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A Study of Sotatercept in Japanese Pulmonary Arterial Hypertension (PAH) Participants (MK-7962-020)

Phase 3
Active, not recruiting
Conditions
Pulmonary Arterial Hypertension
Interventions
Biological: Sotatercept
Registration Number
NCT05818137
Lead Sponsor
Merck Sharp & Dohme LLC
Brief Summary

This local Phase 3 study is planned to confirm the efficacy and safety in Japanese PAH participants. The primary population of this study is Japanese PAH participants with World Health Organization Functional Class (WHO FC) II or III while the study includes PAH participants with WHO FC I or IV as other populations. There are no hypotheses for this study.

Detailed Description

Not available

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
46
Inclusion Criteria
  • Documented diagnostic right heart catheterization (RHC) at any time prior to screening confirming the diagnosis of World Health Organization (WHO) pulmonary arterial hypertension (PAH) Group 1 in any of the following subtypes:

    • Idiopathic PAH
    • Heritable PAH
    • Drug/toxin-induced PAH
    • PAH associated with connective tissue disease
    • PAH associated with simple, congenital systemic-to-pulmonary shunts at least 1 year following repair
  • PAH classified as WHO functional class (FC) I or symptomatic PAH classified as WHO FC II to IV

  • On stable doses of background PAH therapy and diuretics (if applicable) for at least 90 days prior to screening

Exclusion Criteria

  • Diagnosis of PH WHO Groups 2, 3, 4, or 5

  • Diagnosis of the following PAH Group 1 subtypes:

    • Human immunodeficiency virus (HIV)-associated PAH
    • PAH associated with portal hypertension
    • Schistosomiasis-associated PAH
    • PAH with features of significant venous/capillary pulmonary veno-occlusive disease/pulmonary capillary hemangiomatosis (PVOD/PCH) involvement
  • Is on the waiting list for lung transplant

  • Pregnant or breastfeeding women

  • History of full or partial pneumonectomy

  • Pulmonary function test (PFT) values of forced vital capacity (FVC) < 60% predicted at the screening visit or within 6 months prior to the screening visit.

  • Initiation of an exercise program for cardiopulmonary rehabilitation within 90 days prior to the screening visit or planned initiation during the study.

  • History of more than mild obstructive sleep apnea that is untreated

  • Known history of portal hypertension or chronic liver disease, including hepatitis B and/or hepatitis C (with evidence of recent infection and/or active virus replication), defined as mild to severe hepatic impairment.

  • History of restrictive, constrictive, or congestive cardiomyopathy.

  • History of atrial septostomy within 180 days prior to the screening visit.

  • Personal or family history of long QT syndrome (LQTS) or sudden cardiac death.

  • Left ventricular ejection fraction (LVEF) < 45% on historical Echocardiogram (ECHO) within 6 months prior to the screening visit.

  • Any symptomatic coronary disease events within 6 months prior to the screening visit.

  • Cerebrovascular accident within 3 months prior to the screening visit.

  • Significant (≥ 2+ regurgitation) mitral regurgitation or aortic regurgitation valvular disease, mitral stenosis and more than mild aortic valve stenosis.

  • Prior exposure to sotatercept or luspatercept or history of allergic or anaphylactic reaction or hypersensitivity to recombinant proteins or excipients in investigational product

  • Received intravenous inotropes (e.g., dobutamine, dopamine, norepinephrine, vasopressin) within 30 days prior to the screening visit

  • Currently enrolled in or have completed any other investigational product study within 30 days

  • Weight at the screening is over 85 kg

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
SotaterceptSotaterceptParticipants on background PAH therapy will receive sotatercept subcutaneous (SC) injections at a starting dose of 0.3 mg/kg with a target dose of 0.7 mg/kg every 3 weeks up to 24 weeks. Thereafter, participants may choose to continue receiving the treatment until approval of sotatercept in Japan.
SotaterceptSotaterceptParticipants on background PAH therapy will receive sotatercept subcutaneous (SC) injections at a starting dose of 0.3 mg/kg with a target dose of 0.7 mg/kg every 3 weeks up to 24 weeks. Thereafter, participants may choose to receive the sotatercept treatment at same dose and schedule in the extension treatment period from Week 24 to Week 42.
Primary Outcome Measures
NameTimeMethod
Change From Pulmonary Vascular Resistance (PVR) From Baseline at Week 24Baseline and Week 24

PVR was the resistance against blood flow from the pulmonary artery to the left atrium. PVR was measured in dyn\*sec/cm\^5 by right heart catheterization (RHC). RHC was performed during the screening period (baseline) and Week 24. Per protocol, the change in PVR from baseline at Week 24 was reported for the primary treatment period.

Number of Participants Who Experienced an Adverse Event (AE)Up to ~24 weeks

An AE was any untoward medical occurrence in a participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. Per protocol, the number of participants who experienced an AE were reported for the primary treatment period.

Number of Participants Who Discontinued Study Intervention Due to AEsUp to ~24 weeks

An AE was any untoward medical occurrence in a participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. Per protocol, the number of participants who discontinued study treatment due to AEs were reported for the primary treatment period.

Secondary Outcome Measures
NameTimeMethod
Change From Baseline in Six-Minute Walk Distance (6MWD) at Week 24Baseline and Week 24

The 6MWD was the distance walked in 6 minutes as a measure of functional capacity was measured during the screening period (baseline) and at Week 24. This was assessed using the 6-minute walk test (6MWT). Per protocol, the change from baseline in 6MWD at Week 24 was reported for the primary treatment period.

Percentage of Participants With Improvement in World Health Organization Functional Class (WHO FC) at Week 24Baseline and Week 24

The severity of participant's pulmonary arterial hypertension (PAH) symptoms will be graded using the WHO FC system. WHO functional classification for PAH ranges from Class I (no limitation in physical activity, no dyspnea with normal activity), Class II (slight limitation of physical activity), Class III (marked limitation of physical activity) and Class IV (cannot perform a physical activity without any symptoms, dyspnea at rest). Participants who improve in WHO FC were classified into "Improved", "No change" and "Worsened". Improvement = reduction in FC, worsened = increase in FC and no change = no change in FC. Per protocol, the percentage of participants with improvement in WHO FC at Week 24 were presented for the primary treatment period.

Change From Baseline in N-terminal proB-type Natriuretic Peptide (NT-proBNP) at Week 24Baseline and Week 24

NT-proBNP is an established marker of ventricular dysfunction in participants with PAH. NT-proBNP was measured at Day 1 (baseline) and at Week 24. The change from baseline in NT-proBNP at Week 24 was reported for the primary treatment period.

Trial Locations

Locations (17)

Hokkaido University Hospital ( Site 2001)

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Sapporo, Hokkaido, Japan

Chiba Saiseikai Narashino hospital ( Site 2004)

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Narashino, Chiba, Japan

Nagoya University Hospital ( Site 2010)

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Nagoya-Shi, Aichi, Japan

Kurume University Hospital ( Site 2014)

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Kurume, Fukuoka, Japan

Kure Kyosai Hospital ( Site 2017)

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Kure, Hiroshima, Japan

Sapporo Medical University Hospital ( Site 2018)

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Sapporo, Hokkaido, Japan

Kobe University Hospital ( Site 2012)

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Kobe, Hyogo, Japan

National Cerebral and Cardiovascular Center ( Site 2011)

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Suita, Osaka, Japan

Hamamatsu University Hospital ( Site 2016)

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Hamamatsu, Shizuoka, Japan

Tohoku University Hospital ( Site 2002)

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Sendai-shi, Miyagi, Japan

Kyorin University Hospital ( Site 2005)

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Mitaka, Tokyo, Japan

Chiba University Hospital ( Site 2003)

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Chiba, Japan

Kyushu University Hospital ( Site 2015)

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Fukuoka, Japan

National Hospital Organization Okayama Medical Center ( Site 2013)

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Okayama, Japan

Keio university hospital ( Site 2007)

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Tokyo, Japan

International University of Health and Welfare Mita Hospital ( Site 2008)

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Tokyo, Japan

The University of Tokyo Hospital ( Site 2006)

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Bunkyo-ku, Tokyo, Japan

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