Study to Evaluate Sotatercept (MK-7962) in Children With Pulmonary Arterial Hypertension (PAH) (MK-7962-008)

NCT05587712 | Recruiting Phase 2 DMC FDA Drug

• 5 other identifiers: 7962-008MK-7962-0082023-504861-22-00U1111-1290-28582022-000478-25
• Study Type: interventional
• Target: 42
• Locations: 12 countries
• Sites: 35

Brief Summary

The primary objectives of the study are to evaluate the safety and tolerability, and pharmacokinetics (PK) of sotatercept over 24 weeks of treatment in children ≥1 to \<18 years of age with PAH World Health Organization (WHO) Group 1 on standard of care (SoC). There is no formal hypothesis.

Conditions

Pulmonary Arterial Hypertension

Outcome Measures

Primary Outcomes (12)

• Serum Trough Concentration (Ctrough) of Sotatercept

  Ctrough was the lowest concentration of Sotatercept in serum just before the next dose. Blood samples will be collected at multiple time points to estimate the Ctrough of Sotatercept.

  Predose Day 1, Day 21, Day 42, Day 63, Day 84, Day105, Day 126, Day 147, Day 168, Day 189. Postdose Day 7, Day 14, Day 64, Day 69 and Day 76

• Area Under the Curve at Steady State (AUCss) of Sotatercept

  Blood samples will be collected at multiple time points to estimate the AUCss of Sotatercept.

  Predose Day 1, Day 21, Day 42, Day 63, Day 84, Day105, Day 126, Day 147, Day 168, Day 189. Postdose Day 7, Day 14, Day 64, Day 69 and Day 76

• Area Under the Curve from 0 to 3 weeks (AUC0-3 weeks) of Sotatercept

  Blood samples will be collected at Predose Day 1, Day 7, Day 14, and Predose Day 21 to estimate the AUC0-3 weeks of Sotatercept.

  Predose Day 1, Day 7, Day 14, and Predose Day 21

• Percentage of Participants Who Experience at Least 1 Adverse Event (AE)

  An AE is any unfavourable and unintended sign, symptom, or disease temporally associated with the use of a medicinal product or protocol-specified procedure, whether or not considered related to the medicinal product or protocol-specified procedure. Any worsening of a preexisting condition considered related to the medicinal product or protocol-specified procedure. Any worsening of a preexisting condition that is temporally associated with the use of the Sponsor's product, is also an AE. The percentage of participants with 1 or more AEs will be assessed.

  Up to 24 weeks

• Percentage of Participants Who Discontinue Study Drug Due to an AE

  An AE is any unfavourable and unintended sign, symptom, or disease temporally associated with the use of a medicinal product or protocol-specified procedure, whether or not considered related to the medicinal product or protocol-specified procedure. Any worsening of a preexisting condition considered related to the medicinal product or protocol-specified procedure. Any worsening of a preexisting condition that is temporally associated with the use of the Sponsor's product, is also an adverse event. The percentage of participants who discontinued the study drug due to an AE regardless of study completion status will be assessed.

  Up to 24 weeks

• Laboratory Parameter (Hematology): Concentration of Hemoglobin

  Hematological parameters will be investigated in blood samples from participants by means of clinical laboratory assays and evaluated by the investigator. The concentration of hemoglobin will be presented.

  Up to 24 weeks

• Laboratory Parameter (Hematology): Hematocrit

  Hematological parameters will be investigated in blood samples from participants by means of clinical laboratory assays and evaluated by the investigator. The hematocrit will be presented.

  Up to 24 weeks

• Laboratory Parameter (Hematology): Red Blood Cell (RBC) Count

  Hematological parameters will be investigated in blood samples from participants by means of clinical laboratory assays and evaluated by the investigator. The RBC count will be presented.

  Up to 24 weeks

• Laboratory Parameter (Hematology): Reticulocyte Count

  Hematological parameters will be investigated in blood samples from participants by means of clinical laboratory assays and evaluated by the investigator. The reticulocyte count will be presented.

  Up to 24 weeks

• Laboratory Parameter (Hematology): Platelet Count

  Hematological parameters will be investigated in blood samples from participants by means of clinical laboratory assays and evaluated by the investigator. The platelet count will be presented.

  Up to 24 weeks

• Blood Pressure (BP)

  BP will be assessed while the participant was seated after a period of rest in a quiet setting with no distractions (eg, television and cell phones).

  Up to 24 weeks

• Titer of Anti-drug Antibody (ADA) to Sotatercept

  ADA to Sotatercept will be assessed.

  Up to 24 weeks

Secondary Outcomes (11)

• Mean Change from Baseline in 6-Minute Walk Distance (6MWD) (Cohorts 1 and 2)

  Baseline and Week 24

• Mean Change from Baseline in Tricuspid Annular Plane Systolic Excursion (TAPSE)

  Baseline and Week 24

• Mean Change from Baseline in Pulmonary Artery Systolic Pressure (PASP)

  Baseline and Week 24

• Mean Change from Baseline in Right Ventricular Fractional Area Change (RVFAC)

  Baseline and Week 24

• Mean Change from Baseline in Eccentricity Index

  Baseline and Week 24

Study Arms (1)

Children ≥1 to <18 years old

EXPERIMENTAL

Participants will receive a subcutaneous (SC) injection every 3 weeks (Q3W) of 0.3 mg/kg. Dosage may be adjusted based on protocol-specific guidelines.

Drug: Sotatercept

Interventions

Sotatercept DRUG

SC injection every 3 weeks (Q3W) of 0.3 mg/kg. Dosage may be adjusted based on protocol-specific guidelines.

Also known as: MK-7962

Children ≥1 to <18 years old

Eligibility Criteria

• Age: 1 Year - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Documented, historic diagnostic right heart catheterization (RHC) any time before Screening confirming the diagnosis of PAH WHO Group 1 in any of the following subtypes:
• Idiopathic pulmonary arterial hypertension (IPAH)
• Heritable PAH
• Drug/toxin-induced PAH
• PAH associated with connective tissue disease
• PAH-congenital heart disease (CHD) with shunt closure \>6 months before Screening and subsequently confirmed by RHC before Screening
• PAH with coincidental shunt.
• Must be on a stable dose(s) of background PAH therapy (phosphdiesterase-5 (PDE5) inhibitors, endothelin receptor antagonists (ERAs), soluble guanylate cyclase stimulators (sGCS), or prostanoids \[including subcutaneous and intravenous\])
• If male, agree to the following during the intervention period and for at least 16 weeks (112 days) after the last dose of study intervention:
• Abstains from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long-term and persistent basis) and agrees to remain abstinent or
• Uses contraception unless confirmed to be azoospermic (vasectomized or secondary to medical cause, documented from the site personnel's review of the participant's medical records, medical examination, or medical history interview) as detailed below:
• Uses a male condom plus partner use of an additional contraceptive method when having penile-vaginal intercourse with a woman of childbearing potential (WOCBP) who is not currently pregnant Note: Men with a pregnant or breastfeeding partner must agree to remain abstinent from penile-vaginal intercourse or use a male condom during each episode of penile-vaginal penetration.
• If female, must be either not a WOCBP or use a contraceptive method that is highly effective or be abstinent from heterosexual intercourse during the intervention period and for at least 16 weeks (112 days) after the last dose of study intervention
• If male, agrees to refrain from donating blood or sperm for the duration of the study and for 16 weeks (112 days) after the last dose of study intervention
• If female, agrees to refrain from donating blood, eggs, or ovum for the duration of the study and for at least 16 weeks (112 days) after the last dose of study intervention

You may not qualify if:

• History of left-sided heart disease, including valvular disease (eg, moderate or greater mitral or aortic regurgitation or stenosis), left ventricular outflow tract obstruction, and/or left heart failure (eg, restrictive or dilated cardiomyopathy)
• Severe (as based on the opinion of the investigator) congenital or developmental abnormalities of the lung, thorax, and/or diaphragm
• History of Eisenmenger syndrome, Potts shunt, or atrial septostomy
• Unrepaired or residual cardiac shunt
• Diagnosis of pulmonary veno-occlusive diseases, pulmonary capillary hemangiomatosis, or overt signs of capillary and/or venous involvement
• PAH associated with portal hypertension
• Known visceral (lung, liver, or brain) arteriovenous malformation(s)
• History of full or partial pneumonectomy
• Untreated more than mild obstructive sleep apnea
• History of known pericardial constriction
• Family history of sudden cardiac death or long QT syndrome
• Any current or prior history of symptomatic coronary disease (myocardial infarction, percutaneous coronary intervention, coronary artery bypass graft surgery, or cardiac anginal chest pain) within 6 months before Screening
• Cerebrovascular accident within 3 months before Screening
• Prior exposure to sotatercept or luspatercept or has had an allergic reaction to any of their excipients

Sponsors & Collaborators

• Merck Sharp & Dohme LLC: lead

Study Sites (35)

The Regents of the University of California - Los Angeles (UCLA Pediatrics) ( Site 1606)

Los Angeles, California, 90095, United States

RECRUITING

Stanford University School of Medicine ( Site 1603)

Palo Alto, California, 94304, United States

RECRUITING

UCSF Benioff Children's Hospital San Francisco ( Site 1611)

San Francisco, California, 94158, United States

RECRUITING

Children's Hospital Colorado ( Site 1609)

Aurora, Colorado, 80045, United States

RECRUITING

Children's National Medical Center ( Site 1600)

Washington D.C., District of Columbia, 20010, United States

RECRUITING

Cincinnati Children's Hospital Medical Center ( Site 1602)

Cincinnati, Ohio, 45229, United States

RECRUITING

Children's Hospital of Philadelphia (CHOP) ( Site 1608)

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Monroe Carell Jr. Children's Hospital ( Site 1601)

Nashville, Tennessee, 37232, United States

COMPLETED

Seattle Children's Hospital ( Site 1605)

Seattle, Washington, 98105, United States

RECRUITING

Children's Wisconsin ( Site 1610)

Milwaukee, Wisconsin, 53226, United States

RECRUITING

The Children's Hospital at Westmead ( Site 0001)

Westmead, New South Wales, 2145, Australia

RECRUITING

Clinica Somer-Unidad de Investigacion y Docencia ( Site 0205)

Rionegro, Antioquia, 054040, Colombia

RECRUITING

Fundación Valle del Lili ( Site 0200)

Cali, Valle del Cauca Department, 760032, Colombia

RECRUITING

Clínica Imbanaco S.A.S ( Site 0203)

Cali, Valle del Cauca Department, 760042, Colombia

RECRUITING

CHU de Toulouse - Hôpital des Enfants ( Site 0302)

Toulouse, Haute-Garonne, 31059, France

RECRUITING

Assistance Publique Hôpitaux de Marseille - Hôpital de la Timone ( Site 0303)

Marseille, Provence-Alpes-Côte d'Azur Region, 13005, France

RECRUITING

Hôpital Universitaire Necker Enfants Malades ( Site 0300)

Paris, 75015, France

RECRUITING

Universitaetsklinikum Heidelberg ( Site 0401)

Heidelberg, Baden-Wurttemberg, 69120, Germany

RECRUITING

Klinikum der Universität München Großhadern ( Site 0404)

München, Bavaria, 81337, Germany

RECRUITING

Medizinische Hochschule Hannover ( Site 0405)

Hanover, Lower Saxony, 30625, Germany

RECRUITING

Schneider Children's Medical Center ( Site 0603)

Petah Tikva, 4920235, Israel

RECRUITING

Sheba Medical Center ( Site 0601)

Ramat Gan, 5265601, Israel

RECRUITING

University Medical Center Groningen ( Site 0900)

Groningen, 9713 GZ, Netherlands

RECRUITING

Centrum Zdrowia Dziecka w Warszawie-Klinika Kardiologii ( Site 1103)

Warsaw, Masovian Voivodeship, 04-730, Poland

RECRUITING

Uniwersyteckie Centrum Kliniczne-Klinika Kardiologii Dziecięcej i Wad Wrodzonych Serca ( Site 1102)

Gdansk, Pomeranian Voivodeship, 80-952, Poland

RECRUITING

Wits Clinical Research-Chris Hani Baragwanath Hospital ( Site 1201)

Johannesburg, Soweto, Gauteng, 2013, South Africa

RECRUITING

Hospital Universitario Ramón y Cajal ( Site 1300)

Madrid, Madrid, Comunidad de, 28034, Spain

RECRUITING

Hospital Universitari i Politecnic La Fe ( Site 1303)

Valencia, Valencia, 46026, Spain

RECRUITING

Hospital Universitari Vall d'Hebron ( Site 1302)

Barcelona, 08035, Spain

RECRUITING

HOSPITAL GENERAL UNIVERSITARIO GREGORIO MARAÑON ( Site 1301)

Madrid, 28007, Spain

RECRUITING

Hacettepe Universite Hastaneleri ( Site 1400)

Ankara, 06100, Turkey (Türkiye)

RECRUITING

Gazi University Health Research and Application Center Gazi -Çocuk Sağlığı ve Hastalıkları Anabilim ( Site 1402)

Ankara, 06560, Turkey (Türkiye)

RECRUITING

Ankara Bilkent Şehir Hastanesi. ( Site 1403)

Ankara, 06800, Turkey (Türkiye)

RECRUITING

Mehmet Akif Ersoy Research and Training Hospital ( Site 1404)

Istanbul, 34303, Turkey (Türkiye)

RECRUITING

Great Ormond Street Hospital For Children NHS Foundation Trust ( Site 1500)

London, London, City of, WC1N 3JH, United Kingdom

RECRUITING

Related Links

• Merck Clinical Trials Information
• Plain Language Summary

MeSH Terms

Conditions

Pulmonary Arterial Hypertension

Interventions

ACE-011

Condition Hierarchy (Ancestors)

Hypertension, Pulmonary; Lung Diseases; Respiratory Tract Diseases

Study Officials

• Medical Director

  Merck Sharp & Dohme LLC

  STUDY DIRECTOR

Central Study Contacts

Toll Free Number

CONTACT

1-888-577-8839; Trialsites@msd.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 17, 2022
• First Posted: October 20, 2022
• Study Start: January 19, 2023
• Primary Completion (Estimated): September 21, 2028
• Study Completion (Estimated): September 21, 2028
• Last Updated: April 20, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

CO (3); DE (3); GB (1); TU (4); US (10); ES (4); ZA (1); FR (3); AU (1); IL (2); NL (1); PL (2)