Dr Wilfried Mutombo, Coordinating investigator, Fexinidazole study for HAT
At the time DNDi and its partners are extending the clinical trial on fexinidazole, the first new oral treatment tested for sleeping sickness, to the early stages of the disease in adults and to children between 6 and 14 years of age, it is important to remember how clinical trials in remote areas of a country such as the Democratic Republic of the Congo (DRC) can be a daunting challenge, but can also bring lasting benefits to local communities and researchers, and to the health system overall.
Conducting clinical research in compliance with international standards in such conditions is possible, and necessary, so as to ensure that the tested treatments will be safe and effective, and answer to the real needs of patients. However, such research requires adequate capacities and infrastructure, including trained staff, and adequate wards and onsite labs. Because sleeping sickness occurs in very remote areas of the country, there are major operational challenges to be overcome to conduct such trials according to Good Clinical Practice (GCP), to ensure the high quality of the research results.
As infrastructure is often limited in endemic and remote areas, building an environment conducive to efficient clinical trials is required. In DRC and Central African Republic, we selected nine referral treatment units that we renovated for the trial. Due to the lack of electricity and to minimum infrastructure, buildings were refurbished and solar energy equipment and generators installed to ensure regular supply of electricity, in order to guarantee the cold chain required to store some reagents, and for the function of laboratory equipment and computers. Emergency medical equipment such as defibrillators and technical equipment such as the Picocolo anaylser, a fully automated system for blood testing, were provided. Internet access was installed to enable transmission of electronic case report forms to particularly monitor safety parameters.
In addition to physical infrastructure, a lack of trained staff can also be a major obstacle. Therefore, staff received refresher courses in human African trypanosomiasis (HAT) diagnostic techniques and treatment procedures, pharmacovigilance, and training in GCP guidelines through the HAT Platform, which is a regional disease-specific clinical research network supported by DNDi.
Strengthening infrastructures and human resources in endemic countries facilitates the conduct of GCP clinical trials, but also generates greater capacity and knowledge in our countries to conduct our own research that addresses our own public health needs. It also helps us to offer better conditions of treatment for the patients and health staff. Benefits are felt on all sides. As a doctor who treated sleeping sickness patients, a former local investigator of the previous study carried out by DNDi and its partners on NECT, which was the first new, improved treatment option in 25 years for stage 2 HAT and today as the coordinating investigator of the trial on fexinidazole, I can attest to the huge impact of these two studies on the ability and medical practices of doctors, nurses, and lab technicians. For instance, with the study on NECT, a treatment that is partly given intravenously, health staff increased their skills to perform and monitor infusions, a capacity that helps hospital practice well beyond the HAT wards. They are now also more familiar and competent with managing drug stocks.
With the study on fexinidazole, health staff was trained in performing and in interpreting electrocardiogram (ECG) results, and patients who are not part of the study can now also benefit from the ECG when necessary. Another important benefit is the medical waste management, which was not a common practice in rural Congolese hospitals until clinical research began in these regions.
As a conclusion, in neglected-disease endemic countries, where DNDi’s clinical trials take place, and particularly in Africa, the challenges we faced have been overcome by strengthening capacities, working in close collaboration with local and international partners, and keeping the reality of the patients in focus in the trial set up. These components are of critical importance to ensure further successful trials. In addition, the capacity to conduct clinical research in endemic and remote areas has now been built and, while initially there is a certain degree of burden on staff to undergo training and change practice in some cases, by maintaining this capacity in the endemic countries, staff, hospitals, and health systems more broadly can reap many lasting benefits… ultimately for patients!
Dr Wilfried Mutombo
Coordinating investigator, Fexinidazole study for HAT