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

Condition(s) targeted: Trypanosomiasis, African

Intervention: Eflornithine plus Nifurtimox combination therapy (Drug)

Phase: Phase 2/Phase 3

Status: Terminated

Sponsored by: Epicentre

Official(s) and/or principal investigator(s):
Patrice Piola, MD MSc, Principal Investigator, Affiliation: Epicentre
Gerardo Priotto, MD MPH, Study Director, Affiliation: Epicentre

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

Study design: Open Label, Uncontrolled, Single Group Assignment

Primary outcome: 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)

Secondary outcome:

Occurrence and severity of serious clinically apparent adverse events

Occurrence and severity of biochemical (ALAT, creatinine, bilirubin) and haematological (abnormal total and differential leukocyte count, haemoglobin) adverse events

Minimum age: N/A. Maximum age: N/A. Gender(s): Both.

Criteria:

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

Yumbe District Hospital, Yumbe, Yumbe District, Uganda

Related publications:

Priotto G, Fogg C, Balasegaram M, Erphas O, Louga A, Checchi F, Ghabri S, Piola P. Three Drug Combinations for Late-Stage Trypanosoma brucei gambiense Sleeping Sickness: A Randomized Clinical Trial in Uganda. PLoS Clin Trials. 2006 Dec 8;1(8):e39.

Starting date: October 2002
Last updated: June 20, 2007