Pharmacokinetic, Efficacy, Safety and Tolerability Study of a Single Dose of Acoziborole in g-HAT Paediatric Patients
Pharmacokinetic, Efficacy, Safety, and Tolerability Study of a Single Dose of Acoziborole Under Fasting Conditions in Paediatric Patients From 1 to 14 Years of Age and With g-HAT: a Multicentre, Open-label Study
Drugs for Neglected Diseases
35 participants
Jul 9, 2022
INTERVENTIONAL
Conditions
Summary
Acoziborole has been studied in an open-label pivotal Phase II/III trial (DNDi-OXA-02-HAT) in the DRC and Guinea. As the numbers of reported cases diminish, resources for surveillance and specialised screening will also taper. This decrease, coupled with the loss of diagnostic skills and disease management expertise, will lead to a weak and less specialised HAT technical environment. The history of g-HAT has shown that outbreaks or re-emergence of the disease have already happened under different circumstances when surveillance was relaxed or simply because the populations at risk live in areas of political instability, limiting access to specialised care. Even with a steady decrease of reported incidence, no model can currently predict that HAT could not re-emerge. Although g-HAT is predominantly a disease of adults, children are also affected at diverse rates depending on the geographical and behavioural characteristics in the different areas of disease transmission. Hence efforts are needed to develop a paediatric formulation from a new generation of oral HAT treatments.
Eligibility
Inclusion Criteria10
- Signed informed consent from one parent or from the legal representative
- Assent from the paediatric patient (for paediatric patients >6 years of age) to participate in the study, collected in the presence of an impartial witness
- Between 1 and 14 years of age and between 10 and ≤40 kg (as per the requirements of step 1 and step 2)
- Male or female
- Evidence of trypanosomes in any body fluid (blood or lymph or CSF)
- Having a permanent address and able to comply with the schedule of follow-up visits
- Agreement to not take part in any other clinical trials during the participation in this study
- For pubescent girls of childbearing potential must agree to have avoid getting pregnant during the screening period and up to 3 months after acoziborole dosing by using an acceptable effective contraception method (sexual abstinence, condom, injectable progestin-only contraceptive)
- Agreement not to continue any treatment (including traditional/herbal medicine) without consulting the investigator
- Agreement not to start a treatment (including traditional/herbal medicine) during 4 months after intake of acoziborole without consulting the Investigator
Exclusion Criteria18
- Previous treatment for g-HAT
- Refusal to participate in the study, expressed by the paediatric patient and/or parent or legal representative
- Complicated severe acute malnutrition as defined by weight for height (-3 SDs Z score)
- Unable to take medication by the oral route
- Clinically significant medical condition (other than HAT) that could, in the opinion of the Investigator, jeopardise the patient's safety or interfere with participation in the study
- Any condition (excluding HAT-specific symptoms) that affects the patient's and/or parent's ability to communicate with the Investigator as required to complete the study
- Prior enrolment in the study or prior intake of acoziborole
- Foreseeable difficulty complying with follow-up, including family of migrant workers, refugee status, itinerant trader, etc.
- Clinically significant laboratory test abnormality, with:
- Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) more than twice the upper limit of normal (ULN)
- Total bilirubin more than 1.5 x ULN
- Severe leukopenia at <2000/mm3
- Potassium <3.5 mmol/L
- Any other clinically significant laboratory test abnormality
- Pregnancy confirmed by a positive urine pregnancy test (during the screening period and/or within 24 hours prior to the start of treatment) for pubescent girls of childbearing potential
- Not tested for malaria and/or not having received appropriate treatment for malaria
- Not having received appropriate treatment for soil-transmitted helminthiasis
- Paediatric patient who is taking praziquantel, erythromycin, ritonavir, lopinavir, or darunavir and could not stop this treatment
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Interventions
Two different mode of administration will be used during the study depending on the body weight and on the step of the study: whole tablets of 320 mg dose for paediatric patients weighing 30 to 40 kg in step 1 whole or crushed tablets for paediatric patients weighing 10 to 40 kg in step 2
Locations(6)
View Full Details on ClinicalTrials.gov
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NCT05433350