Recruiting
Phase 2

Sotatercept

Sponsor:

Merck Sharp & Dohme LLC

Code:

NCT05587712

Conditions

Pulmonary Arterial Hypertension

Eligibility Criteria

Sex: All

Age: 1 - 17

Healthy Volunteers: Not accepted

Interventions

Sotatercept

Study Details

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

Study ID

NCT05587712

Start date

Jan 19, 2023

Status verified date

Apr, 2026

Completion date

Sep 21, 2028

Anticipated

Primary completion date

Sep 21, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 1 - 17

Healthy Volunteers: Not accepted

Inclusion Criteria

  • 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

Exclusion Criteria

  • 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

Study Design

Enrollment

42 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Children ≥1 to <18 years old

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.

Interventions

Sotatercept

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

Primary outcome measure

  • Serum Trough Concentration (Ctrough) of Sotatercept [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: Predose Day 1, Day 7, Day 14, and Predose Day 21 ]
  • Percentage of Participants Who Experience at Least 1 Adverse Event (AE) [ Time Frame: Up to 24 weeks ]
  • Percentage of Participants Who Discontinue Study Drug Due to an AE [ Time Frame: Up to 24 weeks ]
  • Laboratory Parameter (Hematology): Concentration of Hemoglobin [ Time Frame: Up to 24 weeks ]
  • Laboratory Parameter (Hematology): Hematocrit [ Time Frame: Up to 24 weeks ]
  • Laboratory Parameter (Hematology): Red Blood Cell (RBC) Count [ Time Frame: Up to 24 weeks ]
  • Laboratory Parameter (Hematology): Reticulocyte Count [ Time Frame: Up to 24 weeks ]
  • Laboratory Parameter (Hematology): Platelet Count [ Time Frame: Up to 24 weeks ]
  • Blood Pressure (BP) [ Time Frame: Up to 24 weeks ]
  • Titer of Anti-drug Antibody (ADA) to Sotatercept [ Time Frame: Up to 24 weeks ]

Central Contacts and Locations

Central contacts

Toll Free Number

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

Locations

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

Recruiting

Los Angeles, California, United States, 90095

Contacts

Study Coordinator

310-267-7667

Stanford University School of Medicine ( Site 1603)

Recruiting

Palo Alto, California, United States, 94304

Contacts

Study Coordinator

650-497-8000

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

Recruiting

San Francisco, California, United States, 94158

Contacts

Study Coordinator

415-476-3831

Children's Hospital Colorado ( Site 1609)

Recruiting

Aurora, Colorado, United States, 80045

Contacts

Study Coordinator

720-777-6427

Children's National Medical Center ( Site 1600)

Recruiting

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

Contacts

Study Coordinator

202-476-2130

Cincinnati Children's Hospital Medical Center ( Site 1602)

Recruiting

Cincinnati, Ohio, United States, 45229

Contacts

Study Coordinator

513-636-7072

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

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Study Coordinator

267-426-0120

Seattle Children's Hospital ( Site 1605)

Recruiting

Seattle, Washington, United States, 98105

Contacts

Study Coordinator

206-987-2000

Children's Wisconsin ( Site 1610)

Recruiting

Milwaukee, Wisconsin, United States, 53226

Contacts

Study Coordinator

414-266-2000

More Information

Sponsor

Merck Sharp & Dohme LLC

Last update posted

Apr 20, 2026

Last verified

Apr, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Merck Sharp & Dohme LLC on 2026-04-20.