Explore Randomized Controlled Trials and Clinical Studies
Clinical Trials Process
The journey of a clinical study is extensive and meticulously structured. It generally follows four phases:
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Phase I - This initial phase typically involves a small group of healthy volunteers. The main goal is to assess the safety of a drug and identify any side effects.
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Phase II - Involves a larger group of participants (often varied patients with the disease). The effectiveness of the drug begins to be evaluated.
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Phase III - This phase compares the new treatment against the standard treatment or placebo in a much larger group. The results confirm if the treatment is effective and safe for the general public.
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Phase IV - Conducted after the drug or treatment is approved. Ongoing monitoring ensures long-term effectiveness and safety.
Randomised Controlled Trials
When looking at clinical research it is important to use good quality studies.
Randomised controlled trials (RCT’s) are considered the ‘Gold Standard’ in clinical research. The effectiveness of different therapies are compared through a process of blind randomisation to minimise bias. When looking for research papers try to use RCT’s where possible.
RCT’s are often long and complex studies. This means that there may not be a huge number available to help with your research. In the absence of RCT’s Peer review articles can be used. They are studies that have been scrutinised by experts in the subject being studied before publication. This critical evaluation enhances the credibility of research findings.
Meta analysis is a statistical technique that groups studies on a particular topic to draw comprehensive conclusions. They are more reliable and robust than single studies.
Finding good quality papers to help with your research can be difficult. We recommend looking at EBMT website. They are the leading authority on Bone Marrow Transplants in Europe. They have a plethora of articles and evidence to help you with your research into HSCT. The button below will show results from the EBMT Registry that include the keyword "Multiple Sclerosis".
Click on the picture below to access the full article
The following retrospective analysis was published 11th June 2025
UK wide 20 years experience of Autologous HSCT in multiple sclerosis reaffirming its significant benefits even with advanced disability, with some key findings:
1. Long term progression free survival (PFS) >60% at 5+years in all cohorts (RRMS>PPMS), with around half of PPMS patient showing no further disease activity!
2. Viral infections need monitoring and prompt treatments, negative effect on PFS.
3. First report confirming more immunosuppression i.e. higher ATG doses do not confer additional benefit. Harmonised dosing essential!
4. Transplant related Mortality risk <1.5%, mainly seen with advanced disability and needs better optimisation/ careful selection to reduce this further.
5. Key problem is inequity of access with some regions in UK undertaking markedly less activity. Need more awareness.