Selexipag for the Treatment of Schistosomiasis-Associated Pulmonary Arterial Hypertension

Phase 2

• Conditions: Pulmonary Hypertension; Schistosomiasis
• Interventions: Drug: Selexipag

• Registration Number: NCT04589390
• Lead Sponsor: University of Sao Paulo General Hospital

Brief Summary

Pulmonary arterial hypertension (PAH) is a severe, progressive and potentially fatal disease that impairs the pulmonary circulation and leads to right ventricular failure. One of the world most prevalent etiologies of PAH is schistosomiasis-associated pulmonary arterial hypertension (Sch-PAH). New drugs have emerged to treat other forms of PAH, but their benefits cannot be automatically translated for Sch-PAH patients, since this etiology was not included in the pivotal PAH trials. One of the most promising therapies for the treatment of PAH to emerge in recent years is selexipag, an oral IP receptor agonist, which acts on the prostacyclin pathway. The present study aims to evaluate the efficacy, safety and tolerability of selexipague for the treatment of schistosomiasis-associated pulmonary arterial hypertension.

Detailed Description

Not available

Study Timeline

• Status Verified: 2020-10
• Study First Submitted: 2020-10-08
• Study First Submitted QC: 2020-10-08
• Last Update Submitted: 2020-10-08
• Study Start: 2020-10-15
• Study First Posted: 2020-10-19
• Last Update Posted: 2020-10-19
• Primary Completion: 2021-11-30
• Study Completion: 2022-03-31

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

• Patients with symptomatic Sch-PAH. Sch-PAH diagnosis necessarily include the three criteria below

  1. Invasive confirmation of PAH, according to the criteria defined in the Pulmonary Hypertension Sixth World Symposium: mean pulmonary artery pressure higher than 20 mmHg, at rest, and the presence of pulmonary vascular resistance (PVR) equal to or greater than 3 W, and a pulmonary capillary pressure considered normal (equal to or lower than 15 mmHg (1)).
  2. At least one epidemiological criteria for chronic schistosomiasis: patient from a highly prevalent region for schistosomiasis or previous history of parasitic treatment for schistosomiasis or the presence of Schistosoma mansoni eggs in the patient's feces
  3. Evidence of long-term hepatosplenic involvement by schistosomiasis, via compatible ultrasound findings (peri-portal fibrosis or enlarged left lobe) All patients will necessarily already be receiving at least one specific treatment for PAH, either with phosphodiesterase V inhibitor or with an endothelin receptor antagonist, with a stable dose for at least 12 weeks before inclusion in the study.

Exclusion Criteria

• Patient without clinical condition to perform the 6-minute walk test
• Patient with gastro-intestinal bleeding for over 12 weeks

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Selexipag	Selexipag	Selexipag will be up titrated for a period that will last 12 weeks (Phase 2). The initial dose will be 200 mcg of selexipag every 12 hours, with weekly dose increases of 200 mcg, up to the maximum dose of 1600 mcg every 12 hours or until the classic side effects of the prostacyclin pathway drugs (headache, mandibular pain), among others) arise. The dose will then be reduced by 200 mcg per dose, and this will be the maximum dose considered for that particular patient, maintained in Phase 3 (16 weeks).

Primary Outcome Measures

Name	Time	Method
Pulmonary vascular resistance	16 weeks

Secondary Outcome Measures

Name	Time	Method
6MWT	16 weeks	Lenght in the six minute walking distance test
FC	16 weeks	New York Heart Association Functional Class
BNP	16 weeks	Brain Natriuretic Peptide
HSP 70	16 weeks	Heat shock protein 70

Trial Locations

Locations (1)

Universidade de São Paulo; 🇧🇷 São Paulo, SP, Brazil