Trial · NCT03087955
Efficacy and Safety of Acoziborole (SCYX-7158) in Patients With Human African Trypanosomiasis Due to T.b. Gambiense
NCT03087955, Phase 2, Phase 3, Completed, Drugs for Neglected Diseases; results first posted 2025-09-17
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy and Safety of Acoziborole (SCYX-7158) in Patients With Human African Trypanosomiasis Due to T.b. Gambiense. Trials, record NCT03087955. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03087955
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety of Acoziborole (SCYX-7158) in Patients With Human African Trypanosomiasis Due to T.b. Gambiense
- NCT number
- NCT03087955
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 208 participants, actual
- Lead sponsor
- Drugs for Neglected Diseases
- Conditions
- Trypanosomiasis, African, Gambiense Trypanosomiasis, Sleeping Sickness
- Interventions
- Acoziborole
- First posted
- 2017-03-23
- Last updated
- 2025-09-17
- Results first posted
- 2025-09-17
- Limitations and caveats
The principal limitation of this study was the lack of comparator or control group, instead comparisons were made with a yardstick based on historical data. The sample size was based on the maximum feasible enrolment within a reasonable timeframe, because of the challenges of enrolling patients with HAT in clinical trials given the drastic decline in prevalence, and in order to expose as many participants to the test drug as possible.
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| 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 | 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. |
| Secondary | Percentage of Participants With Late-stage HAT Whose Treatment Outcome Was a Success at Month 12 According to Adapted WHO Criteria | 12 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 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. |
| Secondary | Percentage of Participants With Late-stage HAT Whose Treatment Outcome Was a Success at Month 6 According to Adapted WHO Criteria | 6 months post-dose | 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. |
| Secondary | Percentage of Participants With Early- and Intermediate-stage HAT Whose Treatment Outcome Was a Success at Month 18 According to Adapted WHO Criteria | 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 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. |
| Secondary | Percentage of Participants With Early- and Intermediate-stage HAT Whose Treatment Outcome Was a Success at Month 12 According to Adapted WHO Criteria | 12 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 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. |
| Secondary | Percentage of Participants With Early- and Intermediate-stage HAT Whose Treatment Outcome Was a Success at Month 6 According to Adapted WHO Criteria | 6 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 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. |
| 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 | 18 months post-dose | 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 |
| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | From the single dose acoziborole administration until 6 months post-dose for non-serious AEs and 18 months post-dose for serious AEs | 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. |
| Secondary | Number of Participants With Serious TEAEs | From the single dose acoziborole administration until 18 months post-dose | Occurrence of any serious TEAEs during the observation period and until 18 months post-dose. |
| 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 | Pre-dose and 4, 9, 24, 48, 72, 96, and 240 hours post-dose | 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. |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes12
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| 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 | 18 months post-dose | The modified intention-to-treat (mITT) set included all participants who received at least 1 tablet of acoziborole, excluding participants who fled the region due to armed conflict or natural disaster or due to force majeure and for whom no failure was detected early\* and no data were available at Month 12 and Month 18.\*\* \* Parasite, need for rescue medication, death, or more than 50 WBC/μL in CSF at Month 6.~\*\* Due to armed conflict, natural disaster, or force majeure affecting the site. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Late-stage HAT Whose Treatment Outcome Was a Success at Month 12 According to Adapted WHO Criteria | 12 months post-dose | The modified intention-to-treat (mITT) set included all participants who received at least 1 tablet of acoziborole, excluding participants who fled the region due to armed conflict or natural disaster or due to force majeure and for whom no failure was detected early\* and no data were available at Month 12 and Month 18.\*\* \* Parasite, need for rescue medication, death, or more than 50 WBC/μL in CSF at Month 6.~\*\* Due to armed conflict, natural disaster, or force majeure affecting the site. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Late-stage HAT Whose Treatment Outcome Was a Success at Month 6 According to Adapted WHO Criteria | 6 months post-dose | The modified intention-to-treat (mITT) set included all participants who received at least 1 tablet of acoziborole, excluding participants who fled the region due to armed conflict or natural disaster or due to force majeure and for whom no failure was detected early\* and no data were available at Month 12 and Month 18.\*\* \* Parasite, need for rescue medication, death, or more than 50 WBC/μL in CSF at Month 6.~\*\* Due to armed conflict, natural disaster, or force majeure affecting the site. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Early- and Intermediate-stage HAT Whose Treatment Outcome Was a Success at Month 18 According to Adapted WHO Criteria | 18 months post-dose | The modified intention-to-treat (mITT) set included all participants who received at least 1 tablet of acoziborole, excluding participants who fled the region due to armed conflict or natural disaster or due to force majeure and for whom no failure was detected early\* and no data were available at Month 12 and Month 18.\*\* \* Parasite, need for rescue medication, death, or more than 50 WBC/μL in CSF at Month 6.~\*\* Due to armed conflict, natural disaster, or force majeure affecting the site. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Early- and Intermediate-stage HAT Whose Treatment Outcome Was a Success at Month 12 According to Adapted WHO Criteria | 12 months post-dose | The modified intention-to-treat (mITT) set included all participants who received at least 1 tablet of acoziborole, excluding participants who fled the region due to armed conflict or natural disaster or due to force majeure and for whom no failure was detected early\* and no data were available at Month 12 and Month 18.\*\* \* Parasite, need for rescue medication, death, or more than 50 WBC/μL in CSF at Month 6.~\*\* Due to armed conflict, natural disaster, or force majeure affecting the site. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Early- and Intermediate-stage HAT Whose Treatment Outcome Was a Success at Month 6 According to Adapted WHO Criteria | 6 months post-dose | The modified intention-to-treat (mITT) set included all participants who received at least 1 tablet of acoziborole, excluding participants who fled the region due to armed conflict or natural disaster or due to force majeure and for whom no failure was detected early\* and no data were available at Month 12 and Month 18.\*\* \* Parasite, need for rescue medication, death, or more than 50 WBC/μL in CSF at Month 6.~\*\* Due to armed conflict, natural disaster, or force majeure affecting the site. | percentage of participants | NUMBER |
| 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 | 18 months post-dose | The modified intention-to-treat (mITT) set included all participants who received at least 1 tablet of acoziborole, excluding participants who fled the region due to armed conflict or natural disaster or due to force majeure and for whom no failure was detected early\* and no data were available at Month 12 and Month 18.\*\* \* Parasite, need for rescue medication, death, or more than 50 WBC/μL in CSF at Month 6.~\*\* Due to armed conflict, natural disaster, or force majeure affecting the site. | percentage of participants | NUMBER |
| SECONDARY | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | From the single dose acoziborole administration until 6 months post-dose for non-serious AEs and 18 months post-dose for serious AEs | The Treated set included all participants who received at least one tablet of acoziborole. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Serious TEAEs | From the single dose acoziborole administration until 18 months post-dose | The Treated set included all participants who received at least one tablet of acoziborole. | Participants | COUNT_OF_PARTICIPANTS |
| 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 | Pre-dose and 4, 9, 24, 48, 72, 96, and 240 hours post-dose | The pharmacokinetic (PK) analysis set included participants who received an oral dose of 960 mg acoziborole, as far as the information about time and amount was available, and for whom at least one blood sample for PK was available. | h*ng/mL | MEAN |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 outcomesResults by group18
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Late-stage HAT Whose Treatment Outcome Was a Success at Month 18 According to Adapted World Health Organization (WHO) Criteria | Late-stage HAT | 95.2 | 95% Confidence Interval 91.2 to 97.7 | percentage of participants | |
| Percentage of Participants With Late-stage HAT Whose Treatment Outcome Was a Success at Month 12 According to Adapted WHO Criteria | Late-stage HAT | 95.8 | 95% Confidence Interval 91.9 to 98.1 | percentage of participants | |
| Percentage of Participants With Late-stage HAT Whose Treatment Outcome Was a Success at Month 6 According to Adapted WHO Criteria | Late-stage HAT | 94.6 | 95% Confidence Interval 90.4 to 97.3 | percentage of participants | |
| Percentage of Participants With Early- and Intermediate-stage HAT Whose Treatment Outcome Was a Success at Month 18 According to Adapted WHO Criteria | Early- and Intermediate-stage HAT | 100.0 | 95% Confidence Interval 94.1 to 100.0 | percentage of participants | |
| Percentage of Participants With Early- and Intermediate-stage HAT Whose Treatment Outcome Was a Success at Month 12 According to Adapted WHO Criteria | Early- and Intermediate-stage HAT | 100.0 | 95% Confidence Interval 94.1 to 100.0 | percentage of participants | |
| Percentage of Participants With Early- and Intermediate-stage HAT Whose Treatment Outcome Was a Success at Month 6 According to Adapted WHO Criteria | Early- and Intermediate-stage HAT | 100.0 | 95% Confidence Interval 94.1 to 100.0 | percentage of participants | |
| 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 | Late-stage HAT | 96.0 | 95% Confidence Interval 91.0 to 98.0 | percentage of participants | |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Late-stage HAT | 127 | Participants | ||
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Early- and Intermediate-stage HAT | 28 | Participants | ||
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Any Stage HAT | 155 | Participants |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Late-stage HAT | 167 | |
| Overall Study | STARTED | Early- and Intermediate-stage HAT | 41 | |
| Overall Study | COMPLETED | Late-stage HAT | 160 | |
| Overall Study | COMPLETED | Early- and Intermediate-stage HAT | 41 | |
| Overall Study | NOT COMPLETED | Late-stage HAT | 7 | |
| Overall Study | NOT COMPLETED | Early- and Intermediate-stage HAT | 0 |
Source ClinicalTrials.gov
Adverse events304
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Late-stage HAT | 6 | 167 | 6 |
| other | Anaemia | Blood and lymphatic system disorders | Early- and Intermediate-stage HAT | 1 | 41 | 1 |
| other | Splenomegaly | Blood and lymphatic system disorders | Late-stage HAT | 1 | 167 | 1 |
| other | Splenomegaly | Blood and lymphatic system disorders | Early- and Intermediate-stage HAT | 0 | 41 | 0 |
| other | Arrhythmia | Cardiac disorders | Late-stage HAT | 0 | 167 | 0 |
| other | Arrhythmia | Cardiac disorders | Early- and Intermediate-stage HAT | 1 | 41 | 1 |
| other | Atrioventricular block first degree | Cardiac disorders | Late-stage HAT | 4 | 167 | 4 |
| other | Atrioventricular block first degree | Cardiac disorders | Early- and Intermediate-stage HAT | 0 | 41 | 0 |
| other | Bradycardia | Cardiac disorders | Late-stage HAT | 1 | 167 | 1 |
| other | Bradycardia | Cardiac disorders | Early- and Intermediate-stage HAT | 0 | 41 | 0 |
1 to 10 of 304Source ClinicalTrials.gov
See all 304 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Late-stage HAT | 4 | 167 |
| Total, all-cause mortality | Early- and Intermediate-stage HAT | 0 | 41 |
| Total, other adverse events | Late-stage HAT | 127 | 167 |
| Total, other adverse events | Early- and Intermediate-stage HAT | 27 | 41 |
| Total, serious adverse events | Late-stage HAT | 18 | 167 |
| Total, serious adverse events | Early- and Intermediate-stage HAT | 3 | 41 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 36460027 | RESULT |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Late-stage HAT vs Early- and Intermediate-stage HAT: deaths from any cause | Trial comparisons |
| Late-stage HAT vs Early- and Intermediate-stage HAT: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
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