Longitudinal Study of the Natural History of Duchenne Muscular Dystrophy
Questions and Answers
What is the CINRG Duchenne Natural History Study?
The CINRG Duchenne Natural History Study (DNHS) is the most comprehensive study of Duchenne clinical outcomes and disease progression to date. Over 400 boys and men across the world with Duchenne participated in the study, providing information about their strength and mobility, heart and lung function, clinical care, behavior, community participation and quality of life, making it the largest Duchenne natural history study ever. The information gathered through this study has shaped the way researchers and healthcare providers view the natural course of disease and serves as a guide for current and future research. So far, over 20 research papers have been published using the data collected during this study. These papers have analyzed and discussed topics related to the quality of care for boys with Duchenne and processes that affect the severity of the disease. Data collected from this study serves as a control group for several ongoing clinical research trials by several different sponsors, and summaries from this study have been included in Duchenne drug approval packages submitted to the Food and Drug Administration and other regulatory bodies. The quality and variety of information collected through the CINRG DNHS has proved essential to expanding our knowledge about this disease and improving the quality of life for boys with Duchenne.
What stage is this research?
The original study ran from 2005 to 2016, and has now concluded. Given the importance of natural history data, we are now working with a number of industry sponsors to start an expanded Duchenne natural history study (eDNHS).
New enrollment will start in 2019. The new eDNHS study plans to follow 500 participants with Duchenne of all ages in an effort to collect current and ongoing data that can be used in Duchenne research. Participants who were involved with the original CINRG DNHS are welcome and encouraged to participate once enrollment opens.
The eDNHS study we will re-open the enrollment to all Duchenne participants of all ages (from birth and on).
What is the goal or purpose of this study?
The purpose of this study is to maintain the largest long-term assessment of people with Duchenne muscular dystrophy. In this study, the investigators associated with the Cooperative International Neuromuscular Research Group (CINRG) will take a detailed look at Duchenne participant’s physical abilities, the medical problems they experience, and how they use health care services. Physical abilities will be compared to a group of healthy controls.
This study is also helping determine whether certain biomarkers are present in people with Duchenne and not in healthy controls.
Who is sponsoring this study?
The original DNHS study was sponsored by the U.S. Department of Education, National Institutes of Health (NIH), the Department of Defense, and Parent Project Muscular Dystrophy.
Industry sponsors will be announced upon launch of the new eDHNS study.
Who enrolled in this study?
Duchenne participants with a confirmed diagnosis by genetic test and/or muscle biopsy.
Where does this study take place?
The original DNHS study enrolled participants in over 20 participating CINRG centers. The new eDNHS study will be offered at participating CINRG and CINRG-affiliated sites around the world. More site and study updates are available so be sure to check www.clinicaltrials.gov or the website for the CINRG group: www.cinrgresearch.org for additional information.
Where can I learn more about this study?
You can learn more about this study at www.cinrgresearch.org and www.clinicaltrials.gov (NCT00468832 for the original DNHS).