Pharmacokinetics/Safety of Miltefosine Allometric Dose for the Treatment of Visceral Leishmaniasis in Children in Eastern Africa

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02431143
Recruitment Status : Completed
First Posted : April 30, 2015
Last Update Posted : October 11, 2016
Information provided by (Responsible Party):
Drugs for Neglected Diseases

April 20, 2015
April 30, 2015
October 11, 2016
May 2015
April 2016   (Final data collection date for primary outcome measure)
  • 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 ]
Same as current
Complete list of historical versions of study NCT02431143 on Archive Site
Not Provided
Not Provided
Not Provided
Not Provided
Pharmacokinetics/Safety of Miltefosine Allometric Dose for the Treatment of Visceral Leishmaniasis in Children in Eastern Africa
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.

Not Provided
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Visceral Leishmaniasis
Drug: Miltefosine
Experimental: Miltefosine
allometric dosing
Intervention: Drug: Miltefosine
Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Same as current
September 2016
April 2016   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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
  • 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
Sexes Eligible for Study: All
4 Years to 12 Years   (Child)
Contact information is only displayed when the study is recruiting subjects
Kenya,   Uganda
LEAP 0714
Not Provided
Not Provided
Drugs for Neglected Diseases
Drugs for Neglected Diseases
Not Provided
Principal Investigator: Dr. Rashid Juma, MD Kenya Medical Research Institute
Drugs for Neglected Diseases
October 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP