SchistosomiasisPhase 3NCT03845140
L-PZQ ODT in Schistosoma Infected Children
A Phase 3 study testing L-PZQ ODT 50 mg/kg, Biltricide® and L-PZQ ODT 60 mg/kg in people with Schistosomiasis, run by Merck KGaA, Darmstadt, Germany at 2 sites in 2 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 3 months to 6 years
- Conditions the study is for
- Schistosomiasis
- What is being tested?
- L-PZQ ODT 50 mg/kg, Biltricide® and L-PZQ ODT 60 mg/kg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 288 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 2 Sep 2019to 11 Oct 2021
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 months to 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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. 912. 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
Show all 11Show fewer
- 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
Who cannot join
- 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
Show all 9Show fewer
- 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
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: 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®)
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.
- Conditions
- Schistosomiasis
- Being tested
- L-PZQ ODT 50 mg/kg, Biltricide®, L-PZQ ODT 60 mg/kg
- Keywords
- Schistosomiasis, Children, L-praziquantel, Biltricide®
- Medical subject headings
- Schistosomiasis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
L-PZQ ODT 50 mg/kgDrug
Participants received single oral dose of L-PZQ ODT 50 mg/Kg on Day 1.
Biltricide®Drug
Participants received single oral dose of Biltricide® 40 mg/kg on Day 1.
L-PZQ ODT 60 mg/kgDrug
Participant received single oral dose of L-PZQ ODT 60 mg/kg on Day 1.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: L-PZQ ODT 50 mg/kg |
| 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. Receives: Drug: Biltricide® |
| 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. Receives: Drug: L-PZQ ODT 50 mg/kg |
| 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. Receives: Drug: L-PZQ ODT 50 mg/kg |
| 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. Receives: Drug: L-PZQ ODT 50 mg/kg |
| 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. Receives: Drug: L-PZQ ODT 60 mg/kg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cohort 1a and Cohort 1b: Number of Participants With Clinical Cure Determined by Kato-Katz Method
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.
Measured: at Week 3
Secondary outcomes (30)
Cohort 2 and Cohort 3: Number of Participants With Clinical Cure Determined by Kato-Katz Method
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.
Measured: at Week 3
Cohort 4a and Cohort 4b: Number of Participants With Clinical Cure Determined by Urine Filtration Technique
Clinical cure was defined as no parasite egg in the urine samples at follow up as determined by the urine filtration technique. Number of participants with clinical cure were reported.
Measured: Week 3 and Week 5
Cohort 1a, Cohort 1b, Cohort 2 and Cohort 3: Egg Reduction Rate (Percent [%]) Determined by Kato-Katz Method
Percentage of reduction in group mean egg count was calculated as relative difference between the post-treatment arithmetic mean egg count and pre-treatment arithmetic mean egg count at baseline count, (pre-treatment mean egg count minus post-treatment mean egg count divided by pre-treatment mean egg count) *100. Egg counts were determined by the Kato-Katz method.
Measured: Pre-treatment, Week 3 post-treatment
Cohort 4a and Cohort 4b: Egg Reduction Rate (Percent [%]) Determined by Urine Filtration Technique
Percentage of reduction in group mean egg count was calculated as relative difference between the post-treatment arithmetic mean egg count and pre-treatment arithmetic mean egg count at baseline count, (pre-treatment mean egg count minus post-treatment mean egg count divided by pre-treatment mean egg count) *100. Egg counts were determined by the urine filtration technique.
Measured: Pre-treatment, Weeks 3 and 5 post-treatment
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
Clinical cure is defined as absence of test line in the POC-CCA test cassette (that is no Schistosoma antigens detected). Number of participants with clinical cure were reported.
Measured: at Week 3
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs and Treatment-Related TEAEs
Adverse Event (AE) was defined any untoward medical occurrence in a participant administered with a study drug, which does not necessarily had a causal relationship with this treatment. Serious AE was defined AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial/prolonged inpatient hospitalization; congenital anomaly/birth defect. TEAEs: TEAEs were defined as those events with onset dates/time occurring after study intervention administration or events that worsen after study intervention administration. TEAEs included serious TEAEs and non-serious TEAEs. Treatment-related TEAEs: reasonably related to the study intervention.
Measured: up to Day 40
Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Severity According to Qualitative Toxicity Scale
Severity of TEAEs were graded using Qualitative Toxicity Scale, as follows: Mild: Participant is aware of the event or symptom, but the event or symptom is easily tolerated; Moderate: Participant experiences sufficient discomfort to interfere with or reduce his or her usual level of activity; Severe: Significant impairment of functioning: the participant is unable to carry out his or her usual activities. Number of participants with TEAEs by severity were reported.
Measured: up to Day 40
Change From Baseline in Hematology Parameter: Erythrocytes Mean Corpuscular Hemoglobin
Blood samples were collected in a fasted condition (after a fast of at least 12 hours) to analyze the hematology parameter: erythrocytes mean corpuscular hemoglobin. Change from baseline in hematology parameter: erythrocytes mean corpuscular hemoglobin at Day 1 were reported.
Measured: Baseline, Day 1
Change From Baseline in Hematology Parameters: Erythrocytes Mean Corpuscular Hemoglobin (HGB) Concentration and Hemoglobin
Blood samples were collected in a fasted condition (after a fast of at least 12 hours) to analyze the hematology parameters: erythrocytes mean corpuscular HGB concentration and hemoglobin. Change from baseline in hematology parameters: erythrocytes mean corpuscular HGB concentration and hemoglobin at Day 1 were reported.
Measured: Baseline, Day 1
Change From Baseline in Hematology Parameter: Erythrocytes Mean Corpuscular Volume
Blood samples were collected in a fasted condition (after a fast of at least 12 hours) to analyze the hematology parameter: erythrocytes mean corpuscular volume. Change from baseline in hematology parameter: erythrocytes mean corpuscular volume at Day 1 were reported.
Measured: Baseline, Day 1
Change From Baseline in Hematology Parameter: Erythrocytes
Blood samples were collected in a fasted condition (after a fast of at least 12 hours) to analyze the hematology parameter: erythrocytes. Change from baseline in hematology parameter: erythrocytes at Day 1 were reported.
Measured: Baseline, Day 1
Change From Baseline in Hematology Parameters: Hematocrit, Lymphocytes/Leukocytes, Mixed Cells/Leukocytes, Neutrophils/Leukocytes
Blood samples were collected in a fasted condition (after a fast of at least 12 hours) to analyze the hematology parameters: hematocrit, lymphocytes/leukocytes, mixed cells/leukocytes, neutrophils/leukocytes. Change from baseline in hematology parameters: hematocrit, lymphocytes/leukocytes, mixed cells/leukocytes, neutrophils/leukocytes at Day 1 were reported.
Measured: Baseline, Day 1
Change From Baseline in Hematology Parameters: Leukocytes and Platelets
Blood samples were collected in a fasted condition (after a fast of at least 12 hours) to analyze the hematology parameters: leukocytes and platelets. Change from baseline in hematology parameters: leukocytes and platelets at Day 1 were reported.
Measured: Baseline, Day 1
Change From Baseline in Chemistry Parameters: Alanine Aminotransferase and Aspartate Aminotransferase
Blood samples were collected in a fasted condition (after a fast of at least 12 hours) to analyze the chemistry parameters: Alanine Aminotransferase and Aspartate Aminotransferase. Change from baseline in chemistry parameters: Alanine Aminotransferase and Aspartate Aminotransferase at Day 1 were reported.
Measured: Baseline, Day 1
Change From Baseline in Chemistry Parameters: Bilirubin, Creatinine and Direct Bilirubin
Blood samples were collected in a fasted condition (after a fast of at least 12 hours) to analyze the chemistry parameters: bilirubin, creatinine and direct bilirubin. Change from baseline in chemistry parameters: bilirubin, creatinine and direct bilirubin at Day 1 were reported.
Measured: Baseline, Day 1
Change From Baseline in Chemistry Parameter: C Reactive Protein
Blood samples were collected in a fasted condition (after a fast of at least 12 hours) to analyze the chemistry parameter: C reactive protein. Change from baseline in chemistry parameter: C reactive protein at Day 1 were reported.
Measured: Baseline, Day 1
Change From Baseline in Chemistry Parameters: Glucose, Urea and Urea Nitrogen
Blood samples were collected in a fasted condition (after a fast of at least 12 hours) to analyze the chemistry parameters: glucose, urea and urea nitrogen. Change from baseline in chemistry parameters: glucose, urea and urea nitrogen at Day 1 were reported.
Measured: Baseline, Day 1
Change From Baseline in Chemistry Parameter: Total Protein
Blood samples were collected in a fasted condition (after a fast of at least 12 hours) to analyze the chemistry parameter: total Protein. Change from baseline in chemistry parameter: total Protein at Day 1 were reported.
Measured: Baseline, Day 1
Change From Baseline in Urinalyses Parameter: Specific Gravity of Urine
Urine samples were collected in a fasted condition (after a fast of at least 12 hours) to analyze the urinalyses parameters: specific gravity. Change from baseline in urinalyses parameter: specific gravity Day 1 was reported.
Measured: Baseline, Day 1
Change From Baseline in Urinalyses Parameter: Potential of Hydrogen (pH) of Urine
Urine samples were collected in a fasted condition (after a fast of at least 12 hours) to analyze the urinalyses parameter: pH. Change from baseline in urinalyses parameter: pH at Day 1 was reported.
Measured: Baseline, Day 1
Change From Baseline in Urinalyses Parameter: Urobilinogen
Urine samples were collected in a fasted condition (after a fast of at least 12 hours) to analyze the urinalyses parameter: urobilinogen. Change from baseline in urinalyses parameter: urobilinogen at Day 1 were reported.
Measured: Baseline, Day 1
Change From Baseline in Vital Signs: Diastolic Blood Pressure and Systolic Blood Pressure
Diastolic blood pressure and systolic blood pressure were measured after at least 5 minutes of rest for the participant in a quiet sitting without distractions. Change from baseline in vital signs: diastolic blood pressure and systolic blood pressure at Week 3 were reported.
Measured: Baseline, Week 3
Change From Baseline in Vital Signs: Pulse Rate
Pulse rate was measured after at least 5 minutes of rest for the participant in a quiet sitting without distractions. Change from baseline in vital sign: pulse rate at Week 3 was reported.
Measured: Baseline, Week 3
Change From Baseline in Vital Sign: Respiratory Rate
Respiratory rate was measured after at least 5 minutes of rest for the participant in a quiet sitting without distractions. Change from baseline in vital sign: respiratory rate at Week 3 was reported.
Measured: Baseline, Week 3
Change From Baseline in Vital Signs: Temperature
Temperature was measured after at least 5 minutes of rest for the participant in a quiet sitting without distractions. Change from baseline in vital sign: temperature at Week 3 was reported.
Measured: Baseline, Week 3
Number of Participants With Reaction to Study Intervention Administration
Reaction to study intervention administration were recorded to describe tolerability as assessed by nurse/site staff for all children enrolled in the study. Reactions categorized as spitting, crying, diarrheas, sleepiness, abdominal pain, fever, vomiting and other. Number of participants with reaction to study intervention administration reported.
Measured: Day 1
Cohort 1a, Cohort 1b, Cohort 4a and Cohort 4b: Palatability Assessment Based on Visual Analog Scale (VAS) Score
Palatability of the study intervention was assessed using a human gustatory sensation test (100-millimeter [mm] visual analog scale [VAS]) incorporating a facial hedonic scale, where lower score (0) indicates "not acceptable/not liked at all" and higher score (100) indicates "very acceptable/liked very much".
Measured: Day 1
Maximum Observed Plasma Concentration (Cmax) of Praziquantel (PZQ) Enantiomers: R-PZQ and S-PZQ
Cmax was obtained directly from the plasma concentration versus time curve.
Measured: Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose
Time to Reach Maximum Plasma Concentration (Tmax) of Praziquantel (PZQ) Enantiomers: R-PZQ and S-PZQ
Tmax was obtained directly from the plasma concentration versus time curve.
Measured: 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
Area under the plasma concentration versus time curve from time zero to the last sampling time t at which the concentration was at or above the lower limit of quantification (LLOQ). AUC0-t was calculated according to the mixed log-linear trapezoidal rule.
Measured: Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Feb 2019
- Study start2 Sep 2019
- Primary completion11 Oct 2021
- Study completion11 Oct 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for SchistosomiasisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 288 participants (actual)
Sponsor and investigators
- Lead sponsor
- Merck KGaA, Darmstadt, Germany
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Responsible, Study director, Merck KGaA, Darmstadt, Germany
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03845140
- Sponsor's protocol ID
- MS200661_0003
- Status last verified
- Sep 2023
- First submitted
- 15 Feb 2019
- Last updated
- 21 Mar 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: We are committed to enhancing public health through responsible sharing of clinical trial data. Following approval of a new product or a new indication for an approved product in both the US and European Union, the study sponsor and/or its affiliated companies will share study protocols, anonymized patient data and study level data, and redacted clinical study reports with qualified scientific and medical researchers, upon request, as necessary for conducting legitimate research. Further information on how to request data can be found on our website bit.ly/IPD21
Publications
N'Goran EK, Odiere MR, Assande Aka R, Ouattara M, Aka NAD, Ogutu B, Rawago F, Bagchus WM, Bodding M, Kourany-Lefoll E, Tappert A, Yin X, Bezuidenhout D, Badenhorst H, Huber E, Dalken B, Haj-Ali Saflo O. Efficacy, safety, and palatability of arpraziquantel (L-praziquantel) orodispersible tablets in children aged 3 months to 6 years infected with Schistosoma in Cote d'Ivoire and Kenya: an open-label, partly randomised, phase 3 trial. Lancet Infect Dis. 2023 Jul;23(7):867-876. doi: 10.1016/S1473-3099(23)00048-8. Epub 2023 Mar 6.
PubMed 36893784
Study at a glance
From the registry record, last updated by the sponsor on 21 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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®)
- ClinicalTrials.gov ID
- NCT03845140
- Acronym / sponsor ID
- MS200661_0003
- Conditions
- Schistosomiasis
- Keywords
- Schistosomiasis, Children, L-praziquantel, Biltricide®
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 288 participants (actual)
- Lead sponsor
- Merck KGaA, Darmstadt, GermanyIndustry (pharma, biotech, devices)
- Study start
- 2 Sep 2019 (actual)
- Primary completion
- 11 Oct 2021 (actual)
- Study completion
- 11 Oct 2021 (actual)
- First posted
- 19 Feb 2019 (actual)
- Results
- Posted 21 Mar 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Cohort 1a: 4 to 6 years L-PZQ ODT 50 mg/kg | Experimental | Drug: L-PZQ ODT 50 mg/kgParticipants 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. |
| Cohort 1b: 4 to 6 years Biltricide® 40 mg/kg | Active comparator | Drug: Biltricide®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. |
| Cohort 2: 2 to 3 years L-PZQ ODT 50 mg/kg | Experimental | Drug: L-PZQ ODT 50 mg/kgParticipants 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. |
| Cohort 3: 3 to 24 months L-PZQ ODT 50 mg/kg | Experimental | Drug: L-PZQ ODT 50 mg/kgParticipants 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. |
| Cohort 4a: 3 months to 6 years L-PZQ ODT 50 mg/kg | Experimental | Drug: L-PZQ ODT 50 mg/kgParticipants 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. |
| Cohort 4b: 3 months to 6 years L-PZQ ODT 60 mg/kg | Experimental | Drug: L-PZQ ODT 60 mg/kgParticipants 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. |
Who may qualify
- Ages
- 3 months to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (11 listed)
- 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. 912. 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:
- Participants have a minimum hemoglobin level of 10 gram per deciliter
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| L-PZQ ODT 50 mg/kgParticipants received single oral dose of L-PZQ ODT 50 mg/Kg on Day 1. | Drug | Cohort 1a: 4 to 6 years L-PZQ ODT 50 mg/kg, Cohort 2: 2 to 3 years L-PZQ ODT 50 mg/kg, Cohort 3: 3 to 24 months L-PZQ ODT 50 mg/kg, Cohort 4a: 3 months to 6 years L-PZQ ODT 50 mg/kg |
| Biltricide®Participants received single oral dose of Biltricide® 40 mg/kg on Day 1. | Drug | Cohort 1b: 4 to 6 years Biltricide® 40 mg/kg |
| L-PZQ ODT 60 mg/kgParticipant received single oral dose of L-PZQ ODT 60 mg/kg on Day 1. | Drug | Cohort 4b: 3 months to 6 years L-PZQ ODT 60 mg/kg |
Primary outcome
Cohort 1a and Cohort 1b: Number of Participants With Clinical Cure Determined by Kato-Katz Method
Time frame: 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.
30 secondary outcomes
Cohort 2 and Cohort 3: Number of Participants With Clinical Cure Determined by Kato-Katz Method
Time frame: at Week 3
Cohort 4a and Cohort 4b: Number of Participants With Clinical Cure Determined by Urine Filtration Technique
Time frame: Week 3 and Week 5
Cohort 1a, Cohort 1b, Cohort 2 and Cohort 3: Egg Reduction Rate (Percent [%]) Determined by Kato-Katz Method
Time frame: Pre-treatment, Week 3 post-treatment
Cohort 4a and Cohort 4b: Egg Reduction Rate (Percent [%]) Determined by Urine Filtration Technique
Time frame: Pre-treatment, Weeks 3 and 5 post-treatment
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
Time frame: at Week 3
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious TEAEs and Treatment-Related TEAEs
Time frame: up to Day 40
Number of Participants With Treatment Emergent Adverse Events (TEAEs) by Severity According to Qualitative Toxicity Scale
Time frame: up to Day 40
Change From Baseline in Hematology Parameter: Erythrocytes Mean Corpuscular Hemoglobin
Time frame: Baseline, Day 1
Change From Baseline in Hematology Parameters: Erythrocytes Mean Corpuscular Hemoglobin (HGB) Concentration and Hemoglobin
Time frame: Baseline, Day 1
Change From Baseline in Hematology Parameter: Erythrocytes Mean Corpuscular Volume
Time frame: Baseline, Day 1
Change From Baseline in Hematology Parameter: Erythrocytes
Time frame: Baseline, Day 1
Change From Baseline in Hematology Parameters: Hematocrit, Lymphocytes/Leukocytes, Mixed Cells/Leukocytes, Neutrophils/Leukocytes
Time frame: Baseline, Day 1
Change From Baseline in Hematology Parameters: Leukocytes and Platelets
Time frame: Baseline, Day 1
Change From Baseline in Chemistry Parameters: Alanine Aminotransferase and Aspartate Aminotransferase
Time frame: Baseline, Day 1
Change From Baseline in Chemistry Parameters: Bilirubin, Creatinine and Direct Bilirubin
Time frame: Baseline, Day 1
Change From Baseline in Chemistry Parameter: C Reactive Protein
Time frame: Baseline, Day 1
Change From Baseline in Chemistry Parameters: Glucose, Urea and Urea Nitrogen
Time frame: Baseline, Day 1
Change From Baseline in Chemistry Parameter: Total Protein
Time frame: Baseline, Day 1
Change From Baseline in Urinalyses Parameter: Specific Gravity of Urine
Time frame: Baseline, Day 1
Change From Baseline in Urinalyses Parameter: Potential of Hydrogen (pH) of Urine
Time frame: Baseline, Day 1
Change From Baseline in Urinalyses Parameter: Urobilinogen
Time frame: Baseline, Day 1
Change From Baseline in Vital Signs: Diastolic Blood Pressure and Systolic Blood Pressure
Time frame: Baseline, Week 3
Change From Baseline in Vital Signs: Pulse Rate
Time frame: Baseline, Week 3
Change From Baseline in Vital Sign: Respiratory Rate
Time frame: Baseline, Week 3
Change From Baseline in Vital Signs: Temperature
Time frame: Baseline, Week 3
Number of Participants With Reaction to Study Intervention Administration
Time frame: Day 1
Cohort 1a, Cohort 1b, Cohort 4a and Cohort 4b: Palatability Assessment Based on Visual Analog Scale (VAS) Score
Time frame: Day 1
Maximum Observed Plasma Concentration (Cmax) of Praziquantel (PZQ) Enantiomers: R-PZQ and S-PZQ
Time frame: Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose
Time to Reach Maximum Plasma Concentration (Tmax) of Praziquantel (PZQ) Enantiomers: R-PZQ and S-PZQ
Time frame: 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
Time frame: Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, 12 hours post-dose
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Mar 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 36893784N'Goran EK, Odiere MR, Assande Aka R, Ouattara M, Aka NAD, Ogutu B, Rawago F, Bagchus WM, Bodding M, Kourany-Lefoll E, Tappert A, Yin X, Bezuidenhout D, Badenhorst H, Huber E, Dalken B, Haj-Ali Saflo O. Efficacy, safety, and palatability of arpraziquantel (L-praziquantel) orodispersible tablets in children aged 3 months to 6 years infected with Schistosoma in Cote d'Ivoire and Kenya: an open-label, partly randomised, phase 3 trial. Lancet Infect Dis. 2023 Jul;23(7):867-876. doi: 10.1016/S1473-3099(23)00048-8. Epub 2023 Mar 6.
- Trial Awareness and Transparency websiteLink provided by the sponsor.
- Medical Information Location Map - Med Info ContactsLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. We are committed to enhancing public health through responsible sharing of clinical trial data. Following approval of a new product or a new indication for an approved product in both the US and European Union, the study sponsor and/or its affiliated companies will share study protocols, anonymized patient data and study level data, and redacted clinical study reports with qualified scientific and medical researchers, upon request, as necessary for conducting legitimate research. Further information on how to request data can be found on our website bit.ly/IPD21
Related trials for Schistosomiasis
Not yet recruitingPhase 3
A Phase III Trial to Evaluate the Safety and Efficacy of Anti-inflammatory Drugs in Addition to Praziquantel in Girls and Women With Female Genital Schistosomiasis
Female Genital Schistosomiasis
Not yet recruitingPhase 2/3
Different Treatment Frequencies for Hepatic Fibrosis Due to Schistosomiasis
Schistosomiasis, Schistosoma Mansoni
Not yet recruiting
Assessment of Right Ventricular-Pulmonary Arterial Coupling in Schistosomiasis-Associated Pulmonary Arterial Hypertension
Schistosomiasis
Recruiting
Expanding Access to Preventive Chemotherapy Among Mobile and Migrant Populations
Onchocerciasis (River Blindness), Schistosomiasis
Not yet recruitingObservational study
Nanobody-based Schistosomiasis Urine Test Kit Research
Schistosomiasis Haematobia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Responsible study director
Merck KGaA, Darmstadt, Germany
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing L-PZQ ODT 50 mg/kg, Biltricide® and L-PZQ ODT 60 mg/kg in people with Schistosomiasis, run by Merck KGaA, Darmstadt, Germany at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Côte d’Ivoire, Kenya. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 21 Mar 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cohort 1a and Cohort 1b: Number of Participants With Clinical Cure Determined by Kato-Katz Method (time frame: at Week 3). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03845140, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Mar 2024.