Investigation of the Safety and Pharmacology of Dry Powder Inhalation of Treprostinil

Overview

  • Study type

    Interventional
  • Study phase

    III
  • Study IDs

  • Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

  • Site IRB
    • Rochester, Minnesota: 18-002201
    NCT ID: NCT03399604
    Sponsor Protocol Number: 18-002201

About this study

The primary objective of this study is to evaluate the long-term safety and tolerability of LIQ861, a dry powder formulation of treprostinil, in patients with Pulmonary Arterial Hypertension (PAH). A secondary objective of this study is to evaluate the comparative bioavailability of treprostinil between two formulations of inhaled therapy.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility criteria

Inclusion Criteria:

  • An Institutional Review Board (IRB) approved informed consent is signed and dated by the patient prior to any study-related activities.
  • The patient is 18 years of age or older.
  • If the patient is a female of childbearing potential, then the patient hasa negative pregnancy test at the Baseline Visit and agrees to practice adequate birth control throughout the duration of the study. If the patient is postmenopausal or has documented surgical sterilization, a pregnancy test and birth control is not necessary. It is the Investigator’s responsibility for determining whether the patient has adequate birth control for study participation.
  • The patient has been diagnosed with PAH belonging to the following subgroups of the updated Nice Clinical Classification Group 1, which include:
    • Idiopathic PAH (1.1); or
    • Heritable PAH (1.2); or
    • Drug and toxin induced PAH (1.3); or
    • PAH associated with connective tissue disease (1.4.1), HIV infection (1.4.2), or congenital heart disease (1.4.4) with simple systemic-to-pulmonary shunt at least 1 year after surgical repair.
  • The patient is NYHA Functional Class II - IV at Screening and:
    • has documented stable doses of Tyvaso for at least 3 months prior to screening and is willing and able to transition from their prescribed dose of inhaled therapy to study drug; or
    • has documented stable doses of no more than two approved oral therapies for at least 3 months prior to screening and is willing and able to add LIQ861 to their treatment regimen.
  • The patient can complete a baseline six-minute walk distance (6MWD) ≥ 150 m.
  • The patient has had evidence of FEV1 ≥ 60% and FEV1/FVC ratio ≥ 60% during the 6-month period prior to enrollment.

Exclusion Criteria:

 

  • The patient's clinical condition is such that, in the opinion of the Investigator, they are not expected to remain clinically stable for the duration of the study.
  • Patients with pulmonary hypertension (PH) in the Updated Nice Classification Groups 2-5, or PAH Group 1 subgroups not covered by the inclusion criteria (e.g., associated with portal hypertension [1.4.3] or with schistosomiasis [1.4.5])
  • The patient is currently taking oral prostacyclin analogues or agonists, including treprostinil and selexipag
  • The patient has had any PAH medication (except for anticoagulants) discontinued within 14 days of Baseline.
  • The patient has had a new type of chronic therapy (including but not limited to oxygen, a different class of vasodilator, diuretic, digoxin, and digitalis) for pulmonary hypertension added within 30 days of Baseline.
  • The patient has uncontrolled systemic hypertension as evidenced by persistent systolic blood pressure greater than 160 mmHg or diastolic blood pressure greater than 100 mmHg.
  • The patient has a history of hemodynamically significant left-sided heart disease including, but not limited to: aortic or mitral valve disease, pericardial constriction, restrictive or congestive cardiomyopathy, or coronary artery disease (CAD).
  • The patient has had an atrial septostomy.
  • The patient has any serious or life-threatening disease other than conditions associated with PAH (e.g., malignancy requiring aggressive chemotherapy, end stage renal disease, etc.).
  • The patient is taking any excluded medications listed in the Investigator’s Brochure, namely inhibitors and inducers of CYP2C8.
  • The patient has a hypersensitivity or allergy to any of the ingredients of LIQ861 or other clinically relevant allergies (clinical relevance per Investigator judgment).
  • The patient has had a pulmonary infarction (defined as infarction in more than one lung segment documented by V/Q scan or pulmonary angiography) within two weeks of Screening.
  • The patient has had a stroke or transient ischemic attack (TIA) within six months of Screening.
  • The patient has evidence of an active uncontrolled sepsis or systemic infection during Screening.
  • The patient is pregnant or lactating.
  • The patient has any musculoskeletal disease or any other disease that would limit ambulation.
  • The patient has participated in an investigational product or device study within the 30 days prior to Screening.
  • The patient has current evidence of drug abuse in the opinion of the Investigator.
  • The patient has severe hepatic impairment as evidenced by any history of ascites AND encephalopathy.
  • The patient has severe renal impairment (eGFR < 35).

Additional Exclusion Criteria for PK Sub-Study:

  • The patient meets any of Primary Exclusion Criteria above.
  • The patient has moderate or severe renal impairment (eGFR < 60).

 

 

 

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Hilary DuBrock, M.D.

Open for enrollment

Contact information:

Louise Durst R.N.

(507)284-1838

durst.louise@mayo.edu

.
CLS-20438955

Mayo Clinic Footer