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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.

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: Christopher Spurney)
  3. University of Tennessee, Memphis, TN (Principal Investigator: Tulio Bertorini, MD)
  4. Hadassah, Hebrew University Hospital, Jerusalem, Israel (Principal Investigator: Yoram Nevo, MD) Pending regulatory approval
  5. Carolinas Medical Center, Charlotte, NC (Principal Investigator: Susan Sparks, MD)
  6. Alberta Children's Hospital, Calgary, Canada (Principal Investigator: Jean Mah, MD)
  7. National Center of Neurology and Psychiatry , Tokyo, Japan (Principal Investigator: Shin'Ichi Takeda, MD)
  8. Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL (Principal Investigator: Nancy Kuntz, MD)
  9. University of California Davis, Sacramento, CA (Principal Investigator: Craig McDonald, MD)
  10. University of California Los Angeles, Sacramento, CA (Principal Investigator: Perry Shieh, MD)
  11. Other CINRG sites may be added to this list

Questions: Please contact Andrea Smith at 412-383-7207 (smithal7@upmc.edu) or Lauren Hache at 412-224-2030 (LHache@childrensnational.org )

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

This study is listed at clinicaltrials.gov:
http://www.clinicaltrials.gov/ct2/show/NCT01539772?term=becker+muscular+dystrophy&recr=Open&rank=3


We are currently performing a three year study to learn more about the disease progression and quality of life in patients with Becker muscular dystrophy (BMD).

Title: Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping (PITT0112)

Study Details: The purpose of this study is to understand the disease progression and quality of life in patients with BMD. The information collected will help us understand the clinical progression and quality of life that would result from treating individuals with DMD with exon skipping therapies.

Participants will be seen at a participating CINRG center. This study will include four study visits over the course of three years. The first visit will be at baseline, and then study visits will occur at 12, 24, and 36 months.

Inclusion Criteria and Participants:
A total of 80 participants with BMD ages 4 years and older will be enrolled. Participants must have a documented out-of-frame deletion in the dystrophin gene.

Status: Enrolling.

Participating CINRG Sites:

  1. University of Pittsburgh / Children's Hospital of Pittsburgh of UPMC located in Pittsburgh, PA (Principal Investigator: Paula R. Clemens, MD)
  2. Children's National Medical Center, Washington, DC (Principal Investigator: Mathula Thangarajh, MD)
  3. University of Tennessee, Memphis, TN (Principal Investigator: Tulio Bertorini, MD)
  4. Washington University, St. Louis, MO (Principal Investigator: Anne Connolly, MD)
  5. University of Minnesota, Minneapolis, MN (Principal Investigator: Peter Karachunski)
  6. Carolinas Medical Center, Charlotte, NC (Principal Investigator: Susan Sparks, MD)
  7. Alberta Children's Hospital, Calgary, Canada (Principal Investigator: Jean Mah, MD)
  8. Texas Children's Hospital, Houston, TX (Principal Investigator: Timothy Lotze, MD)
  9. Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL (Principal Investigator: Nancy Kuntz, MD)
  10. University of California Davis, Sacramento, CA (Principal Investigator: Craig McDonald, MD)
  11. Duke University, Durham, NC (Principal Investigator: Edward Smith, MD)
  12. Additional CINRG sites may be added to this list

Questions: Please contact Lauren Hache at 412-224-2030 (LHache@childrensnational.org) or Andrea Smith at 412-383-7207 (smithal7@upmc.edu). .

Sponsorship: This study is sponsored by the National Institutes of Health (NIH)

This study is listed at clinicaltrials.gov:
http://www.clinicaltrials.gov/ct2/show/NCT01539772?term=becker+muscular+dystrophy&recr=Open&rank=3


We are collecting blood and skin samples of participants who are diagnosed with Duchenne muscular dystrophy (DMD) and carry a specific changes in the dystrophin gene to create a tissue bank for future research purposes.

Title: Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping (CHAR0312)

Study Details: The purpose of this tissue bank is to collect blood and skin samples from participants who are diagnosed with Duchenne muscular dystrophy (DMD) and carry a specific dystrophin change (deletion) that would work with exon-skipping therapies in patients with DMD. Blood and skin samples will be held in a tissue bank at Carolinas Medical Center for future DMD research.

Inclusion Criteria and Participants: 60 participants will be enrolled in this study. Participants must be over the age of 4 and must have a confirmed diagnosis of DMD with and an eligible dystrophin deletion. Eligible dystrophin deletions are those which would be amenable to skipping of exons 45, 51, or 53. These include:

Mutations eligible for exon 45:
Exon 44
Exon 46
Exon 46-47
Exon 46-48
Exon 46-49
Exon 46-51
Exon 46-53
Exon 46-55
Exon 46-60
Mutations eligible for exon 51 drug:
Exon 13-50
Exon 29-50
Exon 43-50
Exon 45-50
Exon 47-50
Exon 48-50
Exon 49-50
Exon 50
Exon 52
Exon 52-63
Mutation eligible for exon 53 drug:
Exon 10-52
Exon 43-52
Exon 45-52
Exon 47-52
Exon 48-52
Exon 49-52
Exon 50-52

Status: Enrolling.

Participating CINRG Sites:

  1. Carolinas Medical Center, Charlotte, NC (Principal Investigator: Susan Sparks, MD)
  2. University of Pittsburgh / Children's Hospital of Pittsburgh of UPMC, Pittsburgh, PA (Principal Investigator: Paula R. Clemens, MD)
  3. University of Tennessee, Memphis, TN (Principal Investigator: Tulio Bertorini, MD)
  4. Alberta Children's Hospital, Calgary, Canada (Principal Investigator: Jean Mah, MD)
  5. Texas Children's Hospital, Houston, TX (Principal Investigator: Timothy Lotze, MD)
  6. Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL (Principal Investigator: Nancy Kuntz, MD)
  7. University of California Davis, Sacramento, CA (Principal Investigator: Craig McDonald, MD)
  8. Johns Hopkins/Kennedy Krieger, Baltimore, MD (Principal Investigator: Kathryn Wagner, MD)
  9. Additional sites to be added pending regulatory approval

Questions: Please contact Lauren Hache at 412-224-2030 (LHache@childrensnational.org) or Andrea Smith at 412-383-7207 (smithal7@upmc.edu).

Sponsorship: This study is sponsored by the National Institutes of Health (NIH)

This study is listed at clinicaltrials.gov: http://clinicaltrials.gov/ct2/show/NCT01772043?term=dmd+tissue+bank&rank=1

Last Updated: August 18, 2014

 

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