
    		 Group B
    Arm description
    		 Group B received 5-20 mg LJPC-401 QW plus Standard of Care.
    Arm type
    		 Experimental

    Investigational medicinal product name
    LJPC-401
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intramuscular use
    Dosage and administration details
    The dose level in Group B was escalated the first 3 weeks of Period 1 per the following schedule: Week 1 (Period 1): LJPC-401 5 mg QW and SOC Week 2 (Period 1): LJPC-401 10 mg QW and SOC Week 3 through Week 52 (Period 1 through Period 2): LJPC-401 20 mg QW and SOC Doses were given as one or more intramuscular injections. If more than one injection was needed, injections were spread over different parts of the body.

    Number of subjects in period 2
    		Group A Group B
    Started
    26
    26
    Completed
    15
    17
    Not completed
    11
    9
         Consent withdrawn by subject
    4
    2
         Physician decision
    -
    1
         Sponsor Decision
    7
    6

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Group A
    Reporting group description
    		 Group A did not receive treatment other than Standard of Care during Period 1. During Period 2, Group A received 10 mg BIW LJPC-401 plus Standard of Care.

    Reporting group title
    Group B
    Reporting group description
    		 Group B received 5-20 mg LJPC-401 QW plus Standard of Care.

    Reporting group values
    		 Group A Group B Total
    Number of subjects
    		 42 42 84
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 0 0 0
        Adolescents (12-17 years)
    		 0 0 0
        Adults (18-64 years)
    		 42 42 84
        From 65-84 years
    		 0 0 0
        85 years and over
    		 0 0 0
    Gender categorical
    Units: Subjects
        Female
    		 24 15 39
        Male
    		 18 27 45

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    Group A
    Reporting group description
    		 Group A did not receive treatment other than Standard of Care during Period 1. During Period 2, Group A received 10 mg BIW LJPC-401 plus Standard of Care.

    Reporting group title
    Group B
    Reporting group description
    		 Group B received 5-20 mg LJPC-401 QW plus Standard of Care.
    Reporting group title
    Group A
    Reporting group description
    		 Group A did not receive treatment other than Standard of Care during Period 1. During Period 2, Group A received 10 mg BIW LJPC-401 plus Standard of Care.

    Reporting group title
    Group B
    Reporting group description
    		 Group B received 5-20 mg LJPC-401 QW plus Standard of Care.

    Primary: Cardiac T2*MRI at Week 26

    		 Close Top of page
    End point title
    		 Cardiac T2*MRI at Week 26
    End point description
    End point type
    Primary
    End point timeframe
    Week 26
    End point values
    		 Group A Group B
    Number of subjects analysed
    27
    29
    Units: Cardiac Iron Level
        least squares mean (standard deviation)
    0.67 ± 3.473
    0.45 ± 3.898
    Statistical analysis title
    		 Cardiac T2* MRI Change from Baseline
    Comparison groups
    Group A v Group B
    Number of subjects included in analysis
    56
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.8092
    Method
    		 General Linear Model
    Confidence interval

    Secondary: Cardiac T2*MRI at Week 52

    		 Close Top of page
    End point title
    		 Cardiac T2*MRI at Week 52
    End point description
    End point type
    Secondary
    End point timeframe
    Week 52
    End point values
    		 Group A Group B
    Number of subjects analysed
    17
    18
    Units: Cardiac Iron Level
        least squares mean (standard deviation)
    1.17 ± 6.106
    1.94 ± 4.601
    Statistical analysis title
    		 Cardiac T2* MRI Change from Baseline at Week 52
    Comparison groups
    Group A v Group B
    Number of subjects included in analysis
    35
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.7436
    Method
    		 General Linear Model
    Confidence interval

    Adverse events

    		 Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    From the time of consent through End of Study.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    20.1
    Reporting groups
    Reporting group title
    Group A
    Reporting group description
    		 Group A did not receive treatment other than Standard of Care during Period 1. During Period 2, Group A received 10 mg BIW LJPC-401 plus Standard of Care.

    Reporting group title
    Group B
    Reporting group description
    		 Group B received 10 mg LJPC-401 QW plus Standard of Care.

    Serious adverse events
    		 Group A Group B
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 42 (7.14%)
    4 / 41 (9.76%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Cardiac disorders
    Atrial fibrillation
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 41 (2.44%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Enteritis
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 41 (2.44%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Cholecystitis acute
         subjects affected / exposed
    1 / 42 (2.38%)
    0 / 41 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Adenovirus infection
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 41 (2.44%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastroenteritis
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 41 (2.44%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory tract infection
         subjects affected / exposed
    1 / 42 (2.38%)
    0 / 41 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Salmonellosis
         subjects affected / exposed
    0 / 42 (0.00%)
    1 / 41 (2.44%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Hypocalcaemia
         subjects affected / exposed
    1 / 42 (2.38%)
    0 / 41 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Group A Group B
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    30 / 42 (71.43%)
    38 / 41 (92.68%)
    Injury, poisoning and procedural complications
    Allergic transfusion reaction
         subjects affected / exposed
    1 / 42 (2.38%)
    4 / 41 (9.76%)
         occurrences all number
    1
    4
    Cardiac disorders
    Palpitations
         subjects affected / exposed
    3 / 42 (7.14%)
    2 / 41 (4.88%)
         occurrences all number
    4
    3
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    1 / 42 (2.38%)
    4 / 41 (9.76%)
         occurrences all number
    1
    8
    Headache
         subjects affected / exposed
    7 / 42 (16.67%)
    6 / 41 (14.63%)
         occurrences all number
    10
    16
    Paraesthesia
         subjects affected / exposed
    1 / 42 (2.38%)
    3 / 41 (7.32%)
         occurrences all number
    3
    4
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    4 / 42 (9.52%)
    2 / 41 (4.88%)
         occurrences all number
    8
    4
    Injection site erythema
         subjects affected / exposed
    5 / 42 (11.90%)
    10 / 41 (24.39%)
         occurrences all number
    30
    81
    Injection site pain
         subjects affected / exposed
    8 / 42 (19.05%)
    12 / 41 (29.27%)
         occurrences all number
    37
    76
    Injection site pruritus
         subjects affected / exposed
    1 / 42 (2.38%)
    7 / 41 (17.07%)
         occurrences all number
    1
    9
    Injection site rash
         subjects affected / exposed
    1 / 42 (2.38%)
    3 / 41 (7.32%)
         occurrences all number
    2
    6
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    3 / 42 (7.14%)
    1 / 41 (2.44%)
         occurrences all number
    5
    1
    Abdominal pain upper
         subjects affected / exposed
    3 / 42 (7.14%)
    4 / 41 (9.76%)
         occurrences all number
    4
    4
    Constipation
         subjects affected / exposed
    3 / 42 (7.14%)
    1 / 41 (2.44%)
         occurrences all number
    4
    1
    Diarrhoea
         subjects affected / exposed
    7 / 42 (16.67%)
    3 / 41 (7.32%)
         occurrences all number
    11
    3
    Nausea
         subjects affected / exposed
    2 / 42 (4.76%)
    5 / 41 (12.20%)
         occurrences all number
    4
    10
    Vomiting
         subjects affected / exposed
    2 / 42 (4.76%)
    3 / 41 (7.32%)
         occurrences all number
    5
    5
    Fatigue
         subjects affected / exposed
    0 / 42 (0.00%)
    3 / 41 (7.32%)
         occurrences all number
    0
    5
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    3 / 42 (7.14%)
    5 / 41 (12.20%)
         occurrences all number
    5
    6
    Nasal congestion
         subjects affected / exposed
    1 / 42 (2.38%)
    4 / 41 (9.76%)
         occurrences all number
    1
    5
    Oropharyngeal pain
         subjects affected / exposed
    3 / 42 (7.14%)
    5 / 41 (12.20%)
         occurrences all number
    3
    6
    Rhinorrhoea
         subjects affected / exposed
    1 / 42 (2.38%)
    6 / 41 (14.63%)
         occurrences all number
    1
    7
    Rash
         subjects affected / exposed
    1 / 42 (2.38%)
    4 / 41 (9.76%)
         occurrences all number
    1
    4
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    8 / 42 (19.05%)
    4 / 41 (9.76%)
         occurrences all number
    10
    5
    Infections and infestations
    Gastroenteritis
         subjects affected / exposed
    1 / 42 (2.38%)
    4 / 41 (9.76%)
         occurrences all number
    1
    5
    Influenza
         subjects affected / exposed
    2 / 42 (4.76%)
    4 / 41 (9.76%)
         occurrences all number
    2
    4
    Nasopharyngitis
         subjects affected / exposed
    2 / 42 (4.76%)
    3 / 41 (7.32%)
         occurrences all number
    3
    3
    Sinusitis
         subjects affected / exposed
    0 / 42 (0.00%)
    4 / 41 (9.76%)
         occurrences all number
    0
    4
    Upper respiratory tract infection
         subjects affected / exposed
    10 / 42 (23.81%)
    13 / 41 (31.71%)
         occurrences all number
    14
    21
    Urinary tract infection
         subjects affected / exposed
    0 / 42 (0.00%)
    4 / 41 (9.76%)
         occurrences all number
    0
    5

    More information

    		 Close Top of page

    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    16 Nov 2017
    - Revised primary endpoint - Clarified that secondary endpoints using data from visits other than Week 26 - Updated stratification to include baseline T2* MRI results - Added details of structure and responsibilities of the DMC
    02 Jul 2018
    - Clarified and expanded secondary and exploratory endpoints - Revised LJPC-401 dosing (introduced re-randomization to two dose levels in Group A) - PK substudy design revised - Removed blinding from the study
    16 Nov 2018
    - Enrollment eligibility revised to allow patients 14 years of age and older - Study design amended to add an additional cohort and to investigate a larger range of dosing regimens - Revised statistical methods and analyses section to coincide with study design revisions - Clarified that a periodic review of safety data will occur approximately every 4 months - Interim efficacy analysis was set to occur after 20 subjects in each arm (previously 25) completed Week 26
    20 Feb 2019
    - Revised dosing frequencies in Period 2 for Groups A and B (study design reverted to 2 cohorts) - Deleted previously added Group C - Revised Group A dosage in Period 2 - Revised Group B dosage in Period 2

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    14 Jan 2020
    Following an interim analysis, the study was stopped early due to lack of efficacy and enrollment was discontinued. Not all the planned exploratory analyses were conducted. Analyses for the Per Protocol population were not performed.
    -

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Fri May 03 12:51:19 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA