NCT04974749已完成3 期
An Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of REPLAGAL® in Treatment-naïve Chinese Subjects With Fabry Disease
Takeda6 个研究点 分布在 1 个国家目标入组 20 人开始时间: 2022年5月1日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 20
- 试验地点
- 6
- 主要终点
- Number of Participants With Serious Treatment-emergent Adverse Events (TEAEs)
研究概览
简要总结
The main aim of the study is to assess the safety of REPLAGAL. Study participants will receive REPLAGAL as an intravenous infusion every other week for 52 weeks. Participants will visit their study clinic many times throughout the study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 7 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant and/or legally authorized representative must voluntarily sign an Institutional Review Board/Independent Ethics Committee approved written informed consent form (ICF) after all relevant aspects of the study have been explained and discussed with the participant. For the participants less than (<) 18 years old, participants will give assent AND their parent(s)/legally authorized representative should sign the ICF accordingly.
- •The participant has confirmed diagnosis of Fabry disease as determined by the investigator, according to medical record including:
- •For male participant, Fabry disease is confirmed by a deficiency of α-galactosidase A (GLA) activity and a mutation in the GLA gene
- •For female participant, Fabry disease is confirmed by a mutation in the GLA gene.
- •The participant is 7 to 65 years of age, inclusive, at screening.
- •Female participants of childbearing potential must have a negative pregnancy test at screening.
- •Female participants of childbearing potential must agree to use a medically acceptable method of contraception at all times during the study and for at least 14 days after the final investigational product infusion.
- •The participant is deemed, as determined by the investigator, to have adequate general health to undergo the specified protocol-related procedures and to have no safety or medical contraindications for participation.
- •The participant has not received any treatment (approved or investigational) specific to Fabry disease, such as ERT, chaperone therapy, or substrate reduction therapy.
- •The adult participant (greater than or equal to [>=] 18 years old) must have an estimated glomerular filtration rate (eGFR) of 45 to 120 milliliter per minute per 1.73 meter square (mL/min/1.73 m^2) (inclusive). Serum creatinine is tested and the eGFR is calculated by central laboratory using the Chronic Kidney Disease Epidemiology (CKD-EPI) equation.
排除标准
- •In the opinion of the investigator, the participant's life expectancy is less than or equal to (<=) 5 years.
- •The participant has undergone or is scheduled to undergo kidney transplantation or is currently on dialysis or has any signs or symptoms of end stage renal disease.
- •The participant has a urine protein/creatinine ratio of greater than (>) 500 milligram per gram (mg/g).
- •The participant has a clinically relevant history of allergy or signs or symptoms of severe hypersensitivity, which in the investigator's judgment, will substantially increase the participant's risk if he or she participates in the study.
- •In the opinion of the investigator, the participant has non-Fabry disease-related cause of end organ (renal, cardiovascular, central nervous system) dysfunction/failure or is receiving medications that may affect the rate of disease progression, as assessed by renal measures.
- •The participant has a positive test result at screening for hepatitis B surface antigen with detectable hepatitis B viral deoxyribonucleic acid (DNA) load, hepatitis C virus (HCV) antibody with confirmation by HCV ribonucleic acid polymerase chain reaction testing, or human immunodeficiency virus antibody.
- •The participant has received prior treatment with any of the following medications, with the exception of non-systemic use:
- •Chloroquine
- •Amiodarone
- •Monobenzone
- •Gentamicin
- •The participant is pregnant or lactating.
- •The participant has a body mass index >35 kilogram per square meter (kg/m^2).
- •The participant is treated or has been treated with any investigational drug for indication other than Fabry disease within 30 days of study start.
- •The participant and/or the participant's parent(s)/legal guardian is unable to understand the nature, scope, and possible consequences of the study.
- •The participant is unable to comply with the protocol, example, uncooperative with protocol schedule, refusal to agree to all of the study procedures, inability to return for evaluations, or is otherwise unlikely to complete the study, as determined by the investigator.
研究组 & 干预措施
REPLAGAL
Experimental
Participants received REPLAGAL 0.2 milligrams per kilogram (mg/kg) body weight, intravenous (IV) infusion, every other week (EOW) from Day 1 (Week 0) up to Week 52.
干预措施: REPLAGAL (Biological)
结局指标
主要结局
Number of Participants With Serious Treatment-emergent Adverse Events (TEAEs)
时间窗: From start of study drug administration up to 14 days after end of treatment (EOT) [up to Week 54]
An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that did not necessarily have a causal relationship with this investigational product (IP) or medicinal product. Serious AE was any untoward medical occurrence (whether considered to be related to investigational product or not) that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital abnormality/birth defect, and was an important medical event. A TEAE was defined as any event emerging at or after the initiation of treatment with an IP or any existing event that worsened in either intensity or frequency following exposure to the IP until the end of the safety follow-up period.
次要结局
- Number of Participants With TEAEs(From start of study drug administration up to 14 days after EOT (up to Week 54))
- Number of Participants With Infusion-related Reactions (IRRs)(From start of study drug administration up to Week 52)
- Number of Participants With Positive Anti-drug Antibodies (ADA) to REPLAGAL(Baseline up to Week 52)
- Number of Participants With Positive Neutralizing Antibodies (NAb) to REPLAGAL(Baseline up to Week 52)
- Number of Participants With Clinically Meaningful Changes in Laboratory Parameters(From start of study drug administration up to Week 52)
- Number of Participants With Clinically Meaningful Changes in Vital Signs(From start of study drug administration up to Week 52)
- Number of Participants With Clinically Meaningful Changes in Electrocardiogram (ECG) Parameters(From start of study drug administration up to Week 52)
- Renal Function as Assessed by Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 52(Baseline, Week 52)
- Change From Baseline in eGFR Values at Weeks 8, 16, 28, and 40(Baseline, Weeks 8, 16, 28, and 40)
- Change From Baseline in Left Ventricular Mass Index (LVMI) at Weeks 16 and 52(Baseline, Weeks 16 and 52)
- Change From Baseline in Left Ventricular Ejection Fraction (LVEF) at Weeks 16 and 52(Baseline, Weeks 16 and 52)
- Change From Baseline in Urine Protein/Creatinine Ratio(Baseline, Weeks 8, 16, 28, 40, and 52)
- Change From Baseline in Brief Pain Inventory (BPI) Short Form Pain Severity Total Score(Baseline, Weeks 8, 16, 28, 40, and 52)
- Change From Baseline in BPI Short Form Pain Interference Total Score(Baseline, Weeks 8, 16, 28, 40, and 52)
- Percent Change From Baseline in Plasma Globotriaosylsphingosine (Lyso-Gb3) Level(Baseline, Weeks 8, 16, 28, 40, and 52)
- Number of Participants With Hearing Loss as Assessed by Audiology Testing(Baseline up to Week 52)
- Area Under Serum Concentration-time Curve From the Time of Dosing to the Last Measurable Concentration (AUC0-last) of REPLAGAL(Pre-infusion (0 minutes), during infusion (20 minutes) and end of infusion (40 minutes), and post-infusion at 50, 60, 90, 120, 240, and 360 minutes at Weeks 0 and 28)
- Area Under Serum Concentration-time Curve From Time Zero Extrapolated to Infinity (AUC0-inf) of REPLAGAL(Pre-infusion (0 minutes), during infusion (20 minutes) and end of infusion (40 minutes), and post-infusion at 50, 60, 90, 120, 240, and 360 minutes at Weeks 0 and 28)
- Serum Clearance of Administered Dose (CL) of REPLAGAL(Pre-infusion (0 minutes), during infusion (20 minutes) and end of infusion (40 minutes), and post-infusion at 50, 60, 90, 120, 240, and 360 minutes at Weeks 0 and 28)
- Serum Clearance of Administered Dose Normalized Based on Body Weight of REPLAGAL(Pre-infusion (0 minutes), during infusion (20 minutes) and end of infusion (40 minutes), and post-infusion at 50, 60, 90, 120, 240, and 360 minutes at Weeks 0 and 28)
- Maximum Observed Serum Concentration (Cmax) of REPLAGAL(Pre-infusion (0 minutes), during infusion (20 minutes) and end of infusion (40 minutes), and post-infusion at 50, 60, 90, 120, 240, and 360 minutes at Weeks 0 and 28)
- Terminal Elimination Half-life (T1/2z) of REPLAGAL(Pre-infusion (0 minutes), during infusion (20 minutes) and end of infusion (40 minutes), and post-infusion at 50, 60, 90, 120, 240, and 360 minutes at Weeks 0 and 28)
- Time to Reach Maximum Observed Serum Concentration (Tmax) of REPLAGAL(Pre-infusion (0 minutes), during infusion (20 minutes) and end of infusion (40 minutes), and post-infusion at 50, 60, 90, 120, 240, and 360 minutes at Weeks 0 and 28)
- Volume of Distribution at Steady State (Vss) of REPLAGAL(Pre-infusion (0 minutes), during infusion (20 minutes) and end of infusion (40 minutes), and post-infusion at 50, 60, 90, 120, 240, and 360 minutes at Weeks 0 and 28)
- Volume of Distribution at Steady State Normalized Based on Body Weight of REPLAGAL(Pre-infusion (0 minutes), during infusion (20 minutes) and end of infusion (40 minutes), and post-infusion at 50, 60, 90, 120, 240, and 360 minutes at Weeks 0 and 28)
- Dose Normalized Area Under Serum Concentration-time Curve From Time Zero to the Last Sampling Time (AUC0-last/Dose) of REPLAGAL(Pre-infusion (0 minutes), during infusion (20 minutes) and end of infusion (40 minutes), and post-infusion at 50, 60, 90, 120, 240, and 360 minutes at Weeks 0 and 28)
- Dose Normalized Area Under the Serum Concentration-time Curve From Time Zero Extrapolated to Infinity (AUC0-inf/Dose) of REPLAGAL(Pre-infusion (0 minutes), during infusion (20 minutes) and end of infusion (40 minutes), and post-infusion at 50, 60, 90, 120, 240, and 360 minutes at Weeks 0 and 28)
- Dose Normalized Maximum Observed Serum Concentration (Cmax/Dose) of REPLAGAL(Pre-infusion (0 minutes), during infusion (20 minutes) and end of infusion (40 minutes), and post-infusion at 50, 60, 90, 120, 240, and 360 minutes at Weeks 0 and 28)
研究者
研究点 (6)
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