Phase 2 Open-label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sotatercept (MK-7962) in Children from 1 to Less Than 18 Years of Age With PAH on Standard of Care
- Conditions
- pulmonary arterial hypertension10057166
- Registration Number
- NL-OMON53701
- Lead Sponsor
- Merck Sharp & Dohme (MSD)
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Pending
- Sex
- Not specified
- Target Recruitment
- 1
The main inclusion criteria are listed here. For a complete list of inclusion
please refer to the research protocol.
1. Male or female participants >=1 to <18 years of age at the time of providing
documented informed consent/assent:
• Cohort 1: Age >=12 to <18 years of age
• Cohort 2: Age >=6 to <12 years of age
• Cohort 3: Age >=2 to <6 years of age
• Cohort 4: Age >=1 to <2 years of age
2. Documented, historic diagnostic RHC any time before Screening confirming the
diagnosis of PAH WHO Group 1.
3. For the above-mentioned historical RHC, diagnostic criteria will be mean
pulmonary artery pressure >=20 mmHg at rest, pulmonary capillary wedge pressure
or left ventricular end-diastolic pressure <=15 mmHg, and PVR indexed to body
surface area, >= 3.0 WU.m2
4. PAH classified as WHO FC I or symptomatic PAH classified as WHO FC II to IV
5. Participants must be on a stable dose(s) of background PAH therapy
6. Arterial BP at Screening within normal range for the age, gender, and height
percentiles
7. Left ventricular ejection fraction >=50% on the ECHO at Screening
The main exclusion criteria are listed here. For a complete list of exclusion
please refer to the research
protocol.
1. 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)
2 Severe (as based on the opinion of the investigator) congenital or
developmental abnormalities of the lung, thorax, and/or diaphragm
3. History of Eisenmenger syndrome, Potts shunt, atrial septostomy within 180
days prior to the screening visit, or atrial septostomy with Eisenmenger
physiology
4. Unrepaired or residual cardiac shunt with Qp/Qs >1.5
5. Diagnosis of pulmonary veno-occlusive diseases, pulmonary capillary
hemangiomatosis, or overt signs of capillary and/or venous involvement
6. PAH associated with portal hypertension
7. Known visceral (lung, liver, or brain) arteriovenous malformation(s)
8. History of full or partial pneumonectomy
9. Untreated more than mild obstructive sleep apnea
10. History of known pericardial constriction
11. Family history of sudden cardiac death or long QT syndrome
12. 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
13. Cerebrovascular accident within 3 months before Screening
14. Prior exposure to sotatercept or luspatercept or has had an allergic
reaction to any of their excipients
15. Currently enrolled in or has completed a study with any other
investigational products (small molecule drugs or biologics) within 30 days or
5 half-lives of that investigational product (whichever is longer) before
Screening
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>• To evaluate the safety and tolerability of sotatercept over 24 weeks of<br /><br>treatment<br /><br>• To evaluate the PK of sotatercept over 24 weeks of treatment</p><br>
- Secondary Outcome Measures
Name Time Method <p>• To evaluate the pharmacodynamics of sotatercept over 24 weeks of treatment</p><br>