Ongoing research into rare diseases through clinical trials provides valuable insights into a disease, how it affects the body and how new medical treatments may help. Clinical trials are particularly important for medical conditions such as thymidine kinase 2 deficiency (TK2d) that are relatively unknown and where treatment options are scarce. Before accepting to participate in a clinical trial, though, it is important to understand the potential risks and benefits.
What is a clinical trial?
A clinical trial is a strictly controlled study of a new treatment on a carefully selected human population to prove its efficacy and safety. There are four different phases, with each phase progressing further towards approval of the medicine or therapy.
Clinical trials are time-consuming, costly and often unsuccessful, but they are an essential part of developing new therapeutic options.
What are the potential benefits of participating in a clinical trial?
The main benefit is receiving a drug that may potentially halt disease progression, reduce the symptom burden, improve quality of life or maybe even provide a cure. A clinical trial can be a life-changing chance to have early access to innovative medical treatments that wouldn’t otherwise be available for many years to come (sometimes up to 10 years).
In people living with TK2d, treatment options are mainly limited to managing symptoms and providing supportive care, with only one recently approved medicine available to help slow disease progression.
Advances in medical research cannot occur without clinical trials, and clinical trials cannot proceed without participants. By participating in a clinical trial, you are contributing to medical research that will benefit future patients with TK2d.
Learn more about TK2d treatment and care
What are the potential disadvantages?
The doctors and researchers involved in the clinical trial are studying the safety, dosing and efficacy of the drug based on the response of the trial participants. Particularly in the early phases of clinical trials, there is a lack of information on what to expect. Side effects can be difficult to manage and unpredictable. There is also a risk of unknown long-term side effects.
A clinical trial is conducted within strict conditions, and there are many follow-up visits and tests to monitor side effects, the effect of the treatment on the body and the effectiveness of the treatment. This is time-consuming and can be logistically challenging, especially in a condition like TK2d that limits mobility.
It is also worth noting that in some clinical trials there is a risk that you will receive a placebo rather than the active drug.
Finally, there is no guarantee that the clinical trial will work. In fact, the majority of clinical trials fail, with only a 7.9% success rate.
The takeaway
The decision to participate in a clinical trial is a personal one, and needs to be based on your family’s individual situation, including the stage of disease progression, your overall physical health and age. Your healthcare team will support you while you weigh the pros and cons of participating in a clinical trial.
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