Pan African Clinical Trials Registry
South African Medical Research Council, South African Cochrane Centre
PO Box 19070, Tygerberg, 7505, South Africa
Telephone: +27 21 938 0835 or +27 21 938 0967
Email: pactradmin@mrc.ac.za Website: pactr.samrc.ac.za
Trial no.: PACTR201704002215604 Date of Registration: 19/04/2017
Trial Status: Retrospective registration - This trial was registered after enrolment of the first participant
TRIAL DESCRIPTION
Public title Fexinidazole in Human African Trypanosomiasis Due to T.b. Gambiense at Any Stage
Official scientific title An Open-label Study Assessing Effectiveness, Safety and Compliance With Fexinidazole
Brief summary describing the background and objectives of the trial Human African Trypanosomiasis (HAT) is a potentially fatal, neglected disease. Currently, the choice of drug used to treat HAT is based on the stage of the disease: pentamidine in patients with early-stage HAT and nifurtimox-eflornithine combined therapy (NECT) for patients with late-stage HAT. Patients with HAT must therefore undergo lumbar puncture for disease staging before treatment can begin. Fexinidazole is a 2-substituted 5-nitroimidazole, orally active in vitro and in vivo against the two parasites that cause HAT. A pivotal study comparing fexinidazole and NECT, including 394 adult patients with late-stage HAT is currently in the follow-up phase. In addition, a study in adult patients with early-stage HAT, as well as a study in children over 6 years of age with early- or late-stage HAT are currently in the follow-up phase. The aim of the present study is to provide additional information on the effectiveness and safety of fexinidazole and to assess its use under conditions as close as possible to those in real life, both in patients treated on an out-patient basis and in the hospital setting, depending on clinical status.
Type of trial CCT
Acronym (If the trial has an acronym then please provide)
Disease(s) or condition(s) being studied Human African Trypanosomiasis due to T.b. gambiense,Infections and Infestations
Sub-Disease(s) or condition(s) being studied Human African Trypanosomiasis due to T.b. gambiense
Purpose of the trial Treatment: Drugs
Anticipated trial start date 10/11/2016
Actual trial start date 10/11/2016
Anticipated date of last follow up 29/11/2019
Actual Last follow-up date
Anticipated target sample size (number of participants) 174
Actual target sample size (number of participants) 15
Recruitment status Recruiting
Publication URL
Secondary Ids Issuing authority/Trial register
STUDY DESIGN
Intervention assignment Allocation to intervention If randomised, describe how the allocation sequence was generated Describe how the allocation sequence/code was concealed from the person allocating the participants to the intervention arms Masking If masking / blinding was used
Non-randomised Open-label(Masking Not Used)
INTERVENTIONS
Intervention type Intervention name Dose Duration Intervention description Group size Nature of control
Experimental Group Fexinidazole For patients with a weight between 20 and 34 kg: 1800mg for 4d then 1200 mg fot 6d. For patients with a weight of 35kg and above: 1800 mg for 4d, than 1200 mg for 6d 10 days Fexinidazole, tablets of 600 mg 174
ELIGIBILITY CRITERIA
List inclusion criteria List exclusion criteria Age Category Minimum age Maximum age Gender
* Male or female patient, including breastfeeding or pregnant women in the second or third trimester. * ¿ 6 years of age. * ¿ 20 kg body weight. * Signed Informed Consent Form * Trypanosomes detected in any body fluid. * Physically able to ingest at least one solid meal per day. * Able to take oral medication. * Karnofsky Performance Status > 40%. * Able to comply with the schedule of follow-up visits and with the study constraints. * Easily reachable during the out-patient follow-up period. * Willing to undergo lumbar punctures. Eligibility Criteria for Out-patient Treatment: - Accepting to be treated on an out-patient basis. - Karnofsky Performance Status > 50%. - Good understanding of the method of administration of fexinidazole by the patient and/or caregiver (checked using a questionnaire at the time of IMP dispensing). - Residing close to the investigational centre, i.e. approximately one hour by road and/or boat, during the treatment period*. - Easily reachable during the treatment period. - No medical or psychiatric contraindications for treatment as out-patient. - No pregnancy or breastfeeding. - No neurological symptoms. *The patient (and caregiver) is permitted to reside in a place different from his/her main place of residence during the treatment period. * Active clinically relevant medical conditions other than HAT that, in the Investigator¿s opinion, could jeopardise patient safety or interfere with participation in the study, including but not limited to significant liver or cardiovascular diseases, HIV infection, CNS trauma or seizure disorders, coma or altered consciousness not related to HAT. * Severe renal or hepatic impairment defined as: o elevated creatinine at > 3 times the upper limit of normal (ULN) o elevated ALT, AST or bilirubin at > 3 ULN * Severely deteriorated general condition, such as cardiovascular shock, respiratory distress or terminal illness. * Any condition (except symptoms of HAT) that compromises ability to communicate with the Investigator as required for completion of the study. * Any contraindication to imidazole products (known hypersensitivity to imidazoles). * Treatment for HAT within 2 years prior to inclusion. * Prior enrolment in the study or prior intake of fexinidazole. * Foreseeable difficulty in complying with the schedule of follow-up visits (migrants, refugees, itinerant traders, etc.). Temporary Non-inclusion Criteria: * Recovery period after antimalarial treatment and/or treatment of helminthiasis (at least 3 days). * Uncontrolled diabetes or hypertension or any patients requiring clinical stabilisation; wait until appropriate treatment to control the disease has been initiated. * First trimester of pregnancy. * Traumatic lumbar puncture at Screening i.e. red blood cells visible in CSF; wait for 48 hours before repeating lumbar puncture. 6 Year(s) 100 Year(s) Both
ETHICS APPROVAL
Has the study received appropriate ethics committee approval Date the study will be submitted for approval Date of approval Name of the ethics committee
Yes 13/09/2016 Comité d'Ethique de l'université protestante au Congo
Ethics Committee Address
Street address City Postal code Country
Croisement des l'avenue de Libération et du Boulevard Triomphal, Kinshasa/ Kinshasa Democratic Republic of the Congo
OUTCOMES
Type of outcome Outcome Timepoint(s) at which outcome measured
Primary Outcome Success or failure for patients any stage HAT 12 and 18 months follow up
Secondary Outcome Occurrence of grade ¿ 3 adverse events (AEs) between the first intake of fexinidazole and the end of the observation period or the follow-up period (18 months) for non-serious AEs assessed as related to fexinidazole
Secondary Outcome Occurrence of any serious adverse event (SAE) between the first intake of fexinidazole and the end of the follow-up period (18 months)
Secondary Outcome PK measure: Presence of fexinidazole and/or its main metabolites in the blood for PK analyses Day 11
Secondary Outcome Outpatients compliance: Number of tablets left over and patient's responses to the questionnaire Day 11
Secondary Outcome Outpatients compliance: Patient's responses to the questionnaire Day 11
Secondary Outcome Feasibility of self-management of treatment intake in outpatients based on interview and left over tablets at D11 Day 11
Secondary Outcome questionnaire on acceptability of packaging for outpatients only questionnaire to be completed by patients and caregivers Day 0 and Day 11
Secondary Outcome Whole blood concentration of fexinidazole and its metabolites from dry blood spot of inpatients day8, day9, day10, 3hour after treatment administration, Day10, 7h15 after treatment administration, Day10, 24h after treatment administration, Day10, 48h after treatment administration
RECRUITMENT CENTRES
Name of recruitment centre Street address City Postal code Country
Hôpital de Bagata Bagata Democratic Republic of the Congo
Hôpital Général de Bandundu Bandundu Democratic Republic of the Congo
Hôpital de Masi-Manimba Masi-Manimba Democratic Republic of the Congo
Hôpital de Mushie Mushie Democratic Republic of the Congo
Hôpital de Dipumba Mbuji-Mayi Democratic Republic of the Congo
Hôpital Général de Référence Roi Baudouin Kinshasa Democratic Republic of the Congo
FUNDING SOURCES
Name of source Street address City Postal code Country
DNDi Chemin Louis Dunant Geneva 1202 Switzerland
SPONSORS
Sponsor level Name Street address City Postal code Country Nature of sponsor
Primary Sponsor DNDi Chemin Louis Dunant 15 Geneva 1202 Switzerland Charities/Societies/Foundation
COLLABORATORS
Name Street address City Postal code Country
PNLTHA Kinshasa Democratic Republic of the Congo
CONTACT PEOPLE
Role Name Email Phone Street address
Principal Investigator Victor Kande Betu Kumeso kandevictor@yahoo.fr +243 999 904 293 36 Avenue de la Justice
City Postal code Country Position/Affiliation
Gombe/Kinshasa Democratic Republic of the Congo Programme des MTN , Secrétariat Général de la Santé
Role Name Email Phone Street address
Public Enquiries Antoine Tarral atarral@dndi.org +41229069260 Chemin Louis Dunant 15
City Postal code Country Position/Affiliation
Geneva 1202 Switzerland Head of HAT Clinical Program/DNDi
Role Name Email Phone Street address
Scientific Enquiries Antoine Tarral atarral@dndi.org +41229069260 Chemin Louis Dunant 15
City Postal code Country Position/Affiliation
Geneva 1202 Switzerland Head of HAT Clinical Program/DNDi
REPORTING
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