Open Research Studies

Below is a list and brief description of ongoing CINRG research studies that are enrolling. This list is updated at frequent intervals. Additional information is available at Clinical Trials.gov, by sending an email to info@cinrgresearch.org and requesting information on current CINRG studies.

Robert Leshner, MD) We are currently performing a one-day study that is looking at the quality of life, biochemical changes (changes in the blood), and strength of patients with different types of limb girdle muscular dystrophies and Becker muscular dystrophy.

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 a diagnosis of Limb girdle muscular dystrophy, type 2a (LGMD2A)
• 11 participants with a diagnosis of Limb girdle muscular dystrophy, type 2a (LGMD2B)
• 11 participants with a diagnosis of Limb girdle muscular dystrophy, type 2a (LGMD2I)
• 11 participants with a diagnosis of Becker muscular dystrophy (BMD)
• 11 controls (no neuromuscular disorder)

Status: Actively enrolling.

Participating CINRG Sites:

1. Children's National Medical Center located in Washington, DC (Principal Investigator: Carolina Tesi-Rocha, MD)
2. 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

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We are currently performing a two-year clinical trial that is looking to see if people with certain types of muscular dystrophy who are given Coenzyme Q10 (a nutritional supplement) or lisinopril (a drug) have less heart problems compared to people who receive enhanced standard care with being given a drug or nutritional supplement.

Title: Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies (PITT0908)

Study Details: The purpose of the research study is to test an angiotensin converting enzyme (ACE) inhibitor, lisinopril, and an anti-oxidant, coenzyme Q10 (CoQ10), to ameliorate the decline in cardiac muscle function that occurs in muscular dystrophies.

The research study will include 120 participants aged 8 and up years old with Duchenne, Becker, or autosomal recessive limb-girdle (specifically: LGMD 2C-2F and 2I) muscular dystrophies. Participants will be seen at a participating CINRG center. This will be a 24-month, prospective, randomized study comparing a lisinopril arm, a CoQ10 arm (serum level of ≥ 2.5 µg/mL) and a combination arm with an enhanced standard of care arm.

Inclusion Criteria and Participants: 120 participants will be enrolled in this study. Participants must be over the age of 8 and must have a confirmed diagnosis of Duchenne, Becker, or autosomal recessive limb-girdle muscular dystrophies. Participants already taking an ACE inhibitor or CoQ10 are not eligible to participate.

Status: Enrollment pending regulatory approvals.

Participating CINRG Sites:

1. University of Pittsburgh / Children's Hospital of Pittsburgh of UPMC, Pittsburgh, PA (Principal Investigator: Paula R. Clemens, MD)
2. Children's National Medical Center, Washington, DC (Principal Investigator: Carolina Tesi-Rocha, MD)
3. University of Tennessee, Memphis, TN (Principal Investigator: Tulio Bertorini, MD ) Pending regulatory approval
4. University of Puerto Rico, San Juan, Puerto Rico (Principal Investigator: Jose Carlo, MD) Pending regulatory approval
5. Hadassah, Hebrew University Hospital, Jerusalem, Israel (Principal Investigator: Yoram Nevo, MD) Pending regulatory approval
6. Washington University, St. Louis, MO (Principal Investigators: Ann Connolly, MD and Alan Pestronk, MD) Pending regulatory approval
7. Unidad de Enfermedades Neuromusculares, Argentina (Principal Investigator: ) Pending regulatory approval
8. Children's Hospital of Westmead, Sydney, Australia (Principal Investigator: Andrew Kornberg, MD ) Pending regulatory approval
9. University of Minnesota Minneapolis, MN (Principal Investigator: John Day, MD ) Pending regulatory approval
10. Alberta Children's Hospital, Calgary, Canada (Principal Investigator: Jean Mah, MD ) Pending regulatory approval
11. National Center of Neurology and Psychiatry , Tokyo, Japan (Principal Investigator: Shin'Ichi Takeda, MD ) Pending regulatory approval
12. Children's Memorial Hospital of Northwestern University, Chicago, IL (Principal Investigator: Nancy Kuntz, MD ) Pending regulatory approval
13. Other CINRG sites may be added to this list

Questions: Please contact Lauren Hache at 412-383-7207 / hachel@upmc.edu

Sponsorship: This study is sponsored by the Department of Defense (DoD).