Use, safety and Tolerability of IntraVenous epOprostenoL (Veletri®) in patients with severe pulmonary arterIal hypertension: A 6-month, open label, multicenter,observational, non-interventional study (TIVOLI)

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

  Other
• Non-interventional study design, other:
  Open-label, observational and prospective study

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  EPOPROSTENOL
• Medical condition to be studied:
  Pulmonary hypertension

Population studied

• Short description of the study population:
  Patients with PAH will receive medically indicated i.v. epoprostenol (Veletri®) according to a systematic drug management including initiation, administration, storage, handling of the infusion system and patient education. Patients in need of treatment escalation according to the current guidelines receiving at least dual oral combination treatment with two PAH-targeted drugs and having an unsatisfying long-term clinical response or are still in an intermediate or high risk group will be eligible for the study.; ; Eligibility criteria; Only patients with confirmed PAH already on targeted therapy who needed treatment escalation with i.v. epoprostenol were eligible to participate if they fulfilled the following criteria: This trial included adult patients of both sexes with confirmed PAH able and willing to give written informed consent. Eligible patients needed treatment escalation with i.v. epoprostenol according to current guidelines, with PAH confirmed by right heart catheter (RHC) before enrolment (i.e., mPAP ≥25 mmHg at rest, PAWP ≤15 mmHg).; Exclusion criteria comprised a known intolerance to epoprostenol or one of its excipients, pregnancy or lactation. Moreover, patients participating in any other clinical drug trial within four weeks prior to screening and/or patients scheduled to receive any investigational medicinal product (IMP) during the course of this trial were not eligible
• Age groups:
  Adults (18 to < 46 years)

  Adults (46 to < 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 hypertension patients
• Estimated number of subjects:
  15

Study design details

• Main study objective:
  To collect and explore real-life data on the use, safety and tolerability of a systematic drug management of intravenous epoprostenol (Veletri®) treatment in patients with PAH. Real-life safety and tolerability data will be obtained by listings of the frequency of i.v. epoprostenol (Veletri®)-associated adverse drug reactions (ADRs), serious adverse events (SAEs).
• Outcomes:
  Frequency of adverse events and serious adverse events, To assess the real world clinical effectiveness of i.v. epoprostenol (Veletri®) - demography - vital signs - exercise capacity as measured by six minute walking distance - N-terminal pro-brain natriuretic peptide (NTproBNP) - WHO functional class - Borg dyspnea score - blood gas analysis - right heart size and function - haemodynamics - symptoms of PAH - outcome
• Data analysis plan:
  Descriptive statistics: - All data (demographic and other baseline characteristics, continuous data at each visit and their change to baseline) will be listed and trial summary tables will be provided. The data from all centers will be pooled. - Descriptive statistics will be displayed (arithmetic mean, median, standard deviation, standard error, 95% confidence limits of mean and median, first and third quartiles, minimum, and maximum for quantitative variables). Frequency tables for qualitative data will be provided.

Documents

Study results

2022-02-24 VELETRI observational study report clean (2)