Clinical trial NCT03087955: Prospective Study on Efficacy and Safety of Acoziborole (SCYX-7158) in Patients Infected by Human African Trypanosomiasis Due to T.b. Gambiense
Official title: Efficacy and Safety Study of Acoziborole (SCYX-7158) in Patients With Human African Trypanosomiasis (HAT) Due to Trypanosoma Brucei Gambiense: a Multicentre, Open-label, Prospective Study
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Trypanosomiasis, African
Gambiense Trypanosomiasis
Sleeping Sickness
Lead sponsor: Drugs for Neglected Diseases
Sponsor class: OTHER
First posted: 2017-03-23
Enrollment: 260
Sex: ALL
Minimum age (years): 15
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The goal of this study is to assess efficacy and safety of Acoziborole (SCYX-7158) given as a single dose oral treatment for adult patients (above or equal 15) in the fasting state with T.b. Gambiense HAT


Eligibility criteria: Inclusion Criteria:

* Male or female patient
* 15 years of age or older
* Signed informed consent form (as well as assent from illiterate and under-age patients, and those unable to give consent)
* Karnofsky Performance Status above 50
* Able to ingest oral tablets
* Having a permanent address or being traceable by other persons
* Able to comply with the schedule of follow-up visits and requirements of the study
* Agreement to be hospitalised in order to receive treatment
* For patients with late-stage HAT:

  * Confirmation of g-HAT by detection of the parasite in the blood and/or the lymph and/or the CSF, at the investigational centre
  * If trypanosomes are found in the blood or lymph, but not in the CSF, the CSF WBC, measured at the investigational centre, must be above 20/μL for the patient to be included in the cohort of patients with late-stage HAT
* For patients with early- or intermediate-stage HAT:

  * Confirmation of g-HAT by detection of the parasite in the blood and/or the lymph, at the investigational centre
  * Absence of parasites in the CSF
  * The CSF WBC, measured at the investigational centre, must be between 6 and 20/μL for the patient to be included in the cohort of patients with intermediate-stage HAT and equal to or below 5/μL for the patient to be included in the cohort of patients with early-stage HAT.

Exclusion Criteria:

* Severe malnourishment, defined as body-mass index (BMI) below 16
* Pregnancy or breastfeeding (for women of child-bearing potential, confirmed pregnancy on a urine pregnancy test performed within 24 hours prior to administration of SCYX-7158)
* Clinically significant medical condition that could, in the opinion of the Investigator, jeopardise the patient's safety or interfere with participation in the study, including, but not limited to significant liver or cardiovascular disease, suspected or proven active infection, central nervous system trauma or seizure disorder, coma or consciousness disturbances
* Severely deteriorated health status, e.g. due to cardiovascular shock, respiratory distress syndrome or end-stage disease
* Previously treated for HAT (except prior treatment with pentamidine)
* Prior enrolment in the study
* Foreseeable difficulty complying with follow-up, including migrant worker, refugee status, itinerant trader etc.
* Current alcohol abuse or drug addiction
* Not tested for malaria and/or not having received appropriate treatment for malaria
* Not having received appropriate treatment for soil-transmitted helminthiasis
* Clinically significant abnormal laboratory values including Aspartate AminoTransferase(AST) and/or AlanineAminoTransferase (ALT) more than 2 times the upper limit of normal (ULN), total bilirubin more than 1.5 ULN, severe leukopenia at less than 2000/mm3, Potassium below 3.5 mmol/L, any other clinically significant abnormal laboratory value

Primary outcomes:
measure: Success or failure for patients in late stage HAT
timeFrame: 18 months follow up
Secondary outcomes:
measure: Success or failure for all stage HAT patients
timeFrame: 6, 12 and 18 months follow up
measure: Time to Failure in Patients with Late-stage HAT
timeFrame: 18 months follow up
measure: Occurence of adverse events
timeFrame: From day 1 until 6 months follow-up
measure: Occurence of serious advers events
timeFrame: Between the day 1 and the end of the follow-up period (18 month)
measure: Pharmacokinetics measure
timeFrame: Days 1, 2, 3, 4, 5, 11, Month 3 and Month 6 follow up visits
measure: Pharmacokinetics measure
timeFrame: Days 1, 2, 3, 4, 5, 11, Month 3 and Month 6 follow up visits
measure: Electrocardiogram measure
timeFrame: Days 1, 2, 3, 4, 5 and 11
measure: Electrocardiogram measure
timeFrame: Days 1, 2, 3, 4, 5 and 11
measure: Electrocardiogram measure
timeFrame: Days 1, 2, 3, 4, 5 and 11
measure: Electrocardiogram measure
timeFrame: Days 1, 2, 3, 4, 5 and 11
measure: Electrocardiogram measure
timeFrame: Days 1, 2, 3, 4, 5 and 11
Other outcomes:
Locations:
city: Nkara
country: Congo, The Democratic Republic of the
facility: Centre de Traitement de NKara
state: Bandundu
city: Kimpese
country: Congo, The Democratic Republic of the
facility: Centre de Traitement de Kimpese
state: Bas Congo
city: Gandajika
country: Congo, The Democratic Republic of the
facility: Hôpital Général de Référence de NGandajika
state: Kasai Oriental
city: Mbuji-Mayi
country: Congo, The Democratic Republic of the
facility: Hôpital de Dipumba
state: Kasai-Oriental
city: Katanda
country: Congo, The Democratic Republic of the
facility: Hôpital secondaire de Katanda
state: Kasaï-Oriental
city: Bagata
country: Congo, The Democratic Republic of the
facility: Hopital General de réference de Bagata
state: Kwilu
city: Masi-Manimba
country: Congo, The Democratic Republic of the
facility: Hôpital Général de référence de Masi-Manimba
state: Kwilu
city: Kwamouth
country: Congo, The Democratic Republic of the
facility: Hôpital Généal de référence de Kwamouth
state: Mai-Ndombe
city: Bandundu
country: Congo, The Democratic Republic of the
facility: Hopital Général de réference de Bandundu
city: Isangi
country: Congo, The Democratic Republic of the
facility: Hôpital de référence d'Isangi
city: Kinshasa
country: Congo, The Democratic Republic of the
facility: Hôpital Général de Référence Roi Baudouin
city: Dubréka
country: Guinea
facility: Centre de Traitement de la THA de Dubreka
state: Dubreka
Study officials:
affiliation: Ministère de la Santé
name: Victor Kande Betu Kumeso, Dr
role: PRINCIPAL_INVESTIGATOR
References: