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.: PACTR202104770362022 Date of Registration: 08/04/2021
Trial Status: Retrospective registration - This trial was registered after enrolment of the first participant
TRIAL DESCRIPTION
Public title Phase 2 interventional, multicenter, randomized open-label study to determine the effective and tolerable dose of KAF156 and Lumefantrine Solid Dispersion Formulation in combination, given once daily for 1, 2 and 3-days to adults and children with uncomplicated Plasmodium falciparum malaria
Official scientific title A Phase 2 interventional, multicenter, randomized open-label study to determine the effective and tolerable dose of KAF156 and Lumefantrine Solid Dispersion Formulation in combination, given once daily for 1, 2 and 3-days to adults and children with uncomplicated Plasmodium falciparum malaria
Brief summary describing the background and objectives of the trial Standard antimalarial drugs such as chloroquine (CQ), pyrimethamine (PYR), sulfadoxine (SFDX) and mefloquine (MEF) have become largely ineffective in many malaria endemic regions (exceptions are the artemisinin-based combination therapies (ACTs) such as Novartis’ Coartem®/Riamet® and Eurartesim®, current standard-of-care for P. falciparum malaria). Unfortunately, some recent reports suggest that decades of continuous use of artemisinin and bisquinoline derivatives as monotherapies may have fostered the emergence of drug resistance in Plasmodium species in Southeast Asia. There is therefore a strong medical need for new chemical entities with a new mode of action as additional treatment options for this very common disease with substantial morbidity and mortality. Simplifying regimens by developing treatments that can be used in a once daily dose for less than 3-day administration can improve treatment success and reduce probability of developing resistance via improved adherence and thus accelerate malaria eradication. The purpose of this study is to determine the effective and tolerable dose of KAF156, a new antimalarial molecule that can be administered in combination with a Solid Dispersion Formulation of lumefantrine (LUM-SDF) for the shortest treatment duration possible.
Type of trial RCT
Acronym (If the trial has an acronym then please provide) NA
Disease(s) or condition(s) being studied Infections and Infestations
Sub-Disease(s) or condition(s) being studied Malaria
Purpose of the trial Treatment: Drugs
Anticipated trial start date 01/12/2016
Actual trial start date 02/08/2017
Anticipated date of last follow up 31/07/2021
Actual Last follow-up date 14/06/2021
Anticipated target sample size (number of participants) 525
Actual target sample size (number of participants) 524
Recruitment status Completed
Publication URL https://clinicaltrials.gov/ct2/show/study/NCT03167242?term=CKAF156A2202&draw=2&rank=1
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
Parallel: different groups receive different interventions at same time during study Randomised Simple randomization using a randomization table created by a computer software program Central randomisation by phone/fax Open-label(Masking Not Used)
INTERVENTIONS
Intervention type Intervention name Dose Duration Intervention description Group size Nature of control
Experimental Group PK Run in Cohort KAF156 200 mg and LUM-SDF 960 mg - QD for 1 day QD for Day 1 LUM-SDF sachet(s) should be emptied in mouth directly and then patients should drink some water. Subsequently (preferably in 2 min, but in any case < 15 min), KAF156 will be administered orally. Tablet(s) to be swallowed with water 12
Experimental Group Part A Cohort 1 KAF156 400 mg and LUM-SDF 960 mg QD for Day 1 LUM-SDF sachet(s) should be emptied in mouth directly and then patients should drink some water. Subsequently (preferably in 2 min, but in any case < 15 min), KAF156 will be administered orally. Tablet(s) to be swallowed with water. 51
Experimental Group Part A Cohort 2 KAF156 800 mg and LUM-SDF 960 mg QD for Day 1 LUM-SDF sachet(s) should be emptied in mouth directly and then patients should drink some water. Subsequently (preferably in 2 min, but in any case < 15 min), KAF156 will be administered orally. Tablet(s) to be swallowed with water 51
Experimental Group Part A Cohort 3 KAF156 400 mg and LUM-SDF 960 mg QD for 2 days LUM-SDF sachet(s) should be emptied in mouth directly and then patients should drink some water. Subsequently (preferably in 2 min, but in any case < 15 min), KAF156 will be administered orally. Tablet(s) to be swallowed with water. 52
Experimental Group Part A Cohort 4 KAF156 200 mg and LUM-SDF 480 mg QD for 3 days LUM-SDF sachet(s) should be emptied in mouth directly and then patients should drink some water. Subsequently (preferably in 2 min, but in any case < 15 min), KAF156 will be administered orally. Tablet(s) to be swallowed with water 54
Experimental Group Part A Cohort 5 KAF156 400 mg and LUM-SDF 480 mg QD for 3 days LUM-SDF sachet(s) should be emptied in mouth directly and then patients should drink some water. Subsequently (preferably in 2 min, but in any case < 15 min), KAF156 will be administered orally. Tablet(s) to be swallowed with water. 51
Experimental Group Part A Cohort 6 KAF156 400 mg and LUM-SDF 960 mg QD for 3 days LUM-SDF sachet(s) should be emptied in mouth directly and then patients should drink some water. Subsequently (preferably in 2 min, but in any case < 15 min), KAF156 will be administered orally. Tablet(s) to be swallowed with water. 52
Control Group Part A Cohort 7 Coartem BID for 3 days NA 27 Placebo
Experimental Group Part B Cohort 1 KAF156 400 mg and LUM-SDF 960 mg QD for 1 day LUM-SDF sachet(s) should be emptied in mouth directly and then patients should drink some water. Subsequently (preferably in 2 min, but in any case < 15 min), KAF156 will be administered orally. Tablet(s) to be swallowed with water 50
Experimental Group Part B Cohort 2 KAF156 400 mg and LUM-SDF 960 mg QD for 2 days LUM-SDF sachet(s) should be emptied in mouth directly and then patients should drink some water. Subsequently (preferably in 2 min, but in any case < 15 min), KAF156 will be administered orally. Tablet(s) to be swallowed with water. 50
Control Group Part B Cohort 4 Coartem BID for 3 days NA 25 Placebo
Experimental Group Part B Cohort 3 KAF400mg, LUM 960mg QD for 3 days LUM-SDF sachet(s) should be emptied in mouth directly and then patients should drink some water. Subsequently (preferably in 2 min, but in any case < 15 min), KAF156 will be administered orally. Tablet(s) to be swallowed with water. 50
ELIGIBILITY CRITERIA
List inclusion criteria List exclusion criteria Age Category Minimum age Maximum age Gender
1. PK Run-in Part and Part A: male and female patients ≥ 12 years and with a body weight ≥ 35.0 kg Part B: after determining the effective/tolerated doses and regimens in adolescent and adult patients, male and female patients ≥ 2 and < 12 years and with a body weight ≥ 10.0 kg will be included 2. Microscopic confirmation of P. falciparum by Giemsa-stained thick and thin films (refer to the laboratory manual for details) 3. P. falciparum parasitaemia of more than 1000 and less than 150 000 parasites/µL at the time of pre-screening (i.e., Study Visit 1) 4. Axillary temperature ≥ 37.5 ºC or oral/tympanic/rectal temperature ≥ 38.0ºC; or similar history of fever during the previous 24 hours (history of fever must be documented) 5. Negative pregnancy test for women of child bearing potential (WOCBP) 6. Written informed consent must be obtained before any assessment is performed. If the patient is unable to read and write, then a witnessed consent according to local ethical standards is permitted. Patients < 18 years old, who are capable of providing assent, must provide assent with parental/legal guardian consent or as per local ethical guidelines 7. The patient or his/her parent/legal guardian (in case of pediatric patients) is able to understand and comply with protocol requirements, instructions and protocol-stated restrictions and is likely to complete the study as planned 1. Mixed Plasmodium infections 2. Signs and symptoms of severe malaria according to WHO 2015 criteria unless characterized by high parasitaemia only 3. Active infections including tuberculosis 4. Patients with concurrent febrile illnesses (e.g., typhoid fever) 5. History of, or current alcohol misuse/abuse defined as five or more drinks on the same occasion on each of 5 or more days in the past 30 days 6. Known relevant liver disease e.g. chronic hepatitis, cirrhosis, compensated or decompensated, history of hepatitis B or C, hepatitis B or A vaccination in last 3 months, known gallbladder or bile duct disease, acute or chronic pancreatitis 7. Any confirmed or suspected immunosuppressive or immunodeficient condition, including human immunodeficiency virus (HIV) infection 8. Severe malnutrition (body mass index (BMI) < 16.0 for patients ≥ 12 years, and less than 70% of median normalized WHO reference weight for children < 12 years) 9. Severe vomiting, defined as more than 3 times in the 24 hours prior to inclusion in the study or severe diarrhea defined as more than 3 watery stools per day 10. Pregnant or nursing (lactating) women 11. Sexually active patients not willing to practice effective contraception 12. Women of child bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for the duration of the study. Highly effective contraception methods include: • Total abstinence (when this is in line with the preferred and usual lifestyle of the patient). Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception • Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy) total hysterectomy or tubal ligation at least six weeks before taking investigational drug. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment • Male sterilization (at least 6 months prior to screening). For female patients on the study, the vasectomized male partner should be the sole partner for that patient • Use of oral, (estrogen and progesterone), injected or implanted hormonal methods of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS) or other forms of hormonal contraception that have comparable efficacy (failure rate < 1%), for example hormone vaginal ring or transdermal hormone contraception In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking investigational drug Women are considered post-menopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g., age appropriate, history of vasomotor symptoms) or have had surgical bilateral oophorectomy (with or without hysterectomy), total hysterectomy or tubal ligation at least six weeks ago. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment is she considered not of child bearing potential 13. Sexually active males must use a condom during intercourse while taking drug and for the duration of the study and should not father a child in this period. A condom is required to be used also by vasectomized men in order to prevent delivery of the drug via seminal fluid 14. Active duodenal ulcer, ulcerative colitis, Crohn’s disease, chronic (i.e., > 2 weeks) use of non-steroidal anti-inflammatory drugs (NSAIDs) 15. Clinically relevant abnormalities of electrolyte balance which require correction, e.g., hypokalemia, hypocalcemia or hypomagnesemia. 16. Anemia (Hemoglobin level < 8 g/dL) 17. Any surgical or medical condition which might significantly alter the absorption, distribution, metabolism, or excretion of drugs, or which may jeopardize the patient in case of participation in the study. The investigator should make this determination in consideration of the patient’s medical history and/or clinical or laboratory evidence of any of the following: • AST/ALT > 2 x the upper limit of normal range (ULN), regardless of the level of total bilirubin • AST/ALT > 1.5 and ≤ 2 x ULN and total bilirubin is > ULN • Total bilirubin > 2 x ULN, regardless of the level of AST/ALT 18. Resting QTcF > 450 ms (males), QTcF > 460 ms (females) at screening 19. Creatinine > 2 x ULN in the absence of dehydration. In the case of dehydration, the creatinine should be < 2 x ULN after oral/parenteral rehydration 20. History of malignancy of any organ system (other than localized basal cell carcinoma of the skin or in situ cervical cancer), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases 21. Known chronic underlying disease such as sickle cell disease, and severe cardiac, renal, or hepatic impairment 22. Known active or uncontrolled thyroid disease 23. Inability to tolerate oral medication (in tablet and/or liquid form) 24. Patients with prior antimalarial therapy or antibiotics with antimalarial activity within minimum of their five (5) plasma half-lives (or within 4 weeks of screening if half-life is unknown) 25. Use of other investigational drugs within 5 half-lives of enrollment, or within 30 days or until the expected pharmacodynamic effect has returned to baseline, whichever is longer 26. Patients taking medications prohibited by the protocol (see Section 5.6.8, Table 5-5) 27. Previous participation in any malaria vaccine study or received malaria vaccine in any other circumstance 28. History or family history of long QT syndrome or sudden cardiac death, or any other clinical condition known to prolong the QTc interval, such as history of symptomatic cardiac arrhythmias, clinically relevant bradycardia or severe heart disease 29. Use of agents known to prolong the QT interval unless it can be permanently discontinued for the duration of study 30. History of hypersensitivity to any of the study drugs or its excipients or to drugs of similar chemical classes 80 and over: 80+ Year,Adolescent: 13 Year(s)-17 Year(s),Adult: 18 Year(s)-44 Year(s),Aged: 65 Year(s)-79 Year(s),Child: 6 Year-12 Year,Middle Aged: 45 Year(s)-64 Year(s),Preschool Child: 2 Year-5 Year 2 Year(s) 80 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 15/03/2017 Comite d Ethique pour la Recherche en Sante
Ethics Committee Address
Street address City Postal code Country
Ouagadougou Ouagadougou 00226 Burkina Faso
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 22/06/2018 Comite National d Ethique des Sciences de la Vie et de la Sante
Ethics Committee Address
Street address City Postal code Country
Boulevard de l Universite Abidjan 00000 Cote Divoire
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 17/03/2017 Comite National d Ethique pour la Recherche CNER
Ethics Committee Address
Street address City Postal code Country
B.P 2217 Libreville 00000 Gabon
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 04/07/2017 The Gambia Government MRC Joint EC
Ethics Committee Address
Street address City Postal code Country
PO Box 273 Banjul 00000 Gambia
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 03/07/2017 KEMRI Scientific and Ethics Review Unit SERU
Ethics Committee Address
Street address City Postal code Country
P.O. Box 54840, Off Mbagathi Road Nairobi 00000 Kenya
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 07/07/2018 Walter Reed Army Institute of Research Institutional Review Board
Ethics Committee Address
Street address City Postal code Country
503 Robert Grant Avenue Maryland 20910 United States of America
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 30/12/2016 FMPOS
Ethics Committee Address
Street address City Postal code Country
Bamako Bamako 00000 Mali
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 14/06/2017 CNBS
Ethics Committee Address
Street address City Postal code Country
C.Postal 264 Maputo 00000 Mozambique
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 08/03/2017 Mulago Hospital Research and Ethics Committee MHREC
Ethics Committee Address
Street address City Postal code Country
National Referral Hospital Mulago Hill Road PO Box 7051 Kampala 00000 Uganda
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 09/05/2017 Uganda Virus Research Institute LEC
Ethics Committee Address
Street address City Postal code Country
Plot 51 to 59 Nakiwogo Road PO Box 49 Entebbe 00000 Uganda
OUTCOMES
Type of outcome Outcome Timepoint(s) at which outcome measured
Primary Outcome To determine the effective doses of KAF156 combined with LUM-SDF given daily over 1, 2 or 3 days for treatment of uncomplicated malaria caused by P.falciparum PCR-corrected adequate clinical and parasitological response ACPR at Day 29 ie 28 days post-dose in Parts A and B
Secondary Outcome To evaluate the safety and tolerability of KAF156/LUM-SDF Standard safety/tolerability assessments: AE incidence and severity, liver and kidney function tests and electrocardiogram ECG abnormalities in the 43 day study period
Secondary Outcome To further assess the effect of treatment with KAF156, LUM-SDF by assessing uncorrected ACPR and corrected ACPR at additional time points, as well as fever and parasite clearance times PCR-Uncorrected ACPR at Days 15, 29 and 43 i.e., 14, 28 and 42 days postdose. PCR-corrected ACPR at Days 15 and 43 i.e., 14 and 42 days post-dose. Incidence rate of recrudescence and reinfection
Secondary Outcome To assess the key PK parameters of KAF156 and lumefantrine PK assessments between day 1 and 15 depending on assigned cohort
RECRUITMENT CENTRES
Name of recruitment centre Street address City Postal code Country
Institut de Recherche en Science de la Sante Centre Medical St Camille de Nanoro Nanoro Burkina Faso
Centre National de Recherche et de Formation sur le Paludisme Niangoloko Unite de Recherche Clinique de BP 37, S/C Centre Medical de Niangoloko Niangoloko Burkina Faso
Centre National de Recherche et de Formation sur le Paludisme Banfora Unite de Recherche Clinique de BP 09, CHR de Banfora Banfora Burkina Faso
Pasteur Institute of Ivory Coast D Anonkoua Koute 13 BP 972 Abidjan 13 Abidjan Cote Divoire
Pasteur Institute of Ivory Coast Ayame site D Anonkoua Koute 13 BP 972 Abidjan 13 Abidjan Cote Divoire
Centre de Recherches Medicales de Lambarene CERMEL Albert Schweizer Hospital Lambarene Cote Divoire
MRC Gambia Unit Atlantic Boulevard Fajara, P.O. Box 273 Banjul Gambia
KEMRI Siaya Clinical Research Annexe PO Box 1578 Siaya Kenya
KEMRI Kombewa Kombewa Clinical Research Centre, Walter Reed Project, Off Kisumu Bondo Road, Opposite Kombewa Sub-county Hospital Kombewa Kenya
Malaria Research and Training Center Bougoula Hameau s.c MRTC.DEAP.FMOS-FAPH. Point G Bamako Sikasso Mali
Chokwe Health Research and Training Centre Centro de Investigacao e Treino em Saude de Chokwe, 1 Bairro, Zona do Orli, CP 30 Chokwe Mozambique
MRC.UVRI and LSHTM Uganda Research Unit UVRI Uganda Research Unit on AIDS Masaka Uganda
Infectious Diseases Research Collaboration IDRC Tororo Tororo Hospital, Station Road Tororo Town Tororo Uganda
FUNDING SOURCES
Name of source Street address City Postal code Country
Novartis Pharma AG Lichtstrasse 35 CH4056 Basel Switzerland
SPONSORS
Sponsor level Name Street address City Postal code Country Nature of sponsor
Primary Sponsor Novartis Pharma AG Lichtstrasse 35 CH4056 Basel Switzerland Commercial Sector/Industry
COLLABORATORS
Name Street address City Postal code Country
Medicines for Malaria Venture MMV 20, Route de Pre-Bois 1215 Geneva 15 Geneva Switzerland
CONTACT PEOPLE
Role Name Email Phone Street address
Principal Investigator Moussa Lingani NA@NA.NA +22670675780 Centre Medical St Camille de Nanoro
City Postal code Country Position/Affiliation
Nanoro Burkina Faso Site Principal Investigator
Role Name Email Phone Street address
Principal Investigator Alphonse Ouedraog NA@NA.NA +22650324695 Unite de Recherche Clinique de BP 37
City Postal code Country Position/Affiliation
Niangoloko Burkina Faso Site Principal Investigator
Role Name Email Phone Street address
Principal Investigator Alfred Tiono NA@NA.NA +000000000 Unite de Recherche Clinique de BP 09
City Postal code Country Position/Affiliation
Banfora Burkina Faso Site Principal Investigator
Role Name Email Phone Street address
Principal Investigator Offianan Andre Toure na@na.na +22502580337 D Anonkoua Koute, 13 BP 972
City Postal code Country Position/Affiliation
Abidjan Cote Divoire Site Principal Investigator
Role Name Email Phone Street address
Principal Investigator Martin Grobusch na@na.na +31205664380 Albert Schweizer Hospital
City Postal code Country Position/Affiliation
Lambarene Gabon Site Principal Investigator
Role Name Email Phone Street address
Principal Investigator Umberto D Alessandro na@na.na +2204495917 P.O. Box 273
City Postal code Country Position/Affiliation
Banjul Gambia Site Principal Investigator
Role Name Email Phone Street address
Principal Investigator Grace Kaguthi na@na.na +254729489743 PO Box 1578
City Postal code Country Position/Affiliation
Siaya Kenya Site Principal Investigator
Role Name Email Phone Street address
Principal Investigator Bernhards Ogutu na@na.na +254733966065 Walter Reed Project, Off Kisumu Bondo Road
City Postal code Country Position/Affiliation
Kombewa Kenya Site Principal Investigator
Role Name Email Phone Street address
Principal Investigator Bakary Fofana na@na.na +22366789450 s.c MRTC.DEAP.FMOS FAPH. Point G Bamako
City Postal code Country Position/Affiliation
Sikasso Mali Site Principal Investigator
Role Name Email Phone Street address
Principal Investigator Ricardo Thompson na@na.na +25821431103 1 Bairro, Zona do Orli, CP 30
City Postal code Country Position/Affiliation
Chokwe Mozambique Site Principal Investigator
Role Name Email Phone Street address
Principal Investigator Sylvia Kusemererwa na@na.na +256481421211 UVRI Uganda Research Unit on AIDS
City Postal code Country Position/Affiliation
Masaka Uganda Site Principal Investigator
Role Name Email Phone Street address
Principal Investigator Adoke Yeka na@na.na +256772473533 Station Road Tororo Town
City Postal code Country Position/Affiliation
Tororo Uganda Site Principal Investigator
Role Name Email Phone Street address
Public Enquiries Emma Jane Moseley EmmaJane.Moseley@quintiles.com +27795106755 1st Floor, Montrose place 2 Bella Rosa Street Rosenpark
City Postal code Country Position/Affiliation
Bellville South Africa Clinical Lead
Role Name Email Phone Street address
Scientific Enquiries Emma Jane Moseley Emma.Moseley@quintiles.com +27795106755 1st Floor, Montrose place 2 Bella Rosa Street Rosenpark
City Postal code Country Position/Affiliation
Bellville South Africa Clinical Lead
REPORTING
Share IPD Description Additional Document Types Sharing Time Frame Key Access Criteria
Yes CSR report available Informed Consent Form,Study Protocol CSR report available CSR report available
URL Results Available Results Summary Result Posting Date First Journal Publication Date
https://clinicaltrials.gov/ct2/show/study/NCT03167242?term=CKAF156A2202&draw=2&rank=1 Yes 10/03/2022
Result Upload 1: Result Upload 2: Result Upload 3: Result Upload 4: Result Upload 5:
Result - 10/03/2022
Result URL Hyperlinks Link To Protocol
Result URL Hyperlinks
Changes to trial information