Parent Project Muscular Dystrophy Clinical Trials Educational Video Library includes several short videos (filmed in 2016) featuring M. Carrie Miceli, PhD and physician, advocate, parent and industry colleagues on the following topics:

  • Benefits of Clinical Trials
  • Managing Expectations
  • Trial Decisions
  • Trial Logistics
  • Natural History Trials
  • Trial Failures

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Status: Recruiting
Conditions
  • Muscular Dystrophy, Duchenne
Interventions
  • Other: Cardiac MRI with contrast
  • Other: Cardiac MRI without contrast
  • Other: Blood Test
  • Other: Heart Rate
  • Other: Pulmonary Function Test
  • Other: Genetic Testing
  • Other: Repeat MRI scan
Locations
  • University of California, Los Angeles (UCLA), Los Angeles, California, United States
  • Children's Hospital of Orange County, Orange, California, United States

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Status: Not yet recruiting
Conditions
  • Duchenne Muscular Dystrophy
Interventions
  • Other: Dystrophin levels
Locations
  • University of California, Los Angeles (UCLA), Los Angeles, California, United States

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Status: Recruiting
Conditions
  • Muscular Dystrophy
Interventions
  • Drug: Tadalafil 20 MG
  • Other: beetroot juice extract
Locations
  • Cedars-Sinai Medical Center, Los Angeles, California, United States

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Status: Enrolling by invitation
Conditions
  • Duchenne Muscular Dystrophy
Interventions
  • Drug: Casimersen
  • Drug: Golodirsen
Locations
  • University of California Los Angeles, Los Angeles, California, United States
  • Ann and Robert H Lurie Childrens Hospital of Chicago, Chicago, Illinois, United States
  • Johns Hopkins University School of Medicine, Baltimore, Maryland, United States
  • Fondazione Policlinico Universitario A Gemelli, Milano, Italy
  • Great Ormond Street Hospital (GOSH), London, United Kingdom
  • Royal Victoria Infirmary, Newcastle Upon Tyne, United Kingdom

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Status: Recruiting
Conditions
  • Duchenne Muscular Dystrophy (DMD)
Interventions
  • Drug: Idebenone 150 mg film-coated tablets
  • Drug: placebo
Locations
  • University of Alabama, Birmingham, Alabama, United States
  • Phoenix Children's Hospital, Phoenix, Arizona, United States
  • Banner University of Arizona Medical Center, Tucson, Arizona, United States
  • Arkansas Children's Hospital, Little Rock, Arkansas, United States
  • Childrens Hospital of Los Angeles, Los Angeles, California, United States
  • David Geffen School of Medicine at UCLA, Los Angeles, California, United States
  • UC Davis Department of Physical Medicine and Rehabilitation, Sacramento, California, United States
  • Loma Linda University Healthcare, San Bernardino, California, United States
  • Shriners Hospitals for Children-Tampa, Tampa, Florida, United States
  • Rare Disease Research, Atlanta, Georgia, United States
  • and 55 more

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Status: Recruiting
Conditions
  • Duchenne Muscular Dystrophy
Interventions
  • Drug: SRP-4045
  • Drug: SRP-4053
  • Drug: Placebo
Locations
  • Neuromuscular Research Center, Phoenix, Arizona, United States
  • Children's Hospital Los Angeles, Los Angeles, California, United States
  • David Geffen School of Medicine, UCLA, Los Angeles, California, United States
  • UC Davis Medical Center, Sacramento, California, United States
  • Rady Children's Hospital San Diego/ UCSD, San Diego, California, United States
  • Stanford University School of Medicine/Medical Center, Stanford, California, United States
  •  
  • Connecticut Children's Medical Center, Hartford, Connecticut, United States
  • University of Florida, Gainesville, Florida, United States
  • NW Florida Clinical Research Group, LLC, Gulf Breeze, Florida, United States
  • Miami, Florida, United States
  • and 45 more

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Status: Not yet recruiting
Conditions
  • Duchenne Muscular Dystrophy
Interventions
  • Drug: Ataluren
Locations
  • Phoenix Childrens Hospital, Phoenix, Arizona, United States
  •  
  • University of California, Los Angeles (UCLA), Los Angeles, California, United States
  • University of California (UC) Davis Medical Center, Sacramento, California, United States
  • Rush University Medical Center, Chicago, Illinois, United States
  • University of Kansas Medical Center, Kansas City, Kansas, United States
  • University of Minnesota, Minneapolis, Minnesota, United States
  • Columbia University College of Physicians & Surgeons, New York, New York, United States
  • Texas Children's Hospital, Houston, Texas, United States
  • University of Texas Heath Science Center at San Antonio, San Antonio, Texas, United States
  • Children's Hospital of the King's Daughters, Norfolk, Virginia, United States

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Status: Enrolling by invitation
Conditions
  • Duchenne Muscular Dystrophy
Interventions
  • Genetic: PF-06939926
Locations
  • Ronald Reagan UCLA Medical Center Drug Information Center, Los Angeles, California, United States
  • UCLA (David Geffen School of Medicine), Los Angeles, California, United States
  • UCLA Mattel Children's Hospital, Los Angeles, California, United States
  • UCLA Medical Center, Los Angeles, California, United States
  • Duke Neurology, Durham, North Carolina, United States
  • Duke University Medical Center, Lenox Baker Children's Hospital, Durham, North Carolina, United States
  • Biospecimen Repository & Processing Core - BPRC, Durham, North Carolina, United States
  • Duke Children's Hospital & Health Center, Durham, North Carolina, United States
  • Duke Early Phase Clinical Research Unit, Durham, North Carolina, United States
  • Duke University Investigational Drug Services, Durham, North Carolina, United States

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Status: Recruiting
Conditions
  • Muscular Dystrophy Duchenne
  • Muscular Dystrophies
  • Muscular Disorders, Atrophic
  • Muscular Diseases
  • Musculoskeletal Disease
  • Neuromuscular Diseases
  • Nervous System Diseases
  • Genetic Diseases, X-Linked
  • Genetic Diseases, Inborn
Interventions
  • Drug: Ataluren
  • Drug: PLACEBO
Locations
  • Phoenix Childrens Hospital, Phoenix, Arizona, United States
  • Children's Hospital of Los Angeles, Los Angeles, California, United States
  • University of California, San Francisco (UCSF) - Benioff Children's Hospital - Oakland, Oakland, California, United States
  • Stanford University Medical Center, Palo Alto, California, United States
  • University of California (UC) Davis Medical Center, Sacramento, California, United States
  • Loma Linda University Children's Hospital, San Bernardino, California, United States
  • Yale New Haven Hospital, New Haven, Connecticut, United States
  • Child Neurology Center of Northwest Florida, Gulf Breeze, Florida, United States
  • Indiana University Health - Riley Child Neurology, Indianapolis, Indiana, United States
  • University of Kansas Medical Center, Kansas City, Kansas, United States

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Status: Recruiting
Conditions
  • Duchenne Muscular Dystrophy
Interventions
  • Drug: RO7239361
  • Drug: Placebo for RO7239361
Locations
  • Neuromuscular Research Center, Phoenix, Arizona, United States
  • Arkansas Children's Hospital; Pediatrics, Little Rock, Arkansas, United States
  • David Geffen School of Medicine at UCLA; Clinical Trials Contract Unit, Los Angeles, California, United States
  • Stanford University, Palo Alto, California, United States
  • University of California Davis Medical Center, Sacramento, California, United States
  • Yale University School of Medicine ; Pulmonary & Critical Care, New Haven, Connecticut, United States
  • University of Florida, Gainesville, Florida, United States
  • Nemours Children's Hospital, Orlando, Florida, United States
  • Rare Disease Research, LLC, Atlanta, Georgia, United States
  • Rush University Medical Center - PPDS, Chicago, Illinois, United States

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Status: Recruiting
Conditions
  • Duchenne Muscular Dystrophy
Interventions
  • Drug: givinostat
  • Drug: placebo
Locations
  • University of California - Davis Medical Center - Devis Physical Medicin & Rehabilitation, Davis, California, United States
  • David Geffen School of Medicine - UCLA Neurology, Los Angeles, California, United States
  • Rady Children's Hospital center - UCSD Department of Neuroscience, San Diego, California, United States
  • Children's Hospital Colorado, Aurora, Colorado, United States
  • Connecticut Children's Medical Center - Division Neurology, Hartford, Connecticut, United States
  • Child Health Research Institute - Department of Pediatrics, Gainesville, Florida, United States
  • Nemours Children's Hospital, Orlando, Florida, United States
  • University of Iowa Children's Hospital, Iowa City, Iowa, United States
  • Wayne State University - University Pediatrics - Children's Hospital of Michigan, Detroit, Michigan, United States
  • University of Minnesota - Department of Neurology, Minneapolis, Minnesota, United States

Learn More

Status: Recruiting
Conditions
  • Muscular Dystrophy, Duchenne
Interventions
  • Drug: SRP-5051
Locations
  • David Geffen School of Medicine at UCLA, Los Angeles, California, United States
  • Neuromuscular Research Center, Sacramento, California, United States
  • University of Florida, Gainesville, Florida, United States
  • NW FL Clinical Research Group, LLC, Gulf Breeze, Florida, United States
  • Rare Disease Research, LLC, Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
  • University of Kansas Medical Center, Kansas City, Kansas, United States
  • Washington University in St. Louis, Saint Louis, Missouri, United States
  • The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, United States
  • Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania, United States
  • Children's Medical Center Dallas, Dallas, Texas, United States •London Health Sciences Centre, London, Ontario, Canada

Learn More

Status: Recruiting
Conditions
  • Muscular Dystrophies
  • Muscular Dystrophy, Duchenne
  • Muscular Disorders, Atrophic
  • Muscular Diseases
  • Neuromuscular Diseases
  • Nervous System Diseases
  • Genetic Diseases, X-Linked
  • Genetic Diseases, Inborn
Interventions
  • Biological: CAP-1002
  • Drug: Placebo
Locations
  • Ronald Reagan UCLA Medical Center, Los Angeles, California, United States
  • University of California, Davis, Sacramento, California, United States
  • Children's Hospital Colorado, Aurora, Colorado, United States
  • Nemours Children's Hospital, Orlando, Florida, United States
  • Rare Disease Research, Atlanta, Georgia, United States
  • University of Iowa Stead Family Children's Hospital, Iowa City, Iowa, United States
  • University of Massachusetts Medical Center, Worcester, Massachusetts, United States
  • Washington University, Saint Louis, Missouri, United States
  • Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States
  • Oregon Health and Science University, Portland, Oregon, United States

 

This patient registry will determin the rate at which children suffer from sudden cardiac death and whether therapy with internal cardiac defibrillators is beneficial. This is an international, multisite research study.

Qualified participants must be at least 10 years old and have Duchenne muscular dystrophy and a weak heart (ejection fraction less than or equal to 35%) or have a heart device (pacemaker, ICD).

There is not cost to participate in the registry and no payment provided. Participation is voluntary.
This study is sponsored by Nationwide Children's Hospital

If you feel you may be eligible, please contact:
Dr Nancy Halnon
Division of Pediatric Cardiology-Mattel Children's Hospital, UCLA
300 Medical Plaza, Suite 300
Los Angeles, CA 90095
(310) 267-7667

If you wish to contact the investigators by email, please be aware that e-mail communications may not be secure. Do not include any sensitive health information if you choose to communicate with the study team by email.

Protocol ID:IRB#14-001101 | UCLA IRB Approved | Approval Date: 9/5/2014 | Through: 9/4/2015 | Committee: Medical IRB 1

Download more information here

The purpose of this study is to improve the understanding of the changes in function over time in boys with Duchenne Muscular Dystrophy (DMD) using objective outcome measures. Parents complete quality of life, behavior and function questionnaires while child participate in evaluations. If you have questions, please contact Eileen Fowler at (310) 825-4028 or efowler@mednet.ucla.edu

Who is appropriate for the study?

  • Diagnosis of DMD
  • Male
  • Four years of age or older
  • Can be on or off corticosteroids
  • Able to walk independently for 10 minutes at self-selected speed
  • Able to understand directions for testing procedures
  • Able to complete up to four hours of testing

 

Click here for more information

Recruitment announcement: Genetic modifiers of Duchenne and Becker Muscular Dystrophy

Take our survey

UCLA researchers within the Center for Duchenne Muscular Dystrophy (CDMD) are seeking individuals with Duchenne Muscular Dystrophy to participate in a DNA research study to identify genes and gene variants that may modify the disease process.

All ages may participate. Of high priority, is the recruitment of boys with DMD who require the full time use of a wheelchair prior to age 8, and boys past age 13 still able to walk independently. Individuals affected with Duchenne or Beckers with early onset cardiomyopathy are also of interest. Other people affected by DMD or BMD may be included at the discretion of the study’s principal investigator (PI). Please contact Dr. Stanley Nelson.

Participation consists of completion of

  • a brief one page questionnaire that is emailed to you, and returned by email
  • blood draw or saliva collected near your home, at UCLA or at your local doctors office
  • a signed consent form

UCLA researchers will isolate and purify DNA from the blood/saliva and store this DNA with a coded identifier. The whole genome (all genes) will be sequenced to search for genetic variants that may make some DMD individuals more or less severely affected. The stored DNA can be used for future analysis, and data will be shared with other researchers. Contact information will be stored so that updates to your disease course can be provided. The confidentiality of identifiable data is assured and only the PI and his proxys will have access to them.

For more information please contact:

Stanley F. Nelson, MD (Principal Investigator)
Center for Duchenne Muscular Dystrophy
Professor of Human Genetics
David Geffen School of Medicine at UCLA
Los Angeles, CA 90095
Phone: (310) 991-2635
Email: snelson@ucla.edu

Emilie Douine, MS
Center for Duchenne Muscular Dystrophy
David Geffen School of Medicine at UCLA
Los Angeles, CA 90095
Phone: (310) 267-2416
Email: edouine@mednet.ucla.edu

Why Genetic modifiers of Duchenne? A brief explanation.

This study seeks to study the effect of genetics on the progression of Duchenne muscular dystrophy. To accomplish this, we are seeking boys and men with DMD who have unusual progression in terms of ambulation or cardiomyopathy.

Even though the dystrophin gene (DMD) responsible for Duchenne and Becker was identified in 1987, we still have lots of questions about the progression of the disease. From observational studies, we know the major milestones of Duchenne occur in a predictable order: trouble walking, at an early age, transition to fulltime wheelchair around ages 9 to 13 and heart or respiratory troubles in later years.

What is surprising to researchers is the wide variability in some of these stages. For instance, the age when a DMD patient begins using a wheelchair full time can vary by more than 10 years! Figure 1 shows this for patients in the DuchenneConnect Registry. This is true even among brothers with Duchenne. Such cases are extreme, but they provide fertile ground for research to find other contributing factors.

How to account for such a disparity? As many studies have shown, corticosteroid use can account for 2-3 years of delay in walking ability (Figure 2). Environment certainly plays a role. But another aspect is the genetic contribution from one or more of the other 20,000 genes in the human genome. It is known that some genes can affect how well another gene is activated or repressed. Others genes may be able to compensate for the missing dystrophin protein while others yet may worsen the onset of the disease.

Every human has genetic variants spread across their genome. Even identical twins will have a few variants that the other doesn’t have! We can capitalize on these individual variants by grouping together, for instance, relatively mildly affected Duchenne individuals. If, as a group, they tend to have lots of variants in particular genes more often than the general population, then we have a clue that this gene may be important. This same idea can be applied to severely affected individuals as well, hopefully giving us an idea of another gene involved in Duchenne.

So how do we define severe or mild? This is a difficult question, but for this project we decided to use age at which Duchenne individuals transition to full time use of a wheelchair. This is also called age at loss of ambulation. We like this measure because it is easily remembered and used by many other researchers.

We also can look at other dimension of Duchenne. For cardiomyopathy, for example, we look at boys with an ejection fraction of less than 55% before the age of 17 (a normal heart's ejection fraction may be between 55% and 70%). This is again, unusual among the general group of patients with DMD.

The shape of the plot for age at loss of ambulation (Figure 1) fits very well with a typical bell shaped curve (Figure 3). This is typical of traits under the control of several genes. By focusing on the more extreme tails (the red regions of Figure 3), we are trying to find a smaller number of genes (maybe just one) that contribute a large amount to the trait of interest, age at loss of ambulation in this case. With smaller the number of genes involved, the easier it is to hone in on promising candidates. Crucially, the success of studies like these relies on recruiting enough participants. The larger the study sample, the more power we have to see what genes contribute greatest to disease progression. Once we identify a gene or genes that look promising, we can look for drugs, therapies or strategies that target that gene of interest.

I hope this gives you some insight into our study. Feel free to ask questions and please enroll and pass along to others who might be interested.

Download more information here

UCLA researchers are seeking individuals with Duchenne Muscular Dystrophy and their family members to participate in a research study funded by the National Institutes of Health (NIH) & California Institute for Regenerative Medicine (CIRM).

We want to biopsy skin and/or draw blood to create permanent cell lines for laboratory studies of cells. We will convert the skin biopsy sample into a stem cell lines and make muscle cells in culture, which will permit researchers to have valuable tools to explore the causes of muscular dystrophy and research new therapies.

All ages may participate. To participate, individuals must complete and return informed consent form, available on request from Dr. Nelson. Individuals may be requested to either have skin biopsy performed to remove 3mm of skin and/or have blood draw performed to provide genomic DNA for gene sequencing. Estimated time of participation is 1 hour, and both procedures can typically be performed locally with shipment of samples to UCLA.

If you are interested, please contact:

Stanley F. Nelson, MD
Center for Duchenne Muscular Dystrophy
Professor of Human Genetics
David Geffen School of Medicine at UCLA
Los Angeles, CA
Phone: 310 991 2635
Email: snelson@ucla.edu

Click here for more information

Download the flyer here

The purpose of this study is to develop sensitive outcome measurements for use in clinical trials and to improve understanding of changes in strength, motor function and lean body mass over time in boys with Duchenne Muscular Dystrophy (DMD). Participants will receive evaluations of strength and motor function and a non-invasive body scan every 6 months for 18 months.

Who is appropriate for the study?

  • Diagnosis of DMD
  • Male
  • Four years of age or older
  • Can be on or off corticosteroids
  • Able to walk with or without assistive devices
  • Able to understand directions for testing procedures
  • Able to lie still for approximately 2 minutes

 

If you are interested, please contact:

Eileen Fowler, PhD
Center for Duchenne Muscular Dystrophy
Professor, UCLA Orthopaedic Surgery
Phone: (310) 825-4028
Email: efowler@mednet.ucla.edu

Click here for more information