Clinical Trial: Efficacy and Tolerability of Subcutaneously Administered Treprostinil Sodium in Patients With Severe (Non-operable) Chronic Thromboembolic Pulmonary Hypertension (CTREPH)

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

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 Thromboembo

Brief 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.

Detailed Summary:

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.


Sponsor: SciPharm SàRL

Current Primary Outcome: 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 ]

Original Primary Outcome: To determine the effect on 6MWT distance after 24 weeks following sc Treprostinil Sodium or Control in patients with Severe (inoperable) Chronic Thromboembolic Pulmonary Hypertension

Current Secondary Outcome:

  • 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 ]


Original Secondary Outcome:

  • To assess the time to clinical worsening of CTEPH, defined as hospitalization with requirement for additional pulmonary hypertension treatment for worsening CTEPH, loss in functional NYHA class and/or death/transplantation due to worsening CTEPH.
  • To assess the effect on maximal Borg score during 6MWT
  • To assess the effect on WHO functional class
  • To assess the effect on QOL by the MINNESOTA instru
  • To assess the effect on brain natriuretic peptide (BNP) and N-terminal pro-BNP levels
  • To assess the effect on asymmetric dimethylarginine (ADMA) levels


Information By: SciPharm SàRL

Dates:
Date Received: August 12, 2011
Date Started: March 2009
Date Completion: December 2016
Last Updated: August 8, 2016
Last Verified: August 2016