Overview

This trial is active, not recruiting.

Condition non-operable chronic thromboembolic pulmonary hypertension
Treatment treprostinil sodium
Phase phase 3
Sponsor SciPharm SàRL
Start date March 2009
End date December 2016
Trial size 100 participants
Trial identifier NCT01416636, 116-02

Summary

The primary purpose of this study it to determine the effect on 6 minute walking test (MWT) distance after 24 weeks treatment with subcutaneous (SC) Treprostinil Sodium in patients with Severe (inoperable) Chronic Thromboembolic Pulmonary Hypertension.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking double blind (subject, caregiver, investigator, outcomes assessor)
Primary purpose treatment
Arm
(Experimental)
treprostinil sodium
(Active Comparator)
treprostinil sodium

Primary Outcomes

Measure
To determine the effect of subcutaneous Treprostinil sodium on 6MWT distance after 24 weeks in patients with severe non-operable chronic thromboembolic pulmonary hypertension severe (inoperable) Chronic Thromboembolic Pulmonary Hypertension
time frame: 24 weeks

Secondary Outcomes

Measure
To assess the time to clinical worsening
time frame: 24 weeks
To assess the effect on maximal Borg score, heart rate and oxygen saturation during 6MWT
time frame: 24 weeks
To assess the effect on WHO NYHA (World health organization- New York Heart Association) functional class
time frame: 24 weeks
To assess the effect on QOL (Quality of Life) by the MINNESOTA questionnaire
time frame: 24 weeks
To assess the effect on N-terminal pro-BNP levels
time frame: 24 weeks
To assess the effect on hemodynamic parameters (PVR, mPap, mRap (Mean right atrial pressure, CO (Cardiac Output), CI (Cardiac Index))
time frame: 24 weeks
To assess the effect on signs & symptoms of the CTEPH
time frame: 24 weeks
To assess the treatment - emergent Adverse Events (AE's), Serious Adverse Events (SAE's) , AE's leading to discontinuation and relevant laboratory abnormalities
time frame: 24 weeks

Eligibility Criteria

Male or female participants at least 18 years old.

1. Subject must be competent to understand the information given in the written informed consent and from the investigator and must sign and date the informed consent prior to any study mandated procedure. 2. Subject must be at least 18 years of age and can be of any ethnical origin 3. Women of child bearing potential must be surgically sterile or postmenopausal (amenorrhea for at least 12 months) or using an acceptable form of contraception. Reliable contraception is defined as a method which results in a low failure rate, i.e., less than 1% per year when used correctly such as, implants, injectables, oral contraceptive medications, sexual abstinence, or a vasectomised partner. 4. Subject must have a current diagnosis of CTEPH, as defined by the following criteria: - A test result of perfusion scintigraphy and pulmonary angiography and/or multislice CT not older than 6 months, consistent with the diagnosis CTEPH. In case of recurrent PH after PEA, test results from before the surgery are acceptable if a typical specimen was harvested during PEA substantiating the diagnosis of CTEPH. - A right heart catheterization, not older than 6 months, consistent with the diagnosis CTEPH but specifically with a PAPm of > 25 mmHg, and a PVR of > 300 dyn.s.cm-5 - At least three months of effective anticoagulation therapy (without improvement / to exclude subacute pulmonary emboli) 5. Subject must have CTEPH classified as severe, as defined by the following criteria: - An un-encouraged 6MWT distance of between 150 and 400 meters - Classification in the WHO/NYHA functional class III or IV 6. The subject must not be suitable to undergo a PEA and is therefore defined as non-operable, due to at least one of the following reasons: - Clot is not accessible - Discrepancy between severity of PH and morphologic lesion - Subject is not a good surgical candidate for other reasons: PVR > 1500 dynes.s.cm-5 Age Comorbidity No functional lung parenchyma - Unsuccessful PEA in the past with residual/recurrent CTEPH - No consent for PEA given by subject 7. Subject must be willing and able to follow all study procedures Exclusion: 1. Subject with any form of pulmonary arterial hypertension or any disease known to cause PAH (WHO Group I) 2. Subjects with a total lung capacity (TLC) of < 70% predicted or a forced expiratory volume/forced capacity (FEV1/FVC < 50%) 3. Subject who received any prostanoids, within the 30 days before screening or be scheduled to receive prostanoids during the course of the study 4. Subject with a new type of chronic therapy (a different category of vasodilator or diuretic) for PAH added within the last month, except anticoagulants 5. Subject with an increased risk for hemorrhage or stroke or with a major cardiovascular event during the past 6 months. 6. Unstable subjects for any reason (according to the investigators discretion) 7. Subject who received any investigational medication within 30 days prior to the screening visit of this study or be scheduled to receive another investigational drug during the course of this study 8. Subject with a known intolerance to any drug relevant for this trial, especially to Treprostinil sodium or prostanoids 9. Subject with a history or suspicion of non compliance 10. Subject who has any musculoskeletal disease or any other disease that would limit ambulation 11. Subject with other cardiovascular, liver, renal, hematologic, gastrointestinal immunologic, endocrine, metabolic, or central nervous system disease that, in the opinion of the investigator, may adversely affect the safety of the subject and /or efficacy of the study drug or limit the lifespan of the subject 12. Female who is considering pregnancy or who is pregnant and/or lactating 13. Subject who is an investigator or any other team member involved directly or indirectly in the conduct of the clinical study. 14. Subject who is an inmate of a psychiatric ward, prison or is suspected not to be able to give consent of his free will

Additional Information

Official title A Double Blind Controlled Clinical Study to Investigate the Efficacy and Tolerability of Subcutaneous Treprostinil Sodium in Patients With Severe Non-operable Chronic Thromboembolic Pulmonary Hypertension (CTREPH)
Principal investigator Irene Lang, MD
Description Chronic thromboembolic pulmonary hypertension (CTEPH) is characterized by non-resolving organized thromboembolic obstructing the pulmonary vascular bed (Lang, 1994). These thrombi are resistant to thrombolytic therapy and chronic plasmatic anticoagulation. An increase in pulmonary vascular resistance (PVR), right ventricular overload, and eventually right ventricular failure ensue. The treatment of choice for CTEPH is pulmonary endarterectomy (PEA), providing a potential cure for the disease (Klepetko, 2004)). However, about 50 % of patients are not candidates for surgery, mainly because of distal location of thromboemboli. Despite recent advances in the treatment of pulmonary arterial hypertension (PAH) (McLaughlin, 2004), medical treatments have not been recommended for inoperable CTEPH, because of the concept that a predominantly major vessel obstructive arteriopathy would not be suitable for vasodilators. Furthermore, a major drawback of i.v. prostacyclin therapy is the need for a permanent central venous access that increases the risk of infection (0.22-0.68 per patient per year) (Kuhn, 2003), thrombosis and new major vessel thromboembolism.
Trial information was received from ClinicalTrials.gov and was last updated in August 2016.
Information provided to ClinicalTrials.gov by SciPharm SàRL.