Circulating Activin-A and FSTL3 in Precapillary Pulmonary Hypertension (ACTIBIO)

Investigation of Circulating ACTIvin-A and FSTL3 as Prognostic and Predictive BIOmarkers in Precapillary Pulmonary Hypertension

The ACTIOBIO study aims to evaluate the prognostic value of Activin-A and FSTL3 in a large cohort of patients with precapillary pulmonary hypertension, including pulmonary arterial hypertension (Group 1), pulmonary hypertension associated with lung diseases (Group 3), and chronic thromboembolic pulmonary hypertension (Group 4).

Study Overview

• Status: Active, not recruiting
• Conditions: Pulmonary Hypertension
• Intervention / Treatment: Other: Biological collection

Detailed Description

In recent years, major advances have been achieved in understanding the cellular and molecular mechanisms underlying pulmonary arterial hypertension (PAH), a major subtype of precapillary PH. These discoveries have led to the development of novel therapeutic strategies, including agents targeting dysregulated pathways of the transforming growth factor beta (TGF-β) superfamily.

Parallel to therapeutic advances, risk stratification at diagnosis and during follow-up has become a cornerstone of PH management. Validated multidimensional tools now integrate clinical, exercise, biomarker, imaging, and hemodynamic parameters to estimate prognosis, guide initial treatment, and define therapeutic goals for follow-up. However, as new therapeutic targets emerge and influence disease trajectory, there is a growing need for novel biomarkers to refine these tools, making them less invasive and more accurate.

Beyond prognostic markers, identifying biomarkers that predict treatment response is essential to move toward precision medicine in PH. Such biomarkers would help tailor therapy to individual patient profiles and optimize long-term outcomes.

The main objective of this study is to analyze the prognostic value of Activin-A and Follistatin-like 3 (FSTL3) in a large cohort of patients with precapillary pulmonary hypertension, including pulmonary arterial hypertension (PAH, group 1), PH associated with lung disease (group 3), and chronic thromboembolic PH (group 4).

• Study Type: Interventional
• Enrollment (Estimated): 550
• Phase: Not Applicable

Contacts and Locations

Study Locations

• France
  • France
    • Le Kremlin-Bicêtre, France, France, 94270
      • Hôpital Bicêtre (AP-HP.Université Paris-Saclay), Service de Pneumologie et Soins Intensifs

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

1. Male or female ≥ 18 years of age

2. Precapillary PH documented by right heart catheterization:

   • mean pulmonary arterial pressure (mPAP) > 20 mmHg,
   • pulmonary artery wedge pressure (PAWP) ≤ 15 mmHg;
   • pulmonary vascular resistance (PVR) > 2 WU
3. Pulmonary arterial hypertension (group 1) or PH associated with lung disease (group 3) or chronic thromboembolic PH (group 4)
4. Patient registered in the French PH registry
5. Patient capable of understanding the study procedures
6. Affiliation to a social security regime (or exemption)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: No Intervention	Other: Biological collection; Serum, plasma, ADN

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Lung-transplant-free survival at 2 years	The primary endpoint is time fto lung-transplantation or death within two years following inclusion	Two years

Collaborators and Investigators

• Sponsor: Assistance Publique - Hôpitaux de Paris
• Investigators: Principal Investigator: Laurent SAVALE, Assistance Publique - Hôpitaux de Paris (AP-HP), Hôpital Bicètre

Study record dates

Study Major Dates

• Study Start (Actual): March 1, 2024
• Primary Completion (Estimated): April 1, 2026
• Study Completion (Estimated): April 1, 2028

Study Registration Dates

• First Submitted: November 19, 2025
• First Submitted That Met QC Criteria: November 19, 2025
• First Posted (Actual): November 28, 2025

Study Record Updates

• Last Update Posted (Actual): November 28, 2025
• Last Update Submitted That Met QC Criteria: November 19, 2025
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Keywords: biomarkers; Pulmonary Hypertension; FSTL3; Activin-A
• Additional Relevant MeSH Terms: Vascular Diseases; Cardiovascular Diseases; Respiratory Tract Diseases; Lung Diseases; Hypertension; Hypertension, Pulmonary
• Other Study ID Numbers: APHP231330; N° IDRCB: 2023-A02244-41 (Registry Identifier: ANSM)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No