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)

Recruiting now · Phase 4

Conditions studied: Pulmonary Arterial Hypertension PAH, Pulmonary Arterial Hypertension WHO Group I, Pulmonary Arterial Hypertension

In brief

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.

Key facts

Study ID: NCT06658522
Run by: Amsterdam UMC, location VUmc
People needed: 20
Starts: 2025-05-14
Expected to finish: 2026-05-01
Last updated by the study team: 2025-09-22

Who can join

Age: 18 and older, up to 70. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Adult patients between 18-70 years of age
Able to provide signed informed consent
WHO FC II to IV
NTproBNP \> 300 ng/L
PAH etiology belonging to one of the following groups (Nice classification):
Idiopathic PAH
Heritable PAH
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)
For patients treated with oral diuretics, treatment dose must have been stable at least 1 month prior to RHC during the screening period
All patients are on stable background therapy at least 3 months prior to RHC during the screening period
Women of childbearing potential must have a negative pregnancy test at screening and agree to use reliable methods of contraception
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

You may not qualify if…

Any contraindication to treatment with sotatercept
Body weight \< 40 kg
Body mass index (BMI) \> 35kg/m2
Pregnancy, breastfeeding, or intention to become pregnant during the study
Recently started (\< 8 weeks prior to informed consent signature) or planned cardio-pulmonary rehabilitation program
Known concomitant life-threatening disease with a life expectancy \< 12 months
Any condition likely to affect protocol or treatment compliance
Hospitalization for PAH within 3 months prior to informed consent signature
Left atrial volume per body surface area ≥ 43mL/m2 by echocardiography or CMR
Valvular disease grade 2 or higher
History of pulmonary embolism or deep vein thrombosis
Documented moderate to severe chronic obstructive pulmonary disease
Documented moderate to severe restrictive lung disease
Historical evidence of significant coronary artery disease
Diabetes mellitus
Active cancer
Systolic blood pressure \< 90 mmHg
Need for dialysis
Responders to acute vasoreactivity testing based on medical history
Treatment with another investigational drug (planned, or taken within the 3 months prior to study treatment initiation)
Claustrophobia
Permanent cardiac pacemaker, automatic internal cardioverter
Metallic implant (e.g. defibrillator, neurostimulator, hearing aid, infusion device)
Atrial fibrillation, multiple premature ventricular or atrial contractions , or any other condition that would interfere with proper cardiac gating during CMR.
History of major bleeding

Where it is running

Amsterdam UMC, location VUMC — Amsterdam, North Holland, Netherlands (enrolling)

Full record on ClinicalTrials.gov

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