Clinical Study to Assess the Tolerability, Feasibility and Effectiveness of Nifurtimox and Eflornithine (NECT) for the Treatment of Trypanosoma Brucei Gambiense Human African Trypanosomiasis (HAT) in the Meningo-encephalitic Phase

Study Overview
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Brief Summary
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Multicenter, open label, uncontrolled phase IIIb study of therapeutic use of the combination of nifurtimox and eflornithine (NECT) for the treatment of Trypanosoma brucei gambiense human African trypanosomiasis (HAT) in the meningo-encephalitic phase. Overall objectives: Assess the clinical tolerability, feasibility and effectiveness of NECT co-administration to treat patients with T.b. gambiense human African trypanosomiasis (HAT) in the meningo-encephalitic phase in actual real-life conditions (regular treatment centers of the National HAT Control Programme, NGO treatment centers). Primary objective: Assess the clinical response of the NECT co-administration under field conditions. Secondary objectives: Assess the incidence and type of adverse events (AE), and the capacity of the treatment centers to deal with these. Assess the feasibility of the implementation of the NECT coadministration by the health center. Assess the effectiveness of the NECT co-administration at 24* months after treatment.

Official Title
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Clinical Study to Assess the Clinical Tolerability, Feasibility and Effectiveness Under Field Conditions of the Combination of Nifurtimox and Eflornithine (NECT) for the Treatment of T.b.Gambiense Human African Trypanosomiasis (HAT) in the Meningo-encephalitic Stage

Conditions
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Human African Trypanosomiasis

Intervention / Treatment
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* Drug: Nifurtimox-Eflronithine Combination Treatment (NECT)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: All patients diagnosed as stage 2 HAT according to the diagnostic protocols in use at the treatment center (generally, if presence of parasites in blood, lymph node fluid or CSF and an elevated white blood cell count in the CSF, but this can vary from center to center) will be included if a written Informed Consent is given by the patient or a legally acceptable representative if the patient is a minor or unable to communicate. Pregnancy and breastfeeding women: On a case by case basis according to the guidelines of the National HAT Control Programme or the NGO, the Investigator will decide to treat the patient or to defer the treatment. In case of inclusion, the mother-child pairs or the children of lactating mothers will be closely monitored during treatment and follow up. Children under 2 years of age: On a case by case basis, the Investigator will decide to treat an infant with NECT or an alternative treatment (preferably eflornithine). In case of inclusion, these infants will be closely monitored during treatment and follow up like all children less than 12 years of age. Exclusion Criteria: Unable to take oral medication, and impossibility to use a nasogastric tube. Treatment failure after nifurtimox-eflornithine treatment. Any other condition or reason for which the Investigator (or the responsible treating staff member) judges that another or no HAT treatment is warranted.
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

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


Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: Nifurtimox-Eflronithine Combination Treatment (NECT)|co-administration of nifurtimox (10 days, 15 mg/kg/day, p.o. TID) and eflornithine (7 days, 400 mg/kg/day, i.v. BID)|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of patients discharged alive from the hospital or the treatment center |  |  | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of serious AE (SAE) and severe AE (CTC score 3 and 4) related to treatment, and overall frequency and nature of AE and need for additional medications to manage these. |  | up to 24 months after treatment | 
| Effectiveness: The clinical cure rate (Survival without clinical and/or parasitological signs of HAT) |  | 24 months after treatment | 
| Number of temporary treatment interruptions, number of premature treatment cessations, length of hospitalization stay (including observation period), treatment compliance (deviation in dosing), and other feasibility indicators. |  | during the treatment/hospitalisation time | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Human African Trypanosomiasis, HAT, sleeping sickness, combination treatment, nifurtimox, eflornithine, Human African Trypanosomiasis in the meningo-encephalitic phase (stage 2 HAT)