What Is an Investigator-Initiated Trial?
When most people think of a clinical trial, they picture a pharmaceutical company designing, funding and running the study. That is how many trials work, and company-sponsored trials remain the core of developing any medicine. But there is another important model in clinical development: the investigator-initiated trial, or IIT.
IN SHORT
An IIT is a clinical trial that begins with doctors rather than a company.
What is an IIT?
An IIT is a clinical trial that begins with doctors rather than a company. A physician or research team at a hospital, university or academic research organization spots an important scientific or clinical question that no existing trial is addressing, and decides to answer it.
The investigators write the trial plan, obtain the regulatory and ethics approvals, recruit patients and run the trial. Their institution is the sponsor, which means it carries the legal responsibility for the trial and oversees how it is run.
The company that manufactures or develops the medicine is not the sponsor and does not control the trial or its results. Depending on the agreement, it may provide the medicine and other support, but the question, the design, the conduct and the reporting remain with the investigators. Results from academic trials have also contributed to the approval of medicines, although that always depends on the data and on regulatory review.
Why do investigators start IITs?
Doctors and researchers see unmet medical needs every day. Drawing on their clinical experience and scientific work, they may spot a new interesting combination, a different stage of disease or a biological question that deserves testing. An IIT lets them explore that idea in a properly designed prospective clinical trial, following the principles of evidence-based medicine.
It also takes real commitment. Designing and running a trial demands years of work, expertise and institutional resources, and investigators do not spend them on ideas they do not believe in. Their choice to test a medicine reflects their own scientific conviction that the approach is worth examining. That is not evidence that the medicine works, and it says nothing about how any trial will turn out. Only the data can answer that.
IITs and bexmarilimab
Faron sponsors its own trials, such as BEXMAB and BEXERA. Alongside them, independent investigators are studying, or preparing to study, bexmarilimab in several settings:
Investigator-initiated trials of bexmarilimab
| TRIAL | WHAT IT STUDIES | LED BY |
|---|---|---|
| BEXAR | Bexmarilimab together with the chemotherapy doxorubicin in metastatic soft-tissue sarcoma | Dr César Serrano, Vall d’Hebron, Barcelona. Sponsored by MEDSIR |
| BLAZE | Bexmarilimab together with the immunotherapy nivolumab in metastatic lung cancer or melanoma that has stopped responding to earlier anti-PD-1 treatment | Institute of Cancer Research with the Royal Marsden, United Kingdom |
| City of Hope trial In planning | Bexmarilimab together with decitabine-cedazuridine, an oral medicine already used in myelodysplastic syndromes (MDS), in MDS that has returned or stopped responding to treatment | Dr Brian Ball, City of Hope, United States |
| BEAM-X Being prepared | Bexmarilimab in acute myeloid leukemia (AML) after a stem-cell transplant, when traces of leukemia remain in blood tests but the disease has not yet relapsed | Nordic AML Group |
Each trial asks a different question, and each began with investigators who saw a scientific and clinical rationale for studying bexmarilimab. That breadth is worth noting: experienced researchers in different institutions, countries and diseases have each decided, independently, that this is a question they want to answer.
In every case, the investigators and their institutions are responsible for the trial. Faron collaborates with them, and may supply bexmarilimab, but does not sponsor, lead or direct their work.
Why IITs matter for Faron
Company-sponsored and investigator-initiated trials play different roles. Faron’s own trials are the core of its development program, designed to answer the questions that matter most for the medicine’s development. IITs complement them by letting independent and experienced investigators examine further questions across new diseases, treatment combinations and stages of illness.
Who does what
| COMPANY-SPONSORED TRIAL | INVESTIGATOR-INITIATED TRIAL | |
|---|---|---|
| Who has the idea | The company | Doctors and researchers |
| Sponsor, with the legal responsibility | The company | A hospital, university or academic research organization |
| Who runs the trial | The company and its partners | The investigators and their institution |
| Who pays for running it | The company | Mostly the academic sponsor |
| Faron’s role | Leads the trial and carries the responsibility | Collaborates and supports, with scientific input, potential financial support and supply of bexmarilimab. No operational lead |
| Rules that apply | Regulatory and ethics approvals, informed consent and Good Clinical Practice | Regulatory and ethics approvals, informed consent and Good Clinical Practice |
They also make careful use of resources. Running a company-sponsored trial means paying for hospital sites, monitoring, safety reporting, regulatory work and data management. In an IIT, those responsibilities and most of that cost sit with the academic sponsor. Faron’s support is agreed separately for each trial. It can include scientific input, financial support and supplying bexmarilimab, but Faron takes no operational lead and carries no responsibility for how the trial is run.
In return, and subject to the collaboration terms, Faron may gain access to clinical data and to the results of laboratory analyses of patient samples. For a fraction of the cost of sponsoring such trials ourselves, these exploratory studies of novel combinations and tumor types have the potential to generate valuable new findings and add to the evidence around bexmarilimab.
What an IIT is not
Not a guarantee of results. Every clinical trial carries uncertainty. Bexmarilimab is an investigational medicine, and its safety and efficacy have not been established.
Not controlled by Faron. The investigators and their institutions decide how an IIT is conducted, analyzed and reported.
Not a shortcut. Like company-sponsored trials, IITs require regulatory and ethics approvals, informed consent and compliance with Good Clinical Practice. The standard is the same. What differs is who sponsors and runs the trial.
Looking beyond a single trial
No single trial can tell the whole story of a new medicine. Evidence builds up from different researchers, patients, hospitals and clinical settings. IITs add independent research expertise to that picture, alongside Faron’s own trials. We find the continued interest of investigators encouraging, while recognizing that every trial will ultimately be judged on the data it produces. Importantly, IITs complement Faron sponsored drug development by exploring important clinical questions and generating new evidence in areas of unmet need.
As our clinical program progresses, we will continue to share more about the science, the development process and the work needed to turn an idea into evidence that can matter for patients with high unmet need.
— Petri Bono, Chief Medical Officer
Forward-looking statements: This blog post contains forward-looking statements, including statements about the planning, design, timing and potential results of clinical trials, among them trials sponsored and run by independent investigators, and about the development of bexmarilimab. Forward-looking statements are based on Faron’s current expectations and are subject to risks and uncertainties that could cause actual results to differ materially. These include the uncertain outcome of clinical trials; the fact that investigator-initiated trials are sponsored and conducted by third parties, so that their planning, start, timing, conduct, results and reporting are outside Faron’s control; the need for regulatory and ethics approvals; and the availability of funding. Faron undertakes no obligation to update these statements, except as required by applicable law and regulation.
Bexmarilimab is an investigational medicine and has not been approved by any regulatory authority for any use.