Phase 3
N=208
Efficacy and Safety of Acoziborole (SCYX-7158) in Patients With Human African Trypanosomiasis Due to T.b. Gambiense
Trypanosomiasis, African · Gambiense Trypanosomiasis · Sleeping Sickness
Bottom Line
View on ClinicalTrials.gov: NCT03087955 ↗Enrolled (actual)
208
Serious AEs
10.1%
Results posted
Sep 2025
Primary outcome: Primary: Percentage of Participants With Late-stage HAT Whose Treatment Outcome Was a Success at Month 18 According to Adapted World Health Organization (WHO) Criteria — 95.2 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Acoziborole (Drug)
- Age
- Pediatric, Adult, Older Adult · 15+ yrs
- Sex
- All
- Sponsor
- Drugs for Neglected Diseases
- Primary completion
- Sep 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Late-stage HAT Whose Treatment Outcome Was a Success at Month 18 According to Adapted World Health Organization (WHO) Criteria |
95.2 | — |
| SECONDARY Percentage of Participants With Late-stage HAT Whose Treatment Outcome Was a Success at Month 12 According to Adapted WHO Criteria |
95.8 | — |
| SECONDARY Percentage of Participants With Late-stage HAT Whose Treatment Outcome Was a Success at Month 6 According to Adapted WHO Criteria |
94.6 | — |
| SECONDARY Percentage of Participants With Early- and Intermediate-stage HAT Whose Treatment Outcome Was a Success at Month 18 According to Adapted WHO Criteria |
100.0 | — |
| SECONDARY Percentage of Participants With Early- and Intermediate-stage HAT Whose Treatment Outcome Was a Success at Month 12 According to Adapted WHO Criteria |
100.0 | — |
| SECONDARY Percentage of Participants With Early- and Intermediate-stage HAT Whose Treatment Outcome Was a Success at Month 6 According to Adapted WHO Criteria |
100.0 | — |
| SECONDARY Estimated Percentage of Participants With Late-stage HAT Whose Treatment Outcome Was Not a Proven Failure at Month 18, Based on the Kaplan-Meier Analysis of Time to Proven and Definitive Failure |
96.0 | — |
| SECONDARY Number of Participants With Treatment-emergent Adverse Events (TEAEs) |
127; 28; 155 | — |
| SECONDARY Number of Participants With Serious TEAEs |
18; 3; 21 | — |
| SECONDARY Acoziborole Area Under the Curve From Time Zero to 240 Hours Post Dose (AUC0-240h) in Whole Blood Considering Concentration-time Data up to 240 Hours After a Single Administration |
2217617.78; 2051104.88; 2184795.53 | — |
| SECONDARY Mean Acoziborole Concentration in CSF After 240 Hours in Participants With Late-stage HAT |
100.8 | — |
| SECONDARY Mean Acoziborole Concentration in CSF After 240 Hours in Participants With Early- and Intermediate-stage HAT |
81.4 | — |
Summary
The goal of this study is to assess efficacy and safety of acoziborole in adult participants with Trypanosoma brucei gambiense (T.b. gambiense) HAT, either early- or intermediate-stage HAT (first arm) or late-stage HAT (second arm). Participants will receive 3 tablets of 320 mg as a single oral dose of acoziborole in the fasting state on Day 1. Participants will stay in the hospital for observation for 15 days. In total, participants will be followed for 18 months.
Eligibility Criteria
Inclusion Criteria
- Male or female patient
- 15 years of age or older
- Signed informed consent form (as well as assent from illiterate and under-age patients, and those unable to give consent)
- Karnofsky Performance Status above 50
- Able to ingest oral tablets
- Having a permanent address or being traceable by other persons
- Able to comply with the schedule of follow-up visits and requirements of the study
- Agreement to be hospitalised in order to receive treatment
- For patients with late-stage HAT:
- Confirmation of g-HAT by detection of the parasite in the blood and/or the lymph and/or the CSF, at the investigational centre
- If trypanosomes are found in the blood or lymph, but not in the CSF, the CSF WBC, measured at the investigational centre, must be above 20/μL for the patient to be included in the cohort of patients with late-stage HAT
- For patients with early- or intermediate-stage HAT:
- Confirmation of g-HAT by detection of the parasite in the blood and/or the lymph, at the investigational centre
- Absence of parasites in the CSF
- The CSF WBC, measured at the investigational centre, must be between 6 and 20/μL for the patient to be included in the cohort of patients with intermediate-stage HAT and equal to or below 5/μL for the patient to be included in the cohort of patients with early-stage HAT.
Exclusion Criteria
- Severe malnourishment, defined as body-mass index (BMI) below 16
- Pregnancy or breastfeeding (for women of child-bearing potential, confirmed pregnancy on a urine pregnancy test performed within 24 hours prior to administration of acoziborole)
- Clinically significant medical condition that could, in the opinion of the Investigator, jeopardise the patient's safety or interfere with participation in the study, including, but not limited to significant liver or cardiovascular disease, suspected or proven active infection, central nervous system trauma or seizure disorder, coma or consciousness disturbances
- Severely deteriorated health status, e.g. due to cardiovascular shock, respiratory distress syndrome or end-stage disease
- Previously treated for HAT (except prior treatment with pentamidine)
- Prior enrolment in the study
- Foreseeable difficulty complying with follow-up, including migrant worker, refugee status, itinerant trader etc.
- Current alcohol abuse or drug addiction
- Not tested for malaria and/or not having received appropriate treatment for malaria
- Not having received appropriate treatment for soil-transmitted helminthiasis
- Clinically significant abnormal laboratory values including aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) more than 2 times the upper limit of normal (ULN), total bilirubin more than 1.5 ULN, severe leukopenia at less than 2000/mm^3, Potassium below 3.5 mmol/L, any other clinically significant abnormal laboratory value
Data sourced from ClinicalTrials.gov (NCT03087955). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.