A national, multicenter, prospective, single-arm registry PASS of pulmonary hypertension patients treated with Riociguat (Adempas®) in China (MK-4836-001) (EXPERT China)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Drug utilisation

  Effectiveness study (incl. comparative)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  ADEMPAS
• Medical condition to be studied:
  Pulmonary arterial hypertension

  Pulmonary hypertension

Population studied

• Short description of the study population:
  Patients who have been prescribed Adempas® for a medically appropriate use will be eligible to be included into this registry. Indications and contraindications according to the local Chinese label for Adempas® should be carefully considered.; ; Inclusion Criterion/Criteria; • Patients who have been diagnosed with PAH or CTEPH; • Female and male patients who start or are on treatment with Adempas®; • WHO Function Class II-III for patients newly treated with Adempas®; • Written informed consent; ; Exclusion Criterion/Criteria; • Patients currently participating in an interventional clinical trial (If a patient is currently in the CHEST or PATENT (riociguat) long term extension trials, then the; patient can be considered for transition into EXPERT China study after the last dosing of riociguat.); • Female patient who is pregnant; • Patients with severe hepatic impairment (Child Pugh grade C); • Patients with SBP<95 mmHg when newly treated with Adempas®; • Patients who have been diagnosed with idiopathic interstitial pneumonia; • Co-administration with specific PDE 5 inhibitors (such as sildenafil, tadalafil or vardenafil) or nonspecific PDE 5 inhibitors (such as dipyridamole or theophylline) ; • Co-administration with nitrates or nitric oxide donors (such as amyl nitrite) in any form; • Any condition which, in the opinion of the investigator may confound the results or result in unwarranted risk in administering Adempas® to the patient.
• Age groups:
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)

      • Adults (65 to < 75 years)
      • Adults (75 to < 85 years)
      • Adults (85 years and over)
• Special population of interest:
  Other
• Special population of interest, other:
  Pulmonary Arterial Hypertension patients
• Estimated number of subjects:
  80

Study design details

• Main study objective:
  The primary objective is the assessment of long-term safety of Adempas® in real-world clinical practice.
• Outcomes:
  Incidence of adverse events Incidence of serious adverse events Incidence of all-cause mortality, Incidence of adverse events (AE) and Serious Adverse Events (SAE) in the different PH indications (PAH, CTEPH) Incidence of AE of special interest (Symptomatic hypotension and hemoptysis) Change from baseline in clinical parameters at follow up of PH participants Number of hospitalizations/outpatient visits Dosage and change in treatment for subgroups
• Data analysis plan:
  Analyses will be of an explorative and descriptive nature. There is no formal hypothesis testing. All background variables and outcome parameters will be analyzed descriptively with appropriate statistical methods: Categorical variables by frequency tables, and continuous variables by summary statistics. Continuous variables will also be described by absolute value and as change from baseline per analysis time point, if applicable. All analyses will be performed for the total study population (overall analysis) and separately for PH subtype and relevant subgroups, (e.g. age, gender, incident and prevalent patients, functional class at baseline, titrated dose), if patient numbers are sufficient.