Our Clinical Trials

Prevail Therapeutics is developing PR001, a potentially disease-modifying, single-dose gene therapy for Parkinson’s disease with GBA1 mutation (PD-GBA). We have initiated the PROPEL trial, a Phase 1/2 clinical trial of PR001 for the treatment of PD-GBA patients. More information can be found here. We also plan to initiate a Phase 1/2 clinical trial in neuronopathic Gaucher patients in 2020. Prevail is also developing a pipeline of therapies for patients with other neurodegenerative diseases.

Our clinical studies will be the primary route by which patients receive access to our investigational drug(s). We intend to post information related to our ongoing applicable clinical trials on

Our Expanded Access Policy

Prevail is committed to developing safe and effective medicines and treatments for patients worldwide. Before marketing drugs to patients, Prevail designs and conducts clinical trials to generate valid scientific evidence and to protect the rights, safety, and welfare of research participants.

Once available, Prevail’s clinical studies will be the primary route by which patients receive access to Prevail’s investigational drug(s). Prevail intends to post information related to its ongoing applicable clinical trials on

“Expanded access” or “compassionate use” is a process through which a patient and the patient’s physician may seek to obtain an investigational product outside of a clinical study. These programs only apply to medicines not yet approved by the national drug regulatory authority in the country of the patient’s residence. Regulations and guidance issued by the U.S. Food and Drug Administration provide that access to an investigational product as part of an expanded access or compassionate use program may be appropriate only when all of the following conditions are satisfied:

  • The patient has a serious or immediately life-threatening disease or condition;
  • The patient is ineligible or otherwise unable to enter ongoing clinical trials of the investigational product in question;
  • There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition;
  • The potential benefit to the patient justifies the potential risks of the use of the product and the risks are not unreasonable in the context of the disease or condition to be treated; and
  • Providing the investigational drug for the requested use will not interfere with the initiation or conduct of clinical trials that could support marketing approval of the expanded access use or otherwise compromise the potential development of the expanded access use.

At this time, Prevail does not make its products available through an expanded access or compassionate use pathway. Prevail believes that conducting clinical trials is the most appropriate way to achieve Prevail’s goal of developing its investigational products and bringing such products to market as quickly and safely as possible. However, Prevail may revise this policy at any time.

Patient Inquiries

Our therapies are not yet available to patients. We are working to bring forward these potential medicines as quickly as possible.

If you have questions about Prevail’s expanded access policy or clinical trials, please write to Prevail will try to acknowledge receipt of your message within five (5) business days of receiving your question, but it may take longer.