Visceral LeishmaniasisPhase 2NCT02431143
Pharmacokinetics/Safety of Miltefosine Allometric Dose for the Treatment of Visceral Leishmaniasis in Children in Eastern Africa
A Phase 2 study testing Miltefosine in people with Visceral Leishmaniasis, run by Drugs for Neglected Diseases at 2 sites in 2 countries.
Trial summary
This is a multicenter, non-comparative, open-label clinical trial to assess the Pharmacokinetics (PK) and safety of miltefosine using an allometric dose algorithm in the treatment of children with primary Visceral Leishmaniasis (VL) in eastern Africa. Read more about this study
- Who is it for?
- Ages 4 to 12 years
- Conditions the study is for
- Visceral Leishmaniasis
- What is being tested?
- Miltefosine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 2 sitesin 2 countries
- Study dates
- May 2015to Sep 2016
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 4 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients with clinical signs and symptoms of VL and confirmatory parasitological microscopic diagnosis
- Patients aged > 4 to < 12 years who are able to comply with the study protocol.
- Patients for whom written informed consent has been signed by parents(s) or legal guardian
- Weight < 30 kg
Who cannot join
- Patients who are relapse cases
- Patients who have received any anti-leishmanial drugs in the last 6 months
- Patients with severe malnutrition (for children aged <5 years, weight-for-height WHO reference curves by gender, z score <-3; for children 5-12 years, BMI-for-age WHO reference curves for gender, z score < -3)
- Patients with positive HIV diagnosis
- Patients with previous history of hypersensitivity reaction to miltefosine
- Patients suffering from a concomitant severe infection such as Tuberculosis (TB) or any other serious underlying disease (cardiac, renal, hepatic) which would preclude evaluation of the patient's response to study medication
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- Patients suffering from other conditions associated with splenomegaly such as schistosomiasis
- Pregnant or lactating women or female patient in childbearing age (reached menarche)
- Patients with haemoglobin < 5g/dl
- Patients with White Blood Cells (WBC) < 1 x 10³/mm³
- Patients with platelets < 40,000/mm³
- Patients with abnormal liver function (ALT and AST) tests of more than three times the normal range.
- Patients with bilirubin more than 1.5 times the upper normal range
- Patients with serum creatinine above the upper limit of normal (ULN) for age and gender.
- Patients with clinical signs of severe VL disease such as jaundice and bleeding
- Patients who cannot comply with the planned scheduled visits and procedures of the study protocol
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: An Open-label Clinical Trial to Assess the Pharmacokinetics and Safety of Miltefosine Allometric Dose for the Treatment of Children With Primary Visceral Leishmaniasis in Eastern Africa
This is a multicenter, non-comparative, open-label clinical trial to assess the Pharmacokinetics (PK) and safety of miltefosine using an allometric dose algorithm in the treatment of children with primary Visceral Leishmaniasis (VL) in eastern Africa. Efficacy and Pharmacodynamics (PD) will be assessed as secondary outcomes.
The proposed study aims to assess whether drug exposure in children can be increased to equivalent adult drug exposure by using the miltefosine allometric dose given BID for 28 days in paediatric VL patients aged 4-12y and whether this dose is tolerable. The present study is also expected to provide the basis for minimum time to reach sufficient drug exposure for miltefosine activity to guide optimal treatment duration to be used in combination therapy for visceral leishmaniasis. The PK data will be assessed in this trial using a compartmental population PK approach.
- Conditions
- Visceral Leishmaniasis
- Being tested
- Miltefosine
- Medical subject headings
- Leishmaniasis, Visceral
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MiltefosineDrug
| Study group | Role | What they receive |
|---|---|---|
| Miltefosine | Experimental | allometric dosing Receives: Drug: Miltefosine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pharmacokinetics Parameters (Area Under the Curve (AUC) - composite outcome)
Area Under the Curve calculation is based on several timepoints from first drug intake up to complete elimination of the drug.
Measured: During treatment, at 1 and 6 months follow-up
Safety (composite outcome) adverse events
- Frequency of Serious Adverse Events (SAEs) and Adverse Events (AEs) requiring treatment discontinuation, 2. Frequency and severity of adverse events
Measured: until day 210
Pharmacokinetics Parameters (Css/Cmax)
Measured: Day 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Apr 2015 (estimated)
- Study startMay 2015
- Primary completionApr 2016
- Study completionSep 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Visceral LeishmaniasisStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Drugs for Neglected Diseases
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Dr. Rashid Juma, MD, Principal investigator, Kenya Medical Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02431143
- Sponsor's protocol ID
- LEAP 0714
- Status last verified
- Oct 2016
- First submitted
- 20 Apr 2015
- Last updated
- 11 Oct 2016
- Results posted
- No
- Data monitoring committee
- No
Publications
Mbui J, Olobo J, Omollo R, Solomos A, Kip AE, Kirigi G, Sagaki P, Kimutai R, Were L, Omollo T, Egondi TW, Wasunna M, Alvar J, Dorlo TPC, Alves F. Pharmacokinetics, Safety, and Efficacy of an Allometric Miltefosine Regimen for the Treatment of Visceral Leishmaniasis in Eastern African Children: An Open-label, Phase II Clinical Trial. Clin Infect Dis. 2019 Apr 24;68(9):1530-1538. doi: 10.1093/cid/ciy747.
PubMed 30188978
Study at a glance
From the registry record, last updated by the sponsor on 11 Oct 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Clinical Trial to Assess the Pharmacokinetics and Safety of Miltefosine Allometric Dose for the Treatment of Children With Primary Visceral Leishmaniasis in Eastern Africa
- ClinicalTrials.gov ID
- NCT02431143
- Acronym / sponsor ID
- LEAP 0714
- Conditions
- Visceral Leishmaniasis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Drugs for Neglected DiseasesUniversity, hospital or other
- Study start
- May 2015
- Primary completion
- Apr 2016 (actual)
- Study completion
- Sep 2016 (actual)
- First posted
- 30 Apr 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a multicenter, non-comparative, open-label clinical trial to assess the Pharmacokinetics (PK) and safety of miltefosine using an allometric dose algorithm in the treatment of children with primary Visceral Leishmaniasis (VL) in eastern Africa. Efficacy and Pharmacodynamics (PD) will be assessed as secondary outcomes.
The proposed study aims to assess whether drug exposure in children can be increased to equivalent adult drug exposure by using the miltefosine allometric dose given BID for 28 days in paediatric VL patients aged 4-12y and whether this dose is tolerable. The present study is also expected to provide the basis for minimum time to reach sufficient drug exposure for miltefosine activity to guide optimal treatment duration to be used in combination therapy for visceral leishmaniasis. The PK data will be assessed in this trial using a compartmental population PK approach.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Miltefosine | Experimental | Drug: Miltefosineallometric dosing |
Who may qualify
- Ages
- 4 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Patients with clinical signs and symptoms of VL and confirmatory parasitological microscopic diagnosis
- Patients aged > 4 to < 12 years who are able to comply with the study protocol.
- Patients for whom written informed consent has been signed by parents(s) or legal guardian
- Weight < 30 kg
Key exclusion criteria (16 listed)
- Patients who are relapse cases
- Patients who have received any anti-leishmanial drugs in the last 6 months
- Patients with severe malnutrition (for children aged <5 years, weight-for-height WHO reference curves by gender, z score <-3; for children 5-12 years, BMI-for-age WHO reference curves for gender, z score < -3)
- Patients with positive HIV diagnosis
- Patients with previous history of hypersensitivity reaction to miltefosine
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 |
|---|---|---|
| Miltefosine | Drug | Miltefosine |
Primary outcome
Pharmacokinetics Parameters (Area Under the Curve (AUC) - composite outcome)
Time frame: During treatment, at 1 and 6 months follow-up
Area Under the Curve calculation is based on several timepoints from first drug intake up to complete elimination of the drug.
Safety (composite outcome) adverse events
Time frame: until day 210
1. Frequency of Serious Adverse Events (SAEs) and Adverse Events (AEs) requiring treatment discontinuation, 2. Frequency and severity of adverse events
Pharmacokinetics Parameters (Css/Cmax)
Time frame: Day 28
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Oct 2016.
- Derived from this study: PubMed 30188978Mbui J, Olobo J, Omollo R, Solomos A, Kip AE, Kirigi G, Sagaki P, Kimutai R, Were L, Omollo T, Egondi TW, Wasunna M, Alvar J, Dorlo TPC, Alves F. Pharmacokinetics, Safety, and Efficacy of an Allometric Miltefosine Regimen for the Treatment of Visceral Leishmaniasis in Eastern African Children: An Open-label, Phase II Clinical Trial. Clin Infect Dis. 2019 Apr 24;68(9):1530-1538. doi: 10.1093/cid/ciy747.
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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.
Dr. Rashid Juma, MD principal investigator
Kenya Medical Research Institute
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Miltefosine in people with Visceral Leishmaniasis, run by Drugs for Neglected Diseases 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 4 inclusion and 16 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 11 Oct 2016. 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 Kenya, Uganda. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Pharmacokinetics Parameters (Area Under the Curve (AUC) - composite outcome) (time frame: During treatment, at 1 and 6 months follow-up). The study lists 3 primary outcomes in all. 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 NCT02431143, 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 11 Oct 2016.