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Phase 2 N=21 Treatment

A Study of Sotatercept for the Treatment of Pulmonary Arterial Hypertension

Pulmonary Arterial Hypertension

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

OutcomeResultp-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
View full record on ClinicalTrials.gov →

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.

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