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 Two Part, Phase 2 Open-label, Multi-Centre, Dose Escalation Hemodynamic Study to Evaluate Dose-Response and Safety of Inhaled LIQ861 (Treprostinil) in Pulmonary Arterial Hypertension (WHO Group 1) Subjects

    Summary
    EudraCT number
    		 2018-003414-40
    Trial protocol
    		 DE   FR  
    Global end of trial date
    12 May 2021

    Results information
    Results version number
    		 v1(current)
    This version publication date
    25 Aug 2022
    First version publication date
    25 Aug 2022
    Other versions
    Summary report(s)
    		 LTI-201 Summary

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    LTI-201
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Liquidia Technologies, Inc
    Sponsor organisation address
    419 Davis Dr., Suite 100, Morrisville, NC, United States, 27560
    Public contact
    Liquidia Technologies Headquarters, Liquidia Technologies, Inc., +1 919 328-4400, ClinicalTrials@liquidia.com
    Scientific contact
    Liquidia Technologies Headquarters, Liquidia Technologies, Inc., +1 919 328-4400, ClinicalTrials@liquidia.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
    26 Oct 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    12 May 2021
    Global end of trial reached?
    Yes
    Global end of trial date
    12 May 2021
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of this study is to characterize hemodynamic dose-response relationships for LIQ861.
    Protection of trial subjects
    Subject signed Informed Consent; Protocol was approved by Ethic Committees and Regulatory Authorities and we adhered to all ICH guidelines.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    02 Jan 2019
    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
    Germany: 15
    Worldwide total number of subjects
    15
    EEA total number of subjects
    15
    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)
    11
    From 65 to 84 years
    4
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 Patients in the LTI-201 study were from PAH centers in France (enrolled 0) and Germany (enrolled 15) from October 30, 2019 (first patient screened) thru December 7, 2020 (last patient screened).

    Pre-assignment
    Screening details
    		 The following screening assessments were performed to assess eligibility criteria: Record medical history, demographics, conmeds, vital signs, functional class assessment, height and weight. Blood was drawn for laboratory assessment and pulmonary function testing performed. 6 Minute Walk test was collected for study eligibility.

    Period 1
    Period 1 title
    Part A
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Cohort 1 - Day 1
    Arm description
    		 Cohort 1 - Day 1 received 5 mg of LIQ861
    Arm type
    		 Experimental

    Investigational medicinal product name
    treprostinil
    Investigational medicinal product code
    LIQ861 5 mg
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Inhalation use
    Dosage and administration details
    Dosage: LIQ861 5 mg capsule contains 26.5 mcg treprostinil Mode of administration: dry powder inhalation by using the RS00 Model 8 dry-powder inhaler

    Arm title
    		 Cohort 2 - Day 1
    Arm description
    		 LIQ861 10 mg Day 1
    Arm type
    		 Experimental

    Investigational medicinal product name
    treprostinil
    Investigational medicinal product code
    LIQ861 10 mg
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Inhalation use
    Dosage and administration details
    Dosage: LIQ861 10 mg capsule contains 53 mcg treprostinil Mode of administration: dry powder inhalation by using the RS00 Model 8 dry-powder inhaler

    Number of subjects in period 1
    		Cohort 1 - Day 1 Cohort 2 - Day 1
    Started
    8
    7
    Completed
    8
    7
    Period 2
    Period 2 title
    Part B
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    No

    Arm title
    		 Cohort 2 - Day 1
    Arm description
    		 LIQ861 10 mg Day 1
    Arm type
    		 Experimental

    Investigational medicinal product name
    treprostinil
    Investigational medicinal product code
    LIQ861 10 mg
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Inhalation use
    Dosage and administration details
    Dosage: LIQ861 10 mg capsule contains 53 mcg treprostinil Mode of administration: dry powder inhalation by using the RS00 Model 8 dry-powder inhaler

    Arm title
    		 Cohort 2 - Week 16
    Arm description
    		 LIQ861 10 mg - Week 16
    Arm type
    		 Experimental

    Investigational medicinal product name
    treprostinil
    Investigational medicinal product code
    LIQ861
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Inhalation use
    Dosage and administration details
    Dosage: Subjects titrated to symptom relief (dosed between 5 mg and 30 mg LIQ861) Mode of administration: dry powder inhalation by using the RS00 Model 8 dry-powder inhaler

    Number of subjects in period 2
    		Cohort 2 - Day 1 Cohort 2 - Week 16
    Started
    7
    7
    Completed
    7
    0
    Not completed
    0
    7
         Early Termination of Study
    -
    7

    Baseline characteristics

    		 Close Top of page

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    Cohort 1 - Day 1
    Reporting group description
    		 Cohort 1 - Day 1 received 5 mg of LIQ861

    Reporting group title
    Cohort 2 - Day 1
    Reporting group description
    		 LIQ861 10 mg Day 1
    Reporting group title
    Cohort 2 - Day 1
    Reporting group description
    		 LIQ861 10 mg Day 1

    Reporting group title
    Cohort 2 - Week 16
    Reporting group description
    		 LIQ861 10 mg - Week 16

    Subject analysis set title
    Cohort 1
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Group of patient's dosed with 5 mg of LIQ861

    Subject analysis set title
    Cohort 2
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Subjects received 10 mg of LIQ861. Only Day 1 data is available as study was terminated early.

    Primary: Pulmonary Vascular Resistance - Day 1

    		 Close Top of page
    End point title
    		 Pulmonary Vascular Resistance - Day 1 [1]
    End point description
    The change in PVR measured from Baseline (pre-dose) to maximal response (eMAX) at Day 1 will be considered the primary efficacy endpoint.
    End point type
    Primary
    End point timeframe
    Day 1
    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 testing was done. All variables were analyzed descriptively, ie, continuous variables by summary statistics and categorical variables by frequency tables using number and percentage of subjects.
    End point values
    		 Cohort 1 Cohort 2
    Number of subjects analysed
    Units: dynes/sec/cm-5
        arithmetic mean (standard error)
    -143.3 ± 24.1
    -139.1 ± 39.1
    No statistical analyses for this end point

    Primary: Incidence and Severity of AEs

    		 Close Top of page
    End point title
    		 Incidence and Severity of AEs [2]
    End point description
    The primary safety endpoint in Parts A and B and the open-label extension period will be the incidence and severity of AEs and Serious Adverse Events (SAEs) grouped by Medical Dictionary for Regulatory Activities (MedDRA) System Organ Class, dose level, time on drug, and relationship to dose.
    End point type
    Primary
    End point timeframe
    Safety endpoints changes from Day 1 to end of treatment.
    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 testing was done. All variables were analyzed descriptively, ie, continuous variables by summary statistics and categorical variables by frequency tables using number and percentage of subjects.
    End point values
    		 Cohort 1 Cohort 2
    Number of subjects analysed
    Units: Incidence
    27
    15
    No statistical analyses for this end point

    Primary: Pulmonary Vascular Resistance - Week 16

    		 Close Top of page
    End point title
    		 Pulmonary Vascular Resistance - Week 16 [3]
    End point description
    The change in PVR measured from Baseline (pre-dose) to maximal response (eMAX) at Week 16 will be considered the primary efficacy endpoint.
    End point type
    Primary
    End point timeframe
    Week 16
    Notes
    [3] - 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 testing was done. All variables were analyzed descriptively, ie, continuous variables by summary statistics and categorical variables by frequency tables using number and percentage of subjects.
    End point values
    		 Cohort 1
    Number of subjects analysed
    Units: dynes/sec/cm-5
        arithmetic mean (standard error)
    -137 ± 54.9
    No statistical analyses for this end point

    Other pre-specified: Mean Pulmonary Arterial Pressure - Day 1

    		 Close Top of page
    End point title
    		 Mean Pulmonary Arterial Pressure - Day 1
    End point description
    End point type
    Other pre-specified
    End point timeframe
    Day 1
    End point values
    		 Cohort 1 Cohort 2
    Number of subjects analysed
    Units: mmHg
        arithmetic mean (standard error)
    -7.3 ± 2.4
    -8.4 ± 2.8
    No statistical analyses for this end point

    Other pre-specified: PK Parameters: Cmax

    		 Close Top of page
    End point title
    		 PK Parameters: Cmax
    End point description
    End point type
    Other pre-specified
    End point timeframe
    Day 1
    End point values
    		 Cohort 1 Cohort 2
    Number of subjects analysed
    Units: ng/ml
        geometric mean (full range (min-max))
    0.213 (.128 to 0.347)
    0.496 (0.305 to 0.909)
    No statistical analyses for this end point

    Other pre-specified: PK Parameters: AUC (tau)

    		 Close Top of page
    End point title
    		 PK Parameters: AUC (tau)
    End point description
    End point type
    Other pre-specified
    End point timeframe
    Day 1
    End point values
    		 Cohort 1 Cohort 2
    Number of subjects analysed
    Units: H*ng/ml
        geometric mean (full range (min-max))
    0.198 (0.127 to 0.390)
    0.469 (0.261 to 0.841)
    No statistical analyses for this end point

    Other pre-specified: Mean Pulmonary Arterial Pressure - Week 16

    		 Close Top of page
    End point title
    		 Mean Pulmonary Arterial Pressure - Week 16
    End point description
    End point type
    Other pre-specified
    End point timeframe
    Week 16
    End point values
    		 Cohort 1
    Number of subjects analysed
    Units: mmHg
        arithmetic mean (standard error)
    -9.0 ± 4.3
    No statistical analyses for this end point

    Adverse events

    		 Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    Day 1 to 30 days after end of treatment.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22.1
    Reporting groups
    Reporting group title
    Cohort 1
    Reporting group description
    		 -

    Reporting group title
    Cohort 2
    Reporting group description
    		 -

    Serious adverse events
    		 Cohort 1 Cohort 2
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 7 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Hypoxia
         subjects affected / exposed
    1 / 8 (12.50%)
    0 / 7 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Cohort 1 Cohort 2
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    7 / 8 (87.50%)
    6 / 7 (85.71%)
    Investigations
    Brain natriuretic peptide increased
         subjects affected / exposed
    2 / 8 (25.00%)
    0 / 7 (0.00%)
         occurrences all number
    2
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    2 / 8 (25.00%)
    1 / 7 (14.29%)
         occurrences all number
    2
    1
    Gastrointestinal disorders
    Diarrhea
         subjects affected / exposed
    1 / 8 (12.50%)
    1 / 7 (14.29%)
         occurrences all number
    1
    1
    Nausea
         subjects affected / exposed
    2 / 8 (25.00%)
    0 / 7 (0.00%)
         occurrences all number
    2
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    4 / 8 (50.00%)
    1 / 7 (14.29%)
         occurrences all number
    4
    1
    Hypoxia
         subjects affected / exposed
    0 / 8 (0.00%)
    1 / 7 (14.29%)
         occurrences all number
    0
    1
    Throat irritation
         subjects affected / exposed
    1 / 8 (12.50%)
    3 / 7 (42.86%)
         occurrences all number
    1
    3

    More information

    		 Close Top of page

    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    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.
    The study was prematurely terminated due to the impact of COVID on recruitment. Due to the early termination, only the first two cohorts were enrolled and the LIQ861 dose of subjects in Part B was down titrated at the investigator's discretion.
    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-Thu May 02 15:05:23 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA