Generating the Evidentiary Data Package for Dystrophin Biomarker Qualification
Abstract
Duchenne muscular dystrophy patients are missing the muscle protein dystrophin. Therapies to restore this missing protein are currently being developed and tested. It is important to test if these drugs are working to get Food and Drug Administration (FDA) approval. Because the drugs are restoring the missing dystrophin protein, detection of the new dystrophin is important. Additionally, it is also important to know exactly how much new dystrophin is being made. Currently, we do not know how much new dystrophin is needed before the patient will improve or stabilize. The goal of this current project is to look at a lot of patients that have a wide range of dystrophin protein levels and determine that protein level with high accuracy. To gather this information, we will use the already established resources of patient muscle biopsies, so no new patient enrollment is needed. Next, we will compare the dystrophin level with the patient well-being to determine a relationship between dystrophin protein amount and the patient s overall health. This study will help reveal the amount of new dystrophin needed to be made by these drugs to help patients. The relationship between patient s health and dystrophin protein level will be submitted to the FDA for review in their public Biomarker Qualification Program. Through this interaction with the FDA, we will make these data available to all researchers and patients to help all future drug programs that aim to treat Duchenne patients by restoring dystrophin protein. FDA Biomarker Qualification of dystrophin protein would aid clinical trials by reducing the length, and therefore costs, of the trials. Shorter time for a clinical trial means a reduced burden to the patient by having fewer visits and faster transition of the drug through the preliminary stages of approval. Biomarker Qualification is a Department of Defense Fiscal Year 2015 Duchenne Muscular Dystrophy Research Program Focus Area. The qualified biomarker would be publically available to all researchers and companies to share and use. The overall goal is to help Duchenne patients and families by streamlining the clinical trial process for new drugs.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Jan 31, 2017
- Source ID
- W81XWH1610572
Entities
People
- Kristy Brown
Organizations
- United States Army