Clinical research brings new treatments to patients. New treatments can be new medicines, new cell therapies and other forms of advanced therapies, new surgical procedures or other new approaches to managing a patient. Randomised controlled prospective clinical trials are generally agreed to be the golden-standard for clinical research. A randomised controlled trial compares two or more treatments or approaches in a controlled setting.
A prospective clinical trial is required if the trial protocol includes an intervention that is outside of the patient’s routine medical care, such as an unlicensed medicine. All prospective clinical trials must follow a protocol that has prior ethics committee and competent authority approval and must be conducted in compliance with the EU Clinical Trials Directive (2001/20/EC) and the EU Good Clinical Practice directive (2005/28/EC). All EBMT-sponsored prospective clinical trials will be approved by the Clinical Study Unit (CSU).
EBMT-sponsored prospective clinical trials are run by the EBMT Clinical Study Unit in Leiden, often in conjunction with a CRO. The CSU supports multinational, multicentre trials. The services that the CSU can provide vary between trials and may include study design and operational feasibility, protocol and patient information leaflet writing, ethics committee and competent authority submissions, insurance, site selection and set up, monitoring pharmacovigilance, database set up, data management, and day-to-day project management and trial master file set up and maintenance. The services that the CSU will provide for an individual study will be agreed with the Primary Investigator.
If you would like to know more about the EBMT Clinical Study Unit, or are interested in sponsoring a trial, please contact the Associate Director Clinical Research or the Clinical Studies Operationals Manager.
How to submit a clinical trial proposal
- The Investigator proposes his or her trial idea to the appropriate Working Party (WP) Chair. All EBMT-sponsored and EBMT-labelled prospective clinical trials must be submitted via a WP chair.
Two of the Working Parties, the Inborn Errors WP and the Infectious Diseases WP, have specific procedures for proposing new studies. For all other WPs, contact the Chair or the Secretary.
- Preparation and review of the proposal within the WP and with a WP statistician.
The WP statistician will write the statistical section. The Clinical Studies Operations Manager and the study Statistician can be approached during this period for advice.
- The WP Chair submits the proposal to the Clinical Study Unit with budget estimates. A Word version and a signed PDF of the proposal, along with a budget estimate spreadsheet must be submitted to the the Associate Director Clinical Research. Confirmation of receipt will be provided and the WP Statistician must be able to provide clarifications and missing material if required.
- The Clinical Study Unit will review the proposal in terms of Science, Statistics, , and Operational feasibility. The Business Unit will be responsible for the study budget review together with the CSU.
- The Clinical Study Unit will present the proposed trial to the Board. You will will be provided with feedback after the Board meeting.
- The Board may select 1-2 trials for EBMT sponsorship per year and the Clinical Study Unit will inform the WP Chair. There will be a second review of the selected trials when the full protocol and contracts are available.
Golden rules for setting up a clinical trial
- Ensure everyone is on board before you finalise the protocol - this must have been reviewed by the Working Party, the WP statistician and any relevant national coordinating groups. This avoids subsequent avoidable amendments to the protocol.
- Work with the CSU and BU early to develop a budget for the trial so that you can raise funds to support it. Include a payment for centres. All EBMT-sponsored clinical trials must have adequate funding.
- Be clear in the questions that will be answered by the trial. This ensures that the CRFs can be well designed so that the correct data are collected.
- Allow the CSU to advise which centres should participate in a trial, based on transplant data and past experiences.
- Ensure that all inclusion and exclusion criteria are warranted to ensure adequate patient recruitment, and have contingency plans for poor recruitment (back up centres/countries, advertising).
- Support the CSU’s Risk Management Plans (safety, monitoring, quality management) - these ensure that we fulfil our legal obligations and that our trials protect the rights, safety and well-being of subjects, and that the data generated are reliable and robust.
- Support the Clinical Study Coordinators (CSCs) by providing all essential documents and requested information in a timely manner.