Single dose of Acoziborole for treating sleeping sickness in children
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
This study is testing a single dose of Acoziborole to see if it can safely and effectively treat sleeping sickness in children aged 1 to 14.
Quick facts
| Phase | Phase2; Phase3 |
|---|---|
| Study type | Interventional |
| Enrollment | 35 (estimated) |
| Ages | 1 Year to 14 Years |
| Sex | All |
| Sponsor | Drugs for Neglected Diseases Academic / other |
| Locations | 6 sites (Mbuji-Mayi, Kasai-Oriental and 5 other locations) |
| Trial ID | NCT05433350 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the pharmacokinetics, efficacy, safety, and tolerability of a single dose of Acoziborole in pediatric patients suffering from g-HAT, a form of sleeping sickness caused by Trypanosoma brucei gambiense. The study aims to include children aged 1 to 14 years who have confirmed evidence of trypanosomes in their body fluids. Participants will be monitored for their response to the treatment and any adverse effects, contributing to the understanding of Acoziborole's effectiveness in a vulnerable population. The trial is conducted in various locations in the Democratic Republic of the Congo, where the disease remains a public health concern.
Who should consider this trial
Good fit: Ideal candidates for this study are children aged 1 to 14 years who have confirmed trypanosomiasis.
Not a fit: Patients outside the age range of 1 to 14 years or those without evidence of trypanosomes in their body fluids may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a safer and more effective option for children suffering from sleeping sickness.
How similar studies have performed: Other studies have shown promise in treating g-HAT, but the specific application of Acoziborole in pediatric patients is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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 Criteria: * 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
Where this trial is running
Mbuji-Mayi, Kasai-Oriental and 5 other locations
- Hôpital Général de Dipumba — Mbuji-Mayi, Kasai-Oriental, Congo, The Democratic Republic of the (Recruiting)
- CDTC Katanda — Katanda, Kasaï-Oriental, Congo, The Democratic Republic of the (Recruiting)
- HGR Bagata — Bagata, Kwilu, Congo, The Democratic Republic of the (Recruiting)
- Hospital of Masi-Manimba — Masi-Manimba, Kwilu, Congo, The Democratic Republic of the (Recruiting)
- General Hospital of Bandundu — Bandundu, Congo, The Democratic Republic of the (Recruiting)
- General Referral Hospital of Dubreka — Dubreka, Guinea (Recruiting)
Study contacts
- Principal investigator: Victor Kande Betu Ku Mesu, Dr — Ministry of Public Health, Hygiene and Prevention, Kinshasa
- Study coordinator: Junior Matangila, Dr
- Email: jmatangila@dndi.org
- Phone: +243 97 075 68 90
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.