Pulmonary arterial hypertension (PAH) due to idiopathic pulmonary arterial hypertension (IPAH), familial PAH or PAH associated with connective tissue diseases (CTD) (e.g. systemic sclerosis, systemic lupus erythematosus) or to repaired congenital heart defects (RCHD). MedDRA version: 9.1 Level: LLT Classification code 10064911 Term: Pulmonary arterial hypertension MedDRA version: 9.1 Level: LLT Classification code 10065151 Term: Idiopathic pulmonary arterial hypertension MedDRA version: 9.1 L
Conditions
Interventions
Product Name: Aviptadil
Product Code: VIP
Pharmaceutical Form: Powder for nebuliser solution
INN or Proposed INN: Aviptadil
CAS Number: 40077-57-4
Other descriptive name: VIP
Concentration unit: mg/ml
Sponsors
MondoGEN AG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male and female patients between 18 and 80 years of age with IPAH, familial PAH or PAH associated with CTD or RCHD. 2. Patients with NYHA functional class II or III. 3. Patients who have received stabilized therapy with the endothelin receptor antagonist bosentan or the phosphodiesterase type 5 inhibitor sildenafil or a combination of both for at least 1 month prior to screening. 4. Symptomatic PAH diagnosed as either IPAH, familial PAH or PAH associated with CTD or RCHD clinically stable for at least 1 month prior to the inclusion. 5. An unencouraged 6-MWT of more than 200 meters at screening. 6. Cardiac catheterization consistent with PAH, specifically mean pulmonary arterial pressure (PAPm) = 25 mmHg (at rest), PCWP (or left ventricular end diastolic pressure) = 15 mmHg and PVR > 3 mmHg/L/min. 7. Echocardiogram consistent with PAH, specifically evidence of right ventricular hypertrophy or dilation, evidence of normal left ventricular function, and absence of mitral valve stenosis. 8. Have a chest radiograph consistent with the diagnosis of PAH. 9. Able to understand and willing to sign the Informed Consent Form. 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 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Patients with NYHA functional class I or IV. 2. Patients with PAH due to conditions other than IPAH, familial PAH or PAH associated with CTD or RCHD. 3. Patients who are on therapy with prostanoids, endothelin receptor antagonists other than bosentan, phosphodiesterase type 5 inhibitors other than sildenafil or calcium channel blockers. 4. Patients with Eisenmenger's Syndrome (a condition where the patient has a septal defect of the heart or a persistent ductus arteriosus and where resulting pulmonary hypertension creates a reversal of the shunt). 5. Patients who have a new type of chronic therapy (e.g. a different category of vasodilator, diuretic) for PAH added within the last month, except anticoagulants. 6. Patients who have received therapy with an investigational drug within 1 month prior to the start of this study or who are scheduled to receive another investigational drug during the course of this study. 7. Patients who have discontinued regular medication or who have changed dose or class of any medication within the last week, except anticoagulants. 8. Patients with a concomitant disease that could interfere with their ability to perform the 6-MWT. 9. Patients with an acute concomitant disease that could potentially complicate the assessment of PAH disease severity or response to therapeutic intervention. 10. Patients with any illness other than PAH which might reduce life expectancy to less than 6 months. 11. Patients incapable or unwilling to sign the informed consent. 12. Patients pregnant and/or lactating. 13. Patients with any pre-existing disease known to cause pulmonary hypertension unless listed in the inclusion criteria. The following pre-existing diseases are exclusionary: obstructive lung disease, parasitic disease affecting the pulmonary system, sickle cell anemia, mitral valve stenosis and portal hypertension. 14. Patients with a history of drug and/or alcohol abuse, as defined by the Investigator. 15. Females who are pregnant or plan to become pregnant during the study and females who are not using a highly effective method of birth control (failure rate less than 1% per year). 16. Patients with a positive NO vasodilator test at screening.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is to assess the efficacy of a number of doses of Aviptadil acutely and after 12 weeks of treatment.;Secondary Objective: The secondary objective of the study is to assess the safety and tolerability of a number of doses of Aviptadil.;Primary end point(s): Percentage change in pulmonary vascular resistance (PVR; peak PVR reduction compared to baseline PVR) after a single Aviptadil inhalation on Day 2, where baseline PVR is defined as the value measured when stability is reached after instrumentation of the patients and peak PVR is defined as the lowest value of PVR in post-baseline assessments (at 15, 30, 45, 60, 90, 120, 150 or 180 minutes). | — |
Countries
Austria, Belgium, France, Germany, Italy, Netherlands, Spain, United Kingdom
Outcome results
None listed