Pulmonary Arterial Hypertension
Conditions
Brief summary
The goal of this study is to learn about the safety of sotatercept and how well Japanese children tolerate it, when taken along with standard (usual) pulmonary arterial hypertension (PAH) treatment. Researchers also want to learn what happens to it in a person's body over time and whether it lowers resistance in blood vessels in the lungs.
Interventions
BIOLOGICALSotatercept
Sotatercept subcutaneous injection every 3 weeks
Sponsors
Merck Sharp & Dohme LLC
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
The main inclusion criteria include but are not limited to the following: * Has pulmonary arterial hypertension (PAH, World Health Organization Group 1) confirmed by a prior right heart catheterization * Has idiopathic, heritable, drug- or toxin-induced PAH, connective tissue disease-associated PAH, repaired congenital heart disease-associated PAH, or PAH with coincidental shunt * Has PAH classified as World Health Organization Functional Class I, or symptomatic World Health Organization Functional Class II to IV * Has been receiving stable standard-of-care background therapy for PAH for at least 90 days * Is Japanese
Exclusion criteria
The main
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants with Detectable Anti-Drug Antibodies (ADAs) | Up to approximately 24 weeks | Blood samples will be collected to assess antidrug antibodies to sotatercept. The number of participants with detectable ADAs at any time during the study will be presented. |
| Number of Participants with Detectable Neutralizing Antibodies to Sotatercept | Up to approximately 24 weeks | Neutralizing antibody analysis will be performed on blood samples for participants who are confirmed as antidrug antibody-positive. The number of participants with detectable neutralizing antibodies at any time during the study will be presented. |
| Serum Trough Concentration (Ctrough) of Sotatercept | At designated time points and up to approximately 24 weeks | Serum samples collected predose will be used to determine serum trough concentration (Ctrough) of sotatercept. |
| Change From Baseline in Pulmonary Vascular Resistance Index (PVRI) | Baseline and Week 24 | The change from baseline in PVRI will be presented. |
| Number of Participants With One or More Adverse Events | Up to approximately 24 weeks | An adverse event is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. |
| Number of Participants Who Discontinue Study Intervention Due to an Adverse Event | Up to approximately 24 weeks | An adverse event is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. |
| Laboratory Parameter (Hematology): Concentration of Hemoglobin | Up to approximately 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 approximately 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 approximately 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. |
| Laboratory Parameter (Hematology): Reticulocyte Count | Up to approximately 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 approximately 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. |
| Blood Pressure (BP) | Up to approximately 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
| Measure | Time frame | Description |
|---|---|---|
| Laboratory Parameter (Hematology): Reticulocyte Count | From Week 24 up to approximately 58 months | 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 | From Week 24 up to approximately 58 months | 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. |
| Blood Pressure (BP) | From Week 24 up to approximately 58 months | 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). |
| Number of Participants with Detectable ADAs | From Week 24 up to approximately 58 months | Blood samples will be collected to assess antidrug antibodies to sotatercept. The number of participants with detectable ADAs at any time during the study will be presented. |
| Number of Participants with Detectable Neutralizing Antibodies to Sotatercept | From Week 24 up to approximately 58 months | Neutralizing antibody analysis will be performed on blood samples for participants who are confirmed as ADA-positive. The number of participants with detectable neutralizing antibodies at any time during the study will be presented. |
| Mean Change from Baseline in 6-Minute Walk Distance (6MWD) | Baseline and Week 24 | 6MWD will be assessed using the 6-minute walk test (6MWT). |
| 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. |
| Number of Participants With One or More Adverse Events | From Week 24 up to approximately 58 months | An adverse event is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. |
| Number of Participants Who Discontinue Study Intervention Due to an Adverse Event | From Week 24 up to approximately 58 months | An adverse event is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. |
| Laboratory Parameter (Hematology): Concentration of Hemoglobin | From Week 24 up to approximately 58 months | 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 | From Week 24 up to approximately 58 months | 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 | From Week 24 up to approximately 58 months | 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. |
Countries
Japan
Contacts
CONTACTToll Free Number
STUDY_DIRECTORMedical Director
Merck Sharp & Dohme LLC
Outcome results
None listed