Updated by: Jan Barfoot
A clinical trial is a research study in human volunteers to answer specific health questions.
Clinical trials are carried out in four phases:
- Phase I and Phase II trials check on safety – they make sure the treatment does no harm to patients.
- Phase III or IV trials focus on assessing how effective the treatment is, and developing it into a therapy that can be made widely available.
For more detailed information about the clinical trials process, visit these websites, or read our graphic short story, Hope Beyond Hype.
- European Union Clinical Trials Register
- Testing Treatments: Better research for better healthcare (available in many languages)
- Molecules to medicine: Clinical trials for beginners guest blog series on Scientific American
- NHS Clinical Trials
- FDA (US Food & Drug Administration)
- WHO International Clinical Trials Registry
In the first instance, we recommend that you discuss your plans and options with a range of people, for example, your doctor or physician, your broader healthcare team, your family, caregivers or a support or advocacy charity related to your condition.
Consider your options carefully, participating in a clinical trial is not the best option for everyone. The closerlookatstemcells.org website has information to help people make a decision regarding a stem cell clinical trial. To search for ongoing clinical trials - in stem cell research as well as other research areas - you can also try searching one of the clinical trials databases: the EU Clinical Trials Register, NIH's ClinicalTrials.gov, the World Health Organization's International Clinical Trials Registry Platform or Current Controlled Trials. Note that not all trials are listed in these databases.
ClinicalTrials.gov disclaimer - IMPORTANT: Information on ClinicalTrials.gov is provided by the sponsor or principal investigator of the clinical study, and posting to this site does not necessarily reflect endorsement by NIH. ClinicalTrials.gov does not independently verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies. Talk with a trusted healthcare professional before choosing to participate in a clinical study.
Some patient organizations also offer a platform for searching for clinical trials.
For more information about the clinical trials process, see:
- A Closer Look at Stem Cell Treatments - information to help patients and others evaluate stem cell treatments. Includes an outline of the process by which science becomes medicine. Produced by the ISSCR.
- ClinicalTrials.gov - 'About Clinical Studies' section
- What you need to know about stem cell therapies - handbook for patients from University of Alberta, Albany Medical College and the Canadian Stem Cell Network
- ISSCR Patient Handbook on Stem Cell Therapies - 8-page PDF document with patient-oriented information on stem cell therapies and the development of new medical treatments
- Experimental Treatments for Spinal Cord Injuries: What you should know if you are considering participation in a clinical trial - 40 page PDF document produced by International Campaign for Cures of Spinal Cord Injury Paralysis (ICCP) with general information about the clinical trials process as well as specific information about spinal cord injuries.
- Stem cell therapies in MS - 18 page PDF document jointly produced by the national multiple sclerosis societies from UK, Italy, France, USA, Australia and by the International Multiple Sclerosis Federation with general information about the state of the art of stem cell research in multiple sclerosis, including treatment prospects and clinical trial information.
- Clinical trials and stem cells: what patients should consider: a short video (2 minutes) from Stem Cells Australia with Dr Kirsten Herbert offering advice on questions patients should ask when deciding whether to participate in a clinical trial
In the first instance, we recommend that you discuss your plans with a range of people, for example, your doctor or physician, your family, caregivers and a support or advocacy charity related to your condition.
Information and resources provided on the site include:
- Nine Things to Know about Stem Cell Treatments—what is currently possible and what is not?
- The Patient Handbook on Stem Cell Therapies—a take-along handbook with information to help patients and their families evaluate stem cell treatments.
- How Science Becomes Medicine—a description of the process that is required to develop a new medical treatment and the internationally-recognized checks that protect the rights and safety of patients.
- What to Ask—questions to ask providers in order to learn about a treatment’s potential risks and benefits.
Further information is also available in the booklet What you need to know about stem cell therapies - a handbook for patients published in 2014 by the University of Alberta, Albany Medical College and the Canadian Stem Cell Network.
Sometimes it is not possible to access a clinical trial. However there are still options to get involved in research through contributing to a patient registry.
A patient or disease registry collects information about patients who are affected by a particular condition. Usually it is an electronic database. Information collected in patient registries can be used by doctors and researchers to improve medical care for people affected by those conditions.
If a disease is rare, a patient registry can allow doctors and consultants to see data from a lot of patients, rather than just the small number they see personally.
Sometimes in clinical trials a treatment is tested against the data collected from a registry, rather than have a group of patients affected by a condition get the trial treatment and a group that don't. Registries are proving to be useful for accelerating the research into orphan drugs (drugs developed to treat rare conditions or diseases) in specific populations.
Some researchers use registries to look for eligible clinical trial subjects. If you are considering a clinical trial, participating in a registry could help put you in contact with a clinical researcher.
For more information about patient registries and how they are used, TREAT-NMD has good further information and FAQ.
There are several listings of patient registries in Europe, including:
- TREAT-NMD listing of neuromuscular patient registries
- OrphaNet rare disease patient registry and biobank searchable database (available in 7 languages)
Please contact us if you discover others that should be on this list.
This article was compiled by EuroStemCell and reviewed by Phil Rossall, Research Manager (Knowledge Management) Age UK and Fellow, University of Edinburgh (Knowledge Exchange).