Clinical Studies

General

The different phases

Clinical studies are conducted in three successive phases, of varying lengths, at research sites.

Phases I

Phase I studies are the first studies on humans, and take place before the efficacy of a treatment is evaluated. They are conducted on a small group of healthy volunteers. The main aim of this phase is to evaluate the tolerability, the absence of side effects and the way in which the product is absorbed, distributed and metabolised by the body. This phase may, in exceptional cases, not need to be carried out.

Phases II

Phase II studies are conducted on a larger group than phase I studies, comprised of volunteers suffering from the disease targeted by the test product. The aim of this phase is to study whether the expected beneficial effects are observed in the population affected by the targeted disease, as well as the side effects of the test product at the doses associated with beneficial effects.

Phases III

Phase III studies involve a much larger group of patients. The aim is to formally test the nature and extent of the benefits of the new treatment versus a comparator in terms of safety and efficacy. From phase II, but more generally during phase III, the treatment is compared to a standard treatment, called a “reference” treatment, and/or a placebo treatment, that is, with no pharmacological properties.

At the end of phase III, the treatment may be submitted to the competent authorities and reimbursement authorities in order to obtain marketing authorisation. The competent authorities may be, for example, the European Medicines Agency (EMA) or the American Food and Drug Administration (FDA)

What is a clinical study?

A clinical study is a study which is designed to evaluate the safety and efficacy of a new treatment in humans.

Before reaching the clinical study stage, the hypothetical treatment is first tested in the laboratory – in vitro and in vivo (on animal models) – to confirm the relevance and safety of the hypothesis. If the results are satisfactory, it is then tested on human subjects during clinical studies.

The key players

The sponsor

The sponsor initiates and finances the clinical trial and handles the organisation and execution thereof. The sponsor may be a legal person (non-governmental organisation, laboratory or business) or a physical person.

The investigator

The investigator is a doctor who supervises the conduct of the clinical trial. He/She is the link between the sponsor and the patient who is participating in the study. The patient is, therefore, never in direct contact with the sponsor. The investigator supervises the clinical study in a hospital centre, or in a private or public establishment, which is called the investigating site.

The patients

The patients are the participants in the clinical study. They must meet certain eligibility criteria in order to be enrolled in the study.

Advantages and disadvantages

The patient may benefit from several advantages by participating in a clinical study:

Possibility of receiving a promising treatment before it is marketed.
Close medical follow-up by a highly qualified team.
Contribution to the advancement of medical research in the clinical study field.

There are also some constraints and disadvantages:

Strict protocol of visits with which the patient must comply in order to monitor disease progression.
Possible administration of a placebo instead of the test treatment.
Possible adverse side effects.
Uncertainty with respect to the efficacy of the treatment.

Who can participate?

In order to participate, patients must meet the eligibility criteria (also called inclusion criteria) required for the study in which they want to participate. These criteria may, for example, be based on age, diagnosis or the stage of progression of the treated disease.

Patients must be able to understand the requirements of the study and read and sign the informed consent document.

There are also exclusion criteria which exclude some patients from participating in the study. These may, for example, be associated with a list of particular treatments or diseases.

The informed consent

During the process of obtaining informed consent, the doctor ensures that the potential patient has received the necessary information regarding the trial (the aim, the treatment given, the duration and the protocol) and regarding the risks involved. Once the patient has received this information and if he/she is still interested in participating in the study, he/she will sign an informed consent document as proof of his/her consent.

Once the document has been signed, the patient can still withdraw from the study at any time, without having to give a reason or being subject to damages as a result of his/her withdrawal.

cGVHD indication

A successful Phase II study

cGvHD - Chronic Graft versus Host Disease - is a complex autoimmune reaction that develops following bone marrow or more precisely allogeneic hematopoietic stem cell transplants, with a frequency of 30-60%.

In this study, the first-line combination of arsenic trioxide and prednisone in cGvHD showed an Overall Response Rate (ORR) at 6 months of 75%, allowing rapid corticosteroid tapering.
Skin and eating disorders significantly improved whereas other dimensions of quality of life did not change.
In murine models of cGvHD, arsenic trioxide (ATO) increases oxidative stress and regulates T and B cell populations and macrophage polarization.

High response rate and corticosteroid sparing with arsenic trioxide-based first-line therapy in cGvHD after allo-HSCT - Transplantation and Cellular Therapy, Official Publication of the American Society for Transplantation and Cellular Therapy (astctjournal.org)

Study status: Finalized

Sites: Investigation sites throughout France

Detailed information: More information about the study can be found here.

Phase III study in preparation

Following the successful Phase II in cGvHD, and the positive conclusions of the pre-IND (Investigational New Drug) meeting with the FDA (U.S. Food and Drug Administration), a Phase III is being prepared with a protocol including OATO (oral arsenic trioxide).
This will be submitted as part of an IND application and may begin in the first half of 2023. This is a randomized, double-blind, placebo-controlled Phase III study to evaluate the efficacy and safety of oral arsenic trioxide (OATO - ArsciCor) as an adjunct to corticosteroids in first-line chronic graft versus host disease (cGvHD).

Study status: In preparation

Participation

Choosing to participate in a study is an important personal decision. Patients should first talk it through with their doctor and family members in order to discuss the state of their health and different treatment options. If the patient is interested in the study, his/her doctor will then be able to redirect him/her to their nearest clinical trial site.

BioSenic is not authorised to refer patients to clinical study sites or respond to their requests for information regarding participation in ongoing trials.

If you are a physician and you would like to receive information regarding a BioSenic study or you have an eligible patient interested in a study, you can contact BioSenic