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

Condition(s) studied

Investigational drug(s) / intervention(s)

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

Study 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.

Eligibility

Primary outcome measure(s)

• 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.
• 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 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.
• 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): 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.
• 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.
• 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).
• Titer of Anti-drug Antibody (ADA) to Sotatercept — Up to 24 weeks
  ADA to Sotatercept will be assessed.

Trial sites (35)