Children's National Medical Center Human Clinical Core (CINRG)

Center Institution(s)

Research Center for Genetic Medicine
Children's National Medical Center

Title of core facility or other resource

Human Clinical Core (CINRG)

Contact person, email address, phone number

Diana Escolar, MD
descolar@cnmcresearch.org
202-884-6011

Currently available or expected date of availability

Currently available.

Requirements for use (by collaboration or fee-for-service, application required, etc.)

Datasets concerning outcome measures, reliability of these measures, and data from published trials are available with no fee or application.

Assistance with clinical trial design and statistical analyses of clinical trial data can be done as collaboration, or as a fee-for-service, depending on the level of assistance required. The CINRG clinical trial network can also be used to run clinical trials on a collaborative basis. Given the cost and resources required for clinical trials, negotiation with the CINRG management team is required. The interested party should submit a short application with information and rationale for the selection of the proposed treatment to be tested.

Access to frozen muscle tissue or genomic DNA samples is by collaboration.

Brief description of services or resources (a paragraph or two)

The Cooperative International Neuromuscular Research Group (CINRG) is a collaborative network of neuromuscular clinic sites designed to conduct clinical trials and natural history studies in neuromuscular disease. A description of CINRG can be found on their web site (http://www.cinrgresearch.org), including a list of the CINRG sites (24 sites in 9 countries). The Coordinating Center for CINRG is in the Research Center for Genetic Medicine, Children's National Medical Center, although the principal investigators for specific clinical trials can be at other sites.

A. Extensive experience in the design of clinical trials for Duchenne muscular dystrophy and other pediatric and adult neuromuscular disorders

B. Sensitive and reliable outcome measures designed and extensively tested in the pediatric and adult neuromuscular patient populations.

C. Regulatory personnel with experience in pediatric and adult clinical trials.

D. Clinical evaluator trainers and protocols to maintain multi-site outcome reliability.

E. Clinical trial data management, including CRF design, data monitoring, statistical analysis.

F. Extensive natural history data for Duchenne muscular dystrophy.

G. Frozen muscle biopsy tissue bank of 5,000 neuromuscular patients.

H. Gridded genomic DNA for 1,000 muscular dystrophy patients, with most having tested negative for most known genes.

J. Genetic counselor for central diagnostic confirmation for trials.

Services include:

A. Consultation on outcome measures for clinical trials in neuromuscular disease.

B. Consultation on design and conduct of clinical trials.

C. Statistical consultation for interpretation of data from human clinical trials.

D. Molecular diagnostics on muscle biopsies and DNA.