NCT06658522 Right Ventricular Compensation With Sotatercept: A Prospective Single Arm Open Label Phase 4 Study to Evaluate the Effects of Sotatercept on Right Ventricular Function in Pulmonary Arterial Hypertension (RECOMPENSE)

Eligibility & Interventions

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What to Expect as a Participant

You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.

Phase 4 studies follow an already-approved treatment in real-world conditions to monitor long-term safety and effectiveness.

This trial targets 20 participants in total. It began in 2025-05-14 with a primary completion date of 2026-05-01.

Brief Summary

Pulmonary arterial hypertension (PAH) is a progressive disease characterized by vascular remodelling resulting in elevated pressures in the pulmonary artery (PA). This elevated pressure ultimately leads to fulminant right heart failure. Current therapeutic options are limited and are centred around vasodilatory medications such as phosphodiesterase-5 inhibitors and prostacyclin. While these medications allow for the widening of blood vessels that are narrowed due to remodelling, they have no effect on the remodelling itself. Sotatercept is a novel medication which targets the BMPR2/TGF-β pathway and restore a pro- and anti- proliferative balance to ultimately counteract vascular remodelling. Recent phase 2 and 3 trials showed that treatment with sotatercept led to lower resistance and pressure in the pulmonary vasculature and improved exercise tolerance. However, these results were not coupled with an increase in cardiac output, a change that is seen with other PAH-medications with a primarily vasodilatory effect. These results suggest that cardiac work is reduced and cardiac efficiency is improved in patients being treated with sotatercept, in contrast with conventional PAH therapies. This is a potentially beneficial effect that may result in improved disease control in the long-term. Our study aims to explore the effect of sotatercept on cardiac work and function. We hypothesize that the effects of sotatercept are more beneficial for cardiac function than that of traditional PAH medications. All participants included in the trial will undergo a screening visit in which it will be checked that all inclusion criteria and no exclusion criteria are met. The screening visit involves a physical exam, blood draw, 6-minute walk test, right heart catheterization (RHC) and cardiac magnetic resonance imaging (cMRI) with contrast to assess fibrosis. Upon inclusion, all participants will receive a subcutaneous injection of sotatercept starting at a dose of 0.3 mg/kg. Participants will return to the hospital every three weeks for a blood draw, physical examination and an adverse event review. If the laboratory values (specifically haemoglobin and platelet counts) stay stable after the first dose, the dosage will be escalated to 0.7 mg/kg. The dose will not be escalated beyond 0.7 mg/kg. After 24 weeks of receiving sotatercept, there will be an end of treatment visit including a physical exam, 6-minute walk test, right heart catheterization (RHC) and cardiac magnetic resonance imaging (cMRI) with contrast material.

Eligibility Criteria

Inclusion Criteria: 1. Adult patients between 18-70 years of age 2. Able to provide signed informed consent 3. WHO FC II to IV 4. NTproBNP \> 300 ng/L 5. PAH etiology belonging to one of the following groups (Nice classification): * Idiopathic PAH * Heritable PAH 6. Hemodynamic diagnosis of PAH confirmed by RHC during screening showing: * mPAP \> 20 mmHg * Pulmonary capillary wedge pressure (PCWP) or left ventricular end diastolic pressure (LVEDP) ≤ 15 mmHg * PVR ≥ 4WU (320 dyn.sec.cm-5) 7. For patients treated with oral diuretics, treatment dose must have been stable at least 1 month prior to RHC during the screening period 8. All patients are on stable background therapy at least 3 months prior to RHC during the screening period 9. Women of childbearing potential must have a negative pregnancy test at screening and agree to use reliable methods of contraception 10. Males must agree to use a condom during sexual contact with a pregnant female or female of childbearing potential while participating in the study. Males should refrain from donating blood or sperm for the duration for the study and for 16 weeks after last dose of sotatercept Exclusion Criteria: 1. Any contraindication to treatment with sotatercept 2. Body weight \< 40 kg 3. Body mass index (BMI) \> 35kg/m2 4. Pregnancy, breastfeeding, or intention to become pregnant during the study 5. Recently started (\< 8 weeks prior to informed consent signature) or planned cardio-pulmonary rehabilitation program 6. Known concomitant life-threatening disease with a life expectancy \< 12 months 7. Any condition likely to affect protocol or treatment compliance 8. Hospitalization for PAH within 3 months prior to informed consent signature 9. Left atrial volume per body surface area ≥ 43mL/m2 by echocardiography or CMR 10. Valvular disease grade 2 or higher 11. History of pulmonary embolism or deep vein thrombosis 12. Documented moderate to severe chronic obstructive pulmonary disease 13. Documented moderate to severe restrictive lung disease 14. Historical evidence of significant coronary artery disease 15. Diabetes mellitus 16. Active cancer 17. Systolic blood pressure \< 90 mmHg 18. Need for dialysis 19. Responders to acute vasoreactivity testing based on medical history 20. Treatment with another investigational drug (planned, or taken within the 3 months prior to study treatment initiation) 21. Claustrophobia 22. Permanent cardiac pacemaker, automatic internal cardioverter 23. Metallic implant (e.g. defibrillator, neurostimulator, hearing aid, infusion device) 24. Atrial fibrillation, multiple premature ventricular or atrial contractions , or any other condition that would interfere with proper cardiac gating during CMR. 25. History of major bleeding 26. Hemoglobin above ULN for age and gender

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