Clinical Research Studies
Study Title and Description
Age
Diagnosis
Status
(Project 1) State-of-the Art Clinical Endpoints versus
Person- Reported Outcomes in Individuals with
Neuromuscular Disease: Reliability, Validity and
Responsiveness to Change.
12-75
years
BMD, FSHD,
LGMD, MMD,
CMT, SMA
II&III, ALS
Open to
enrollment
6-65
years
Patients with
upper limb
and mobility
impairments,
healthy
controls
Open to
enrollment
PI: Craig McDonald, MD
Coordinator: Alina Nicorici, BS
Before clinical trials can begin in subjects with NMDs, the FDA will
require the development of clinically meaningful outcome measures
that clearly show that a potential therapy will represent a “lifealtering event.” Several potential clinically meaningful outcome
measurements have been proposed to test the efficacy of
therapeutic treatments in NMDs. The purpose of this study is to
determine the interrelationship between the commonly-used
outcome measurements for clinical trials of NMDs, and to assess
the sensitivity, validity, and responsiveness of these measures to
change.
Development of wearable motion analysis systems
to measure physical activities and energy
expenditure.
PI: Jay Han, MD
Coordinator: Alina Nicorici, BS
The purpose of this study is to develop accurate non-invasive
monitors to assess physical activities and energy expenditure.
Researchers at the UC Berkeley CITRIS center have developed a
system of non-invasive sensors known as the Cal-FIT system to
monitor people’s activity patterns. This study is designed to
determine whether the Cal-FIT system will accurately detect
whether a person is engaged in common activities such as
sitting, standing, walking and climbing stairs and measure the
amount of energy expended while the person performs these
activities.
Call or email today to participate in our research studies
916-734-0968 or pmr.research@ucdmc.ucdavis.edu
Please refer to the study title and coordinator.
1
04/2013
Clinical Research Studies
ACH0311: A multicenter collaborative study on the clinical
features, expression profiling, and quality of life of
infantile onset facioscapulohumeral muscular dystrophy
>5
years
iFSHD
Open to
enrollment
≥4
DMD
Open to
enrollment
DMD
Soon to
enroll!
PI: Craig McDonald, MD
Coordinator: Erica Goude, BA CCRP
The purpose of this study is to establish a common method to test
muscles in individuals with infantile onset (iFSHD, diagnosed at
<11 years of age). This study will also look at how iFSHD affects
other parts of the body and how it affects quality of life for people
living with this health condition. Finally, this study will explore
biological variations in individuals that may affect their clinical
symptoms.
For more details please visit ClinicalTrials.gov
FOR-DMD: Duchenne muscular dystrophy: doubleblind randomized trial to find optimum steroid
regimen
years
<8
years
PI: Craig McDonald, MD
Coordinator: Erica Goude, BA CCRP
Randomized controlled trial (60 month) will compare 3
corticosteroid regimens to address the hypothesis that daily
corticosteroids (prednisone vs. deflazacort) will be of greater
benefit in terms of function and subject/parent satisfaction than
intermittent corticosteroids (prednisone) in patients diagnosed with
DMD.
For more details please visit ClinicalTrials.gov
A Prospective Natural History Study of the Progression of
Physical Impairment, Activity Limitation and Quality of
Life in Duchenne Muscular Dystrophy
3-18
years
PI: Erik Henricson, MPH
Coordinator: Erica Goude, BA CCRP
The purpose of this study is to look at ways of measuring how
fast different muscles become weaker in children with DMD. This
will provide valuable information for helping to design treatments
with oligonucleotides and with other medications for DMD. There
is no treatment given in this study, it is called a natural history
study. The study will provide information about how to measure
the progression of DMD which will, in turn, help new treatments
to be developed.
For more details please visit ClinicalTrials.gov
Call or email today to participate in our research studies
916-734-0968 or pmr.research@ucdmc.ucdavis.edu
Please refer to the study title and coordinator.
2
04/2013
Clinical Research Studies
PITT0908 - Clinical Trial of Coenzyme Q10 and Lisinopril in
Muscular Dystrophies
<8
years
DMD, BMD,
LGMD2
Open to
enrollment
4-30
years
DMD,
healthy
controls
Open to
Enrollment
>21
years
BMD, SMA,
LGMD, MMD,
FSHD, CMT
PI: Craig McDonald, MD
Coordinator: Erica Goude, BA CCRP
The purpose of this study is to see if people with certain types
of muscular dystrophy who are given Coenzyme Q10 (a
nutritional supplement) or lisinopril (a drug) have fewer heart
problems compared to people who receive enhanced standard
care without being given a drug or nutritional supplement.
For more details please visit ClinicalTrials.gov
UCD0305: A longitudinal study of the relationship between
impairment, activity limitation, participation and quality of
life in persons with confirmed Duchenne muscular
dystrophy (DMD)
PI: Craig McDonald, MD
Coordinator: Michelle Cregan, CCRP (916)734-6304
The purpose of this study is to continue a world-wide, long-term
study of people with Duchenne muscular dystrophy (DMD) that is
now following 340 families at universities and clinics around the
world. Through this study we are evaluating different measures of
strength and function that researchers can use in tests of new
therapies. For this part of our study, we are seeking two new
cohorts, 1) a new group of children diagnosed with DMD aged 4-8,
and 2) a group of typically-developing male participants’ ages 6 to
30 years old without Duchenne muscular dystrophy (“healthy male
controls”). Another purpose of this study is to collect a sample of
blood to evaluate potential biomarkers that are associated with
disease progression and response to steroid therapy.
For more details please visit ClinicalTrials.gov
Ultrasound in ALS/NMD: (M-mode Ultrasound as a measure
of Respiratory Dysfunction in patients with Amyotrophic
Lateral Sclerosis and other Neuromuscular diseases (NMD)
PI: Nanette Joyce, DO
Coordinator: Michelle Cregan, CCRP (916)734-6304
The purpose of this study is to learn more about the use of an
ultrasound in identifying diaphragm dysfunction and to predict
respiratory failure in patients diagnosed with ALS and other
neuromuscular diseases. These ultrasound diagnostic results will in
turn be compared to the current use of PFTs as a primary indicator
of respiratory function.
Call or email today to participate in our research studies
916-734-0968 or pmr.research@ucdmc.ucdavis.edu
Please refer to the study title and coordinator.
3
04/2013
Open to
enrollment
Clinical Research Studies
Study of Degenerative Disorders: Skin Biopsy
All ages
DMD
PI: Craig McDonald, MD
Open to
Enrollment
Coordinator: Evan de Bie, BS
In collaboration with Stanley F. Nelson, MD at the University of
California, Los Angeles, the purpose of this study is 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
PITT0112: Becker Muscular Dystrophy - A Natural History
Study to Predict Efficacy of Exon Skipping
>4 years BMD
Soon to
Enroll!
5<
Closed to
enrollment
PI: Erik Henricson, MPH
Coordinator: Erica Goude, BA CCRP
This study is looking at patients with BMD to develop a better
understanding of how certain changes in the dystrophin gene affect
the disease progression, physical development, mental
development, and quality of life in patients with BMD.
For more details please visit ClinicalTrials.gov
An Open-Label, Safety Study for Previously treated ataluren
(PTC124) patients with nonsense Mutation
Dystrophinopathy
DMD/BMD
years
PI: Craig McDonald, MD
Coordinator: Erica Goude, BA CCRP
The main goal of this study is to obtain additional information as
to whether ataluren can safely be given to patients with nonsense
mutation-mediated Duchenne/Becker muscular dystrophy
(DMD/BMD) who have participated in earlier ataluren clinical
trials.
For more details please visit ClinicalTrials.gov
Call or email today to participate in our research studies
916-734-0968 or pmr.research@ucdmc.ucdavis.edu
Please refer to the study title and coordinator.
4
04/2013
Clinical Research Studies
An exploratory study to assess two doses of GSK2402968
in the treatment of ambulant boys with Duchenne muscular
dystrophy
5<
DMD
Closed to
enrollment
DMD
Closed to
enrollment
DMD
Closed to
enrollment
years
PI: Craig McDonald, MD
Coordinator: Erica Goude, BA CCRP
We are inviting you to join this study because your son is known
(from earlier tests) to have or may have a mutation around location
51 that might be treatable with GSK2402968. The purpose of this
study is to test the safety of study drug, GSK2402968 at different
doses, and if it could help to prevent or slow down muscles from
losing their strength. We want to find out what effects, good or
bad, it has on boys with Duchenne Muscular Dystrophy who have
certain dystrophin gene changes around exon-51.
For more details please visit ClinicalTrials.gov
Clinical Outcome Validation in Non-Ambulatory
Boys/Men with Duchenne Muscular Dystrophy (DMD)
PI: Craig McDonald, MD
7<
years
Coordinator: Alina Nicorici, BS
The primary objective of this study is to establish optimal and
reliable clinical assessment in non-ambulatory boys and men
with DMD, to test the quality of life using the Individualized
Neuromuscular Quality of life Questionnaire (INQoL) in adults
with DMD, and to test Caregiver burden in primary caregiver of
non-ambulatory boys and men with DMD, using the revised
Impact on Family (IOF) scale.
Clinical Outcomes Validation in Clinical Outcome for
DMD Infants and Children Ages 1 Month to 5 Years
PI: Craig McDonald, MD
6mth5years
Coordinator: Alina Nicorici, BS
The purpose of this study is to develop outcomes measures of
strength and function in young children with DMD. If validated,
these measures will be used in future clinical trials in young
children with DMD.
Call or email today to participate in our research studies
916-734-0968 or pmr.research@ucdmc.ucdavis.edu
Please refer to the study title and coordinator.
5
04/2013
Clinical Research Studies
A Phase III Double-Blind, Randomized, Placebo
Controlled Study of the Efficacy, Safety and Tolerability
of Idebenone in 10 – 18 Year Old Patients with
Duchenne Muscular Dystrophy
10-18
years
DMD
PI: Craig McDonald, MD
Coordinator: Erica Goude, BA CCRP
In this study, idebenone will be tested in order to determine if
it has any effect on breathing, skeletal muscles or the wellbeing and quality of life of DMD patients as compared to
placebo (does not contain active treatment).
For more details please visit ClinicalTrials.gov
Call or email today to participate in our research studies
916-734-0968 or pmr.research@ucdmc.ucdavis.edu
Please refer to the study title and coordinator.
6
04/2013
Closed to
Enrollment