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The Gene Therapy Clinical Trial Experience—What to Know

Curious about gene therapy clinical trials but don’t know where to start? Check out the new content on Hemophilia Forward about the clinical trial experience.


Clinical trials play a crucial role in advancing research and understanding potential treatments. By participating in gene therapy clinical trials, individuals make an important contribution to the development of potential future gene therapies. Results from a clinical trial provide insight into the safety, effectiveness, and appropriateness of the gene therapy being studied. But not all clinical trials are the same, and there are considerations to discuss with your doctor before choosing to participate.


These key points can help get the conversation started:


Investigational gene therapies are studied long before they move to clinical trials. 

The drug-approval process is designed to evaluate the safety and effectiveness of new therapies before they’re available to the public. That’s why it can take years of preclinical research and preliminary studies before investigational therapies are authorized by the FDA for use in a human clinical trial.

Explore the steps of a clinical trial.

The safety and well-being of clinical trial participants are top priorities.

Investigational gene therapies are commonly developed as a one-time dose. This means trial participants are carefully monitored for months or even years after treatment. Talk with your doctor to understand what a clinical trial means for you and your family in the future. 

Is a gene therapy clinical trial right for you? Watch the video.

There are potential benefits and potential risks for any investigational therapy. For example, in hemophilia gene therapy clinical trials, one of the risks people may have is an immune response that could affect how well the gene therapy works after its given. 

Learn more about the potential risks and why it’s important to discuss with your doctor.

FDA=United States Food and Drug Administration.


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