A clinical trial (also called a research study) is one type of study that involves using human volunteers (also called participants). Participating in a clinical trial is an option for many patients with bone marrow failure diseases.
Each clinical trials will have a protocol or written description of the clinical study. A protocol includes the study's objectives, design, and methods. It may also include relevant scientific background and statistical information.
A study will often have a primary purpose. It may be related to:
- Treatment: The protocol is designed to evaluate one or more interventions for treating a disease, syndrome, or condition.
- Prevention: The protocol is designed to assess one or more interventions aimed at preventing the development of a specific disease or health condition.
- Diagnosis: The protocol is designed to evaluate one or more interventions aimed at identifying a disease or health condition.
- Supportive Care: The protocol is designed to evaluate one or more interventions where the primary intent is to maximize comfort, minimize side effects, or mitigate (lessen the effect) against a decline in the subject's health or function. Supportive care interventions are not generally intended to cure a disease.
- Screening: The protocol is designed to assess or examine methods of identifying a condition (or risk factors for a condition) in people who are not yet known to have the condition (or risk factor).
- Health Services Research: The protocol is designed to evaluate the delivery, processes, management, organization, or financing of health care.
- Basic Science: The protocol is designed to examine the basic mechanism of action (e.g., physiology, biomechanics) of an intervention.
- Other: The protocol is described in a “Detailed Description.”
The National Institutes of Health’s www.ClinicalTrials.gov is a web-based registry and results database of clinical trials conducted around the world.
When you search for a study related to a bone marrow failure disease, you can see the study record details. Here are some key points you want to pay attention to when you click on a study to learn more. Just below the title of the study, you will find the:
Study Status—If a study is open to participants, it will indicate “Recruiting” in green on the page. Studies that are not open to patients indicate their recruitment status in red.
Sponsor—This will identify the organization or person who oversees the study and is responsible for analyzing the data.
Collaborator—This identifies any organization other than the sponsor that provides support for a clinical study. This may include funding, design, implementation, data analysis, or reporting. This can include pharmaceutical companies, universities, or even patient advocacy organizations.