NCT00543439已完成3 期
An Open-label Study To Evaluate Prophylaxis Treatment, And To Characterize The Efficacy, Safety, And Pharmacokinetics Of B-domain Deleted Recombinant Factor Viii Albumin Free (Moroctocog Alfa [Af-cc]) In Children With Hemophilia A
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 66
- 试验地点
- 55
- 主要终点
- Mean Annualized Bleed Rate (ABR) by Treatment: On Demand Cohort
研究概览
简要总结
The purpose of this research study is to determine the effectiveness, safety, and pharmacokinetics (PK) of moroctocog alfa (AF-CC) in previously treated subjects, who are younger than 6 years of age, with severe or moderately severe hemophilia A.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Months 至 15 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male subjects, aged less than 6 years, with moderately severe to severe hemophilia A.
- •A negative FVIII inhibitor titer at screening, and a medical history negative for a past FVIII inhibitor.
- •At least 20 exposure days to any FVIII replacement product.
- •Adequate hepatic and renal function
- •CD4 count > 400 cells/uL, and if receiving antiviral therapy must be on a stable regimen
- •Additional criteria for subjects participating in the PK assessment:
- •Male subjects as described immediately above except they must have a FVIII Activity of less than or equal to 1% confirmed by the central laboratory screening test
- •Age < 6 years at time of PK assessment.
- •The subject's size is sufficient to permit PK-related phlebotomy.
- •The subject is able to comply with the procedures conducted during the PK assessment, including a mandatory 72-hour washout period preceding the PK assessment.
排除标准
- •A history of FVIII inhibitor.
- •Presence of a bleeding disorder in addition to hemophilia A.
- •Treatment with any investigational drug or device within 30 days before the time of signing the informed consent form.
- •Major or orthopedic surgery planned to occur during the course of the study.
- •Regular (e.g., daily, every other day) use of antifibrinolytic agents or medications known to influence platelet function such as aspirin or certain nonsteroidal anti-inflammatory drugs (NSAIDs), or regular, concomitant therapy with immunomodulating drugs (e.g., intravenous immunoglobulin [IVIG], routine systemic corticosteroids).
- •Known hypersensitivity to hamster protein.
研究组 & 干预措施
2
Experimental
Routine Prophylaxis Crossover
干预措施: Moroctocog alfa (AF-CC) (Biological)
1
Experimental
On-Demand therapy for 6 months, followed by Routine Prophylaxis treatment for 1 year.
干预措施: Moroctocog alfa (AF-CC) (Biological)
结局指标
主要结局
Mean Annualized Bleed Rate (ABR) by Treatment: On Demand Cohort
时间窗: Day 1 up to Month 6 (OD Cohort, OD Therapy, Period 1); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2)
ABR for each participant was calculated as the number of bleeds requiring administration of moroctocog alfa (AF-CC) divided by the total therapy duration (in days), then multiplied by 365.25 (days in a year).
次要结局
- Mean of Moroctocog Alfa (AF-CC) Infusions Administered To Treat Bleeding Episode: All Participants(Day 1 up to Month 24)
- Number of Treated Spontaneous Bleeds by Time Interval Between Bleed Onset and Prior Moroctocog Alfa (AF-CC) Prophylaxis Dose: Routine Prophylaxis Therapy(Day 1 up to Month 24 (RP Cohort, RP 25 IU/kg and 45 IU/kg, Period 1 and 2); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2))
- Area Under the Curve From Time Zero to Extrapolated Infinite Time (AUCinf) of Factor VIII Activity(0.5, 8, 24, 28 and 32 hours post dose on Day 1)
- Mean Residence Time (MRT) of Factor VIII Activity(0.5, 8, 24, 28 and 32 hours post dose on Day 1)
- Mean Annualized Bleed Rate (ABR) by Treatment: Routine Prophylaxis Cohort(Day 1 up to Month 24 (RP Cohort, Period 1 and Period 2))
- Terminal Phase Half Life (t1/2) of Factor VIII (FVIII) Activity(0.5, 8, 24, 28 and 32 hours post dose on Day 1)
- Incremental Recovery of Factor VIII Activity(Day 1, Month 6)
- Area Under the Curve From Time Zero to Last Measurable Concentration (AUClast) of Factor VIII Activity(0.5, 8, 24, 28 and 32 hours post dose on Day 1)
- Steady-State Volume of Distribution (Vss) of Factor VIII Activity(0.5, 8, 24, 28 and 32 hours post dose on Day 1)
- Number of Participants With Treatment Emergent Adverse Events (AEs) According to Severity(Day 1 up to Month 25)
- Number of Participants With Incidence of Less Than Expected Therapeutic Effect (LETE): Routine Prophylaxis Therapy(Day 1 up to Month 24 (RP Cohort, RP 25 IU/kg and 45 IU/kg, Period 1 and 2); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2))
- Number of Treated Bleeds Classified on Basis of Response to First Infusion of Moroctocog Alfa (AF-CC) as On-Demand Treatment: OD Therapy (OD and RP Cohort)(Day 1 up to Month 24)
- Number of Participants Requiring Prophylaxis Regimen Escalation: Routine Prophylaxis Therapy(Day 1 up to Month 24 (RP Cohort, RP 25 IU/kg and 45 IU/kg, Period 1 and 2); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2))
- Mean of Total Number Moroctocog Alfa (AF-CC) Infusions Received: Routine Prophylaxis Therapy(Day 1 up to Month 24 (RP Cohort, RP 25 IU/kg and 45 IU/kg, Period 1 and 2); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2))
- Mean of Total Number of Infusions of Moroctocog Alfa (AF-CC) Received Per Week to Assess Compliance: Routine Prophylaxis Therapy(Day 1 up to Month 24 (RP Cohort, RP 25 IU/kg and 45 IU/kg, Period 1 and 2); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2))
- Maximum Concentration of Factor VIII Activity(0.5, 8, 24, 28 and 32 hours post dose on Day 1)
- Number of Participants With Treatment-Related Adverse Events(Day 1 up to Month 25)
- Mean Routine Prophylaxis Dose (IU/kg) of Moroctocog Alfa (AF-CC) Received: Routine Prophylaxis Therapy(Day 1 up to Month 24 (RP Cohort, RP 25 IU/kg and 45 IU/kg, Period 1 and 2); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2))
- Mean of Total Number of Days Participants Exposed to Moroctocog Alfa (AF-CC): Routine Prophylaxis Therapy(Day 1 up to Month 24 (RP Cohort, RP 25 IU/kg and 45 IU/kg, Period 1 and 2); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2))
- Clearance (CL) of Factor VIII Activity(0.5, 8, 24, 28 and 32 hours post dose on Day 1)
- Number of Participants With Confirmed FVIII Inhibitor Development(Day 1 up to Month 24)
- Number of Participants With Incidence of Less Than Expected Therapeutic Effect (LETE): On Demand Therapy(Day 1 up to Month 24 (RP Cohort, RP 25 IU/kg and 45 IU/kg, Period 1 and 2); Day 1 up to Month 6 (OD Cohort, OD Therapy, Period 1); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2))
研究者
研究点 (55)
Loading locations...
相似试验
招募中
4 期
Efficacy and Safety of Prophylactic Treatment for Pneumocystis Jirovecii Pneumonia in Patients With Autoimmune Inflammatory Rheumatic DiseasePneumonia, PneumocystisAutoimmune Inflammatory Rheumatic DiseaseAutoimmune DiseasesConnective Tissue DiseasePreventionNCT06499233Tongji Hospital800
进行中（未招募）
1 期
A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS ⅡMucopolysaccharidosis IIHunter SyndromeNCT05422482GC Biopharma Corp12
招募中
1 期
Assessment of Safety, Tolerability and Pharmacokinetics With BAT8010 for Injection in Advanced Malignant Solid Tumors PatientsAdvanced or Metastatic Solid TumorsNCT05848466Bio-Thera Solutions109
进行中（未招募）
1 期
Evaluate BL-M17D1 in Patients w/HER2-Expressing/Mutant Advanced or Metastatic Solid TumorsOvarian CancerGastric CancerNCT06714617SystImmune Inc.120
已完成
1 期
Phase 1 Study of Anti-Macrophage Migration Inhibitory Factor (Anti-MIF) Antibody in Solid TumorsMetastatic Adenocarcinoma of the Colon or RectumMalignant Solid TumorsNCT01765790Baxalta now part of Shire68