Trypanosomiasis, AfricanPhase 2/3OXA002NCT03087955
Efficacy and Safety of Acoziborole (SCYX-7158) in Patients With Human African Trypanosomiasis Due to T.b. Gambiense
A Phase 2/3 study testing Acoziborole in people with Trypanosomiasis, African and Gambiense Trypanosomiasis, run by Drugs for Neglected Diseases at 12 sites in 2 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 15 years and older
- Conditions the study is for
- Trypanosomiasis, African, Gambiense Trypanosomiasis, Sleeping Sickness
- What is being tested?
- Acoziborole
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 208 participants
- Where
- 12 sitesin 2 countries
- Study dates
- 11 Oct 2016to 8 Sep 2020
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 15 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
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- 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.
Who cannot join
- 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
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- 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
Trial locations and contacts
12 sites in 2 countries.
12 sites in 2 countries
Democratic Republic of the Congo11 sites
Hôpital Général de Référence de Bagata
Bagata, Kwilu
Hopital Général de Réference de Bandundu
Bandundu Province
Hôpital Général de Référence de Ngandajika
Gandajika, East Kasai
Hôpital de Référence d'Isangi
Isangi
Hôpital Secondaire de Katanda
Katanda, East Kasai
Centre de Traitement de Kimpese
Kimpese, Bas-Congo Province
Hôpital Général de Référence Roi Baudouin
Kinshasa
Hôpital Général de Référence de Kwamouth
Kwamouth, Mai Ndombe
Hôpital Général de Référence de Masi-Manimba
Masi-Manimba, Kwilu
Hôpital de Dipumba
Mbuji-Mayi, East Kasai
Centre de Traitement de Nkara
Nkara, Bandundu
About this study
Official title: Efficacy and Safety Study of Acoziborole (SCYX-7158) in Patients With Human African Trypanosomiasis (HAT) Due to Trypanosoma Brucei Gambiense: a Multicentre, Open-label, Prospective Study
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.
- Being tested
- Acoziborole
- Medical subject headings
- Trypanosomiasis, African
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AcoziboroleDrug
Also called SCYX-7158
Acoziborole 3 x 320 mg tablets (fasted state)
| Study group | Role | What they receive |
|---|---|---|
| Late-stage HAT | Experimental | Participants with confirmation of HAT by detection of the parasite in the blood and/or lymph and/or cerebrospinal fluid (CSF) at the investigational center. If testing for parasites in CSF was negative, the CSF white blood cell count, measured at the investigational center, had to be above 20 cells/µL for classification as late-stage HAT. Participants received 960 mg acoziborole as a single oral dose. Receives: Drug: Acoziborole |
| Early- and intermediate-stage HAT | Experimental | Participants with confirmation of HAT by detection of the parasite in the blood and/or lymph at the investigational center. Parasites had to be absent from the CSF. The CSF white blood cell count, measured at the investigational center, had to be between 6 and 20 cells/µL for classification as intermediate stage HAT and equal to or below 5 cells/µL for classification as early-stage HAT. Participants received 960 mg acoziborole as a single oral dose. Receives: Drug: Acoziborole |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants With Late-stage HAT Whose Treatment Outcome Was a Success at Month 18 According to Adapted World Health Organization (WHO) Criteria
Success was defined according to an algorithm based on the criteria adapted from the WHO Recommendations of the Informal Consultation on Issues for Clinical Product Development for Human African Trypanosomiasis 2007 (WHO/CDS/NTD/IDM/2007.1). Success was defined as a cure or a probable cure. Failure was defined as a relapse, probable relapse, death, use of rescue medication, loss to follow-up, refusal of all post-treatment lumbar puncture, and, in the absence of lumbar puncture at the Month 18 visit, an unfavorable outcome earlier than Month 18, or signs and symptoms evoking a relapse at Month 18. An estimate of the percentage of participants whose treatment outcome was a success at Month 18 and the 95% Jeffreys confidence interval (CI) of the estimate were provided.
Measured: 18 months post-dose
Secondary outcomes (11)
Percentage of Participants With Late-stage HAT Whose Treatment Outcome Was a Success at Month 12 According to Adapted WHO Criteria
Success was defined according to an algorithm based on the criteria adapted from the WHO Recommendations of the Informal Consultation on Issues for Clinical Product Development for Human African Trypanosomiasis 2007 (WHO/CDS/NTD/IDM/2007.1). Success was defined as a cure or a probable cure. Failure was defined as a relapse, probable relapse, death, use of rescue medication, loss to follow-up, refusal of all post-treatment lumbar puncture, and, in the absence of lumbar puncture at the end of Month 12, an unfavorable outcome earlier than end of Month 12, or signs and symptoms evoking a relapse at end of Month 12. For participants who continued after Month 12, data after Month 12 were considered in the algorithm. An estimate of the percentage of participants whose treatment outcome was a success at Month 12 and the 95% Jeffreys CI of the estimate were provided.
Measured: 12 months post-dose
Percentage of Participants With Late-stage HAT Whose Treatment Outcome Was a Success at Month 6 According to Adapted WHO Criteria
Success was defined according to an algorithm based on to the criteria adapted from the WHO Recommendations of the Informal Consultation on Issues for Clinical Product Development for Human African Trypanosomiasis 2007 (WHO/CDS/NTD/IDM/2007.1). Success was defined as a cure or a probable cure. Failure was defined as a relapse, probable relapse, death, use of rescue medication, loss to follow-up, refusal of all post-treatment lumbar puncture, and, in the absence of lumbar puncture at the end of Month 6, an unfavorable outcome earlier than end of Month 6, or signs and symptoms evoking a relapse at end of Month 6. For participants who continued after Month 6, data after Month 6 were considered in the algorithm. An estimate of the percentage of participants whose treatment outcome was a success at Month 6 and the 95% Jeffreys CI of the estimate were provided.
Measured: 6 months post-dose
Percentage of Participants With Early- and Intermediate-stage HAT Whose Treatment Outcome Was a Success at Month 18 According to Adapted WHO Criteria
Success was defined according to an algorithm based on the criteria adapted from the WHO Recommendations of the Informal Consultation on Issues for Clinical Product Development for Human African Trypanosomiasis 2007 (WHO/CDS/NTD/IDM/2007.1). Success was defined as a cure or a probable cure. Failure was defined as a relapse, probable relapse, death, use of rescue medication, loss to follow-up, refusal of all post-treatment lumbar puncture, and, in the absence of lumbar puncture at the end of Month 18, an unfavorable outcome earlier than end of Month 18, or signs and symptoms evoking a relapse at end of Month 18. An estimate of the percentage of participants whose treatment outcome was a success at Month 18 and the 95% Jeffreys CI of the estimate were provided.
Measured: 18 months post-dose
Percentage of Participants With Early- and Intermediate-stage HAT Whose Treatment Outcome Was a Success at Month 12 According to Adapted WHO Criteria
Success was defined according to an algorithm based on the criteria adapted from the WHO Recommendations of the Informal Consultation on Issues for Clinical Product Development for Human African Trypanosomiasis 2007 (WHO/CDS/NTD/IDM/2007.1). Success was defined as a cure or a probable cure. Failure was defined as a relapse, probable relapse, death, use of rescue medication, loss to follow-up, refusal of all post-treatment lumbar puncture, and, in the absence of lumbar puncture at the end of Month 12, an unfavorable outcome earlier than end of Month 12, or signs and symptoms evoking a relapse at end of Month 12. For participants who continued after Month 12, data after Month 12 were considered in the algorithm. An estimate of the percentage of participants whose treatment outcome was a success at Month 12 and the 95% Jeffreys CI of the estimate were provided.
Measured: 12 months post-dose
Percentage of Participants With Early- and Intermediate-stage HAT Whose Treatment Outcome Was a Success at Month 6 According to Adapted WHO Criteria
Success was defined according to an algorithm based on the criteria adapted from the WHO Recommendations of the Informal Consultation on Issues for Clinical Product Development for Human African Trypanosomiasis 2007 (WHO/CDS/NTD/IDM/2007.1). Success was defined as a cure or a probable cure. Failure was defined as a relapse, probable relapse, death, use of rescue medication, loss to follow-up, refusal of all post-treatment lumbar puncture, and, in the absence of lumbar puncture at the end of Month 6, an unfavorable outcome earlier than end of Month 6, or signs and symptoms evoking a relapse at end of Month 6. For participants who continued after Month 6, data after Month 6 were considered in the algorithm. An estimate of the percentage of participants whose treatment outcome was a success at Month 6 and the 95% Jeffreys CI of the estimate were provided.
Measured: 6 months post-dose
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
Failure was defined as the first objective evidence of proven and definitive (sustainable) failure, defined as death; rescue medication use; trypanosomes in any body fluid at Month 6, 12, or 18; a cerebrospinal fluid (CSF) white blood cell count (WBC) of >50 cells/μL at Month 6 followed by confirmation of failure (defined as CSF WBC >20 cells/μL at Month 12 and/or Month 18 and/or signs and symptoms evoking a relapse at Month 12 and/or Month 18); a CSF WBC >20 cells/μL at Month 12 followed by confirmation of failure (defined as CSF WBC >20 cells/μL at Month 18 and/or signs and symptoms evoking a relapse at Month 18); or a CSF WBC >20 cells/μL at Month 18. This outcome was analyzed using a Kaplan-Meier approach to estimate the cumulative rate of proven and definitive failures. The proven failure-free probability was estimated as an alternative (more liberal) success rate (95% CI) at Month 18 based on the Kaplan-Meier estimate of the rate of participants who were not proven failures
Measured: 18 months post-dose
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Occurrence of any AEs, during the observation period and until 6 months post-dose (for non-serious AEs) and until 18 months post-dose for SAEs. Analysis of AEs was based on the concept of TEAEs, defined as any AEs occurring on or after the date of study-drug administration or worsening in intensity on or after the date of study-drug administration.
Measured: From the single dose acoziborole administration until 6 months post-dose for non-serious AEs and 18 months post-dose for serious AEs
Number of Participants With Serious TEAEs
Occurrence of any serious TEAEs during the observation period and until 18 months post-dose.
Measured: From the single dose acoziborole administration until 18 months post-dose
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
Participants received a single dose of 960 mg acoziborole on Day 1. Acoziborole in whole blood was assessed pre-dose and 4, 9, 24, 48, 72, 96, and 240 hours after the single administration, (on Days 1, 2, 3, 4, 5, and 11). Data up to 240 hours post dose were considered for this analysis. Descriptive statistics of the AUC0-240h were presented. The activity of acoziborole is more exposure-dependent than concentration-dependent, therefore the exposure (AUC) was used as the main PK data for efficacy purposes.
Measured: Pre-dose and 4, 9, 24, 48, 72, 96, and 240 hours post-dose
Mean Acoziborole Concentration in CSF After 240 Hours in Participants With Late-stage HAT
Participants received a single dose of 960 mg acoziborole on Day 1. Acoziborole in CSF of participants with late-stage HAT was assessed 240 hours after the single administration (on Day 11). Descriptive statistics of the acoziborole concentration were presented.
Measured: 240 hours post-dose
Mean Acoziborole Concentration in CSF After 240 Hours in Participants With Early- and Intermediate-stage HAT
Participants received a single dose of 960 mg acoziborole on Day 1. Acoziborole in CSF of participants with early- and intermediate-stage HAT was assessed 240 hours after the single administration (on Day 11). Descriptive statistics of the acoziborole concentration were presented.
Measured: 240 hours post-dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Mar 2017
- Study start11 Oct 2016
- Primary completion8 Sep 2020
- Study completion8 Sep 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Trypanosomiasis, AfricanStudies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 208 participants (actual)
Sponsor and investigators
- Lead sponsor
- Drugs for Neglected Diseases
- Sponsor type
- University, hospital or other
- Collaborators
- Bill and Melinda Gates Foundation
- Responsible party
- Sponsor
- Investigators
- Victor Kande Betu Kumeso, Dr, Principal investigator, Ministère de la Santé, The Democratic Republic of the Congo
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03087955
- Sponsor's protocol ID
- DNDi-OXA-02-HAT
- Acronym
- OXA002
- Status last verified
- Aug 2025
- First submitted
- 7 Mar 2017
- Last updated
- 17 Sep 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: The data underlying the results of this study are available upon request because they contain potentially sensitive personal information, which must be de-identified at the individual level. Interested researchers may request access to de-identified participant data from Vivli, the data-sharing partner of the DNDi, commissioner of this study, at https://vivli.org/ourmember/dndi/.
Publications
Betu Kumeso VK, Kalonji WM, Rembry S, Valverde Mordt O, Ngolo Tete D, Pretre A, Delhomme S, Ilunga Wa Kyhi M, Camara M, Catusse J, Schneitter S, Nusbaumer M, Mwamba Miaka E, Mahenzi Mbembo H, Makaya Mayawula J, Layba Camara M, Akwaso Massa F, Kaninda Badibabi L, Kasongo Bonama A, Kavunga Lukula P, Mutanda Kalonji S, Mariero Philemon P, Mokilifi Nganyonyi R, Embana Mankiara H, Asuka Akongo Nguba A, Kobo Muanza V, Mulenge Nasandhel E, Fifi Nzeza Bambuwu A, Scherrer B, Strub-Wourgaft N, Tarral A. Efficacy and safety of acoziborole in patients with human African trypanosomiasis caused by Trypanosoma brucei gambiense: a multicentre, open-label, single-arm, phase 2/3 trial. Lancet Infect Dis. 2023 Apr;23(4):463-470. doi: 10.1016/S1473-3099(22)00660-0. Epub 2022 Nov 29.
PubMed 36460027
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety Study of Acoziborole (SCYX-7158) in Patients With Human African Trypanosomiasis (HAT) Due to Trypanosoma Brucei Gambiense: a Multicentre, Open-label, Prospective Study
- ClinicalTrials.gov ID
- NCT03087955
- Acronym / sponsor ID
- OXA002 / DNDi-OXA-02-HAT
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 208 participants (actual)
- Lead sponsor
- Drugs for Neglected DiseasesUniversity, hospital or other
- Collaborators
- Bill and Melinda Gates Foundation
- Study start
- 11 Oct 2016 (actual)
- Primary completion
- 8 Sep 2020 (actual)
- Study completion
- 8 Sep 2020 (actual)
- First posted
- 23 Mar 2017 (actual)
- Results
- Posted 17 Sep 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Late-stage HAT | Experimental | Drug: AcoziboroleParticipants with confirmation of HAT by detection of the parasite in the blood and/or lymph and/or cerebrospinal fluid (CSF) at the investigational center. If testing for parasites in CSF was negative, the CSF white blood cell count, measured at the investigational center, had to be above 20 cells/µL for classification as late-stage HAT. Participants received 960 mg acoziborole as a single oral dose. |
| Early- and intermediate-stage HAT | Experimental | Drug: AcoziboroleParticipants with confirmation of HAT by detection of the parasite in the blood and/or lymph at the investigational center. Parasites had to be absent from the CSF. The CSF white blood cell count, measured at the investigational center, had to be between 6 and 20 cells/µL for classification as intermediate stage HAT and equal to or below 5 cells/µL for classification as early-stage HAT. Participants received 960 mg acoziborole as a single oral dose. |
Who may qualify
- Ages
- 15 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (15 listed)
- 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
Key exclusion criteria (11 listed)
- 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)
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 |
|---|---|---|
| AcoziboroleAlso: SCYX-7158Acoziborole 3 x 320 mg tablets (fasted state) | Drug | Early- and intermediate-stage HAT, Late-stage HAT |
Primary outcome
Percentage of Participants With Late-stage HAT Whose Treatment Outcome Was a Success at Month 18 According to Adapted World Health Organization (WHO) Criteria
Time frame: 18 months post-dose
Success was defined according to an algorithm based on the criteria adapted from the WHO Recommendations of the Informal Consultation on Issues for Clinical Product Development for Human African Trypanosomiasis 2007 (WHO/CDS/NTD/IDM/2007.1). Success was defined as a cure or a probable cure. Failure was defined as a relapse, probable relapse, death, use of rescue medication, loss to follow-up, refusal of all post-treatment lumbar puncture, and, in the absence of lumbar puncture at the Month 18 visit, an unfavorable outcome earlier than Month 18, or signs and symptoms evoking a relapse at Month 18. An estimate of the percentage of participants whose treatment outcome was a success at Month 18 and the 95% Jeffreys confidence interval (CI) of the estimate were provided.
11 secondary outcomes
Percentage of Participants With Late-stage HAT Whose Treatment Outcome Was a Success at Month 12 According to Adapted WHO Criteria
Time frame: 12 months post-dose
Percentage of Participants With Late-stage HAT Whose Treatment Outcome Was a Success at Month 6 According to Adapted WHO Criteria
Time frame: 6 months post-dose
Percentage of Participants With Early- and Intermediate-stage HAT Whose Treatment Outcome Was a Success at Month 18 According to Adapted WHO Criteria
Time frame: 18 months post-dose
Percentage of Participants With Early- and Intermediate-stage HAT Whose Treatment Outcome Was a Success at Month 12 According to Adapted WHO Criteria
Time frame: 12 months post-dose
Percentage of Participants With Early- and Intermediate-stage HAT Whose Treatment Outcome Was a Success at Month 6 According to Adapted WHO Criteria
Time frame: 6 months post-dose
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
Time frame: 18 months post-dose
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Time frame: From the single dose acoziborole administration until 6 months post-dose for non-serious AEs and 18 months post-dose for serious AEs
Number of Participants With Serious TEAEs
Time frame: From the single dose acoziborole administration until 18 months post-dose
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
Time frame: Pre-dose and 4, 9, 24, 48, 72, 96, and 240 hours post-dose
Mean Acoziborole Concentration in CSF After 240 Hours in Participants With Late-stage HAT
Time frame: 240 hours post-dose
Mean Acoziborole Concentration in CSF After 240 Hours in Participants With Early- and Intermediate-stage HAT
Time frame: 240 hours post-dose
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 36460027Betu Kumeso VK, Kalonji WM, Rembry S, Valverde Mordt O, Ngolo Tete D, Pretre A, Delhomme S, Ilunga Wa Kyhi M, Camara M, Catusse J, Schneitter S, Nusbaumer M, Mwamba Miaka E, Mahenzi Mbembo H, Makaya Mayawula J, Layba Camara M, Akwaso Massa F, Kaninda Badibabi L, Kasongo Bonama A, Kavunga Lukula P, Mutanda Kalonji S, Mariero Philemon P, Mokilifi Nganyonyi R, Embana Mankiara H, Asuka Akongo Nguba A, Kobo Muanza V, Mulenge Nasandhel E, Fifi Nzeza Bambuwu A, Scherrer B, Strub-Wourgaft N, Tarral A. Efficacy and safety of acoziborole in patients with human African trypanosomiasis caused by Trypanosoma brucei gambiense: a multicentre, open-label, single-arm, phase 2/3 trial. Lancet Infect Dis. 2023 Apr;23(4):463-470. doi: 10.1016/S1473-3099(22)00660-0. Epub 2022 Nov 29.
- Individual participant dataSharing planned: Yes. The data underlying the results of this study are available upon request because they contain potentially sensitive personal information, which must be de-identified at the individual level. Interested researchers may request access to de-identified participant data from Vivli, the data-sharing partner of the DNDi, commissioner of this study, at https://vivli.org/ourmember/dndi/.
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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.
Victor Kande Betu Kumeso, Dr principal investigator
Ministère de la Santé, The Democratic Republic of the Congo
12 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Acoziborole in people with Trypanosomiasis, African and Gambiense Trypanosomiasis, run by Drugs for Neglected Diseases at 12 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 15 inclusion and 11 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 17 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in Democratic Republic of the Congo, Guinea. 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 combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 17 Sep 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Percentage of Participants With Late-stage HAT Whose Treatment Outcome Was a Success at Month 18 According to Adapted World Health Organization (WHO) Criteria (time frame: 18 months post-dose). 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 NCT03087955, 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 17 Sep 2025.