SchistosomiasisPhase 2NCT02806232
An Open Label Dose Finding Safety and Efficacy in Children and Infants Infected With Schistosomiasis (S.Mansoni)
A Phase 2 study testing Biltricide (racemate praziquantel) oral tablets, Racemate Praziquantel ODT and Levo Praziquantel ODT in people with Schistosomiasis, run by Merck KGaA, Darmstadt, Germany at 1 site in Germany.
Trial summary
The Phase II study consisted of two parts, part 1 is open label, randomized, controlled and exploratory dose finding in children aged between 2 and 6 years infected with S. mansoni. Part 2 investigated efficacy and safety with the selected formulation and dosage in S. 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?
- Biltricide (racemate praziquantel) oral tablets, Racemate Praziquantel ODT and Levo Praziquantel ODT
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 444 participants
- Where
- 1 siteGermany
- Study dates
- 12 Jun 2016to 17 Nov 2018
- Phase
- Phase 2
- 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
- Male and female children aged 2 to 6 years (Part 1) and 3 to 24 months (Part 2)
- S. mansoni positive diagnosis defined as positive egg counts in stool (greater than [>]1 egg/1 occasion) according to World Health Organization (WHO) classification : light (1-99 eggs per gram of faeces), moderate (100-399 eggs per gram of faeces) and heavy (greater than or equal to [>=]400 eggs per gram of faeces) infections
- Minimum weight of 8.0 kg in 2- to 6-year-old children and of 4.0 kg in 3- to 24-month infants
- Parents/legal representative ability to communicate well with the Investigator, to understand the protocol requirements and restrictions, and willing their children to comply with the requirements of the entire trial, i.e.
- To be examined by a study physician at screening and 14-21 days after treatment
- To provide stool and urine samples at screening, 24 hours and 8 days after treatment, as well as 14-21 days after treatment
- To provide finger prick blood samples for Pharmacokinetics (PK) studies and blood samples for safety assessments
Who cannot join
- Treatment in the 4 weeks prior to study screening with Praziquantel (PZQ) , other anti-helminthic, antimalarial or anti-retroviral compounds or any other medication that might affect the PK of PZQ such as certain antiepileptics (e.g., carbamazepine or phenytoin), glucocorticosteroids (e.g., dexamethasone), chloroquine, rifampicin or cimetidine
- For children being breast fed, treatment of the mothers/wet nurses with PZQ in the 3 days prior to administration of Investigational medicinal product
- Previous history of adverse reactions associated with PZQ treatment
- Marked increases of the liver transaminases (alanine aminotransferase and/or aspartate aminotransferase) above 3x Upper Limit of Normal (ULN)
- History of acute or severe chronic disease including hepato-splenic schistosomiasis
- Fever defined as temperature above 38.0 degree centigrade
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- Debilitating illnesses such as tuberculosis, malnutrition, etc. as well as a medical history of seizures
- Mixed S. haematobium and S. mansoni infections
- Findings in the clinical examination of schistosome-infected children participating in the study as performed by the study clinician on the treatment day, that in the opinion of the Investigator constitutes a risk or a contraindication for the participation of the subject in the study or that could interfere with the study objectives, conduct or evaluation
- Unlikelihood to comply with the protocol requirements, instructions and trial-related restrictions, e.g., uncooperative attitude, inability to return for follow-up visits, and improbability of completing the trial
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Open-label, Dose-finding, 2-parts, Efficacy Phase II Study With Three Formulations (Racemate Raziquantel Commercial Oral Tablets, New Oral Disintegrating Tablets of Racemate Praziquantel and L-praziquantel) in Schistosomiasis (S. Mansoni) Infected Children Aged 2-6 Years (Part 1), Followed by an Assessment of Efficacy and Safety With the Selected Formulation and Dosage in S. Mansoni Infected Infants Aged 3-24 Months (Part 2)
The Phase II study consisted of two parts, part 1 is open label, randomized, controlled and exploratory dose finding in children aged between 2 and 6 years infected with S. mansoni. Part 2 investigated efficacy and safety with the selected formulation and dosage in S. mansoni infected children aged between 3 months - 2 years.
- Conditions
- Schistosomiasis
- Being tested
- Biltricide (racemate praziquantel) oral tablets, Racemate Praziquantel ODT, Levo Praziquantel ODT
- Keywords
- Praziquantel ODT formulation, Biltricide
- Medical subject headings
- Schistosomiasis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Biltricide (racemate praziquantel) oral tabletsDrug
Biltricide (600 mg tablet) was administered to participants at a dose of 20 mg/kg in Part 1, Cohort 1 and at a dose of 40mg/kg in Part 1, Cohort 2.
Racemate Praziquantel ODTDrug
Racemate Praziquantel (PZQ) (150) mg was administered at a dose of 40 mg/kg in Part 1, Cohort 3 and at a dose of 60 mg/kg in Part 1, Cohort 4.
Levo Praziquantel ODTDrug
Levo PZQ (150 mg) was administered at a dose of 30 mg/kg in Part 1 Cohort 5, 45 mg/kg Part 1 Cohort 6, 60 mg/kg Part 1 Cohort 7, 50 mg/kg Part 2 Cohort 8, and 50 mg/kg Part 2 Cohort 9.
| Study group | Role | What they receive |
|---|---|---|
| Part 1, Cohort 1: Biltricide (racemate praziquantel) 20 mg/kg | Experimental | Participants received Biltricide (600 mg tablet) administered orally at a dose of 20 milligram per kilogram (mg/kg), three times a day on treatment Day 1. Receives: Drug: Biltricide (racemate praziquantel) oral tablets |
| Part 1, Cohort 2: Biltricide (racemate praziquantel) 40 mg/kg | Experimental | Participants received Biltricide (600 mg tablet) administered orally at a dose of 40 mg/kg as a single dose on treatment Day 1. Receives: Drug: Biltricide (racemate praziquantel) oral tablets |
| Part 1, Cohort 3: Racemate Praziquantel 40 mg/kg | Experimental | Participants received Racemate Praziquantel oral dispersible tablet (ODT) (150 mg) administered orally at a dose of 40 mg/kg as a single dose on treatment Day 1. Receives: Drug: Racemate Praziquantel ODT |
| Part 1, Cohort 4: Racemate Praziquantel 60 mg/kg | Experimental | Participants received Racemate Praziquantel ODT (150 mg) administered orally at a dose of 60 mg/kg as a single dose on treatment Day 1. Receives: Drug: Racemate Praziquantel ODT |
| Part 1, Cohort 5: Levo Praziquantel 30 mg/kg | Experimental | Participants received Levo Praziquantel ODT (150 mg tablet) administered orally at a dose of 30 mg/kg as a single dose on treatment Day 1. Receives: Drug: Levo Praziquantel ODT |
| Part 1, Cohort 6: Levo Praziquantel 45 mg/kg | Experimental | Participants received Levo Praziquantel ODT (150 mg tablet) administered orally at a dose of 45 mg/kg as a single dose on treatment Day 1. Receives: Drug: Levo Praziquantel ODT |
| Part 1, Cohort 7: Levo Praziquantel 60 mg/kg | Experimental | Participants received Levo Praziquantel ODT (150 mg tablet) administered orally at a dose of 60 mg/kg as a single dose on treatment Day 1. Receives: Drug: Levo Praziquantel ODT |
| Part 2, Cohort 8: Levo Praziquantel 50 mg/kg | Experimental | Participants aged 13-24 months months received Levo Praziquantel ODT (150 mg) administered orally at a dose of 50 mg/kg as a single dose on treatment day 1. Receives: Drug: Levo Praziquantel ODT |
| Part 2, Cohort 9: Levo Praziquantel 50 mg/kg | Experimental | Participants aged 3 to 12 months received Levo Praziquantel ODT (150 mg) administered orally at a dose of 50 mg/kg as a single dose on treatment day 1. Receives: Drug: Levo Praziquantel ODT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Clinical Cure Determined by Kato-Katz Method
Clinical cure was defined as zero egg counts at 14-21 days post treatment as determined by the Kato-Katz method. Number of participants with clinical cure were reported.
Measured: 14-21 days post treatment
Secondary outcomes (2)
Egg Reduction Rate (Percent)
Percent reduction in egg count was calculated as geometric mean egg count at post-treatment minus geometric mean egg count at baseline (before treatment) divided by geometric mean egg count at baseline.
Measured: Baseline, 14-21 days post treatment
Number of Participants With Clinical Cure Determined by Point-of-Care Circulating Cathodic Antigen (POC-CCA) Test
Clinical Cure defined as no parasite eggs in the stools as assessed by the commercially available POC-CCA assay for S. mansoni. Number of participants with clinical cure were reported.
Measured: Day 2, Day 8 and 14-21 days post treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jun 2016 (estimated)
- Study start12 Jun 2016
- Primary completion30 Oct 2018
- Study completion17 Nov 2018
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.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 444 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
- NCT02806232
- Sponsor's protocol ID
- 200661-0005
- Status last verified
- Oct 2019
- First submitted
- 16 Jun 2016
- Last updated
- 20 Nov 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 20 Nov 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-label, Dose-finding, 2-parts, Efficacy Phase II Study With Three Formulations (Racemate Raziquantel Commercial Oral Tablets, New Oral Disintegrating Tablets of Racemate Praziquantel and L-praziquantel) in Schistosomiasis (S. Mansoni) Infected Children Aged 2-6 Years (Part 1), Followed by an Assessment of Efficacy and Safety With the Selected Formulation and Dosage in S. Mansoni Infected Infants Aged 3-24 Months (Part 2)
- ClinicalTrials.gov ID
- NCT02806232
- Acronym / sponsor ID
- 200661-0005
- Conditions
- Schistosomiasis
- Keywords
- Praziquantel ODT formulation, Biltricide
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 444 participants (actual)
- Lead sponsor
- Merck KGaA, Darmstadt, GermanyIndustry (pharma, biotech, devices)
- Study start
- 12 Jun 2016 (actual)
- Primary completion
- 30 Oct 2018 (actual)
- Study completion
- 17 Nov 2018 (actual)
- First posted
- 20 Jun 2016 (estimated)
- Results
- Posted 20 Nov 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The Phase II study consisted of two parts, part 1 is open label, randomized, controlled and exploratory dose finding in children aged between 2 and 6 years infected with S. mansoni. Part 2 investigated efficacy and safety with the selected formulation and dosage in S. mansoni infected children aged between 3 months - 2 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 9
| Arm | Type | Receives |
|---|---|---|
| Part 1, Cohort 1: Biltricide (racemate praziquantel) 20 mg/kg | Experimental | Drug: Biltricide (racemate praziquantel) oral tabletsParticipants received Biltricide (600 mg tablet) administered orally at a dose of 20 milligram per kilogram (mg/kg), three times a day on treatment Day 1. |
| Part 1, Cohort 2: Biltricide (racemate praziquantel) 40 mg/kg | Experimental | Drug: Biltricide (racemate praziquantel) oral tabletsParticipants received Biltricide (600 mg tablet) administered orally at a dose of 40 mg/kg as a single dose on treatment Day 1. |
| Part 1, Cohort 3: Racemate Praziquantel 40 mg/kg | Experimental | Drug: Racemate Praziquantel ODTParticipants received Racemate Praziquantel oral dispersible tablet (ODT) (150 mg) administered orally at a dose of 40 mg/kg as a single dose on treatment Day 1. |
| Part 1, Cohort 4: Racemate Praziquantel 60 mg/kg | Experimental | Drug: Racemate Praziquantel ODTParticipants received Racemate Praziquantel ODT (150 mg) administered orally at a dose of 60 mg/kg as a single dose on treatment Day 1. |
| Part 1, Cohort 5: Levo Praziquantel 30 mg/kg | Experimental | Drug: Levo Praziquantel ODTParticipants received Levo Praziquantel ODT (150 mg tablet) administered orally at a dose of 30 mg/kg as a single dose on treatment Day 1. |
| Part 1, Cohort 6: Levo Praziquantel 45 mg/kg | Experimental | Drug: Levo Praziquantel ODTParticipants received Levo Praziquantel ODT (150 mg tablet) administered orally at a dose of 45 mg/kg as a single dose on treatment Day 1. |
| Part 1, Cohort 7: Levo Praziquantel 60 mg/kg | Experimental | Drug: Levo Praziquantel ODTParticipants received Levo Praziquantel ODT (150 mg tablet) administered orally at a dose of 60 mg/kg as a single dose on treatment Day 1. |
| Part 2, Cohort 8: Levo Praziquantel 50 mg/kg | Experimental | Drug: Levo Praziquantel ODTParticipants aged 13-24 months months received Levo Praziquantel ODT (150 mg) administered orally at a dose of 50 mg/kg as a single dose on treatment day 1. |
| Part 2, Cohort 9: Levo Praziquantel 50 mg/kg | Experimental | Drug: Levo Praziquantel ODTParticipants aged 3 to 12 months received Levo Praziquantel ODT (150 mg) administered orally at a dose of 50 mg/kg as a single dose on treatment day 1. |
Who may qualify
- Ages
- 3 months to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Male and female children aged 2 to 6 years (Part 1) and 3 to 24 months (Part 2)
- S. mansoni positive diagnosis defined as positive egg counts in stool (greater than [>]1 egg/1 occasion) according to World Health Organization (WHO) classification : light (1-99 eggs per gram of faeces), moderate (100-399 eggs per gram of faeces) and heavy (greater than or equal to [>=]400 eggs per gram of faeces) infections
- Minimum weight of 8.0 kg in 2- to 6-year-old children and of 4.0 kg in 3- to 24-month infants
- To be examined by a study physician at screening and 14-21 days after treatment
- To provide stool and urine samples at screening, 24 hours and 8 days after treatment, as well as 14-21 days after treatment
Key exclusion criteria (10 listed)
- Treatment in the 4 weeks prior to study screening with Praziquantel (PZQ) , other anti-helminthic, antimalarial or anti-retroviral compounds or any other medication that might affect the PK of PZQ such as certain antiepileptics (e.g., carbamazepine or phenytoin), glucocorticosteroids (e.g., dexamethasone), chloroquine, rifampicin or cimetidine
- For children being breast fed, treatment of the mothers/wet nurses with PZQ in the 3 days prior to administration of Investigational medicinal product
- Previous history of adverse reactions associated with PZQ treatment
- Marked increases of the liver transaminases (alanine aminotransferase and/or aspartate aminotransferase) above 3x Upper Limit of Normal (ULN)
- History of acute or severe chronic disease including hepato-splenic schistosomiasis
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 |
|---|---|---|
| Biltricide (racemate praziquantel) oral tabletsBiltricide (600 mg tablet) was administered to participants at a dose of 20 mg/kg in Part 1, Cohort 1 and at a dose of 40mg/kg in Part 1, Cohort 2. | Drug | Part 1, Cohort 1: Biltricide (racemate praziquantel) 20 mg/kg, Part 1, Cohort 2: Biltricide (racemate praziquantel) 40 mg/kg |
| Racemate Praziquantel ODTRacemate Praziquantel (PZQ) (150) mg was administered at a dose of 40 mg/kg in Part 1, Cohort 3 and at a dose of 60 mg/kg in Part 1, Cohort 4. | Drug | Part 1, Cohort 3: Racemate Praziquantel 40 mg/kg, Part 1, Cohort 4: Racemate Praziquantel 60 mg/kg |
| Levo Praziquantel ODTLevo PZQ (150 mg) was administered at a dose of 30 mg/kg in Part 1 Cohort 5, 45 mg/kg Part 1 Cohort 6, 60 mg/kg Part 1 Cohort 7, 50 mg/kg Part 2 Cohort 8, and 50 mg/kg Part 2 Cohort 9. | Drug | Part 1, Cohort 5: Levo Praziquantel 30 mg/kg, Part 1, Cohort 6: Levo Praziquantel 45 mg/kg, Part 1, Cohort 7: Levo Praziquantel 60 mg/kg, Part 2, Cohort 8: Levo Praziquantel 50 mg/kg, Part 2, Cohort 9: Levo Praziquantel 50 mg/kg |
Primary outcome
Number of Participants With Clinical Cure Determined by Kato-Katz Method
Time frame: 14-21 days post treatment
Clinical cure was defined as zero egg counts at 14-21 days post treatment as determined by the Kato-Katz method. Number of participants with clinical cure were reported.
2 secondary outcomes
Egg Reduction Rate (Percent)
Time frame: Baseline, 14-21 days post treatment
Number of Participants With Clinical Cure Determined by Point-of-Care Circulating Cathodic Antigen (POC-CCA) Test
Time frame: Day 2, Day 8 and 14-21 days post treatment
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Nov 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Biltricide (racemate praziquantel) oral tablets, Racemate Praziquantel ODT and Levo Praziquantel ODT in people with Schistosomiasis, run by Merck KGaA, Darmstadt, Germany at 1 site in Germany.
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 7 inclusion and 10 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 20 Nov 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 20 Nov 2019). 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?
Number of Participants With Clinical Cure Determined by Kato-Katz Method (time frame: 14-21 days post treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 20 Nov 2019.