PPMD is excited to learn that Solid Biosciences Inc. announced that the FDA has lifted the clinical hold on IGNITE DMD, the Company’s Phase I/II clinical trial for its investigational microdystrophin gene transfer, SGT-001. In its letter, the FDA acknowledged that Solid satisfactorily addressed all clinical hold questions. Solid has begun activities to resume the clinical trial and plans to reinitiate enrollment as quickly as possible. Read the company’s letter to the community below.
Dear Patient Community,
We’re pleased to share with you that the FDA has lifted the clinical hold on IGNITE DMD, our clinical trial investigating SGT-001 as a potential treatment for Duchenne muscular dystrophy (DMD). SGT-001 is a microdystrophin gene transfer candidate that we believe has the potential to slow or stop the progression of DMD, regardless of a patient’s genetic mutation, age or stage of condition. The FDA acknowledged Solid Biosciences satisfactorily addressed all of its clinical hold questions. We now have the green light to move forward with our clinical trial and continue to evaluate SGT-001’s potential in patients with DMD. We have already begun our activities to resume the study and are working to reinitiate enrollment as quickly as possible.
The FDA originally placed a hold on IGNITE DMD because the first patient dosed with SGT-001 in the clinical trial, a non-ambulatory adolescent, experienced a serious adverse event. Due to the excellent care of Dr. Barry Byrne and his team at the University of Florida, the event was identified early and well-managed. The event showed up in laboratory tests, and the patient had few symptoms throughout this experience. We are happy to share that the event has fully resolved and the young man has returned to his normal activities.
As a company directly touched by DMD, Solid Biosciences is always focused on patient safety. Based on what we have learned from the experience of this patient, we’ve made some modifications to the clinical trial protocol to help reduce the risk or impact of any potential future events. These changes include a modified steroid regimen and additional monitoring measures, as well as guides to facilitate early medical interventions should an event occur.
We also now plan to dose several children before dosing additional adolescents in the clinical trial. While this constitutes a change in the sequence of patients dosed with SGT-001, we have every intention to continue to evaluate SGT-001 in non-ambulatory adolescents as quickly as possible.
In addition, we have also made a change to the muscle biopsy schedule outlined in IGNITE DMD. Previously, our protocol stated that each patient enrolled would undergo three biopsies: one at baseline, one at 12 months and one intermediate biopsy at either three or six or nine months. Each patient will continue to undergo three biopsies; however, we now have added an option to take the intermediate biopsy at day 45 post administration. We believe that this additional time point will provide us with even more information to evaluate the potential efficacy of SGT-001.
We look forward to reviewing this information and answering questions during our presentation at the PPMD Annual Conference next week.
We recognize the decision to participate in clinical research is not made lightly, and we thank the patients and families who choose to do so. We will continue to do our best to minimize the burden of being in a clinical trial and to provide a positive patient experience.
We thank the entire DMD community for its continued support of our mission to make DMD a disease of the past and hope to see many of you at PPMD!
Your Team at Solid Biosciences