NCT03845140已完成3 期
An Open Label, Phase III Efficacy and Safety Study of L-PZQ ODT in Schistosoma Infected Children 3 Months to 6 Years of Age, Including a 2:1 Randomized, Controlled Cohort of Schistosoma Mansoni Infected Children 4 to 6 Years of Age Treated With L PZQ ODT or Commercial PZQ (Biltricide®)
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 288
- 试验地点
- 2
- 主要终点
- Cohort 1a and Cohort 1b: Number of Participants With Clinical Cure Determined by Kato-Katz Method
研究概览
简要总结
The study would evaluate the safety and efficacy of L-praziquantel orodispersible (L-PZQ ODT) tablets in Schistosoma infected children aged 3 months to 6 years.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Months 至 6 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age of the participant is 4 to 6 years of age (Cohorts 1 and 4), 2 to 3 years of age (Cohorts 2 and 4) 3 to less than 24 months of age (Cohorts 3 and 4)
- •Participants are; Schistosoma (S.) mansoni positive (Cohorts 1, 2, and 3); diagnosis defined as positive egg counts in stool greater than or equal to ( >=) 1 egg per 1 occasion) according to World Health Organization (WHO) classification [1]: light (1 to 99 eggs per gram of feces), moderate (100 to 399 eggs per gram of feces) and heavy (>= 400 eggs per gram of feces) infections; S. haematobium positive (Cohort 4); diagnosis defined as positive egg counts in urine (>= 1 egg per 10 milliliter(mL) urine) according to WHO classification (Prevention and Control of Schistosomiasis and Soil Transmitted Helminthiasis. WHO Technical Report Series No.
- •WHO, Geneva, Switzerland, 2002).light (less than (<) 50 eggs per 10 mL of urine) and heavy (>=50 eggs per 10 mL of urine) infections
- •Participants have a minimum body weight of 8.0 Kilograms (Kg) in 2 to 6 years of age children and 5.0 Kg in 3 months to < 24 months of age infants and toddlers
- •Parent's or guardian/legally authorized representative's ability to communicate well with the Investigator and his/her delegate, to understand the protocol requirements and restrictions, and to be willing to have their children comply with the requirements of the entire study, that is:
- •To be examined by a study physician at screening and 17 to 21 days after treatment
- •To provide stool samples at screening and 17 to 21 days after treatment
- •To provide urine samples at screening and 17 to 21 days after treatment
- •To provide venous blood samples for laboratory assessments
- •To be housed in the clinic for 12 to 24 hours
- •To provide venous blood samples for pharmacokinetics (PK) assessments (for participants in the PK subset)
- •Participants have a minimum hemoglobin level of 10 gram per deciliter
排除标准
- •Participants with following medical conditions are excluded from the study; Findings in the clinical examination and/or laboratory safety examination on the treatment day, that in the opinion of the Investigator constitute a risk or a contraindication for the child's participation in the study or that could interfere with the study objectives, conduct or evaluation. This includes but is not restricted to bacterial or viral infections, such as dysentery, gastroenteritis, ascites, jaundice, etc.; Participants with seizures and/or medical history of seizures and/or other signs of potential central nervous system involvement; Participants with known cysticercosis, or with signs or symptoms (for example: subcutaneous nodules) suggestive of cysticercosis; Participants with an acute infection or other acute illness within the 7 days prior to study screening; Debilitating illness such as tuberculosis, malnutrition, etc.
- •Treatment with PZQ within the 4 weeks prior to the study screening
- •Concomitant treatment (within 2 weeks prior to enrollment) with medication that might affect the metabolism of PZQ, such as certain anti epileptics (for example: carbamazepine or phenytoin), glucocorticosteroids (for example: dexamethasone), chloroquine, rifampicin or cimetidine (see Biltricide® Summary of Product Characteristics [SmPC])
- •Treatment within the 2 weeks prior to the study screening with anti malarial medications
- •For infants and toddlers being breast fed, treatment of the mothers/wet nurses with PZQ in the 3 days prior to PZQ ODT administration
- •Participation in any clinical study within 4 weeks prior to administration of PZQ ODT, or anticipated at any time until completion of the End of study visit
- •Participants with marked increases of the liver enzymes: alanine aminotransferase and/or aspartate aminotransferase above 3 times the upper limit of normal (ULN); total bilirubin level above 1.5 times the ULN
- •Participants with hepatosplenic schistosomiasis
- •Fever, defined as temperature above 37.5 degree Celsius axillary or oral mixed S. haematobium and S. mansoni infections
研究组 & 干预措施
Cohort 1a: 4 to 6 years L-PZQ ODT 50 mg/kg
Experimental
Participants aged 4 to 6 years infected with Schistosoma (S.) mansoni received Levorotatory enantiomer of praziquantel (L-PZQ) orodispersible tablets (ODT) (150 milligrams [mg]) orally at a dose of 50 milligram per kilogram (mg/Kg) as a single oral dose after food-intake on Day 1.
干预措施: L-PZQ ODT 50 mg/kg (Drug)
Cohort 1b: 4 to 6 years Biltricide® 40 mg/kg
Active Comparator
Participants aged 4 to 6 years infected with S. mansoni received Racemate Praziquantel tablets (Biltricide®) (600 mg) orally at a dose of 40 mg/kg as a single oral dose after food-intake on Day 1.
干预措施: Biltricide® (Drug)
Cohort 2: 2 to 3 years L-PZQ ODT 50 mg/kg
Experimental
Participants aged 2 to 3 years infected with S. mansoni received L-PZQ ODT (150 mg) tablet orally at a dose of 50 mg/kg as a single oral dose after food-intake on Day 1.
干预措施: L-PZQ ODT 50 mg/kg (Drug)
Cohort 3: 3 to 24 months L-PZQ ODT 50 mg/kg
Experimental
Participants aged 3 to 24 months infected with S. mansoni received L-PZQ ODT (150 mg) tablet orally at a dose of 50 mg/kg as a single oral dose after food-intake on Day 1.
干预措施: L-PZQ ODT 50 mg/kg (Drug)
Cohort 4a: 3 months to 6 years L-PZQ ODT 50 mg/kg
Experimental
Participants aged 3 months to 6 years infected with S. haematobium received L-PZQ ODT (150 mg) tablet orally at a dose of 50 mg/kg as a single oral dose after food-intake on Day 1.
干预措施: L-PZQ ODT 50 mg/kg (Drug)
Cohort 4b: 3 months to 6 years L-PZQ ODT 60 mg/kg
Experimental
Participants aged 3 months to 6 years infected with S. haematobium received L-PZQ ODT (150 mg) tablet orally at a dose of 60 mg/kg as a single oral dose after food-intake on Day 1.
干预措施: L-PZQ ODT 60 mg/kg (Drug)
结局指标
主要结局
Cohort 1a and Cohort 1b: Number of Participants With Clinical Cure Determined by Kato-Katz Method
时间窗: at Week 3
Clinical cure was defined as no parasite egg in the stool at Week 3 as determined by the Kato-Katz method. Number of participants with clinical cure were reported.
次要结局
- Cohort 2 and Cohort 3: Number of Participants With Clinical Cure Determined by Kato-Katz Method(at Week 3)
- Cohort 1a, Cohort 1b, Cohort 2, and Cohort 3: Number of Participants With Clinical Cure Determined by Point-of-Care Circulating Cathodic Antigen (POC-CCA) Test(at Week 3)
- Change From Baseline in Hematology Parameter: Erythrocytes Mean Corpuscular Hemoglobin(Baseline, Day 1)
- Change From Baseline in Hematology Parameter: Erythrocytes(Baseline, Day 1)
- Change From Baseline in Hematology Parameters: Hematocrit, Lymphocytes/Leukocytes, Mixed Cells/Leukocytes, Neutrophils/Leukocytes(Baseline, Day 1)
- Change From Baseline in Chemistry Parameter: C Reactive Protein(Baseline, Day 1)
- Change From Baseline in Chemistry Parameter: Total Protein(Baseline, Day 1)
- Change From Baseline in Urinalyses Parameter: Specific Gravity of Urine(Baseline, Day 1)
- Change From Baseline in Urinalyses Parameter: Potential of Hydrogen (pH) of Urine(Baseline, Day 1)
- Change From Baseline in Urinalyses Parameter: Urobilinogen(Baseline, Day 1)
- Change From Baseline in Hematology Parameters: Erythrocytes Mean Corpuscular Hemoglobin (HGB) Concentration and Hemoglobin(Baseline, Day 1)
- Cohort 1a, Cohort 1b, Cohort 2 and Cohort 3: Egg Reduction Rate (Percent [%]) Determined by Kato-Katz Method(Pre-treatment, Week 3 post-treatment)
- Cohort 4a and Cohort 4b: Egg Reduction Rate (Percent [%]) Determined by Urine Filtration Technique(Pre-treatment, Weeks 3 and 5 post-treatment)
- Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs and Treatment-Related TEAEs(up to Day 40)
- Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Severity According to Qualitative Toxicity Scale(up to Day 40)
- Change From Baseline in Hematology Parameters: Leukocytes and Platelets(Baseline, Day 1)
- Change From Baseline in Chemistry Parameters: Alanine Aminotransferase and Aspartate Aminotransferase(Baseline, Day 1)
- Change From Baseline in Chemistry Parameters: Glucose, Urea and Urea Nitrogen(Baseline, Day 1)
- Change From Baseline in Chemistry Parameters: Bilirubin, Creatinine and Direct Bilirubin(Baseline, Day 1)
- Change From Baseline in Vital Signs: Pulse Rate(Baseline, Week 3)
- Change From Baseline in Vital Sign: Respiratory Rate(Baseline, Week 3)
- Maximum Observed Plasma Concentration (Cmax) of Praziquantel (PZQ) Enantiomers: R-PZQ and S-PZQ(Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose)
- Number of Participants With Reaction to Study Intervention Administration(Day 1)
- Change From Baseline in Vital Signs: Diastolic Blood Pressure and Systolic Blood Pressure(Baseline, Week 3)
- Change From Baseline in Vital Signs: Temperature(Baseline, Week 3)
- Cohort 1a, Cohort 1b, Cohort 4a and Cohort 4b: Palatability Assessment Based on Visual Analog Scale (VAS) Score(Day 1)
- Time to Reach Maximum Plasma Concentration (Tmax) of Praziquantel (PZQ) Enantiomers: R-PZQ and S-PZQ(Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose)
- Area Under the Plasma Concentration-Time Curve From Time Zero to Last Measurable Concentration (AUC0-t) of Praziquantel (PZQ) Enantiomers: R-PZQ and S-PZQ(Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose)
- Cohort 4a and Cohort 4b: Number of Participants With Clinical Cure Determined by Urine Filtration Technique(Week 3 and Week 5)
- Change From Baseline in Hematology Parameter: Erythrocytes Mean Corpuscular Volume(Baseline, Day 1)
研究者
研究点 (2)
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