ReveraGen has provided a letter to participants in their vamorolone trial indicating promising results with strength and mobility of patients on drug. Additionally, thanks to a NIH grant, ReveraGen will be sharing individual results with families from the study. Upon request, participating families will receive information related to your arm of the study, as well as your son’s individual results and how his results compare with others. Details on how to obtain the results can be found in the letter.

PPMD was happy to work with ReveraGen to support their NIH grant request to deliver individual data and we hope that other companies will consider sharing trial results with patients when possible.

Read the Letter from ReveraGen

Dear Study Participant,

On behalf of the staff at ReveraGen, we want to thank you sincerely for your and your child’s participation in a vamorolone clinical trial.

It is only with the help of volunteers like you and your son that we can perform the essential research to find new and effective treatments for people with Duchenne.

You and your child participated in one of the two studies:

• 48 boys joined a study in 2016/2017. This included the trial series VBP15-002 (4 weeks), VBP15-003 (6 months) and VBP15-LTE (2 years).  Some families have completed the full 2.5 years.
• 120 patients are being enrolled in VBP15-004 (2018-present).

Studies to date have shown that vamorolone improves strength and mobility in DMD, while reducing side effects seen with corticosteroids.

We realize that participating in a clinical trial was/is time consuming and has asked a tremendous amount from you and your family. We value the time you are committing to our research efforts. Your son and family’s contributions will help others as a result of the medical and scientific knowledge gained from your participation.

We have recently received a National Institutes of Health grant to return vamorolone trial data to families.

This study will help us understand the preferences of you (patients, parents, caregivers) in what clinical trial information you would like returned to you, and then to accomplish this.

If you decide to enroll in this study, which is completely optional and will not affect your son’s involvement in any trial

• You will receive your son’s individual test results
• Your child’s performance relative to others in the trial
• You will be notified of the arm of the study your son was randomized to in VBP15-004, or the starting dose group if he finished VBP15-002, VBP15-003, or VBP15-LTE.

If you choose to participate in this study, you will be asked to return 2 short questionnaires (one before data return and one after data return). A survey will be sent to your site physician also, to better understand his/her perspective.  Survey responses will be compiled with others and will not be linked to you or your child’s identity.

For more information, please contact Suzanne Gaglianone.