Efficacy and Safety of Fexinidazole in Children at Least 6 Years Old and Weighing Over 20 kg With Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open Study, plug-in to the Pivotal Study

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02184689
• Date of registration: 19/06/2014
• Prospective Registration: No
• Primary sponsor: Drugs for Neglected Diseases
• Public title: Efficacy and Safety of Fexinidazole in Children at Least 6 Years Old and Weighing Over 20 kg With Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open Study, plug-in to the Pivotal Study
• Scientific title: Efficacy and Safety of Fexinidazole in Children at Least 6 Years Old and Weighing Over 20 kg With Human African Trypanosomiasis (HAT) Due to T.b. Gambiense: a Prospective, Multicentre, Open Study, plug-in to the Pivotal Study
• Date of first enrolment: May 3, 2014
• Target sample size: 125
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02184689
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2/Phase 3

Countries of recruitment

Congo, The Democratic Republic of the

Key inclusion & exclusion criteria

Inclusion Criteria:

- Informed consent form signed by one of the parents or the child's legal representative

- Child assent to participate in the study collected in the presence of an impartial
witness

- Age between 6 and 15 years old

- > 20 Kg bodyweight

- Boy or girl

- Able to ingest at least one complete meal per day (or at least one Plumpy'Nut® sachet)

- Able to swallow 600mg fexinidazole tablets

- Karnofsky index > 50

- Presence of trypanosomes in blood and/or lymph and/or CSF

- Permanent address and ability to comply with follow-up visit schedule

Exclusion Criteria:

- Child refusing to be included in the trial

- Bodyweight < 20 Kg

- Severe malnutrition, defined as having a BMI below 16 (- 2 Z-score of the norm
according to WHO 2007 Growth reference data)

- Inability to take oral medication

- Pregnancy or breastfeeding

- Clinically relevant medical condition other than HAT that, in the Investigator's
opinion, may jeopardize subject safety or interfere with participation in the study,
including but not limited to significant liver or cardiovascular disease, active
documented or suspected infection, central nervous system (CNS) trauma or seizure
disorders, coma or altered consciousness

- Severely deteriorated general condition, such as cardiovascular shock, respiratory
distress, or terminal illness

- Any medical condition (except HAT-specific symptoms) hindering communication with the
Investigator as required for the completion of this study

- Any contraindication to imidazole products (known hypersensitivity to imidazoles)

- History of HAT treatment in the past 2 years

- Patients previously enrolled in the study or having already received fexinidazole.

- Expected follow-up difficulties (migrants, refugees, itinerant vendors, etc.).

- Current alcohol or drug abuse

- Clinically significant abnormal laboratory findings, including ASAT and/or ALAT > 2
times ULN // Total bilirubin > 1.5 times ULN // Severe leukopenia (< 2000/mm3) //
Potassium (K+) < 3.5 mmol // Any other clinically significant abnormal laboratory
value (see details in Investigator Manual)

- Pregnancy confirmed by a positive urine pregnancy test obtained within 24 hours (h)
prior to start of study treatment (see Section 5.8.3 Contraception; p36) for girls
over 12 years old and over

- ECG abnormalities assessed by a central cardiologist

- QTcF= 450 ms, as measured automatically (if first measurement is abnormal, a second
assessment will be done at least 10-20 min later, with the patient in resting
position).

- Patients not tested for malaria and/or not treated adequately for this infection

- Patients not treated adequately for soil transmitted helminthic diseases



Age minimum: 6 Years
Age maximum: 14 Years
Gender: All

Health Condition(s) or Problem(s) studied

Human African Trypanosomiasis (HAT)

Intervention(s)

Drug: fexinidazole

Primary Outcome(s)

Outcome (success or failure) at the test of cure (ToC) [Time Frame: 12 months after the end of treatment]

Secondary ID(s)

DNDiHATFEX006

Source(s) of Monetary Support

Please refer to primary and secondary sponsors