Sotatercept to support right ventricular function in pulmonary arterial hypertension
RECOMPENSE: 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
This research will try sotatercept injections in adults with PAH to see if the medicine helps the right side of the heart work better.
Quick facts
| Phase | Phase 4 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 18 Years to 70 Years |
| Sex | All |
| Sponsor | Amsterdam UMC, location VUmc Academic / other |
| Locations | 1 site (Amsterdam, North Holland) |
| Trial ID | NCT06658522 on ClinicalTrials.gov |
What this trial studies
This single-center, single-arm, open-label phase 4 study at Amsterdam UMC will enroll 20 adults with idiopathic or heritable PAH on stable background therapy. Participants will receive subcutaneous sotatercept (starting 0.3 mg/kg, target 0.7 mg/kg) and undergo invasive pressure–volume loop measurements via right heart catheterization to measure load-independent right ventricular function and RV–PA coupling. The study will compare pre-treatment and on-treatment RV contractility, dimensions, and hemodynamics including mPAP and PVR, alongside safety and biomarker monitoring. Results will clarify whether sotatercept's vascular effects translate into direct improvements in intrinsic RV performance.
Who should consider this trial
Good fit: Adults 18–70 years with idiopathic or heritable PAH (WHO Group I), WHO functional class II–IV, NT‑proBNP >300 ng/L, and hemodynamic confirmation by right heart catheterization (mPAP >20 mmHg, PCWP/LVEDP ≤15 mmHg, PVR ≥4 WU) who are on stable background PAH therapy are ideal candidates.
Not a fit: Patients with PAH due to left heart disease or other excluded etiologies, those with PCWP/LVEDP >15 mmHg, PVR <4 WU, outside the 18–70 age range, with unstable background therapy, or with contraindications to right heart catheterization or sotatercept are unlikely to benefit.
Why it matters
Potential benefit: If successful, sotatercept could lower pulmonary vascular resistance and improve right ventricular function, potentially reducing symptoms and slowing progression to right heart failure.
How similar studies have performed: Prior phase 2 and 3 trials of sotatercept showed significant reductions in PVR and improved exercise capacity, but produced only minimal increases in cardiac output, leaving direct effects on intrinsic RV function less clear.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Amsterdam, North Holland
- Amsterdam UMC, location VUMC — Amsterdam, North Holland, Netherlands (Recruiting)
Study contacts
- Principal investigator: Harm Jan Bogaard, M.D., PhD — Amsterdam UMC
- Study coordinator: Harm Jan Bogaard, M.D., PhD
- Email: hj.bogaard@amsterdamumc.nl
- Phone: +31 0 20 444 4444
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.