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 0506 / +27 21 938 0834 Fax: +27 21 938 0836
Email: pactradmin@mrc.ac.za Website: pactr.samrc.ac.za
Trial no.: PACTR202407870029422 Date of Registration: 18/07/2024
Trial Status: Registered in accordance with WHO and ICMJE standards
TRIAL DESCRIPTION
Public title FACA Syrup clinical trial
Official scientific title Phase I and II open, controlled and randomized clinical trial of Faca® pediatric syrup for the management of children with sickle cell disease from 06 months to 5 years of age
Brief summary describing the background and objectives of the trial Sickle cell anemia is a genetic disease that affects approximately 2% of newborns in Burkina Faso, of whom 50 to 75% die before the age of 5. This pathology negatively impacts the quality of life of patients as well as the psychosocial and economic development of affected populations. To deal with sickle cell disease, the Health Sciences Research Institute (IRSS) has developed a phytomedicine in capsule form called FACA® (AMM N°0490720102T221000 for FACA 80 mg capsule, B/100 and AMM N°0490620108T221000 for FACA 160 mg capsule, B/100) for the treatment and prevention of seizures. However, this pharmaceutical form is not suitable for infants and young children; a pediatric syrup formulation of FACA® suitable for children aged 6 months to 5 years has just been developed. After conclusive preclinical pharmacological and toxicological studies on this formulation, it is essential to have data on safety and effectiveness in humans to complete the FACA marketing authorization application file (AMM). ® Pediatric syrup. The overall objective of the present study is to carry out a phase I and then phase II clinical trial in order to evaluate the safety and clinical and biological effectiveness of this new formulation in the management of sickle cell disease in children aged 06 months to 5 years. The overall objective of the present study is therefore to carry out a phase I clinical trial in order to evaluate the safety of this new formulation in the management of sickle cell disease in children aged 6 months to 5 years. Specifically, this will involve: i) Evaluating the safety of use of FACA® Pediatric Syrup and its tolerance threshold in healthy children aged 6 months to 5 years and ii) Determining the adverse effects of FACA® Pediatric Syrup in healthy children aged 6 months to 5 years
Type of trial Non-Randomised
Acronym (If the trial has an acronym then please provide) EC FACA Sirop
Disease(s) or condition(s) being studied Genetic Diseases,Paediatrics
Sub-Disease(s) or condition(s) being studied
Purpose of the trial Treatment: Drugs
Anticipated trial start date 31/07/2024
Actual trial start date 31/07/2024
Anticipated date of last follow up 30/10/2024
Actual Last follow-up date 30/10/2024
Anticipated target sample size (number of participants) 10
Actual target sample size (number of participants) 10
Recruitment status Not yet 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
Single Group Non-randomised Allocation was determined by the holder of the sequence who is situated off site Open-label(Masking Not Used)
INTERVENTIONS
Intervention type Intervention name Dose Duration Intervention description Group size Nature of control
Experimental Group FACA Pediatric Syrup FACA® Pediatric Syrup at a dose of 4.4 mg/kg (5 mL syrup) twice daily will be administered orally to each participant 7 days Participants enrolled in the Phase I trial will be monitored daily at home by a qualified health worker to assist with treatment administration and to monitor their clinical condition. If vomiting occurs 30 min after dosing, a full dose will be re-administered. If vomiting occurs 1 hour later, half a dose will be administered. If vomiting persists, the participant will be withdrawn from the study for appropriate medical management. These events will be recorded. All arrangements will be made to ensure that participants have rapid access to emergency medical services should the need arise. 10
ELIGIBILITY CRITERIA
List inclusion criteria List exclusion criteria Age Category Minimum age Maximum age Gender
Must not have sickle cell disease or sickle cell trait (hemoglobin AA on electrophoresis); No renal or hepatic impairment; Must be at least 06 months and no more than 57 months old; Parents or guardians in charge of the child who have signed the informed consent form; Be able to take FACA® Pediatric Syrup orally; Reside in the study area (Ouagadougou) Known allergies to study drugs or any of their components; Participation in any other ongoing clinical study Participant undergoing another treatment for sickle cell disease at the time of the trial; Refusal to sign informed consent by parents or guardians; Participant presenting signs of severity (chronic complications, stroke, acute chest syndrome, etc.). Participant with severe neurological and/or mental disorders; Patient suffering from any other serious pathologies Infant: 13 Month(s)-24 Month(s),Preschool Child: 2 Year-5 Year 6 Month(s) 59 Month(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 15/01/2024 Agence Nationale de Regulation Pharmaceutique
Ethics Committee Address
Street address City Postal code Country
Rue devHopital Ouagadougou 226 Burkina Faso
OUTCOMES
Type of outcome Outcome Timepoint(s) at which outcome measured
Primary Outcome Frequency, time of occurrence and types of adverse effects/events Significant disturbances in biological parameters [Urea, Blood Count (CBC), Transaminases (TRANSA), Creatinine (CREAT), ...] Pre-intervention: Day 0 ,Post intervention : Day7, Day 30, Day 60 and Day 90
Secondary Outcome Tolerable dose of FACA Pediatric Syrup Pre-intervention: Day 0, Post-intervention: Day 7, Day 30, Day 60 and Day 90
RECRUITMENT CENTRES
Name of recruitment centre Street address City Postal code Country
Centre Hospitalier Universitaire Pediatrique Charles De Gaulles 979 Boulevard des Tensoba, Zone I, Ouagadougou Ouagadougou 226 Burkina Faso
FUNDING SOURCES
Name of source Street address City Postal code Country
Fonds National de la Recherche et de Innovation pour le Developpement Avenue du Pere Joseph Wresinski Ouagadougou 226 Burkina Faso
SPONSORS
Sponsor level Name Street address City Postal code Country Nature of sponsor
Primary Sponsor Institut de Recherche en Sciences de la Sante Rue 29.13 Wemtenga Ouagadougou 226 Burkina Faso Research Institute
COLLABORATORS
Name Street address City Postal code Country
CONTACT PEOPLE
Role Name Email Phone Street address
Principal Investigator Diarra YE OUATTARA yediarra@hotmail.com +22670077060 979 Boulevard des Tensoba
City Postal code Country Position/Affiliation
Ouagadougou 226 Burkina Faso Head of the pediatrics department of Charles De Gaulles Teaching Hospital
Role Name Email Phone Street address
Public Enquiries Geoffroy Gueswinde OUEDRAOGO guioffray@gmail.com +22670091503 Rue 29.13 Wemtenga
City Postal code Country Position/Affiliation
Ouagadougou 226 Burkina Faso Researcher at Institut de Recherche en Sciences de la Sante
Role Name Email Phone Street address
Scientific Enquiries Lazare BELEMNABA lbelemnaba2@gmail.com +22670250346 Rue 29.13 Wemtenga
City Postal code Country Position/Affiliation
Ouagadougou 226 Burkina Faso Directeur of Institut de Recherche en Sciences de la Sante
REPORTING
Share IPD Description Additional Document Types Sharing Time Frame Key Access Criteria
Yes Data obtained through this study could be provided to all qualified researchers after encoding without personal health data included. Analytic Code,Clinical Study Report,Informed Consent Form,Statistical Analysis Plan,Study Protocol Beginning 3 months and ending é years after the end of the study All data collected will be accessible after requirement
URL Results Available Results Summary Result Posting Date First Journal Publication Date
No
Result Upload 1: Result Upload 2: Result Upload 3: Result Upload 4: Result Upload 5:
Result URL Hyperlinks Link To Protocol
Result URL Hyperlinks
Changes to trial information