Developing groundbreaking therapies for people living with rare disorders

Gene therapy targets the underlying cause of a genetic disease. In gene therapy, a functioning gene is delivered to a targeted tissue in the body, enabling the cell or tissue to produce a protein that is either missing or is not functioning. This type of treatment has the potential to be used instead of drugs or surgery.

The safety and efficacy of gene therapy is being studied in clinical trials.

We believe that gene therapy is a potentially transformative treatment approach that, if successful, may help reduce the burden of disease and of ongoing treatments for the people who are affected by rare diseases.

What happens in a clinical trial depends on the ‘protocol.’ A protocol is a detailed plan that explains the purpose of the clinical trial and how it will be run. If you consider joining a clinical trial, these details will be explained to you as part of the informed consent process. They include: 

• the length of the clinical trial 
• the study medicines, procedures, and tests in the clinical trial  
• the schedule of study activities 
• information about who can participate 
• how side effects will be tracked, managed, and reported 
• the rules that must be followed 

A clinical trial may also have aspects such as randomization, placebo, or blinding.

We encourage everybody to consider joining clinical trials. There are many types of clinical trials that study different aspects of health in people with a variety of backgrounds and conditions. Each clinical trial has its own criteria for who may participate. These are called eligibility criteria.  
 
Eligibility criteria include things like your age, sex, overall health, type of condition, and your medical treatment history. As part of the steps to join a clinical trial, the study doctor will confirm whether you meet all eligibility criteria and are able to participate.

Participation in a clinical trial is always voluntary. The decision to join is personal, and it is yours.
 
To learn more about Pfizer’s clinical trials, visit Our Research or search for trials here. 

We list all of our clinical trials that are recruiting participants or plan to recruit shortly in our Find a Trial search engine. We welcome you to explore our clinical trials and the information provided about each study. There is contact information available on each study’s webpage. 

For general inquiries about Pfizer clinical trials, you may call 1-800-887-7002 or email [email protected].

Our Find a Trial search engine only provides information about Pfizer clinical trials. To search for all registered clinical trials, regardless of the study sponsor, you may visit ClinicalTrials.gov.