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    Clinical Trial Results:
    A randomized, participant- and investigator-blinded, placebo-controlled study to investigate efficacy, safety and tolerability of LTP001 in participants with pulmonary arterial hypertension

    Summary
    EudraCT number
    		 2021-000670-28
    Trial protocol
    		 DE   NL   ES  
    Global end of trial date
    25 Apr 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    03 Apr 2025
    First version publication date
    03 Apr 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CLTP001A12201
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05135000
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novartis Pharma AG
    Sponsor organisation address
    Novartis Campus, Basel, Switzerland,
    Public contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111, Novartis.email@Novartis.com
    Scientific contact
    Clinical Disclosure Office, Novartis Pharma AG, 41 613241111, Novartis.email@Novartis.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
    25 Apr 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    25 Apr 2024
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective was to assess the efficacy of LTP001 in participants with pulmonary arterial hypertension (PAH). Due to EudraCT system limitations, which EMA is aware of, data using 999 as data points in this record are not an accurate representation of the clinical trial results. Please use https://www.novctrd.com/CtrdWeb/home.nov for complete trial results.
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    30 Jun 2022
    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: 7
    Country: Number of subjects enrolled
    United States: 3
    Country: Number of subjects enrolled
    Germany: 8
    Country: Number of subjects enrolled
    Netherlands: 6
    Country: Number of subjects enrolled
    Poland: 9
    Country: Number of subjects enrolled
    Argentina: 3
    Country: Number of subjects enrolled
    Spain: 11
    Worldwide total number of subjects
    47
    EEA total number of subjects
    34
    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)
    43
    From 65 to 84 years
    4
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 A total of 56 participants were screened for the study. Out of these, 47 participants were randomized.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 LTP001
    Arm description
    		 Participants received LTP001, 6 mg, oral capsules, once daily in the morning for approximately 24 weeks
    Arm type
    		 Experimental

    Investigational medicinal product name
    LTP001
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    LTP001, 6 mg, once daily in the morning for approximately 24 weeks

    Arm title
    		 Placebo
    Arm description
    		 Participants received LTP001 placebo capsules matching LTP001 orally once daily in the morning for approximately 24 weeks
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    LTP001-matching placebo, once daily, in the morning for approximately 24 weeks

    Number of subjects in period 1
    		LTP001 Placebo
    Started
    35
    12
    Completed
    26
    9
    Not completed
    9
    3
         Adverse event, non-fatal
    2
    -
         Study Terminated by Sponsor
    6
    3
         Lost to follow-up
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    LTP001
    Reporting group description
    		 Participants received LTP001, 6 mg, oral capsules, once daily in the morning for approximately 24 weeks

    Reporting group title
    Placebo
    Reporting group description
    		 Participants received LTP001 placebo capsules matching LTP001 orally once daily in the morning for approximately 24 weeks

    Reporting group values
    		 LTP001 Placebo Total
    Number of subjects
    		 35 12 47
    Age Categorical
    Units: Participants
        18 - <65
    		 32 11 43
        65 - <85
    		 3 1 4
    Age Continuous
    Units: Years
        arithmetic mean (standard deviation)
    		 45.3 ( 13.03 ) 45.3 ( 13.78 ) -
    Sex: Female, Male
    Units: Participants
        Female
    		 29 10 39
        Male
    		 6 2 8
    Race/Ethnicity, Customized
    Units: Subjects
        White
    		 32 11 43
        American Indian or Alaska
    		 2 0 2
        Asian
    		 1 1 2

    End points

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    End points reporting groups
    Reporting group title
    LTP001
    Reporting group description
    		 Participants received LTP001, 6 mg, oral capsules, once daily in the morning for approximately 24 weeks

    Reporting group title
    Placebo
    Reporting group description
    		 Participants received LTP001 placebo capsules matching LTP001 orally once daily in the morning for approximately 24 weeks

    Primary: Change From Baseline in Right Heard Catheterization Pulmonary Vascular Resistance (PVR) at Week 25

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    End point title
    		 Change From Baseline in Right Heard Catheterization Pulmonary Vascular Resistance (PVR) at Week 25 [1]
    End point description
    PVR was defined as the resistance against blood flow from the pulmonary artery to the left atrium measured in dyn.s.cm-5
    End point type
    Primary
    End point timeframe
    Baseline, Week 25
    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: Descriptive statistics are reported.
    End point values
    		 LTP001 Placebo
    Number of subjects analysed
    24
    8
    Units: dynes.sec.cm^-5
        arithmetic mean (standard deviation)
    -1.175 ( 254.0792 )
    -49.685 ( 181.3745 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Six Minute Walk Distance (6MWD)

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    End point title
    		 Change From Baseline in Six Minute Walk Distance (6MWD)
    End point description
    6MWD test measures the distance that a patient can walk on a flat, hard surface in a period of 6 minutes
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 13 and 25
    End point values
    		 LTP001 Placebo
    Number of subjects analysed
    30
    10
    Units: meters
    arithmetic mean (standard deviation)
        Week 13 n=30,10
    3.2 ( 42.45 )
    7.4 ( 29.34 )
        Week 25 n=28,10
    10.4 ( 44.36 )
    21.0 ( 32.33 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Right Atrium (RA) Pressures at Week 25

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    End point title
    		 Change From Baseline in Right Atrium (RA) Pressures at Week 25
    End point description
    The Right Heart Catheterization (RHC) assessment was performed to assess several hemodynamic variables in pulmonary hypertension, including RA pressures.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 25
    End point values
    		 LTP001 Placebo
    Number of subjects analysed
    26
    9
    Units: mmHg
        arithmetic mean (standard deviation)
    -0.4 ( 4.40 )
    -0.6 ( 0.73 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Pulmonary Capillary Wedge Pressure at Week 25

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    End point title
    		 Change From Baseline in Pulmonary Capillary Wedge Pressure at Week 25
    End point description
    Right heart catheterization (RHC) assessment was performed to assess several hemodynamic variables in pulmonary hypertension, including pulmonary capillary wedge pressure (PCWP).
    End point type
    Secondary
    End point timeframe
    Baseline, Week 25
    End point values
    		 LTP001 Placebo
    Number of subjects analysed
    24
    9
    Units: mmHg
        arithmetic mean (standard deviation)
    0.2 ( 3.66 )
    0.9 ( 2.26 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Mean Pulmonary Artery Pressure at Week 25

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    End point title
    		 Change From Baseline in Mean Pulmonary Artery Pressure at Week 25
    End point description
    Right heart catheterization (RHC) assessment was performed to assess several hemodynamic variables in pulmonary hypertension, including pulmonary artery pressure.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 25
    End point values
    		 LTP001 Placebo
    Number of subjects analysed
    26
    9
    Units: mmHg
        arithmetic mean (standard deviation)
    0.4 ( 8.55 )
    -0.6 ( 5.61 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Average Cardiac Output (CO) at Week 25

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    End point title
    		 Change From Baseline in Average Cardiac Output (CO) at Week 25
    End point description
    Right heart catheterization (RHC) assessment was performed to assess several hemodynamic variables in pulmonary hypertension, including cardiac output (CO).
    End point type
    Secondary
    End point timeframe
    Baseline, Week 25
    End point values
    		 LTP001 Placebo
    Number of subjects analysed
    26
    8
    Units: liters per minute
        arithmetic mean (standard deviation)
    0.067 ( 1.0772 )
    0.493 ( 0.9075 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Fractional Area Change (FAC)

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    End point title
    		 Change From Baseline in Fractional Area Change (FAC)
    End point description
    Key right ventricular (RV) function endpoints such as RV fractional area change (RV FAC) were assessed with echocardiography.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 5, 13, and 25
    End point values
    		 LTP001 Placebo
    Number of subjects analysed
    31
    10
    Units: percent
    arithmetic mean (standard deviation)
        Week 5 n=31,9
    0.35 ( 6.944 )
    0.92 ( 5.622 )
        Week 13 n=29,10
    -1.19 ( 6.424 )
    2.62 ( 4.417 )
        Week 25 n=26,8
    -1.85 ( 5.412 )
    -1.45 ( 7.532 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Peak Velocity of Excursion (RV S’)

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    End point title
    		 Change From Baseline in Peak Velocity of Excursion (RV S’)
    End point description
    Key right ventricular (RV) function per echocardiography. The terms Tricuspid Annular Systolic Velocity (TASV) and Peak Velocity of Excursion (RV S’) are synonymous in echocardiography to describe the peak systolic velocity of the lateral tricuspid annulus. Including both TASV and RV S’ as separate secondary endpoints was an oversight in the protocol as the data, calculation, and analyses for both (TASV and RV S’) are identical. Therefore, the TASV nomenclature is used in this results disclosure
    End point type
    Secondary
    End point timeframe
    Baseline, weeks 5, 13, and 25
    End point values
    		 LTP001 Placebo
    Number of subjects analysed
    0 [2]
    0 [3]
    Units: centimeters per second
        arithmetic mean (standard deviation)
    ( )
    ( )
    Notes
    [2] - TASV and RV S’ are synonymous. Data are reported in the TASV endpoint.
    [3] - TASV and RV S’ are synonymous. Data are reported in the TASV endpoint.
    No statistical analyses for this end point

    Secondary: Change From Baseline in Tricuspid Annular Plane Systolic Excursion (TAPSE)

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    End point title
    		 Change From Baseline in Tricuspid Annular Plane Systolic Excursion (TAPSE)
    End point description
    Key right ventricular (RV) function endpoints such as tricuspid annular plane systolic excursion (TAPSE) were assessed with echocardiography.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 5, 13, and 25
    End point values
    		 LTP001 Placebo
    Number of subjects analysed
    33
    11
    Units: centimeters
    arithmetic mean (standard deviation)
        Week 5 n=33,11
    0.061 ( 0.3181 )
    0.042 ( 0.3383 )
        Week 13 n=31,11
    -0.026 ( 0.2594 )
    0.100 ( 0.2933 )
        Week 25 n=27,9
    0.015 ( 0.3697 )
    -0.067 ( 0.2121 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Tricuspid Annular Systolic Velocity (TASV)

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    End point title
    		 Change From Baseline in Tricuspid Annular Systolic Velocity (TASV)
    End point description
    Key right ventricular (RV) function endpoints such as tricuspid annular systolic velocity (TASV) were assessed with echocardiography.
    End point type
    Secondary
    End point timeframe
    Baseline, weeks 5, 13 and 25
    End point values
    		 LTP001 Placebo
    Number of subjects analysed
    33
    11
    Units: centimeters per second
    arithmetic mean (standard deviation)
        Week 5 n=33,11
    0.2 ( 2.14 )
    -0.4 ( 1.50 )
        Week 13 n=32,11
    -0.2 ( 2.38 )
    -0.5 ( 1.63 )
        Week 25 n=26,9
    0.4 ( 2.28 )
    -1.0 ( 3.00 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in EmPHasis-10

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    End point title
    		 Change From Baseline in EmPHasis-10
    End point description
    emPHasis-10 is a questionnaire with 10 questions designed to determine how pulmonary hypertension affects a participant's life. Each item is scored on a scale of 0 to 5, with a total score ranging from 0 to 50. A higher score indicates worse quality of life.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 13 and 25
    End point values
    		 LTP001 Placebo
    Number of subjects analysed
    21
    9
    Units: score
    arithmetic mean (standard deviation)
        Week 13 n=21,9
    -1.490 ( 5.7108 )
    -3.622 ( 3.1712 )
        Week 25 n=17,9
    -4.972 ( 8.7643 )
    -3.000 ( 4.2961 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Pulmonary Arterial Hypertension—Symptoms and Impact (PAH-SYMPACT)

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    End point title
    		 Change From Baseline in Pulmonary Arterial Hypertension—Symptoms and Impact (PAH-SYMPACT)
    End point description
    PAH-SYMPACT is a questionnaire used to assess pulmonary arterial hypertension symptoms and their impact. Individual item scores range from 0 to 4. Total score is calculated as the sum of the scores for the individual items divided by the number of items. A higher score indicates more severe symptoms/impacts.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 13 and 25
    End point values
    		 LTP001 Placebo
    Number of subjects analysed
    11
    4
    Units: score
    arithmetic mean (standard deviation)
        Week 13
    -1.452 ( 6.3897 )
    -3.545 ( 3.2693 )
        Week 25
    -0.680 ( 5.7304 )
    -4.220 ( 6.1990 )
    No statistical analyses for this end point

    Secondary: Maximum Observed Blood Concentrations (Cmax) for LTP001

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    End point title
    		 Maximum Observed Blood Concentrations (Cmax) for LTP001
    End point description
    The maximum (peak) observed blood drug concentration after single dose administration.
    End point type
    Secondary
    End point timeframe
    Day 1 and Week 25 at 15, 45, and 120 minutes post-dose
    End point values
    		 LTP001 Placebo
    Number of subjects analysed
    30
    0 [4]
    Units: ng/mL
    geometric mean (geometric coefficient of variation)
        Day 1
    38.6 ( 58.2 )
    ( )
        Week 25
    39.2 ( 54.7 )
    ( )
    Notes
    [4] - Not analyzed in the placebo group.
    No statistical analyses for this end point

    Secondary: Time to Reach Maximum Blood Concentrations (Tmax) of LTP001

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    End point title
    		 Time to Reach Maximum Blood Concentrations (Tmax) of LTP001
    End point description
    The time to reach maximum (peak) blood drug concentration after single dose administration.
    End point type
    Secondary
    End point timeframe
    Day 1 and Week 25 at 15, 45, and 120 minutes post-dose
    End point values
    		 LTP001 Placebo
    Number of subjects analysed
    30
    0 [5]
    Units: hours
    geometric mean (geometric coefficient of variation)
        Day 1
    1.05 ( 56.6 )
    ( )
        Week 25
    0.979 ( 68.8 )
    ( )
    Notes
    [5] - Not analyzed in the placebo group.
    No statistical analyses for this end point

    Secondary: Time to Clinical Worsening

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    End point title
    		 Time to Clinical Worsening
    End point description
    Time to any of the following: - Death - Hospital stay greater than 24 hours due to worsening of pulmonary arterial hypertension - Worsening of PAH resulting in need for lung transplantation or balloon atrial septostomy - Initiation of parenteral prostanoid therapy, initiation of oxygen therapy, initiation of any other pulmonary arterial hypertension-specific therapies or need for increase of diuretics for more than 4 weeks due to worsening of pulmonary arterial hypertension - Significant drop in six minute walk distance
    End point type
    Secondary
    End point timeframe
    Baseline up to approximately 30 weeks
    End point values
    		 LTP001 Placebo
    Number of subjects analysed
    34
    12
    Units: days
        median (confidence interval 95%)
    211.0 (174.0 to 999)
    175.0 (174.0 to 999)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    LTP001 v Placebo
    Number of subjects included in analysis
    46
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.6843
    Method
    		 Logrank
    Parameter type
    		 Cox proportional hazard
    Point estimate
    0.7
    Confidence interval
         level
    		 80%
         sides
    2-sided
         lower limit
    		 0.2
         upper limit
    		 2.8

    Secondary: Change From Baseline in N-terminal Fragment of the Prohormone B-type Natriuretic Peptide (NT-ProBNP)

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    End point title
    		 Change From Baseline in N-terminal Fragment of the Prohormone B-type Natriuretic Peptide (NT-ProBNP)
    End point description
    NT-proBNP is a blood biomarker to assess right ventricular distress.
    End point type
    Secondary
    End point timeframe
    Baseline to Week 29
    End point values
    		 LTP001 Placebo
    Number of subjects analysed
    27
    11
    Units: picomoles per liter
        arithmetic mean (standard deviation)
    4.609 ( 52.9047 )
    -7.918 ( 21.9731 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of 241 days.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    27.0
    Reporting groups
    Reporting group title
    LTP001 6 mg
    Reporting group description
    		 LTP001 6 mg

    Reporting group title
    Total
    Reporting group description
    		 Total

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo

    Serious adverse events
    		 LTP001 6 mg Total Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    5 / 35 (14.29%)
    7 / 47 (14.89%)
    2 / 12 (16.67%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Cardiac disorders
    Right ventricular failure
         subjects affected / exposed
    1 / 35 (2.86%)
    1 / 47 (2.13%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    1 / 35 (2.86%)
    1 / 47 (2.13%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Vomiting
         subjects affected / exposed
    1 / 35 (2.86%)
    1 / 47 (2.13%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Abdominal pain upper
         subjects affected / exposed
    0 / 35 (0.00%)
    1 / 47 (2.13%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pulmonary arterial hypertension
         subjects affected / exposed
    1 / 35 (2.86%)
    1 / 47 (2.13%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Haemoptysis
         subjects affected / exposed
    1 / 35 (2.86%)
    1 / 47 (2.13%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Psychiatric disorders
    Bipolar disorder
         subjects affected / exposed
    1 / 35 (2.86%)
    1 / 47 (2.13%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Catheter site cellulitis
         subjects affected / exposed
    0 / 35 (0.00%)
    1 / 47 (2.13%)
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Lower respiratory tract infection
         subjects affected / exposed
    1 / 35 (2.86%)
    1 / 47 (2.13%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    COVID-19
         subjects affected / exposed
    1 / 35 (2.86%)
    1 / 47 (2.13%)
    0 / 12 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 LTP001 6 mg Total Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    20 / 35 (57.14%)
    27 / 47 (57.45%)
    7 / 12 (58.33%)
    Vascular disorders
    Haematoma
         subjects affected / exposed
    2 / 35 (5.71%)
    2 / 47 (4.26%)
    0 / 12 (0.00%)
         occurrences all number
    4
    4
    0
    Cardiac disorders
    Palpitations
         subjects affected / exposed
    3 / 35 (8.57%)
    4 / 47 (8.51%)
    1 / 12 (8.33%)
         occurrences all number
    3
    4
    1
    Tachycardia
         subjects affected / exposed
    0 / 35 (0.00%)
    1 / 47 (2.13%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    1
    Nervous system disorders
    Presyncope
         subjects affected / exposed
    2 / 35 (5.71%)
    3 / 47 (6.38%)
    1 / 12 (8.33%)
         occurrences all number
    2
    3
    1
    Headache
         subjects affected / exposed
    6 / 35 (17.14%)
    6 / 47 (12.77%)
    0 / 12 (0.00%)
         occurrences all number
    8
    8
    0
    Dizziness
         subjects affected / exposed
    0 / 35 (0.00%)
    1 / 47 (2.13%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    1
    Blood and lymphatic system disorders
    Iron deficiency anaemia
         subjects affected / exposed
    0 / 35 (0.00%)
    1 / 47 (2.13%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    1
    General disorders and administration site conditions
    Peripheral swelling
         subjects affected / exposed
    2 / 35 (5.71%)
    2 / 47 (4.26%)
    0 / 12 (0.00%)
         occurrences all number
    2
    2
    0
    Influenza like illness
         subjects affected / exposed
    1 / 35 (2.86%)
    2 / 47 (4.26%)
    1 / 12 (8.33%)
         occurrences all number
    1
    2
    1
    Fatigue
         subjects affected / exposed
    2 / 35 (5.71%)
    3 / 47 (6.38%)
    1 / 12 (8.33%)
         occurrences all number
    2
    3
    1
    Chest discomfort
         subjects affected / exposed
    0 / 35 (0.00%)
    1 / 47 (2.13%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    1
    Asthenia
         subjects affected / exposed
    2 / 35 (5.71%)
    2 / 47 (4.26%)
    0 / 12 (0.00%)
         occurrences all number
    2
    2
    0
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    1 / 35 (2.86%)
    2 / 47 (4.26%)
    1 / 12 (8.33%)
         occurrences all number
    1
    2
    1
    Nausea
         subjects affected / exposed
    4 / 35 (11.43%)
    5 / 47 (10.64%)
    1 / 12 (8.33%)
         occurrences all number
    4
    5
    1
    Diarrhoea
         subjects affected / exposed
    2 / 35 (5.71%)
    2 / 47 (4.26%)
    0 / 12 (0.00%)
         occurrences all number
    2
    2
    0
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
         subjects affected / exposed
    1 / 35 (2.86%)
    2 / 47 (4.26%)
    1 / 12 (8.33%)
         occurrences all number
    1
    2
    1
    Cough
         subjects affected / exposed
    2 / 35 (5.71%)
    3 / 47 (6.38%)
    1 / 12 (8.33%)
         occurrences all number
    2
    3
    1
    Skin and subcutaneous tissue disorders
    Rash pruritic
         subjects affected / exposed
    0 / 35 (0.00%)
    1 / 47 (2.13%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    1
    Pruritus
         subjects affected / exposed
    2 / 35 (5.71%)
    2 / 47 (4.26%)
    0 / 12 (0.00%)
         occurrences all number
    2
    2
    0
    Endocrine disorders
    Hyperthyroidism
         subjects affected / exposed
    2 / 35 (5.71%)
    2 / 47 (4.26%)
    0 / 12 (0.00%)
         occurrences all number
    2
    2
    0
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    2 / 35 (5.71%)
    2 / 47 (4.26%)
    0 / 12 (0.00%)
         occurrences all number
    2
    2
    0
    Back pain
         subjects affected / exposed
    2 / 35 (5.71%)
    2 / 47 (4.26%)
    0 / 12 (0.00%)
         occurrences all number
    2
    2
    0
    Arthralgia
         subjects affected / exposed
    3 / 35 (8.57%)
    3 / 47 (6.38%)
    0 / 12 (0.00%)
         occurrences all number
    7
    7
    0
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    0 / 35 (0.00%)
    1 / 47 (2.13%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    1
    Respiratory tract infection
         subjects affected / exposed
    2 / 35 (5.71%)
    2 / 47 (4.26%)
    0 / 12 (0.00%)
         occurrences all number
    2
    2
    0
    Nasopharyngitis
         subjects affected / exposed
    2 / 35 (5.71%)
    2 / 47 (4.26%)
    0 / 12 (0.00%)
         occurrences all number
    3
    3
    0
    Influenza
         subjects affected / exposed
    3 / 35 (8.57%)
    3 / 47 (6.38%)
    0 / 12 (0.00%)
         occurrences all number
    3
    3
    0
    COVID-19
         subjects affected / exposed
    0 / 35 (0.00%)
    1 / 47 (2.13%)
    1 / 12 (8.33%)
         occurrences all number
    0
    1
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    01 Dec 2021
    This amendment: required a negative serum pregnancy test within 7 days prior to first dose administration on Day 1 to rule out pregnancy where required by health authority (e. g., the United Kingdom) or local regulations. In countries where this was not required, a negative urine pregnancy test needed to be available prior to dosing on Day 1; excluded participants who took strong CYP3A4/5 inducers or inhibitors; excluded participants who had a long QT syndrome or who took drugs known to prolong the QT interval; added strong CYP3A4/5 inducers, strong CYP3A4/5 inhibitors, and known drugs which cause QT prolongation to the prohibited medications list; specified that 6MWT was only to be conducted on-site to remove testing variability in the assessment; removed the requirement for participants to record investigational medicinal product (IMP) dosing every day in the eDiary; specified that compliance for IMP administration was to be done by medication reconciliation; added coagulation assays (PT, INR, aPTT) and luteinizing hormone and follicle stimulating hormone (FSH) to the safety laboratory panel.
    02 Mar 2022
    This amendment: updated clinical safety data; added guidance text for 6MWT and multi-sensor device; updated withdrawal of consent language to improve clarity.
    01 Nov 2022
    This amendment: incorporated language regarding the implementation of a data monitoring committee (DMC); updated exclusion criteria with information about sperm donation to reflect the requirements described in the protocol appendix; updated protocol appendix with further guidance details for hepatic and renal alert criteria and event follow-up; updated protocol appendix with considerations about the use of LTP001 in combination with allowed standard therapies.

    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.
    Due to EudraCT system limitations, which EMA is aware of, data using 999 as data points in this record are not an accurate representation of the clinical trial results. Please use https://www.novctrd.com/CtrdWeb/home.nov for complete trial results.
    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.

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