D-RSC Database
The Duchenne Regulatory Science Consortium (D-RSC) Database is open to non-consortium members if approved by a data use committee consisting of neutral members of the consortium.
D-RSC Database
The Duchenne Regulatory Science Consortium (D-RSC) Database is open to non-consortium members if approved by a data use committee consisting of neutral members of the consortium. By sharing this resource, D-RSC aims to extend and amplify the availability of data to accelerate drug development for Duchenne muscular dystrophy. The database includes data from DMD clinical trials, natural history studies, and clinical data collections.
Data content:
The D-RSC Dataset contains the following clinical trials and natural history studies and reflects information the data contributor provided:
| Contributor | Study | Type of Data | Number of Participants | Age Range | Time | Types of Variables |
| Eli Lilly | LY450190 (Tadalafil In DMD) | Placebo arm of clinical trial | 116 | 7-14 years | up to 395 days | Functional measures, Respiratory measures, Cardiac measures |
| Summit Therapeutics | Phaseout DMD | Clinical Trial Data | 39 | 5-10 years | 52-56 weeks | Functional Measures, Respiratory Measures, MRI/MRS, Northstar, PODCI |
| Children’s Hospital of Philadelphia (CHOP) | Clinical Study – Pulmonary Function | Clinical study | 66 | 13-33 years | up to 3 years | Respiratory measures |
| Imaging DMD | Imaging DMD Natural History Study | Natural history study | 93 | 5-18 years | up to 7 years | Functional measures, Myometry |
| UC Davis | DMD Natural History | Natural history study | 72 | 2-31 years | up to 10 years | Functional measures, Respiratory measures, Myometry |
| UC Davis | Test/re-test data for Clinical Outcome Assessment (COA) | COA | 60 | 4-14 years | 1 year | Functional measures, Respiratory measures |
| Parent Project Muscular Dystrophy | Duchenne Connect | Patient Reported Registry | 3921 | Reports 1-115 years | none | Questionnaire |
| Leiden University Medical Center | Longitudinal Proteomic Study | Biomarker Study | 14 | 5-18 years | up to 5 years | FVC, Drug Effects, Protein Biomarkers |
| Pfizer | Domagrozumab (PF-06252616) Study | Placebo arm of clinical trial | 40 | 6–15 years | 49 weeks | Functional Measures, Respiratory Measures, MRI/MRS, Northstar, PUL, Muscle Strength |
Data standardization:
All D-RSC data have been mapped to current Clinical Data Interchange Standards Consortium (CDISC) Standard Data Tabulation Model (SDTM) structures – https://www.cdisc.org/standards/therapeutic-areas/duchenne-muscular-dystrophy. Data is fully de-identified and compliant to the terms and conditions specified by the data owners. Within the public-accessible database, datasets are integrated, so researchers will not be able to identify participants of the studies or the specific trial from which individual participant observations originate.
What is not in the database:
- Active treatment comparator data
Data applicability:
- Enhanced understanding and modeling of disease progression
- Advancement of drug development for DMD
- Development of tools to simulate clinical trials for DMD
- Constructing tools for placebo-arm best-practice modeling within DMD clinical trials
Requirements for accessibility:
- Knowledge of SDTM is required for an effective use of the data. Information and training on SDTM is available to approved researchers through the CDISC website.
- The use of the data is restricted to the approved applicant.
- All research requests will be reviewed by the D-RSC data use committee. Please allow 4-6 weeks for processing.
For access to the new tranche of data from the Duchenne Connect Registry through the Rare Disease Cures Accelerator Data and Analytics Platform (RDCA-DAP), please visit https://portal.rdca.c-path.org/. For more information, and for any questions related to RDCA-DAP access, please email rdcadap@c-path.org and “cc”
d-rscadmin@c-path.org.