Phase 2
N=21
A Study of Sotatercept for the Treatment of Pulmonary Arterial Hypertension
Pulmonary Arterial Hypertension
Bottom Line
View on ClinicalTrials.gov: NCT03738150 ↗Enrolled (actual)
21
Serious AEs
14.6%
Results posted
Feb 2023
Primary outcome: Primary: Change From Baseline in Peak Oxygen Uptake (VO2 Max) at 24 Weeks — 1.28 mL/min/kg
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Sotatercept (Biological); SOC (Other)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
- Primary completion
- Dec 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Peak Oxygen Uptake (VO2 Max) at 24 Weeks |
1.28 | — |
| SECONDARY Change From Baseline in Right Ventricular Stroke Volume (RV SV) at 24 Weeks |
-27.306 | — |
| SECONDARY Change From Baseline in Right Ventricular End-Systolic Volume (RV ESV) at 24 Weeks |
-39.151 | — |
| SECONDARY Change From Baseline in Right Ventricular End-Diastolic Volume (RV EDV) at 24 Weeks |
-66.459 | — |
| SECONDARY Percent Change From Baseline in Right Ventricular Ejection Fraction (RV EF) at 24 Weeks |
-1.3 | — |
| SECONDARY Change From Baseline in Right Ventricular Stroke Volume Index (RV SVI) at 24 Weeks |
-15.073 | — |
| SECONDARY Change From Baseline in Right Ventricular (RV) Mass at 24 Weeks |
-6.240 | — |
| SECONDARY Change From Baseline in Ventilatory Efficiency (VE/VCO2 Slope) at 24 Weeks |
-8.87 | — |
| SECONDARY Change From Baseline in Cardiac Index at 24 Weeks |
0.411 | — |
| SECONDARY Change From Baseline in Mean Pulmonary Arterial Pressure at 24 Weeks |
-14.9 | — |
| SECONDARY Change From Baseline in Arteriovenous O2 Content Difference (Ca-vO2) at 24 Weeks |
0.63 | — |
| SECONDARY Change From Baseline in Pulmonary Vascular Resistance (PVR) at 24 Weeks |
-305.728 | — |
| SECONDARY Change From Baseline in 6-Minute Walk Distance (6MWD) at 24 Weeks |
66.35 | — |
| SECONDARY Change From Baseline in Concentration of Amino-Terminal Brain Natriuretic Propeptide (NT-proBNP) at 24 Weeks |
-623.1 | — |
| SECONDARY Change From Baseline in WHO (World Health Organization) Functional Class at 24 Weeks |
-0.76 | — |
| SECONDARY Number of Participants With One or More Adverse Events (AEs) |
20 | — |
| SECONDARY Number of Participants Who Experienced One or More Events Indicative of Clinical Worsening of Pulmonary Arterial Hypertension (PAH) |
1 | — |
| SECONDARY Observed Trough Concentration (Ctrough) of Sotatercept at Cycle 9 |
5884.79 | — |
| SECONDARY Maximum Plasma Concentration (Cmax) of Sotatercept at Cycle 9 |
9732.785 | — |
| SECONDARY Minimum Plasma Concentration (Cmin) of Sotatercept at Cycle 9 |
6234.303 | — |
| SECONDARY Average Concentration (Cavg) of Sotatercept at Cycle 9 |
8221.907 | — |
| SECONDARY Area Under the Concentration-Time Curve From 0 to T (AUC0-T) of Sotatercept at Cycle 9 |
172659.953 | — |
| SECONDARY Apparent Terminal Half-life (t1/2 ) of Sotatercept at Cycle 9 |
21.919 | — |
| SECONDARY Apparent Serum Clearance (CL) of Sotatercept at Cycle 9 |
0.213 | — |
| SECONDARY Apparent Volume of Distribution (Vz/F) of Sotatercept at Cycle 9 |
3.969 | — |
| SECONDARY Absorption Rate Constant (Ka) of Sotatercept at Cycle 9 |
0.280 | — |
Summary
This study evaluates the effect of sotatercept (ACE-011) in adults with pulmonary arterial hypertension (PAH). Each eligible participant will receive standard of care (SOC) plus sotatercept (ACE-011) for a 24-week treatment period, followed by an 18-month extension period, and an 8-week follow-up period.
Eligibility Criteria
Inclusion Criteria
- Age ≥ 18 years
- Documented findings on RHC at any time prior to Screening consistent with a diagnosis of World Health Organization (WHO) pulmonary hypertension Group 1: PAH of any of the following subtypes:
- Idiopathic PAH
- Heritable PAH
- Drug- or toxin-induced PAH
- PAH associated with connective tissue disease
- PAH associated with simple, congenital systemic-to-pulmonary shunts at least 1 year following shunt repair
- Symptomatic pulmonary hypertension classified as WHO functional class III
- Screening RHC documenting a minimum PVR of ≥ 4 Wood units
- Pulmonary function tests within 6 months prior to Screening as follows:
- Total lung capacity > 70% predicted; or if between 60% to 70% predicted, or not possible to be determined, confirmatory high-resolution computed tomography (CT) indicating no more than mild interstitial lung disease per investigator interpretation or
- Forced expiratory volume (first second) (FEV1)/forced vital capacity (FVC) > 70% predicted
- For subjects with a history of lobectomy or pneumonectomy, and for whom there are no population-based normalization methods, assessment based on residual lung volume will be permitted to assess eligibility.
- Ventilation-perfusion (VQ) scan (or, if unavailable, a negative CT pulmonary angiogram [CTPA] or pulmonary angiography result), any time prior to Screening or conducted during Screening Period with normal or low probability result
- 6MWD ≥ 100 and ≤ 550 meters repeated twice during Screening Period and both values within 15% of each other, calculated from the highest value
- Combination PAH therapy at stable (per investigator) dose levels for at least 90 days prior to Cycle 1 Day 1 (C1D1)
Exclusion Criteria
Participants will be excluded from the study if they meet any of the following criteria:
- Started or stopped receiving any general supportive therapy for pulmonary hypertension (e.g., diuretics, oxygen, anticoagulants, digoxin) within 60 days prior to C1D1 (Cycle 1 Day 1)
- Received intravenous inotropes (e.g., dobutamine, dopamine, norepinephrine, vasopressin) within 30 days prior to C1D1
- History of atrial septostomy within 180 days prior to Screening
- History of more than mild obstructive sleep apnea that is untreated
- History of portal hypertension or chronic liver disease, defined as mild to severe hepatic impairment (Child-Pugh Classes A to C)
- History of human immunodeficiency virus infection-associated PAH
- Prior exposure to sotatercept (ACE-011) or luspatercept (ACE-536)
- Uncontrolled systemic hypertension as evidenced by sitting systolic BP > 160 mm Hg or sitting diastolic BP > 100 mm Hg during Screening after a period of rest
- Systolic BP 480 msec during Screening or C1D1
- History of personal or family history of long QTc syndrome or sudden cardiac death
- History of restrictive or constrictive cardiomyopathy
- Left ventricular ejection fraction 15 mm Hg on RHC during baseline evaluation
- Any current symptomatic coronary disease (myocardial infarction, percutaneous coronary intervention, coronary artery bypass graft surgery, or cardiac anginal chest pain in the past 6 months prior to Screening)
- Acutely decompensated heart failure within 30 days prior to C1D1, as per investigator assessment
- Significant (≥ 2+ regurgitation) mitral regurgitation or aortic regurgitation valvular disease
Data sourced from ClinicalTrials.gov (NCT03738150). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.