Trial record 2 of 2 for:
Study of ACT-064992 on Morbidity and Mortality in Patients With Symptomatic Pulmonary Arterial Hypertension
This study has been completed.
Information provided by (Responsible Party):
First received: April 14, 2008
Last updated: October 23, 2012
Last verified: October 2012
The AC-055-302/SERAPHIN study will be an event-driven Phase III study, comparing two different doses of ACT-064992 (3 and 10 mg) vs placebo in patients with symptomatic PAH. The main study objective is to demonstrate that ACT-064992 prolongs time to the first morbidity or mortality event, and to evaluate the benefit/risk profile of ACT-064992 in the treatment of patients with symptomatic PAH.
Pulmonary Arterial Hypertension
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
||A Multicenter, Double-blind, Randomized, Placebo-controlled, Parallel Group, Event-driven, Phase III Study to Assess the Effects of ACT-064992 on Morbidity and Mortality in Patients With Symptomatic Pulmonary Arterial Hypertension
Primary Outcome Measures:
- To demonstrate that either dose of ACT-064992 prolongs the time to first morbidity or mortality event in patients with symptomatic pulmonary arterial hypertension. [ Time Frame: End of Study ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- To evaluate the safety and tolerability of ACT-064992 in patients with symptomatic pulmonary arterial hypertension [ Time Frame: End of Study ] [ Designated as safety issue: Yes ]
| Study Start Date:
| Study Completion Date:
| Primary Completion Date:
||March 2012 (Final data collection date for primary outcome measure)
ACT-064992 tablet, 3 mg, once daily
Tablet, 3 mg dosage, once daily
ACT-064992 tablet, 10 mg, once daily
Tablet, 10 mg dosage, once daily
Placebo Comparator: 3
Matching ACT-064992 placebo, once daily
Matching ACT-064992 placebo, once daily
|Ages Eligible for Study:
||12 Years and older
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
- Signed informed consent prior to initiation of any study mandated procedure.
- Patients with symptomatic pulmonary arterial hypertension (PAH) in modified WHO functional class II to IV.
Patients with the following types of PAH belonging to groups 1.1 to 1.3 of the Venice classification:
- Idiopathic (IPAH);
- Familial (FPAH); or
- Collagen vascular disease;
- Simple, congenital systemic-to-pulmonary shunts at least 1 year post surgical repair;
- HIV infection; or
- Drugs and toxins.
PAH diagnosis confirmed by hemodynamic evaluation performed prior to randomization and showing all of the following:
- Mean pulmonary artery pressure (mPAP) > 25 mmHg at rest;
- Pulmonary capillary wedge pressure (PCWP) or left ventricular end diastolic pressure (LVEDP) < 15 mmHg; and
- Pulmonary vascular resistance (PVR) at rest >= 320 dyn×sec/cm5.
- 6-minute walk distance (6MWD) >= 50 m.
- Men or women > 12 years of age (women of childbearing potential must have a negative pre-treatment serum pregnancy test and must use a reliable method of contraception).
- PAH associated with portal hypertension, thyroid disorders, glycogen storage disease, Gaucher''s disease, hereditary hemorrhagic telangiectasia, hemoglobinopathies, myeloproliferative disorders or splenectomy.
- PAH associated with non corrected simple congenital systemic-to-pulmonary shunts, and combined and complex systemic-to-pulmonary shunts, corrected or non corrected.
- PAH associated with significant venous or capillary involvement (PCWP > 15 mmHg), known pulmonary veno-occlusive disease, and pulmonary capillary hemangiomatosis.
- Persistent pulmonary hypertension of the newborn.
- Pulmonary Hypertension belonging to groups 2 to 5 of the Venice classification.
- Moderate to severe obstructive lung disease: forced expiratory volume in 1 second/forced vital capacity (FEV1/FVC) < 70% and FEV1 < 65% of predicted value after bronchodilator administration.
- Moderate to severe restrictive lung disease: total lung capacity (TLC) < 60% of predicted value.
- Moderate to severe hepatic impairment, i.e., Child-Pugh Class B or C.
- Estimated creatinine clearance < 30 mL/min
- Serum aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) > 1.5 times the upper limit of normal.
- Hemoglobin < 75% of the lower limit of the normal range.
- Systolic blood pressure < 100 mmHg.
- Acute or chronic physical impairment (other than dyspnea), limiting the ability to comply with study requirements.
- Pregnant or breast-feeding.
- Known concomitant life-threatening disease with a life expectancy < 12 months.
- Body weight < 40 kg.
- Any condition that prevents compliance with the protocol or adherence to therapy.
- Recently started (< 8 weeks prior to randomization) or planned cardio-pulmonary rehabilitation program based on exercise.
- Treatment with endothelin receptor antagonists (ERAs) within 3 months prior to randomization.
- Systemic treatment within 4 week prior to randomization with cyclosporine A or tacrolimus, everolimus, sirolimus (calcineurin or mTOR inhibitors).
- Treatment with CYP3A inducers within 4 weeks prior to randomization
- Known hypersensitivity to drugs of the same class as the study drug, or any of their excipients.
- Planned treatment, or treatment, with another investigational drug within 1 month prior to randomization.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00660179
||Loic Perchenet, PhD
No publications provided
History of Changes
|Other Study ID Numbers:
|Study First Received:
||April 14, 2008
||October 23, 2012
||United States: Food and Drug Administration
United States: Institutional Review Board
Australia: Department of Health and Ageing Therapeutic Goods Administration
Austria: Federal Ministry for Health and Women
Belarus: Ministry of Health
Belgium: Federal Agency for Medicinal Products and Health Products
Canada: Health Canada
Chile: Instituto de Salud Publica de Chile
China: Food and Drug Administration
Finland: Finnish Medicines Agency
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: Federal Institute for Drugs and Medical Devices
Israel: Ministry of Health
Italy: The Italian Medicines Agency
Mexico: Ministry of Health
Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
Russia: Ministry of Health of the Russian Federation
Slovakia: State Institute for Drug Control
South Africa: Medicines Control Council
Taiwan: Department of Health
Ukraine: Ministry of Health
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Keywords provided by Actelion:
pulmonary arterial hypertension SERAPHIN
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on May 23, 2013
Respiratory Tract Diseases