Research Studies in Follow-Up
Below is a list and brief description of ongoing CINRG research studies that are in follow-up. This list is updated at frequent intervals. Additional information is available at Clinical Trials.gov or by sending an email to info@cinrgresearch.org and requesting information on current CINRG studies.
Title: Cardiac Outcome Measures in Children with Muscular Dystrophy (PITT1109)
Study Details: The research study will include 50 participants aged 8 to 18 years old (before 18th birthday) with Duchenne, Becker, or autosomal recessive limb-girdle (specifically: LGMD 2C-2F and 2I) muscular dystrophies. Participants will be seen in one of five Cooperative International Neuromuscular Research Group (CINRG) centers located in the United States. All study assessments will be completed in the same day and include reviewing past medical and surgical history, collecting vital signs, collecting cardiac measures through echocardiographic tests. This study may help lead to a better understanding of cardiac measurements in children with muscular dystrophy.
Inclusion Criteria and Participants: Participants must be between the ages of 8 and 18 years old and must have a confirmed diagnosis of Duchenne, Becker, or autosomal recessive limb-girdle muscular dystrophies.
Status: In analysis
Participating CINRG Sites:
- University of Pittsburgh / Children's Hospital of Pittsburgh of UPMC located in Pittsburgh, PA (Principal Investigator: Paula R. Clemens, MD)
- Children's National Medical Center located in Washington, DC (Principal Investigator: Christopher Spurney, MD)
- Washington University located in St. Louis, MO (Principal Investigator: Anne Connolly, MD)
- Texas Children's Hospital located in Houston, TX (Principal Investigator: Timothy Lotze, MD)
- University of California, Davis located in Sacramento, CA (Principal Investigator: Craig McDonald, MD)
Questions: Please contact Lauren Hache at 206-451-4496 (LHache@childrensnational.org )
Sponsorship: NIH through CTSA program.
This study is listed at clinicaltrials.gov: http://clinicaltrials.gov/ct2/show/NCT01066455?term=cardiac+outcome+measures+in&rank=1
Title: Comparative Study of Clinical Endpoint in DMD: HHM vs. CQMS (CNMC0609)
Study Details: With increases in the numbers of experimental therapies and clinical trials in Duchenne Muscular Dystrophy (DMD), it is important to define reliable and sensitive endpoints that fulfill FDA requirements for relevance to quality of life. The aim of the proposed research is to compare two commonly used pediatric strength testing measures: handheld myometry (HHM) and CINRG Quantitative Measurement System (CQMS), with the goal of identifying a sensitive and valid tool for measuring muscle strength in children with DMD. The data obtained from this study will be used to make recommendations for strength measurement endpoints in prospective muscular dystrophy trials and provide more reliable and accurate recommendations in the clinic for strength assessment.
Inclusion Criteria and Participants: Thirty participants, between the ages of 5 and 18, with a diagnosis of Duchenne Muscular Dystrophy (DMD) will be enrolled into this study. Participants must be able to transfer to and from a wheel-chair with assistance and not had any surgeries or muscle injuries in the previous 8 weeks.
Status: In analysis.
Participating CINRG Sites:
- Children's National Medical Center located in Washington, DC (Principal Investigator: s Duong, MPT)
- Carolina's Medical Center located in Charlotte, NC (Principal Investigator: Mohammed Sanjak, PhD, PT)
- Royal Children's Hospital located in Melbourne, Australia (Principal Investigator: Kate Carroll, PhD, PT)
- Centro Clinico Nemo located in Milano, Italy (Principal Investigator: Ksenija Gorni, MD)
- Washington University located in St. Louis, MO (Principal Investigator: Julaine Florence, DPT)
Questions: Please contact Tina Duong at 202-476-6065 / TDuong@childrensnational.org
Sponsorship: This study is sponsored by the Muscular Dystrophy Association (MDA) Crystal Ball Fund.
This study is listed at clinicaltrials.gov: http://clinicaltrials.gov/ct2/show/NCT01125709?term=duchenne&rank=32
Title: National Initiative for Families with Duchenne (NIFD)
Study Details: The purpose of this research study is collected a large-scale needs assessment of the DMD community that is offered to families and physicians. The survey provides information about medical care that families with DMD received and difficulties the families face in getting the services that they need.
Inclusion Criteria and Participants: A total of 237 families have participated, either through the CINRG natural history study or through the NIFD study directly.
Status: Enrollment closed. Active data analyses are underway.
Participating CINRG Sites: n/a as survey information was gathered from participants across the country.
Questions: For questions about this research study please contact Adrienne Arrieta at 202-476-6497 / AArrieta@childrensnational.org
Sponsorship: The National Initiative for Families with DMD (NIFD) study is a questionnaire instrument developed by researchers from CINRG and Columbia University in New York through funding from the Centers for Disease Control and Prevention (CDC).
Title: Evaluation of Limb Girdle Muscular Dystrophy
Study Details: The purpose of this study is to understand the biochemistry of different types of LGMD and collect information about differences in the pathophysiology (causes) of different muscular dystrophies and the effects of the dystrophy process on your quality of life. The information collected will help us understand the natural progression of the disease and can guide future therapeutic trials.
Inclusion Criteria and Participants:
A total of 55 participants will be enrolled. 11 participants with each of the following diagnoses were enrolled: Limb girdle muscular dystrophy, type 2a (LGMD2A), Limb girdle muscular dystrophy, type 2a (LGMD2B), Limb girdle muscular dystrophy, type 2a (LGMD2I), Becker muscular dystrophy (BMD), and 11 controls (no neuromuscular disorder)
Status: In analysis.
Participating CINRG Sites:
- Children's National Medical Center located in Washington, DC (Principal Investigator: Carolina Tesi-Rocha, MD)
- Carolinas Medical Center located in Charlotte, NC (Principal Investigator: Susan Sparks, MD)
Questions: Please contact Priscilla Russo at 704-446-1910 / Priscilla.Russo@carolinashealthcare.org
Sponsorship: This study is sponsored by the Muscular Dystrophy Association (MDA) Crystal Ball Fund and Parsons family, and the Department of Health and Human Services (National Institutes of Health (NIH)).
This study is listed at clinicaltrials.gov: http://clinicaltrials.gov/ct2/show/NCT00893334?term=evaluation+of+limb+girdle+muscular+dystrophy&rank=1
