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出境医 / 临床实验 / Extension Study for Participants in LIQ861 Trials to Evaluate the Long-term Safety of Dry Powder Inhalation of Treprostinil

Extension Study for Participants in LIQ861 Trials to Evaluate the Long-term Safety of Dry Powder Inhalation of Treprostinil

Study Description
Brief Summary:
The primary objective of this study is to evaluate the long-term safety of LIQ861 in patients with pulmonary arterial hypertension (PAH).

Condition or disease Intervention/treatment Phase
Primary Pulmonary Hypertension Drug: LIQ861 Inhaled Treprostinil Phase 3

Detailed Description:
One of the greatest impediments to patient treatment satisfaction with current inhaled treprostinil therapy is inconvenience. Currently, PAH patients using inhaled treprostinil may require more than 36 breaths per day using a nebulizer requiring daily set up and cleaning. The use of a discrete, hand-held dry powder inhaler to deliver treprostinil to the lungs could represent a major improvement in convenience and patient satisfaction, thereby improving the quality of life for PAH patients. Liquidia is pursuing approval of LIQ861, an inhalation dry powder formulation of treprostinil that is produced using Liquidia's PRINT® Technology (Particle Replication in Nonwetting Templates), as an alternative to current inhaled treprostinil therapy for the treatment of patients with PAH (WHO Group 1).
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 92 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: The study will evaluate the long-term safety of LIQ861 in PAH (WHO Group 1) patients who have completed a Liquidia LIQ861 clinical study. All patients will be treated on an outpatient basis until regulatory approval of LIQ861 or study is terminated by sponsor. Study enrollment will occur after final assessments from a prior study have been completed.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Global, Open-Label Extension Study for Participants in LIQ861 Trials to Evaluate the Long-term Safety of Inhaled LIQ861 (Treprostinil) in Pulmonary Arterial Hypertension (WHO Group 1) Patients
Actual Study Start Date : July 18, 2019
Estimated Primary Completion Date : December 31, 2021
Estimated Study Completion Date : March 30, 2022
Arms and Interventions
Arm Intervention/treatment
Experimental: LIQ861 Inhaled Treprostinil
LIQ861 inhaled treprostinil at capsule strengths of 25 μg, 50 μg, 75 μg and 100 μg. LIQ861 will be administered using the RS00 Model 8 dry powder inhalation (DPI) device (Plastiape S.p.A.; Osnago, Italy) at dose levels of 25 μg capsule strength to 200 μg capsule strength treprostinil four times a day (QID) in individual patients. Titrating to dose levels beyond 200 μg capsule strength QID, under clinical investigator supervision, requires review and approval from the Medical Monitor.
 Drug: LIQ861 Inhaled Treprostinil
LIQ861 bulk powder is generated from a treprostinil/excipient matrix from which particles of precise size and shape are created and filled into a hydroxypropyl methylcellulose (HPMC) capsule (size 3). LIQ861 capsules are provided in capsule strengths of 25 μg, 50 μg, 75 μg and 100 μg treprostinil.
Other Names:
  • Inhaled treprostinil
  • Inhaled prostacyclin
Outcome Measures
Primary Outcome Measures :
  1. Number of participants with treatment emergent adverse events (AEs). [ Time Frame: Baseline until the end of study, approximately 2.5 years (Dec-2021). ]
    Treatment-emergent adverse events and serious adverse events will be grouped by MedDRA System Organ Class, dose level at onset, time on drug at onset, and relationship to dose titration.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Evidence of a personally signed and dated informed consent document exists indicating that the patient has been informed of all pertinent aspects of the study prior to initiation of any study-related procedures.
  2. Patient is willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
  3. Patient has fulfilled all entry criteria at the time of enrollment in original study with LIQ861.
  4. Patient has completed the protocol defined end of study procedures or met a protocol-defined and adjudicated endpoint in the original LIQ861 study in which they were enrolled.
  5. Patient, whether male or female, agrees to use a medically acceptable method of contraception throughout the entire study period from informed consent through the termination visit, if the possibility of conception exists. Eligible male and female patients must also agree not to participate in a conception process (e.g., actively attempt to become pregnant or to impregnate, sperm donation, in vitro fertilization) during the study and for 30 days after the last dose of LIQ861.

Exclusion Criteria:

  1. Patient prematurely discontinued LIQ861 due to a drug-related AE/SAE or tolerability issue in the original LIQ861 study in which they were enrolled, or patient did not complete protocol defined study procedures at an end of study visit (not Early Termination visit) in their original LIQ861 study.
  2. Patient withdrew consent during participation in another LIQ861 study.
  3. Patient is a female who wishes to become pregnant or who has a positive pregnancy test on Day 1 (LTI-302 Study Initiation Visit).
  4. Patient has undergone lung or heart/lung transplant or the initiation of parenteral (intravenous [IV] infusion or subcutaneous injection) therapy with a prostacyclin during the time since participation in their original LIQ861 study.
  5. Any reason exists that, in the opinion of the Investigator or Medical Monitor, precludes the patient from participating in the study, e.g., any previous or intercurrent medical condition that may increase the risk associated with study participation or that would confound study analysis or impair study participation or cooperation.
Contacts and Locations

Locations
Show Show 25 study locations
Sponsors and Collaborators
Liquidia Technologies, Inc.
Nuventra, Inc.
Investigators
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Principal Investigator: Nicholas S Hill, MD Tufts Medical Center
Tracking Information
First Submitted Date  ICMJE June 5, 2019
First Posted Date  ICMJE June 20, 2019
Last Update Posted Date April 15, 2020
Actual Study Start Date  ICMJE July 18, 2019
Estimated Primary Completion Date December 31, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 18, 2019) 		Number of participants with treatment emergent adverse events (AEs). [ Time Frame: Baseline until the end of study, approximately 2.5 years (Dec-2021). ]
Treatment-emergent adverse events and serious adverse events will be grouped by MedDRA System Organ Class, dose level at onset, time on drug at onset, and relationship to dose titration.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Extension Study for Participants in LIQ861 Trials to Evaluate the Long-term Safety of Dry Powder Inhalation of Treprostinil
Official Title  ICMJE A Global, Open-Label Extension Study for Participants in LIQ861 Trials to Evaluate the Long-term Safety of Inhaled LIQ861 (Treprostinil) in Pulmonary Arterial Hypertension (WHO Group 1) Patients
Brief Summary The primary objective of this study is to evaluate the long-term safety of LIQ861 in patients with pulmonary arterial hypertension (PAH).
Detailed Description One of the greatest impediments to patient treatment satisfaction with current inhaled treprostinil therapy is inconvenience. Currently, PAH patients using inhaled treprostinil may require more than 36 breaths per day using a nebulizer requiring daily set up and cleaning. The use of a discrete, hand-held dry powder inhaler to deliver treprostinil to the lungs could represent a major improvement in convenience and patient satisfaction, thereby improving the quality of life for PAH patients. Liquidia is pursuing approval of LIQ861, an inhalation dry powder formulation of treprostinil that is produced using Liquidia's PRINT® Technology (Particle Replication in Nonwetting Templates), as an alternative to current inhaled treprostinil therapy for the treatment of patients with PAH (WHO Group 1).
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description:
The study will evaluate the long-term safety of LIQ861 in PAH (WHO Group 1) patients who have completed a Liquidia LIQ861 clinical study. All patients will be treated on an outpatient basis until regulatory approval of LIQ861 or study is terminated by sponsor. Study enrollment will occur after final assessments from a prior study have been completed.
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Primary Pulmonary Hypertension
Intervention  ICMJE Drug: LIQ861 Inhaled Treprostinil
LIQ861 bulk powder is generated from a treprostinil/excipient matrix from which particles of precise size and shape are created and filled into a hydroxypropyl methylcellulose (HPMC) capsule (size 3). LIQ861 capsules are provided in capsule strengths of 25 μg, 50 μg, 75 μg and 100 μg treprostinil.
Other Names:
  • Inhaled treprostinil
  • Inhaled prostacyclin
Study Arms  ICMJE Experimental: LIQ861 Inhaled Treprostinil
LIQ861 inhaled treprostinil at capsule strengths of 25 μg, 50 μg, 75 μg and 100 μg. LIQ861 will be administered using the RS00 Model 8 dry powder inhalation (DPI) device (Plastiape S.p.A.; Osnago, Italy) at dose levels of 25 μg capsule strength to 200 μg capsule strength treprostinil four times a day (QID) in individual patients. Titrating to dose levels beyond 200 μg capsule strength QID, under clinical investigator supervision, requires review and approval from the Medical Monitor.
Intervention: Drug: LIQ861 Inhaled Treprostinil
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: April 14, 2020) 		92
Original Estimated Enrollment  ICMJE
 (submitted: June 18, 2019) 		150
Estimated Study Completion Date  ICMJE March 30, 2022
Estimated Primary Completion Date December 31, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Evidence of a personally signed and dated informed consent document exists indicating that the patient has been informed of all pertinent aspects of the study prior to initiation of any study-related procedures.
  2. Patient is willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
  3. Patient has fulfilled all entry criteria at the time of enrollment in original study with LIQ861.
  4. Patient has completed the protocol defined end of study procedures or met a protocol-defined and adjudicated endpoint in the original LIQ861 study in which they were enrolled.
  5. Patient, whether male or female, agrees to use a medically acceptable method of contraception throughout the entire study period from informed consent through the termination visit, if the possibility of conception exists. Eligible male and female patients must also agree not to participate in a conception process (e.g., actively attempt to become pregnant or to impregnate, sperm donation, in vitro fertilization) during the study and for 30 days after the last dose of LIQ861.

Exclusion Criteria:

  1. Patient prematurely discontinued LIQ861 due to a drug-related AE/SAE or tolerability issue in the original LIQ861 study in which they were enrolled, or patient did not complete protocol defined study procedures at an end of study visit (not Early Termination visit) in their original LIQ861 study.
  2. Patient withdrew consent during participation in another LIQ861 study.
  3. Patient is a female who wishes to become pregnant or who has a positive pregnancy test on Day 1 (LTI-302 Study Initiation Visit).
  4. Patient has undergone lung or heart/lung transplant or the initiation of parenteral (intravenous [IV] infusion or subcutaneous injection) therapy with a prostacyclin during the time since participation in their original LIQ861 study.
  5. Any reason exists that, in the opinion of the Investigator or Medical Monitor, precludes the patient from participating in the study, e.g., any previous or intercurrent medical condition that may increase the risk associated with study participation or that would confound study analysis or impair study participation or cooperation.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03992755
Other Study ID Numbers  ICMJE LTI-302
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Liquidia Technologies, Inc.
Study Sponsor  ICMJE Liquidia Technologies, Inc.
Collaborators  ICMJE Nuventra, Inc.
Investigators  ICMJE
Principal Investigator: Nicholas S Hill, MD Tufts Medical Center
PRS Account Liquidia Technologies, Inc.
Verification Date April 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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