Eflornithine + Nifurtimox Late-Stage Human African Trypanosomiasis (HAT)in West Nile, Uganda

Study Overview
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Brief Summary
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This case series study follows on a terminated randomised clinical trial in a nearby location of Uganda, in which the combination of eflornithine + nifurtimox showed very promising efficacy and safety. The study's purpose is to evaluate the efficacy and safety of this combination in a larger group of late-stage Human African trypanosomiasis (sleeping sickness) patients.

Official Title
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Efficacy and Safety of an Eflornithine + Nifurtimox Combination for Treatment of Late-Stage Human African Trypanosomiasis (HAT) in West Nile, Uganda

Conditions
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Trypanosomiasis, African

Intervention / Treatment
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* Drug: Eflornithine plus Nifurtimox combination therapy


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Stage 2 infection with Trypanosoma brucei gambiense diagnosed within the previous 14 days, as defined by either of the following: (i) Presence of trypanosomes in blood or lymph node fluid and WBC count in CSF > 5 / mm3, or (ii) Presence of trypanosomes in the CSF with any CSF WBC count Residence in the study area Written informed consent (to be obtained from parent/guardian for children under 18 years and patients with impaired cognition) Exclusion Criteria: Pregnancy or clinical history suggestive thereof Weight < 10 Kg History of any HAT treatment within the previous 24 months Inability to undergo hospitalisation or attend follow-up visits during the 24 months following discharge Severe anemia (Hb< 5g/dl) Active tuberculosis (sputum positive) HIV positive (if patient has been tested and results are known) Severe renal or hepatic failure Bacterial or cryptococcal meningitis Other severe underlying diseases upon admission Refugee status
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: Eflornithine plus Nifurtimox combination therapy|nan|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Treatment failure (death within 30 days of inclusion, or later if judged as related to Human African trypanosomiasis; termination of treatment due to adverse events; evidence of infection relapse at or after discharge, up to 24 months post discharge) |  | 24 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Occurrence and severity of serious clinically apparent adverse events |  | treatment period and up to one month post discharge | 
| Occurrence and severity of biochemical (ALAT, creatinine, bilirubin) and haematological (abnormal total and differential leukocyte count, haemoglobin) adverse events |  | treatment period | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Human African trypanosomiasis, sleeping sickness, Trypanosoma brucei gambiense, combination, eflornithine, nifurtimox, Human African trypanosomiasis (sleeping sickness) due to Trypanosoma brucei gambiense in the late stage (stage 2 or meningo-encephalitic)