A Two Part Clinical Study to Evaluate the Effect of Different Doses and the Safety of Inhaled Treprostinil Dry Powder in Pulmonary Arterial Hypertension Patients
- Conditions
- Pulmonary Arterial Hypertension (WHO Group 1)MedDRA version: 20.0Level: LLTClassification code 10077739Term: Pulmonary arterial hypertension WHO functional class ISystem Organ Class: 100000004855Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
- Registration Number
- EUCTR2018-003414-40-FR
- Lead Sponsor
- iquidia Technologies, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 32
1. An Institutional Review Board (IRB) approved informed consent is signed and dated by the subject prior to any study-related activities.
2. The subject is 18 years of age or older.
3. If the subject is a female of childbearing potential, then the subject has a negative pregnancy test at the Day 1 Visit and agrees to practice a highly effective (failure rate of less than 1% per year when used consistently and correctly) method of birth control throughout the duration of the study. If the subject 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 subject has adequate birth control for study participation.
4. The subject has been diagnosed with Pulmonary Arterial Hypertension (PAH) belonging to one of the following subgroups of the updated Nice Clinical Classification Group 1, which includes:
a. Idiopathic PAH (1.1), or
b. Heritable PAH (1.2), or
c. Drug and toxin induced PAH (1.3), or
d. 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
5. The subject is NYHA Functional Class II - IV at Screening and:
a. has not previously been treated for PAH, or
b. has documented stable doses of no more than 2 approved non-prostacyclin therapies for at least 3 months prior to screening and is willing and able to add LIQ861 to their treatment regimen.
6. The subject can complete a baseline six-minute walk distance (6MWD) = 150 m.
7. The subject has had evidence of Forced Expiratory Volume in 1 Second (FEV1) = 60% and FEV1/ Forced Vital Capacity (FVC) ratio = 60% during the 6-month period prior to consent.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 19
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 13
1. The subject'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.
2. Subjects with pulmonary hypertension (PH) in the Updated Nice Classification Groups 2-5, or Pulmonary Arterial Hypertension (PAH) Group 1 subgroups not covered by the inclusion criteria (e.g., associated with portal hypertension or with schistosomiasis)
3. The subject is currently taking prostacyclin analogues or agonists, including treprostinil, iloprost, and selexipag
4. The subject has had any PAH medication (except for anticoagulants) discontinued within 14 days prior to Day 1.
5. The subject 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 prior to Day 1.
6. The subject has uncontrolled systemic hypertension as evidenced by persistent systolic blood pressure greater than 160 mmHg or diastolic blood pressure greater than 100 mmHg.
7. The subject 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).
8. The subject has had an atrial septostomy.
9. The subject has any serious or life-threatening disease other than conditions associated with PAH.
10. The subject is taking any excluded medications listed in the Investigator’s Brochure, namely inhibitors and inducers of CYP2C8 (See Appendix 3 in the Protocol).
11. The subject has a hypersensitivity or allergy to any of the ingredients of LIQ861, NO, or other clinically relevant allergies (clinical relevance per Investigator judgment).
12. The subject 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.
13. The subject has had a stroke or transient ischemic attack (TIA) within six months of Screening.
14. The subject has evidence of an active uncontrolled sepsis or systemic infection during Screening.
15. The subject is pregnant or lactating.
16. The subject has any musculoskeletal disease or any other disease that would limit ambulation.
17. The subject has participated in an investigational product or device study within the 30 days prior to Screening.
18. The subject has current evidence of drug abuse in the opinion of the Investigator.
19. The subject has severe hepatic impairment as evidenced by any history of ascites AND encephalopathy.
20. The subject has severe renal impairment (estimated glomerular filtration rate [eGFR] < 35).
21. The subject is an employee or an immediate family member to an employee of the sponsor or the Investigator.
22. The subject is not a member or beneficiary of a social security scheme.
23. The subject is not subject to a legal protection measure.
24. The subject has not been deprived of their liberty by a judicial or administrative decision.
25. The subject has a known Hepatitis B, Hepatitis C, or HIV infection.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method