The phase II study, led by Professor Peter Horby of the University of Oxford on behalf of the International Severe Acute Respiratory and Emerging Infection Consortium (ISARIC), is the second drug trial to be funded through the Wellcome Trust’s £3.2 million Ebola therapeutics platform.
TKM-Ebola-Guinea, developed and manufactured by Tekmira Pharmaceuticals, is a synthetic small interfering RNA (siRNA) therapeutic designed specifically to target the strain of the Ebola virus responsible for the present outbreak in Guinea, Liberia and Sierra Leone. It works by blocking certain genes of the virus, thereby reducing viral replication.
An earlier version of the TKM-Ebola drug (targeted at a different strain) has been tested in healthy human volunteers. The efficacy of the Guinea version will now be evaluated in patients with a confirmed diagnosis of Ebola virus infection in a single-arm study called RAPIDE-TKM (Phase II Rapid Assessment of Potential Interventions & Drugs for Ebola-TKM). Results of the trial are expected in the second half of 2015.
The RAPIDE-TKM study will be led by the University of Oxford in partnership with the Sierra Leone College of Medicine and Allied Health Sciences, the Sierra Leone Ministry for Health, the WHO-based Special Programme for Research and Training in Tropical Diseases (TDR), the UK Department for International Development (DFID), Public Health England and GOAL Global.
Professor Peter Horby, Associate Professor of Infectious Diseases and Global Health at University of Oxford, who is leading the trial, said: "There are still around 10 new cases of Ebola being diagnosed every day in Sierra Leone. It’s therefore essential that we push forward with clinical trials while we still have a realistic chance of getting answers about which, if any, of the candidate treatments can save lives in this, and in future outbreaks."
Colonel Professor Foday Sahr, Commanding Officer of the joint medical unit in Sierra Leone and a principal investigator on the study, said: "The start of this trial is a very important event for Sierra Leone and the other countries affected by Ebola, it is an exciting development because research is essential in this outbreak so that we can determine what might work for these cases. This clinical trial is a highly collaborative effort and we are working closely with international partners so that together we can get the evidence that is needed. The team working with the patients in the treatment centres demonstrate this partnership in action because they are a mix of local and overseas staff who are working side by side to deliver these trial treatments to the patients."
Dr Jeremy Farrar, Director of the Wellcome Trust, which is funding the trial, added: "The recent surge in new Ebola infections in Guinea and Sierra Leone should serve as a stark warning that this epidemic is far from over. Almost a year on from the first confirmed case, we’ve reached a crucial stage where several large scale trials are gathering steam, but we still don’t have any proven treatments. It’s therefore heartening to see this latest trial of TKM-Ebola getting underway after so much hard work from the research team and partner agencies. We’re very proud to be supporting their important work."
The Wellcome Trust Ebola therapeutics platform was set up in September 2014 to enable multiple partners to quickly establish clinical trials at existing Ebola treatment centres. TKM-Ebola-Guinea is the second candidate drug to be evaluated through the platform. A previous trial of the antiviral brincidofovir was abandoned earlier this year following a sharp fall in the number of Ebola cases at the study site in Liberia.