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| Name | Class |
|---|---|
| European and Developing Countries Clinical Trials Partnership (EDCTP) | OTHER_GOV |
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The ultimate goal of this study is to show that fexinidazole offers an alternative over the existing treatments of Human African trypanosomiasis due to Trypanosoma brucei rhodesiense (r-HAT): melarsoprol in patients with stage 2 r-HAT and suramin in patients with stage 1 r-HAT. The main questions it aims to answer are:
Participants will receive fexinidazole oral treatment for 10 days. Regular blood draws and lumbar punctures will be performed over 12 months to confirm the cure of the disease. Other assessments will include the recording of adverse events, signs and symptoms of the disease, laboratory tests, vital signs, electrocardiograms.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Patients with stage 1 r-HAT | Experimental | Patients with stage 1 r-HAT were patients without trypanosomes in the cerebrospinal fluid (CSF) but with trypanosomes in the blood and/or lymph and CSF white blood cells (WBC) ≤5 cells/µL. Patients were to receive fexinidazole orally for 10 days. |
|
| Patients with stage 2 r-HAT | Experimental | Patients with stage 2 r-HAT were patients with trypanosomes in the CSF (and/or in blood/lymph) and/or CSF WBC >5 cells/µL. Patients were to receive fexinidazole orally for 10 days. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Fexinidazole | Drug | Tablets of 600 mg; Participants with a weight between 20 and 34 kg received 1200 mg (2 tablets) for 4 days, then 600 mg (1 tablet) for 6 days (with food); Participants with a weight of 35 kg and above received 1800 mg (3 tablets) for 4 days, then 1200 mg (2 tablets) for 6 days (with food) |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Evaluable Patients With Stage 2 r-HAT Who Died by the End of Hospitalization, Considering Only Deaths Possibly Related to r-HAT or Fexinidazole | The percentage of patients who died between the first intake of fexinidazole until the End-of-Hospitalization Visit (Day 12-18), considering only deaths possibly related to r-HAT or study treatment as assessed by the Data Safety Monitoring Board (DSMB), was calculated. The World Health Organization(WHO) verbal autopsy questionnaire was used since anatomopathological techniques were not available at the study sites (unless the death occurred at the hospital, in which case the investigator was present). In case of death that was not related to r-HAT nor to the study treatment during the hospitalization, the patient is considered as non-evaluable for efficacy purposes. Any patient who left the hospital without being medically discharged by the site Investigator before the planned End-of-Hospitalization Visit, and for whom no outcome could be retrieved, is also to be considered as non-evaluable. | 12 to 18 days after start of treatment |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Evaluable Patients With Stage 2 r-HAT, Whose Treatment Outcome is a Failure at the End of Hospitalization | Treatment outcome at end of hospitalization (Day 12-18) is categorized as success or failure. Failure is defined as any of the following: presence of trypanosomes in any body fluid at end of treatment (Day 11), death related to r-HAT or fexinidazole at end of hospitalization according to the DSMB, or absence of clinical improvement leading to the use of rescue medication. Unrelated deaths are neither success nor failure (the patient is considered non-evaluable for efficacy purposes). |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Enock Matovu, Prof | Makerere University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Rumphi District Hospital | Rumphi | PO Box 225 | Malawi | |||
| Lwala Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40288400 | Derived | Matovu E, Nyirenda W, Eriatu A, Alves D, Perdrieu C, Lemerani M, Wamboga C, Lejon V, Seixas J, Signorell A, Reymondier A, Baudin E, Scherrer B, Valverde Mordt O. Fexinidazole as a new oral treatment for human African trypanosomiasis due to Trypanosoma brucei rhodesiense: a prospective, open-label, single-arm, phase 2-3, non-randomised study. Lancet Glob Health. 2025 May;13(5):e910-e919. doi: 10.1016/S2214-109X(25)00016-6. |
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The pre-treatment period of up to 7 days included screening and treatment of concurrent malaria and soil-transmitted helminthiasis. At the end of this period, all participants were treated with fexinidazole, regardless of the stage of human African trypanosomiasis due to Trypanosoma brucei rhodesiense (r-HAT) and treatment setting.
The study was conducted at 1 center in Malawi (Rumphi District Hospital) and 1 center in Uganda (Lwala Hospital). 46 participants were screened and signed informed consent. The screening pool was enlarged to other hospitals and centres in Uganda, Rumphi/Mzimba North District (Malawi), and Nkhotakota District (Malawi), from where patients could be referred to Lwala and Rumphi Hospitals for treatment. 45 participants were enrolled between 29 Sep 2019 and 17 Nov 2021.
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| ID | Title | Description |
|---|---|---|
| FG000 | Patients With Stage 1 r-HAT | Patients without trypanosomes in the CSF but with trypanosomes in the blood and/or lymph and CSF WBC ≤5 cells/µL were classified as stage 1. Fexinidazole: Tablets of 600 mg; Participants with a weight between 20 and 34 kg received 1200 mg (2 tablets) for 4 days, then 600 mg (1 tablet) for 6 days (with food); Participants with a weight of 35 kg and above received 1800 mg (3 tablets) for 4 days, then 1200 mg (2 tablets) for 6 days (with food). |
| FG001 | Patients With Stage 2 r-HAT | Patients with trypanosomes in the CSF (and/or in blood/lymph) and/or CSF WBC >5 cells/µL were classified as stage 2. Fexinidazole: Tablets of 600 mg; Participants with a weight between 20 and 34 kg received 1200 mg (2 tablets) for 4 days, then 600 mg (1 tablet) for 6 days (with food); Participants with a weight of 35 kg and above received 1800 mg (3 tablets) for 4 days, then 1200 mg (2 tablets) for 6 days (with food). |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
All patients who took at least one dose of fexinidazole
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| ID | Title | Description |
|---|---|---|
| BG000 | Patients With Stage 1 r-HAT | Patients with stage 1 r-HAT were patients without trypanosomes in the cerebrospinal fluid (CSF) but with trypanosomes in the blood and/or lymph and CSF white blood cells (WBC) ≤5 cells/µL. |
| BG001 | Patients With Stage 2 r-HAT |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percentage of Evaluable Patients With Stage 2 r-HAT Who Died by the End of Hospitalization, Considering Only Deaths Possibly Related to r-HAT or Fexinidazole | The percentage of patients who died between the first intake of fexinidazole until the End-of-Hospitalization Visit (Day 12-18), considering only deaths possibly related to r-HAT or study treatment as assessed by the Data Safety Monitoring Board (DSMB), was calculated. The World Health Organization(WHO) verbal autopsy questionnaire was used since anatomopathological techniques were not available at the study sites (unless the death occurred at the hospital, in which case the investigator was present). In case of death that was not related to r-HAT nor to the study treatment during the hospitalization, the patient is considered as non-evaluable for efficacy purposes. Any patient who left the hospital without being medically discharged by the site Investigator before the planned End-of-Hospitalization Visit, and for whom no outcome could be retrieved, is also to be considered as non-evaluable. | The analysis is performed in evaluable patients with stage 2 r-HAT. A total of 35 patients with stage 2 r-HAT were enrolled and treated. One patient died during hospitalization, but the death was considered unrelated to r-HAT and/or the study treatment, as evaluated by the Investigator and the DSMB and accepted by the Sponsor. As per protocol, this patient was considered non-evaluable for efficacy purposes. There were 34 patients with stage 2 r-HAT who were evaluable. | Posted | Number | 90% Confidence Interval | percentage of participants |
Non-serious treatment-emergent adverse events (AEs) were recorded from the first intake of fexinidazole (Day 1) until the End-of-Hospitalization Visit (Day 12-18). Treatment-emergent AEs considered as serious or related to fexinidazole treatment were collected from the first intake of fexinidazole (Day 1) until the End-of-Study Visit (Month 12). The population of analysis includes all patients who received at least one dose of fexinidazole.
Adverse events were classified as serious according to the standard definition. In addition, in this study, alanine aminotransferase or aspartate aminotransferase levels higher than 3 times the upper limit of normal associated with total bilirubin levels higher than 2 times the upper limit of normal were considered as serious AEs (SAEs). Furthermore, any suspected transmission of an infectious agent via a medicinal product was also considered as an SAE.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Patients With Stage 1 r-HAT | Patients with stage 1 r-HAT were patients without trypanosomes in the cerebrospinal fluid (CSF) but with trypanosomes in the blood and/or lymph and CSF white blood cells (WBC) ≤5 cells/µL. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acute kidney injury | Renal and urinary disorders | MedDRA 22.0 or later | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Vomiting | Gastrointestinal disorders | MedDRA 22.0 or later | Systematic Assessment |
The main limitation is the benchmark study design. A direct comparison with melarsoprol was impossible due to its toxicity (5% of patients die due to treatment-related encephalopathy) and the rarity of r-HAT cases. The annual number of r-HAT cases in all endemic countries decreased from 110 patients in 2012 to 27 in 2017. Although the primary endpoint required a subjective assessment of the cause of death, this was performed by independent experts from the safety committee to ensure robustness.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Olaf Valverde Mordt | Drugs for Neglected Diseases initiative (DNDi) | +41229069239/+41795431713 | ovalverde@dndi.org |
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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 19, 2022 | Jul 18, 2024 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Aug 25, 2020 | Jul 18, 2024 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D014353 | Trypanosomiasis, African |
| D007239 | Infections |
| D058069 | Neglected Diseases |
| ID | Term |
|---|---|
| D014352 | Trypanosomiasis |
| D056986 | Euglenozoa Infections |
| D011528 | Protozoan Infections |
| D010272 | Parasitic Diseases |
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| ID | Term |
|---|---|
| C038307 | fexinidazole |
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|
| 12 to 18 days after start of treatment |
| Percentage of Evaluable Patients With Stage 2 r-HAT, Whose Treatment Outcome is a Failure at 12 Months | Treatment outcome at test of cure (Month 12) is categorized as success or failure. Failure is defined as any of the following: presence of trypanosomes in any body fluid, death related to r-HAT or fexinidazole according to the DSMB, absence of clinical improvement leading to the use of rescue medication, or WBC count in the CSF >20 cells/μL which was unlikely due to causes other than HAT. Unrelated deaths are neither success nor failure (the patient is considered non-evaluable for efficacy purposes). | 12 months after start of treatment |
| Percentage of Evaluable Patients With Stage 1 r-HAT, Whose Treatment Outcome is a Failure at the End of Hospitalization | Treatment outcome at end of hospitalization (Day 12-18) is categorized as success or failure. Failure is defined as any of the following: presence of trypanosomes in any body fluid at end of treatment (Day 11), death related to r-HAT or fexinidazole at end of hospitalization according to the DSMB, or absence of clinical improvement leading to the use of rescue medication. Unrelated deaths are neither success nor failure (the patient is considered non-evaluable for efficacy purposes). | 12 to 18 days after start of treatment |
| Percentage of Evaluable Patients With Stage 1 r-HAT, Whose Treatment Outcome is a Failure at 12 Months | Treatment outcome at test of cure (Month 12) is categorized as success or failure. Failure is defined as any of the following: presence of trypanosomes in any body fluid, death related to r-HAT or fexinidazole according to the DSMB, absence of clinical improvement leading to the use of rescue medication, or WBC count in the CSF >20 cells/μL which was unlikely due to causes other than HAT. Unrelated deaths are neither success nor failure (the patient is considered non-evaluable for efficacy purposes). | 12 months after start of treatment |
| Percentage of Evaluable Patients With Any r-HAT Stage Who Died by the End of Hospitalization, Considering Only Deaths Possibly Related to r-HAT or Fexinidazole | The percentage of patients who died between the first intake of fexinidazole until the End-of-Hospitalization Visit (Day 12-18), considering only deaths possibly related to r-HAT or study treatment as assessed by the DSMB, was calculated. The WHO verbal autopsy questionnaire was used since anatomopathological techniques were not available at the study sites (unless the death occurred at the hospital, in which case the investigator was present). In case of death that was not related to r-HAT nor to the study treatment during the hospitalization, the patient is considered as non-evaluable for efficacy purposes. Any patient who left the hospital without being medically discharged by the site Investigator before the planned End-of-Hospitalization Visit, and for whom no outcome could be retrieved, is also to be considered as non-evaluable. | 12 to 18 days after start of treatment |
| Percentage of Evaluable Patients With Any r-HAT Stage, Whose Treatment Outcome is a Failure at the End of Hospitalization | Treatment outcome at end of hospitalization (Day 12-18) is categorized as success or failure. Failure is defined as any of the following: presence of trypanosomes in any body fluid at end of treatment (Day 11), death related to r-HAT or fexinidazole at end of hospitalization according to the DSMB, or absence of clinical improvement leading to the use of rescue medication. Unrelated deaths are neither success nor failure (the patient is considered non-evaluable for efficacy purposes). | 12 to 18 days after start of treatment |
| Percentage of Evaluable Patients With Any r-HAT Stage, Whose Treatment Outcome is a Failure at 12 Months | Treatment outcome at test of cure (Month 12) is categorized as success or failure. Failure is defined as any of the following: presence of trypanosomes in any body fluid, death related to r-HAT or fexinidazole according to the DSMB, absence of clinical improvement leading to the use of rescue medication, or WBC count in the CSF >20 cells/μL which was unlikely due to causes other than HAT. Unrelated deaths are neither success nor failure (the patient is considered non-evaluable for efficacy purposes). | 12 months after start of treatment |
| Number of Participants With Any AE (Including Abnormal Laboratory or ECG Finding if Considered Clinically Significant) Until the End of Hospitalization | Treatment-emergent AEs during the observation period were recorded from the first intake of fexinidazole (Day 1) until the End-of-Hospitalization Visit (between Day 12 and Day 18). | 12 to 18 days (between Day 1 and Day 12-18) |
| Number of Participants With Any AE Considered as Serious Until the End of the Follow-up Period | Treatment-emergent AEs considered as serious were collected from the first intake of fexinidazole (Day 1) until the End-of-Study Visit (Month 12). An AE was considered as serious if resulted in death, was life-threatening, required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability or incapacity, was a congenital anomaly or birth defect, or was an important medical event. | 12 months (between Day 1 and Month 12) |
| Number of Participants With Any AE Considered as Possibly Related to Fexinidazole Until the End of the Follow-up Period | Treatment-emergent AEs considered as possibly related to fexinidazole were collected from the first intake of fexinidazole (Day 1) until the End-of-Study Visit (Month 12). | 12 months (between Day 1 and Month 12) |
| Lwala |
| Kadeberamaido |
| PO Box 650 |
| Uganda |
Patients with stage 2 r-HAT were patients with trypanosomes in the CSF (and/or in blood/lymph) and/or CSF WBC >5 cells/µL. |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
| r-HAT stage: Stage 1, Stage 2 | Patients with stage 1 r-HAT (early stage, or haemolymphatic stage) were patients without trypanosomes in the cerebrospinal fluid (CSF) but with trypanosomes in the blood and/or lymph and CSF white blood cells (WBC) ≤5 cells/µL. Patients with stage 2 r-HAT (late stage, or meningoencephalitic stage) were patients with trypanosomes in the CSF (and/or in blood/lymph) and/or CSF WBC >5 cells/µL. | Count of Participants | Participants |
|
| White blood cells (WBC) in cerebrospinal fluid (CSF) | Mean | Standard Deviation | cells/µL |
|
| 12 to 18 days after start of treatment |
|
|
|
|
| Secondary | Percentage of Evaluable Patients With Stage 2 r-HAT, Whose Treatment Outcome is a Failure at the End of Hospitalization | Treatment outcome at end of hospitalization (Day 12-18) is categorized as success or failure. Failure is defined as any of the following: presence of trypanosomes in any body fluid at end of treatment (Day 11), death related to r-HAT or fexinidazole at end of hospitalization according to the DSMB, or absence of clinical improvement leading to the use of rescue medication. Unrelated deaths are neither success nor failure (the patient is considered non-evaluable for efficacy purposes). | The analysis is performed in evaluable patients with stage 2 r-HAT. A total of 35 patients with stage 2 r-HAT were enrolled and treated. One patient died during hospitalization, but the death was considered unrelated to r-HAT and/or the study treatment, as evaluated by the Investigator and the DSMB and accepted by the Sponsor. As per protocol, this patient was considered non-evaluable for efficacy purposes. There were 34 patients with stage 2 r-HAT who were evaluable. | Posted | Number | 90% Confidence Interval | percentage of participants | 12 to 18 days after start of treatment |
|
|
|
|
| Secondary | Percentage of Evaluable Patients With Stage 2 r-HAT, Whose Treatment Outcome is a Failure at 12 Months | Treatment outcome at test of cure (Month 12) is categorized as success or failure. Failure is defined as any of the following: presence of trypanosomes in any body fluid, death related to r-HAT or fexinidazole according to the DSMB, absence of clinical improvement leading to the use of rescue medication, or WBC count in the CSF >20 cells/μL which was unlikely due to causes other than HAT. Unrelated deaths are neither success nor failure (the patient is considered non-evaluable for efficacy purposes). | The analysis is performed in evaluable patients with stage 2 r-HAT. A total of 35 patients with stage 2 r-HAT were enrolled and treated. One patient died during hospitalization, but the death was considered unrelated to r-HAT and/or the study treatment, as evaluated by the Investigator and the DSMB and accepted by the Sponsor. As per protocol, this patient was considered non-evaluable for efficacy purposes. There were 34 patients with stage 2 r-HAT who were evaluable. | Posted | Number | 90% Confidence Interval | percentage of participants | 12 months after start of treatment |
|
|
|
|
| Secondary | Percentage of Evaluable Patients With Stage 1 r-HAT, Whose Treatment Outcome is a Failure at the End of Hospitalization | Treatment outcome at end of hospitalization (Day 12-18) is categorized as success or failure. Failure is defined as any of the following: presence of trypanosomes in any body fluid at end of treatment (Day 11), death related to r-HAT or fexinidazole at end of hospitalization according to the DSMB, or absence of clinical improvement leading to the use of rescue medication. Unrelated deaths are neither success nor failure (the patient is considered non-evaluable for efficacy purposes). | The analysis is performed in evaluable patients with stage 1 r-HAT. None of patients with stage 1 r-HAT died from causes related to r-HAT or study treatment; therefore, all 10 patients were evaluable. Because the number of patients with stage 1 r-HAT is very small, no null hypotheses are testable and only descriptive data are provided. | Posted | Number | percentage of participants | 12 to 18 days after start of treatment |
|
|
|
| Secondary | Percentage of Evaluable Patients With Stage 1 r-HAT, Whose Treatment Outcome is a Failure at 12 Months | Treatment outcome at test of cure (Month 12) is categorized as success or failure. Failure is defined as any of the following: presence of trypanosomes in any body fluid, death related to r-HAT or fexinidazole according to the DSMB, absence of clinical improvement leading to the use of rescue medication, or WBC count in the CSF >20 cells/μL which was unlikely due to causes other than HAT. Unrelated deaths are neither success nor failure (the patient is considered non-evaluable for efficacy purposes). | The analysis is performed in evaluable patients with stage 1 r-HAT. None of patients with stage 1 r-HAT died from causes related to r-HAT or study treatment; therefore, all 10 patients were evaluable. Because the number of patients with stage 1 r-HAT is very small, no null hypotheses are testable and only descriptive data are provided. | Posted | Number | percentage of participants | 12 months after start of treatment |
|
|
|
| Secondary | Percentage of Evaluable Patients With Any r-HAT Stage Who Died by the End of Hospitalization, Considering Only Deaths Possibly Related to r-HAT or Fexinidazole | The percentage of patients who died between the first intake of fexinidazole until the End-of-Hospitalization Visit (Day 12-18), considering only deaths possibly related to r-HAT or study treatment as assessed by the DSMB, was calculated. The WHO verbal autopsy questionnaire was used since anatomopathological techniques were not available at the study sites (unless the death occurred at the hospital, in which case the investigator was present). In case of death that was not related to r-HAT nor to the study treatment during the hospitalization, the patient is considered as non-evaluable for efficacy purposes. Any patient who left the hospital without being medically discharged by the site Investigator before the planned End-of-Hospitalization Visit, and for whom no outcome could be retrieved, is also to be considered as non-evaluable. | The analysis is performed in evaluable patients with any r-HAT stage. A total of 45 patients were enrolled and treated. One patient died during hospitalization, but the death was considered unrelated to r-HAT and/or the study treatment, as evaluated by the Investigator and the DSMB and accepted by the Sponsor. As per protocol, this patient was considered non-evaluable for efficacy purposes. There were 44 patients who were evaluable. | Posted | Number | 90% Confidence Interval | percentage of participants | 12 to 18 days after start of treatment |
|
|
|
|
| Secondary | Percentage of Evaluable Patients With Any r-HAT Stage, Whose Treatment Outcome is a Failure at the End of Hospitalization | Treatment outcome at end of hospitalization (Day 12-18) is categorized as success or failure. Failure is defined as any of the following: presence of trypanosomes in any body fluid at end of treatment (Day 11), death related to r-HAT or fexinidazole at end of hospitalization according to the DSMB, or absence of clinical improvement leading to the use of rescue medication. Unrelated deaths are neither success nor failure (the patient is considered non-evaluable for efficacy purposes). | The analysis is performed in evaluable patients with any r-HAT stage. A total of 45 patients were enrolled and treated. One patient died during hospitalization, but the death was considered unrelated to r-HAT and/or the study treatment, as evaluated by the Investigator and the DSMB and accepted by the Sponsor. As per protocol, this patient was considered non-evaluable for efficacy purposes. There were 44 patients who were evaluable. | Posted | Number | 90% Confidence Interval | percentage of participants | 12 to 18 days after start of treatment |
|
|
|
|
| Secondary | Percentage of Evaluable Patients With Any r-HAT Stage, Whose Treatment Outcome is a Failure at 12 Months | Treatment outcome at test of cure (Month 12) is categorized as success or failure. Failure is defined as any of the following: presence of trypanosomes in any body fluid, death related to r-HAT or fexinidazole according to the DSMB, absence of clinical improvement leading to the use of rescue medication, or WBC count in the CSF >20 cells/μL which was unlikely due to causes other than HAT. Unrelated deaths are neither success nor failure (the patient is considered non-evaluable for efficacy purposes). | The analysis is performed in evaluable patients with any r-HAT stage. A total of 45 patients were enrolled and treated. One patient died during hospitalization, but the death was considered unrelated to r-HAT and/or the study treatment, as evaluated by the Investigator and the DSMB and accepted by the Sponsor. As per protocol, this patient was considered non-evaluable for efficacy purposes. There were 44 patients who were evaluable. | Posted | Number | 90% Confidence Interval | percentage of participants | 12 months after start of treatment |
|
|
|
|
| Secondary | Number of Participants With Any AE (Including Abnormal Laboratory or ECG Finding if Considered Clinically Significant) Until the End of Hospitalization | Treatment-emergent AEs during the observation period were recorded from the first intake of fexinidazole (Day 1) until the End-of-Hospitalization Visit (between Day 12 and Day 18). | The analysis is performed in all patients who took at least one dose of fexinidazole (regardless of r-HAT stage). | Posted | Count of Participants | Participants | 12 to 18 days (between Day 1 and Day 12-18) |
|
|
|
| Secondary | Number of Participants With Any AE Considered as Serious Until the End of the Follow-up Period | Treatment-emergent AEs considered as serious were collected from the first intake of fexinidazole (Day 1) until the End-of-Study Visit (Month 12). An AE was considered as serious if resulted in death, was life-threatening, required in-patient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability or incapacity, was a congenital anomaly or birth defect, or was an important medical event. | The analysis is performed in all patients who took at least one dose of fexinidazole (regardless of r-HAT stage). | Posted | Count of Participants | Participants | 12 months (between Day 1 and Month 12) |
|
|
|
| Secondary | Number of Participants With Any AE Considered as Possibly Related to Fexinidazole Until the End of the Follow-up Period | Treatment-emergent AEs considered as possibly related to fexinidazole were collected from the first intake of fexinidazole (Day 1) until the End-of-Study Visit (Month 12). | The analysis is performed in all patients who took at least one dose of fexinidazole (regardless of r-HAT stage). | Posted | Count of Participants | Participants | 12 months (between Day 1 and Month 12) |
|
|
|
| 0 |
| 10 |
| 0 |
| 10 |
| 6 |
| 10 |
| EG001 | Patients With Stage 2 r-HAT | Patients with stage 2 r-HAT were patients with trypanosomes in the CSF (and/or in blood/lymph) and/or CSF WBC >5 cells/µL. | 1 | 35 | 3 | 35 | 16 | 35 |
| Pneumonia | Infections and infestations | MedDRA 22.0 or later | Systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA 22.0 or later | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 22.0 or later | Systematic Assessment |
|
| Dysphagia | Gastrointestinal disorders | MedDRA 22.0 or later | Systematic Assessment |
|
| Gastritis | Gastrointestinal disorders | MedDRA 22.0 or later | Systematic Assessment |
|
| Electrocardiogram U-wave abnormality | Investigations | MedDRA 22.0 or later | Systematic Assessment |
|
| Electrocardiogram QT prolonged | Investigations | MedDRA 22.0 or later | Systematic Assessment |
|
| Blood pressure increased | Investigations | MedDRA 22.0 or later | Systematic Assessment |
|
| Haemoglobin decreased | Investigations | MedDRA 22.0 or later | Systematic Assessment |
|
| Electrocardiogram T wave abnormal | Investigations | MedDRA 22.0 or later | Systematic Assessment |
|
| Electrocardiogram T wave inversion | Investigations | MedDRA 22.0 or later | Systematic Assessment |
|
| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA 22.0 or later | Systematic Assessment |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA 22.0 or later | Systematic Assessment |
|
| Hypertension | Vascular disorders | MedDRA 22.0 or later | Systematic Assessment |
|
| Malaria | Infections and infestations | MedDRA 22.0 or later | Systematic Assessment |
|
| Bacteraemia | Infections and infestations | MedDRA 22.0 or later | Systematic Assessment |
|
| Anaemia | Blood and lymphatic system disorders | MedDRA 22.0 or later | Systematic Assessment |
|
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 22.0 or later | Systematic Assessment |
|
| Epilepsy | Nervous system disorders | MedDRA 22.0 or later | Systematic Assessment |
|
| Extrapyramidal disorder | Nervous system disorders | MedDRA 22.0 or later | Systematic Assessment |
|
| Chromaturia | Renal and urinary disorders | MedDRA 22.0 or later | Systematic Assessment |
|
| Hypothermia | General disorders | MedDRA 22.0 or later | Systematic Assessment |
|
| Inflammation | General disorders | MedDRA 22.0 or later | Systematic Assessment |
|
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 22.0 or later | Systematic Assessment |
|
| Sinus tachycardia | Cardiac disorders | MedDRA 22.0 or later | Systematic Assessment |
|
The sponsor authorizes publications by a single site provided that multicentric results are published beforehand and that any communication is submitted to sponsor for review at least 28 days ahead of the expected date of publication. Upon sponsor request, any confidential information will have to be removed from the communication before publishing.
| D000079426 |
| Vector Borne Diseases |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
The hypothesis H0 (pdeath = 8.5% or more) was tested with a one-sided exact test for proportions at the 0.05 significance level.