EUCTR2022-000478-25-DE进行中（未招募）1 期
A 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 - Sotatercept in Children with PAH on SoC
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 42
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •1. Male or female participants =1 to <18 years of age at the time of providing documented informed consent/assent
- •2. Documented, historic diagnostic RHC any time before Screening confirming the diagnosis of PAH WHO Group 1 in any of the following subtypes (for participants with a history of cardiac intervention for shunt closure, the RHC confirmation must have been performed more than 6 months after the cardiac intervention):
- •- Heritable PAH
- •- Drug/toxin-induced PAH
- •- PAH associated with CTD
- •- PAH-CHD with shunt closure >6 months before Screening and subsequently confirmed by RHC before Screening
- •- PAH with coincidental shunt
- •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:
- •- WHO FC I to III: single, double, or triple background PAH therapy for at least 12 weeks before SCR and during SCR
- •- WHO FC IV: must be clinically stable and on stable doses of maximum tolerated double or triple background PAH therapy for at least 30 days before SCR and during SCR
- •6. Arterial BP at Screening within normal range for the age, gender, and height percentiles as follows:
- •- Cohort 1: systolic BP <120 mmHg and diastolic BP <80 mmHg
- •- Cohorts 2, 3, and 4: systolic and diastolic BP <90th percentile based on the corresponding age ranges as outlined in National High Blood Pressure Education Program, 2004
- •7. Left ventricular ejection fraction =50% on the ECHO at Screening
- •8. If male, agrees to the following during the intervention period and for at least 16 weeks 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
- •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 WOCBP who is not currently pregnant
- •- Contraceptive use by men should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
- •9. A female participant is eligible to participate if she is not pregnant or breastfeeding, and at least one of the following conditions applies:
- •Not a WOCBP
- •A WOCBP and:
- •- Uses a contraceptive method that is highly effective (with a failure rate of <1% per year), with low user dependency, or be abstinent from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long-term and persistent basis), during the intervention period and for at least 16 weeks after the last dose of study intervention. The investigator should evaluate the potential for contraceptive method failure in relationship to the first dose of study intervention. Contraceptive use by women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
- •- Has a negative highly sensitive pregnancy test (urine or serum as requir
排除标准
- •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
- •16. Screening platelet count <50,000/mm3
- •17. Screening Hgb value above gender-specific ULN (per local laboratory)
- •18. Screening eGFR <30 mL/min/m2 (as defined by the Bedside Schwartz equation) and/or a urine protein:creatinine ratio >1 g/g
- •19. Screening AST and/or ALT >3X ULN
- •20. Screening ECG with Fridericia’s corrected QT interval (QTcF) >500 ms (or >550 ms, if right bundle branch block is present)
- •21. Is or has an immediate family member (eg, spouse, parent/legal guardian, sibling, or child) who is investigational site or Sponsor staff directly involved with this study
研究者
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