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| Name | Class |
|---|---|
| Bill and Melinda Gates Foundation | OTHER |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
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| 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Acoziborole | Drug | Acoziborole 3 x 320 mg tablets (fasted state) |
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| Measure | Description | Time Frame |
|---|---|---|
| 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. | 18 months post-dose |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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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:
For patients with early- or intermediate-stage HAT:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Victor Kande Betu Kumeso, Dr | Ministère de la Santé, The Democratic Republic of the Congo | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Centre de Traitement de Nkara | Nkara | Bandundu | Democratic Republic of the Congo | |||
| Centre de Traitement de Kimpese |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36460027 | Result | 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. |
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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/.
Available upon request (see above)
Available upon request (see above)
The pre-treatment period of up to 15 days included pre-screening, screening, and treatment of concurrent malaria/soil-transmitted helminthiasis. At the end of this period, all participants (regardless of HAT-stage) were treated with acoziborole.
The study was conducted at Human African Trypanosomiasis (HAT) treatment centers in the Democratic Republic of Congo and Guinea. A total of 260 HAT-positive participants signed informed consent and 208 participants (167 with late- and 41 with early- and intermediate-stage HAT) were included in the study and treated. 52 participants were not included, mainly due to exclusion criteria met and/or inclusion criteria not met. Participants were enrolled between 11 October 2016 and 25 March 2019.
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| ID | Title | Description |
|---|---|---|
| FG000 | Late-stage HAT | 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. Acoziborole: Acoziborole 960 mg as a single oral dose (3 x 320 mg tablets; fasted state) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Aug 11, 2020 | Jun 4, 2025 |
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| 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. | 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. | 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. | 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. | 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 | 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. | 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. | 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. | 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. | 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. | 240 hours post-dose |
| Kimpese |
| Bas-Congo Province |
| Democratic Republic of the Congo |
| Hôpital Général de Référence de Ngandajika | Gandajika | East Kasai | Democratic Republic of the Congo |
| Hôpital Secondaire de Katanda | Katanda | East Kasai | Democratic Republic of the Congo |
| Hôpital de Dipumba | Mbuji-Mayi | East Kasai | Democratic Republic of the Congo |
| Hôpital Général de Référence de Bagata | Bagata | Kwilu | Democratic Republic of the Congo |
| Hôpital Général de Référence de Masi-Manimba | Masi-Manimba | Kwilu | Democratic Republic of the Congo |
| Hôpital Général de Référence de Kwamouth | Kwamouth | Mai Ndombe | Democratic Republic of the Congo |
| Hopital Général de Réference de Bandundu | Bandundu Province | Democratic Republic of the Congo |
| Hôpital de Référence d'Isangi | Isangi | Democratic Republic of the Congo |
| Hôpital Général de Référence Roi Baudouin | Kinshasa | Democratic Republic of the Congo |
| Centre de Traitement de la THA de Dubreka | Dubréka | Dubreka | Guinea |
| FG001 | Early- and Intermediate-stage HAT | 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. Acoziborole: Acoziborole 960 mg as a single oral dose (3 x 320 mg tablets; fasted state) |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Late-stage HAT | 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. Acoziborole: Acoziborole 960 mg as a single oral dose (3 x 320 mg tablets; fasted state) |
| BG001 | Early- and Intermediate-stage HAT | 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. Acoziborole: Acoziborole 960 mg as a single oral dose (3 x 320 mg tablets; fasted state) |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| White blood cells (WBC) in cerebrospinal fluid (CSF) | Mean | Standard Deviation | cells/µL |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
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| 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 | 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. | 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. | Posted | Number | 95% Confidence Interval | percentage of participants | 18 months post-dose |
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| Secondary | 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. | 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. | Posted | Number | 95% Confidence Interval | percentage of participants | 12 months post-dose |
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| Secondary | 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. | 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. | Posted | Number | 95% Confidence Interval | percentage of participants | 6 months post-dose |
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| Secondary | 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. | 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. | Posted | Number | 95% Confidence Interval | percentage of participants | 18 months post-dose |
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| Secondary | 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. | 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. | Posted | Number | 95% Confidence Interval | percentage of participants | 12 months post-dose |
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| Secondary | 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. | 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. | Posted | Number | 95% Confidence Interval | percentage of participants | 6 months post-dose |
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| 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 | 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 | 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. | Posted | Number | 95% Confidence Interval | percentage of participants | 18 months post-dose |
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| Secondary | 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. | The Treated set included all participants who received at least one tablet of acoziborole. | Posted | Count of Participants | Participants | From the single dose acoziborole administration until 6 months post-dose for non-serious AEs and 18 months post-dose for serious AEs |
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| Secondary | Number of Participants With Serious TEAEs | Occurrence of any serious TEAEs during the observation period and until 18 months post-dose. | The Treated set included all participants who received at least one tablet of acoziborole. | Posted | Count of Participants | Participants | From the single dose acoziborole administration until 18 months post-dose |
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| 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 | 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. | 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. | Posted | Mean | Standard Deviation | h*ng/mL | Pre-dose and 4, 9, 24, 48, 72, 96, and 240 hours post-dose |
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| Secondary | 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. | The 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. | Posted | Mean | Standard Deviation | ng/mL | 240 hours post-dose |
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| Secondary | 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. | The 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. | Posted | Mean | Standard Deviation | ng/mL | 240 hours post-dose |
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Non-serious AEs were recorded from the time of administration of acoziborole on Day 1 until the 6 month follow-up visit. Serious AEs were collected from the time of providing written informed consent until the end of the follow-up (Month 18). TEAEs were 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.
The Investigator collected all AEs observed directly and those reported spontaneously. AEs and serious AEs were defined according to standard definitions. In specific, in this study, alanine aminotransferase or aspartic aminotransferase levels 3 times above the upper limit of normal (ULN) accompanied by total bilirubin levels 2 times above the ULN were considered serious AEs. Furthermore, any suspected transmission of an infectious agent via a medicinal product was also considered a serious AE.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Late-stage HAT | 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. | 4 | 167 | 18 | 167 | 127 | 167 |
| EG001 | Early- and Intermediate-stage HAT | 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. | 0 | 41 | 3 | 41 | 27 | 41 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Malaria | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Appendicitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Extrapulmonary tuberculosis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Puerperal infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Systemic infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Typhoid fever | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Acute psychosis | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Bipolar I disorder | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Brief psychotic disorder with marked stressors | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Major depression | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Mania | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Organic brain syndrome | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Suicide attempt | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Abdominal adhesions | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Inguinal hernia | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Guillain-Barre syndrome | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Intracranial pressure increased | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Acute pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Choking | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Postpartum haemorrhage | Pregnancy, puerperium and perinatal conditions | MedDRA 20.0 | Systematic Assessment |
| |
| Umbilical cord prolapse | Pregnancy, puerperium and perinatal conditions | MedDRA 20.0 | Systematic Assessment |
| |
| Ovarian cyst | Reproductive system and breast disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Poisoning | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Splenomegaly | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Arrhythmia | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Atrioventricular block first degree | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Chest pain | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Tinnitus | Ear and labyrinth disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Eye pruritus | Eye disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Ocular hyperaemia | Eye disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Anal haemorrhage | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Eructation | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Inguinal hernia | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Chills | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Feeling hot | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Gait disturbance | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Abscess | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Acarodermatitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Appendicitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Diarrhoea infectious | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Dysentery | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Enteritis infectious | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Filariasis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Helminthic infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Infection parasitic | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Infectious colitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Malaria | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Onychomycosis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Paronychia | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Pulmonary tuberculosis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Schistosomiasis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Skin infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Syphilis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Typhoid fever | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Varicella | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Wound infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Procedural dizziness | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Procedural headache | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Scratch | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Skin injury | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Blood calcium decreased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Blood glucose increased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Blood potassium increased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Blood sodium decreased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Electrocardiogram T wave inversion | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Weight increased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Malnutrition | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Obesity | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Overweight | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Type 2 diabetes mellitus | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Bone pain | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dyskinesia | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Formication | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Motor dysfunction | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Abnormal behaviour | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Bulimia nervosa | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Delirium | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Eating disorder | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Enuresis | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Psychotic disorder | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Chromaturia | Renal and urinary disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pollakiuria | Renal and urinary disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Amenorrhoea | Reproductive system and breast disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Menorrhagia | Reproductive system and breast disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Ovarian cyst | Reproductive system and breast disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Vaginal discharge | Reproductive system and breast disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Blister | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Decubitus ulcer | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Papule | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Skin disorder | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Skin ulcer | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Orthostatic hypotension | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
|
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.
The sponsor authorizes publications by a single site provided that multicentric results are published beforehand and any communication is submitted to the sponsor for review at least 28 days before the expected publication date. The sponsor can comment for 28 days. Upon sponsor request, any confidential information will have to be removed before publishing. Publishing can be postponed by 90 additional days, should the sponsor want to protect data through a patent application or any other mean.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Sandra Rembry, PharmD | Drugs for Neglected Diseases | +41229077734 | srembry@dndi.org |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 17, 2021 | Jun 3, 2025 | SAP_001.pdf |
| ID | Term |
|---|---|
| D014353 | Trypanosomiasis, African |
| ID | Term |
|---|---|
| D014352 | Trypanosomiasis |
| D056986 | Euglenozoa Infections |
| D011528 | Protozoan Infections |
| D010272 | Parasitic Diseases |
| D007239 | Infections |
| D000079426 | Vector Borne Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C557508 | SCYX 7158 |
Not provided
Not provided
Not provided
| Male |
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| Congo, The Democratic Republic of the |
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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. Acoziborole: Acoziborole 960 mg as a single oral dose (3 x 320 mg tablets; fasted state) |
|
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| OG002 | Any Stage HAT | Participants with any stage HAT, including all participants irrespective of HAT stage (participants with late-stage HAT and participants with early- and intermediate-stage HAT). Participants received 960 mg acoziborole as a single oral dose. |
|
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| OG002 | Any Stage HAT | Participants with any stage HAT, including all participants irrespective of HAT stage (participants with late-stage HAT and participants with early- and intermediate-stage HAT). Participants received 960 mg acoziborole as a single oral dose. |
|
|
| Early- and Intermediate-stage HAT |
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. Acoziborole: Acoziborole 960 mg as a single oral dose (3 x 320 mg tablets; fasted state) |
| OG002 | Any Stage HAT | Participants with any stage HAT, including all participants irrespective of HAT stage (participants with late-stage HAT and participants with early- and intermediate-stage HAT). Participants received 960 mg acoziborole as a single oral dose. |
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