TT4CL aims to deliver a new, safe, orally available treatment for CL afflicting patients in Africa, Europe and the Middle East. Existing therapies have several drawbacks: a variable efficacy, safety issues, upcoming resistance, stability problems and high cost. Moreover, current drugs on the market exhibit low tolerability and serious toxicities. They have long treatment durations and difficult administration (iv, im, intralesional). Miltefosine is the only oral product on the market and patients have difficulties to access it.
A more effective drug with an improved safety profile, more accessible to patients and possibly with a higher patient compliance because of shorter treatment regimen, will be a major advancement. No other drugs are at comparable stage of clinical development. Our impacts will therefore be on an important aspect of disease control. Despite there being few candidates considered for further development work by agencies such as the DNDi (Geneva), there are no other systemic drugs considered for monotherapy that are sufficiently advanced to enter Phase 1/2 studies for CL. For these reasons, delivery of any effective and safe treatment will result in major advancement in the field and transform local and eventually global management of this disease.
Our clinical study will have a positive impact on the health of affected Iranian people by focusing activities and collecting comparative data from their isolates that would not otherwise be available. The TT4CL project puts forward the implementation of a rigorous and progressive study design intended to allow selection of a safe appropriate efficacious dose at the end of the Phase 1 study, so that further trials, including loading dose and multiple dose phase I can be implemented.
If OlPC shows a better safety and at least a similar efficacy compared with the local standard of care, we intend this product to be implemented in national guidelines for the treatment of CL. By answering an unmet medical need OlPC, as an oral safe drug, has the potential to become the preferred alternative to “no treatment”. Registration for the indication of CL with stringent regulatory authorities will greatly facilitate the subsequent registration of OlPC in endemic, mostly LMICs, so that the drug can be accessed.
The results of the Phase I trials will be compiled in an application for stringent regulatory authorities (the American FDA and the EMA). This will have a spill-over to the development of OlPC for new parasitological and/or fungal indications.
