ROCKET Fragile X Syndrome Clinical Study

//ROCKET Fragile X Syndrome Clinical Study

ROCKET Fragile X Syndrome Clinical Study

The ROCKET study is a clinical research study that will help determine if an investigational medicine, called OV101 or gaboxadol, is safe and effective in treating behavioral characteristics commonly present in people with Fragile X syndrome (FXS). The study will test three different doses of OV101.

Participation in the ROCKET study will last approximately 21 weeks and include six visits to the study medical center and four phone appointments. The study center visits will last the majority of the day.

At the clinic appointments, you will meet with your study team, which will include a study physician, nurse, and other healthcare professionals. They will explain the study, answer your questions, and monitor your care.

The phone appointments will take place between visits to the study medical center. During these calls, a study team member will ask about any changes in your son’s health and behavior. You may also discuss any side effects or new symptoms your son may be experiencing and any new medicines or vitamins he may be taking.


Sex: Male
Age: 13 to 22
Disorder: Confirmed diagnosis of Fragile X syndrome

He also needs to be able to swallow a capsule, or its contents when mixed with soft food; and must have a parent or caregiver who can provide consent, attend scheduled visits, and participate in study assessments and scheduled phone meetings.

U.S. Locations

  • Phoenix, Arizona
  • Sacramento, California
  • Aurora, Colorado
  • Chicago, Illinois
  • Baltimore, Maryland
  • Cincinnati, Ohio
  • Media, Pennsylvania
  • Nashville, Tennessee

Frequently Asked Questions

A clinical research study is a scientific test that helps determine if and how a potential medicine or treatment may work. Clinical research studies, like the ROCKET study, are monitored by the U.S. Food and Drug Administration and reviewed by independent ethics committees to safeguard the rights and welfare of research participants.

People agree to participate in studies for many reasons, including the chance to help others who may be or become affected by certain conditions, give back to the scientific community, play an active role in their health and gain access to new potential medicines.

No. Participating in any clinical study is completely voluntary.

Deciding to volunteer in a study is an important choice. If your son qualifies to participate in the ROCKET study, your study team will ensure that both of you understand the study and its possible risks and benefits. You or your son can decide to withdraw from the ROCKET study at any time.

No. You will not be charged for taking part in the study. The investigational medicine and care required by the study will be provided at no expense by the study sponsor, Ovid Therapeutics™. You will also be reimbursed for travel-related expenses, and travel can be arranged for you. Please talk with your study team for more information.


Luke Rosen
Director of Patient Engagement

Study Director
Amit Rakhit, M.D.

Ovid Therapeutics Inc. Identifier:

If you’d like to learn more or apply for this opportunity, please fill out the form to send your contact information to the study coordinators.

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By | 2019-11-13T13:14:13+00:00 Feb 1, 2019|Opportunities for families|Comments Off on ROCKET Fragile X Syndrome Clinical Study