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

Phase 2

Recruiting

• Conditions: Pulmonary Arterial Hypertension
• Interventions: Drug: Sotatercept

• Registration Number: NCT05587712
• Lead Sponsor: Merck Sharp & Dohme LLC

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-10-17
• Study First Submitted QC: 2022-10-17
• Study First Posted: 2022-10-20
• Study Start: 2023-01-19
• Status Verified: 2025-06
• Last Update Submitted: 2025-06-11
• Last Update Posted: 2025-06-13
• Primary Completion: 2028-09-21
• Study Completion: 2028-09-21

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 42

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Children ≥1 to <18 years old	Sotatercept	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.

Primary Outcome Measures

Name	Time	Method
Laboratory Parameter (Hematology): Concentration of Hemoglobin	Up to 24 weeks	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.
Laboratory Parameter (Hematology): Hematocrit	Up to 24 weeks	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.
Laboratory Parameter (Hematology): Red Blood Cell (RBC) Count	Up to 24 weeks	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.
Serum Trough Concentration (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	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.
Percentage of Participants Who Experience at Least 1 Adverse Event (AE)	Up to 24 weeks	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.
Area Under the Curve at Steady State (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	Blood samples will be collected at multiple time points to estimate the AUCss of Sotatercept.
Area Under the Curve from 0 to 3 weeks (AUC0-3 weeks) of Sotatercept	Predose Day 1, Day 7, Day 14, and Predose Day 21	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.
Percentage of Participants Who Discontinue Study Drug Due to an AE	Up to 24 weeks	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.
Laboratory Parameter (Hematology): Reticulocyte Count	Up to 24 weeks	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.
Laboratory Parameter (Hematology): Platelet Count	Up to 24 weeks	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.
Titer of Anti-drug Antibody (ADA) to Sotatercept	Up to 24 weeks	ADA to Sotatercept will be assessed.
Blood Pressure (BP)	Up to 24 weeks	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).

Secondary Outcome Measures

Name	Time	Method
Mean Change from Baseline in Tricuspid Annular Plane Systolic Excursion (TAPSE)	Baseline and Week 24	A two-dimensional echocardiogram (ECHO) will be performed with the results interpreted by a blinded independent central review (BICR) at baseline and after 24 weeks of treatment. The change from baseline in TAPSE will be reported.
Mean Change from Baseline in Pulmonary Artery Systolic Pressure (PASP)	Baseline and Week 24	A two-dimensional ECHO will be performed with the results interpreted by a BICR at baseline and after 24 weeks of treatment. The change from baseline in PASP will be reported.
Mean Change from Baseline in 6-Minute Walk Distance (6MWD) (Cohorts 1 and 2)	Baseline and Week 24	6MWD will be assessed using the 6-minute walk test (6MWT).
Mean Change from Baseline in Eccentricity Index	Baseline and Week 24	A two-dimensional ECHO will be performed with the results interpreted by a BICR at baseline and after 24 weeks of treatment. The change from baseline in eccentricity index will be reported.
Mean Change from Baseline on Cardiac Output (Cohorts 1 and 2)	Baseline and Week 24	Cardiac magnetic imaging (MRI) will be performed with the results interpreted by a BICR at baseline and after 24 weeks of treatment. The change from baseline in cardiac output will be reported.
Mean Change from Baseline in Right Ventricular Fractional Area Change (RVFAC)	Baseline and Week 24	A two-dimensional ECHO will be performed with the results interpreted by a BICR at baseline and after 24 weeks of treatment. The change from baseline in RVFAC will be reported.
Mean Change from Baseline in Right Ventricular (RV) Function (Cohorts 1 and 2)	Baseline and Week 24	Cardiac magnetic imaging (MRI) will be performed with the results interpreted by a BICR at baseline and after 24 weeks of treatment. The change from baseline in eccentricity index will be reported.
Mean Change from Baseline in Pulmonary Arterial Pressure (PAP) (Cohorts 1 and 2)	Baseline and Week 24	Cardiac magnetic imaging (MRI) will be performed with the results interpreted by a BICR at baseline and after 24 weeks of treatment. The change from baseline in PAP will be reported.
Mean Change from Baseline in Pediatric Quality of Life (PedsQL) Generic Score	Baseline and Week 24	PedsQL Measurement Model is a modular approach to measuring health-related quality of life in healthy children and adolescents and those with acute and chronic health conditions. The change from baseline in the PedsQL generic core scale will be reported.
Mean Change from Baseline in N-terminal Prohormone B-type Natriuretic Peptide (NT-proBNP)	Baseline and Week 24	The change from baseline in plasma NT-proBNP levels will be reported.
Percentage of Participants Who Either Improved or Maintained Their World Health Organization Functional Class (WHO FC)	Baseline and Week 24	The severity of an individual's PAH symptoms will be graded using the WHO FC system. WHO functional classification for PAH range 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). The change from baseline in WHO FC will be classified into "Improved", "No change" and "Worsened". Improvement = reduction in FC, worsened = increase in FC and no change = no change in FC.

Trial Locations

Locations (35)

The Regents of the University of California - Los Angeles (UCLA Pediatrics) ( Site 1606); 🇺🇸 Los Angeles, California, United States

Stanford University School of Medicine ( Site 1603); 🇺🇸 Palo Alto, California, United States

UCSF Benioff Children's Hospital San Francisco ( Site 1611); 🇺🇸 San Francisco, California, United States

Children's Hospital Colorado ( Site 1609); 🇺🇸 Aurora, Colorado, United States

Children's National Medical Center ( Site 1600); 🇺🇸 Washington, District of Columbia, United States

Cincinnati Children's Hospital Medical Center ( Site 1602); 🇺🇸 Cincinnati, Ohio, United States

Children's Hospital of Philadelphia (CHOP) ( Site 1608); 🇺🇸 Philadelphia, Pennsylvania, United States

Monroe Carell Jr. Children's Hospital ( Site 1601); 🇺🇸 Nashville, Tennessee, United States

Seattle Children's Hospital ( Site 1605); 🇺🇸 Seattle, Washington, United States

Children's Wisconsin ( Site 1610); 🇺🇸 Milwaukee, Wisconsin, United States

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