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.: PACTR201501000997429 Date of Approval: 16/01/2015
Trial Status: Registered in accordance with WHO and ICMJE standards
TRIAL DESCRIPTION
Public title Rapid Assessment of Potential Interventions & Drugs for Ebola (RAPIDE) ¿ TKM
Official scientific title Open-label, single arm trial to investigate the efficacy of TKM-130803 with a concurrent observational study of Ebolavirus Disease in an outbreak setting in West Africa.
Brief summary describing the background and objectives of the trial The size and scale of the on-going Ebola Virus Disease (EVD) outbreak is unprecedented, shows no signs of abating, and has been declared a Public Health Emergency of International Concern. Ebola virus is among the most virulent infectious agents known and is associated with intense virus replication, severe disease and high mortality. Whilst several therapeutic interventions have shown promise in pre-clinical studies, none have been tested for efficacy and safety in humans with EVD. The aim of this protocol is to assess the effectiveness of the drug TKM-130803 (produced as TKM-130803 by Tekmira Pharmaceuticals) for the treatment of EVD. The Trial Steering Committee selected TKM as one of the leading drugs suitable for urgent evaluation from a possible list of 10 candidate therapies. The criteria for selection were based upon a Target Product Profile that considered existing clinical and pre-clinical data supporting safety and efficacy, immediate availability and scalability, and an acceptable treatment regimen. The purpose of this trial is to establish whether TKM is (a) very effective, (b) promising or (c) ineffective. If conclusion (a) is reached, we recommend that the high effectiveness of TKM is confirmed in a subsequent phase III trial. If conclusion (b) is reached, we recommend that the promise of TKM is studied further in a randomized comparison with best supportive care. If conclusion (c) is reached, we recommend that other treatments are given priority in clinical research, but that TKM might be reconsidered if better treatments are not found. We are developing trial designs for suitable single-arm confirmatory and randomized comparative trials that will ensure low risks that the sequence of drug development will give invalid conclusions. It is the intention to use this clinical trial as part of a platform for investigation of a number of experimental interventions for the treatment of EVD.
Type of trial RCT
Acronym (If the trial has an acronym then please provide) RAPIDE TKM
Disease(s) or condition(s) being studied Ebola,Infections and Infestations
Sub-Disease(s) or condition(s) being studied Ebola
Purpose of the trial Treatment: Drugs
Anticipated trial start date 16/02/2015
Actual trial start date
Anticipated date of last follow up 30/06/2015
Actual Last follow-up date
Anticipated target sample size (number of participants) 100
Actual target sample size (number of participants)
Recruitment status Completed
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)
Non-randomised Open-label(Masking Not Used)
INTERVENTIONS
Intervention type Intervention name Dose Duration Intervention description Group size Nature of control
Experimental Group TKM-100802 0.3 mg/kg, administered as a 2-hour intravenous infusion (IV) once daily for seven days 7 days TKM 100
ELIGIBILITY CRITERIA
List inclusion criteria List exclusion criteria Age Category Minimum age Maximum age Gender
Laboratory confirmed infection with Ebolavirus Informed consent provided by patient >18 years) or representative (too unwell) Informed consent from parent or guardian (child) Has no underlying condition that could jeopardize patient or staff safety Able to comply with protocol requirements None 0 Year(s) 99 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 23/01/2015 Sierra Leone Ethics and Scientific Review Committee
Ethics Committee Address
Street address City Postal code Country
Ministry of Health and Sanitation Freetown Sierra Leone
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 20/02/2015 Oxford Tropical Research Ethics Committee
Ethics Committee Address
Street address City Postal code Country
Joint Research Office, Block 60, Churchill Hospital Oxford OX3 7LE United Kingdom
OUTCOMES
Type of outcome Outcome Timepoint(s) at which outcome measured
Primary Outcome TKM: To evaluate the impact of TKM treatment on early mortality in EVD Mortality at Day 14
Primary Outcome Observational: To characterize the early mortality of EVD in an ETC Mortality at Day 14
Secondary Outcome TKM 1. To evaluate the impact of TKM-130803 treatment of adults and children on: a) Time to recovery Time to meeting ETC discharge criteria.
Secondary Outcome TKM 1. To evaluate the impact of TKM-130803 treatment of adults and children on: b) Late mortality Mortality at Day 30 after first dose of study treatment
Secondary Outcome TKM 1. To evaluate the impact of TKM-130803 treatment of adults and children on: c) Viral load Daily viral load (to day 7)
Secondary Outcome TKM 1. To evaluate the impact of TKM-130803 treatment of adults and children on: d) EVD symptoms Daily presence and duration of symptoms (Days 0-14)
Secondary Outcome TKM 1. To evaluate the impact of TKM-130803 treatment of adults and children on: e) EVD antibody response Day 30 convalescent anti-Ebolavirus IgG titer
Secondary Outcome To assess the safety of TKM treatment of adults and children. Daily incidence of SARs and key adverse events (Days 0-14)
Secondary Outcome Observational: To characterise the natural history of EVD in an ETC - a) Time to recovery Time to meeting ETC discharge criteria
Secondary Outcome Observational: To characterise the natural history of EVD in an ETC - b) Late mortality Mortality at Day 30 after admission
Secondary Outcome Observational: To characterise the natural history of EVD in an ETC - c) Viral load Daily viral load (to Day 7)
Secondary Outcome Observational: To characterise the natural history of EVD in an ETC - c) Viral load Daily viral load (to day 7)
Secondary Outcome Observational: To characterise the natural history of EVD in an ETC - d) EVD symptoms Daily presence and duration of symptoms (Days 0-14)
Secondary Outcome Observational: To characterise the natural history of EVD in an ETC - e) EVD antibody response Day 30 convalescent anti-Ebolavirus IgG titer
RECRUITMENT CENTRES
Name of recruitment centre Street address City Postal code Country
GOAL Port Loko Sierra Leone
FUNDING SOURCES
Name of source Street address City Postal code Country
Wellcome Trust 215 Euston Road London NW1 2BE United Kingdom
SPONSORS
Sponsor level Name Street address City Postal code Country Nature of sponsor
Primary Sponsor University of Oxford Joint Research Office, Block 60, Churchill Hospital Oxford OX3 7LE United Kingdom University
COLLABORATORS
Name Street address City Postal code Country
Tim Brooks Rare and Imported Pathogens Laboratory United Kingdom
Trudie Lang University of Oxford Oxford OX3 7FZ United Kingdom
Piero L Olliaro University of Oxford Oxford OX3 7FZ United Kingdom
Jake Dunning University of Oxford Oxford OX3 7FZ United Kingdom
John Whitehead Lancaster University Lancaster United Kingdom
CONTACT PEOPLE
Role Name Email Phone Street address
Principal Investigator Peter Horby peter.horby@ndm.ox.ac.uk +441865612977 NDM Research Building
City Postal code Country Position/Affiliation
Oxford OX3 7FZ United Kingdom University of Oxford
Role Name Email Phone Street address
Public Enquiries Raul Pardinaz-Solis raul.pardinaz-solis@ndm.ox.ac.uk +441865612977 NDM Research Building
City Postal code Country Position/Affiliation
Oxford OX3 7FZ United Kingdom University of Oxford
Role Name Email Phone Street address
Scientific Enquiries Peter Horby peter.horby@ndm.ox.ac.uk +441865612977 NDM Research Building
City Postal code Country Position/Affiliation
Oxford OX3 7FZ United Kingdom University of Oxford
REPORTING
Share IPD Description Additional Document Types Sharing Time Frame Key Access Criteria
URL Results Available Results Summary Result Posting Date First Journal Publication Date
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