Enrollment Open

Astellas

Study Population: Pediatric male subjects between 8 and 16 years of age

Diagnosis: Duchenne Muscular Dystrophy (DMD)

The purpose of this research is to find out if study drug ASP0367 (also called as MA-0211) is effective and safe as a treatment for boys with Duchenne Muscular Dystrophy (DMD) when compared to placebo (a pill that looks like the study drug but has no active ingredients). Researchers will look at how study drug ASP0367, an investigational drug, can act in the body

Contact Information
Alina Nicorici
916-734-0968
arnicorici@ucdavis.edu 

Capricor OLE

Study PopulationAnyone previously enrolled in the original Capricor study

Diagnosis: Duchenne Muscular Dystrophy (DMD)

The main purpose of this study is to provide study drug CAP-1002 to subjects who completed the Capricor original study. It is also to evaluate the safety and effectiveness of repeat intravenous (IV) infusions of CAP-1002 in patients with Duchenne Muscular Dystrophy (DMD). 

Contact Information
Colleen Anthonisen
916-734-4307
canthonisen@ucdavis.edu 

DoD Biomarker

Study Population: Males including infants, toddlers, and young children

Diagnosis: Duchenne Muscular Dystrophy (DMD)

The purpose of this study is to identify proteins in blood that can be used to measure the severity and progression of DMD. This may help to determine whether early treatments can benefit young children with DMD. Enrolled patients will have blood drawn once a year until the age of 9.

Contact Information
Rachel Baek
916-734-2439
jrbaek@ucdavis.edu

DoD Stepwatch

Study Population: Boys with and without Duchenne Muscular Dystrophy between the ages of 2 and 16 years old

Diagnosis: Duchenne Muscular Dystrophy (DMD)

This research study focuses on validating the use of patient generated step and activity data to facilitate future data collection to support the development of stride rate patterns as an outcome measure for ambulatory patients with DMD. Participants will be assessed using step monitoring devices and traditionally performed clinical outcome measures, followed by community monitoring. The data collected will identify consumer-level devices that are capable of gathering accurate continuous data on patient stride activity and mobility.

Contact Information
Poonam Prasad
916-734-5057
psprasad@ucdavis.edu

Fibrogen

Study Population: Non-ambulatory males ages 12 & up

Diagnosis: Duchenne Muscular Dystrophy (DMD)

The purpose of this research is to evaluate the safety and potential efficacy of investigational drug, Pamrevlumab, with systemic corticosteroids in non-ambulatory DMD patients, compared to placebo with systemic corticosteroids. This trial is expected to enroll 90 subjects and will receive up to 27 infusions over the course of the study.

Contact Information
Alina Nicorici
916-734-0968
arnicorici@ucdavis.edu

Italfarmaco Extension

Study Population: Patients who previously participated in Italfarmaco

Diagnosis: Duchenne Muscular Dystrophy (DMD)

The purpose of this research is to evaluate the long-term safety and efficacy of Givinostat in patients with Duchenne Muscular Dystrophy who have already participated in a study with Givinostat. This study will last approximately 7 years, until Givinostat receives marketing authorization and is available on the market or until the Sponsor provides different instructions due to safety and/or efficacy issues. 

Contact Information
Omaid Sarwary
916-734-3993
omsarwary@ucdavis.edu

See more information on ClinicalTrials.gov

PTC-016

Study Population: Patients who have previous exposure to Ataluren

Diagnosis: Duchenne Muscular Dystrophy (DMD)

This study evaluates the effects of Ataluren, an orally delivered drug aimed to overcome the effects of a nonsense mutation in the dystrophin gene seen in 10-15% of patients with DMD. Enrolled patients will receive Ataluren at a dose of 10mg/kg, 10mg/kg, and 20mg/kg every 12 weeks.

Contact Information
Poonam Prasad
916-734-5057
psprasad@ucdavis.edu

See more information on ClinicalTrials.gov

Sarepta 103

Study Population: DMD ambulatory subjects who are ≥ 4 to < 8 years of age

Diagnosis: Duchenne Muscular Dystophy (DMD)

This is an open-label study of systemic gene delivery of SRP-9001. SRP-9001 is a gene therapy designed to treat the underlying biological cause of DMD by replacing dysfunctional or missing dystrophin protein with a functional truncated dystrophin, called micro-dystrophin, in cardiac and skeletal muscle. Thus, micro-dystrophin would address the root cause of DMD, alter the course of the disease, and address a significant unmet medical need.

Contact Information
Omaid Sarwary 
916-734-3993

Sarepta Prospective Chart Review

Study Population: Males currently on Exondys 51

Diagnosis: Duchenne Muscular Dystrophy (DMD)

This is a prospective observational study of patients receiving Eteplirsen treatment in routine clinical practice. The objective of this observational study is to collect long-term data on DMD patients receiving Eteplirsen in routine clinical practice. No study medication will be provided by the Sponsor as part of this observational study. A 5-year follow-up period will gather additional data regarding exposure to Eteplirsen and the clinical course of DMD patients receiving Eteplirsen.

Contact Information
Rachel Baek
916-734-2439
jrbaek@ucdavis.edu

Vamorolone Expanded Access Program

Study Population: Males previously enrolled in a Vamorolone trial

Diagnosis: Duchenne Muscular Dystrophy (DMD)

This program will enable children who enrolled were previously in Vamorolone trials to have continued access to Vamorolone therapy. Subjects will receive standard of care treatment and procedures for management of DMD.

Contact Information
Colleen Anthonisen
916-734-4307
canthonisen@ucdavis.edu 

See more information on ClinicalTrials.gov

Enrollment Closed

Italfarmaco

 

Study Population: Males 6 years and older

Diagnosis: Duchenne Muscular Dystrophy (DMD)

Givinostat is an oral medication that inhibits enzymes called HDAC’s and has potential anti-inflammatory and anti-tumor effects. This study will assess the effects of Givinostat on patients and its impact on quality of life. Enrolled patients will undergo a treatment period of 18 months.

Contact Information
Poonam Prasad
916-734-0968
psprasad@ucdavis.edu

See more information on ClinicalTrials.gov

NS Pharma Extension

Study Population: Males between the ages of 4 and 10 years old

Diagnosis: Duchenne Muscular Dystrophy (DMD)

NS-065/NCNP-01 is a medication that works to restore the RNA reading frame on the dystrophin gene in patients with Duchenne muscular dystrophy. This study will assess the safety and tolerability of low (40 mg/kg) and high (80 mg/kg) doses for participants that have completed the original study. Enrolled patients will receive weekly IV infusions for up to 144 weeks.

Contact Information
Omaid Sarwary
916-734-3993
omsarwary@ucdavis.edu

See more information on ClinicalTrials.gov

PTC-041

 

Study Population: Males 5 years and older

Diagnosis: Duchenne Muscular Dystrophy (DMD)

This study assesses the long-term effects of Ataluren on disease progression. This is a 148-week study where enrolled patients will be measured primarily through the effect of Ataluren on ambulation and endurance as assessed by the 6-minute walk test. Subjects enrolled will be on a double-blind treatment period for the first 72 weeks. After the 72 week period, all subjects will be given Ataluren for the remainder of the trial.

Contact Information
Poonam Prasad
916-734-5057
psprasad@ucdavis.edu

See more information on ClinicalTrials.gov

Santhera

 

Study Population: Males 10 years and older

Diagnosis: Duchenne Muscular Dystrophy (DMD)

This study will assess the efficacy of Idebenone compared to placebo in delaying the loss of respiratory function in patients diagnosed with Duchenne muscular dystrophy. Enrolled patients will take two tablets orally, three times a day with meals for a treatment period of 78 weeks.

Contact Information
Alina Nicorici
916-734-0968
arnicorici@ucdavis.edu 

See more information on ClinicalTrials.gov

Santhera Extension

 

Study Population: Patients who previously participated in Santhera

Diagnosis: Duchenne Muscular Dystrophy (DMD)

Santhera Pharmaceuticals is testing the long-term safety and efficacy of the drug Idebenone in DMD patients that have completed the previous Santhera original study. In this trial, a series of physical tests and laboratory parameters will be assessed to evaluate the safety and efficacy profile of Idebenone in DMD patients. Any side effects will be assessed by a physical examination, including tests on the heart and by laboratory tests on blood and urine.

Contact Information
Alina Nicorici
916-734-0968
arnicorici@ucdavis.edu 

See more information on ClinicalTrials.gov

Vamorolone 004

 

Study Population: Ambulant males between the ages of 4 and 7 years old

Diagnosis: Duchenne Muscular Dystrophy (DMD)

The purpose of this clinical trial is to see if an investigational drug called Vamorolone is effective in improving or stabilizing muscle strength and function. This is a randomized, double-blind, parallel group, placebo, and active controlled study. The primary objective of this Phase II trial is to compare and evaluate the efficacy of Vamorolone administered orally at daily doses of 2.0 mg and 6.0 mg.

Contact Information
Rachel Baek
916-734-2439
jrbaek@ucdavis.edu

See more information on ClinicalTrials.gov

Enrollment Open

EPM-01-101

Study Population: Males between 16 through 60 years of age with Becker or Becker-like Muscular Dystrophy

DiagnosisBecker Muscular Dystrophy (BMD) or a milder phenotype of Duchenne Muscular Dystrophy (DMD)

This study aims to evaluate the safety and possible effectiveness of (+)-epicatechin, the study drug, for the treatment of Becker or Becker-like muscular dystrophy. Previously, the (-)-epicatechin, similar to (+)-epicatechin, was shown to increase the levels of certain protiens that may benefit people with Becker or Becker-like muscular dystrophy. The primary objective of this study is to determine a safe and potentially effective dose in future studies. 

Contact Information
Rachel Baek
916-734-2439
jrbaek@ucdavis.edu

See more information on ClinicalTrials.gov

Enrollment Closed

No current trials are closed at this time. 

Enrollment Open

No current trials are open at this time, but please check back at a later date. 

Enrollment Closed

Acceleron Extension

Study Population: Patients who previously participated in the Acceleron A083-02 trial

Diagnosis: Facioscapulohumeral Muscular Dystrophy (FSHD)

The primary objective of this clinical trial is to evaluate the long term effective of safety and tolerability of ACE-083 in patients with FSHD. Additional objectives include to evaluate the changes in muscle volume, motor function, and quality of life during the treatment phase. The study drug is a recombinant fusion protein consisting of a modified form of human follistatin and is administered through an injection.

Contact Information
Colleen Anthonisen
916-734-4307
canthonisen@ucdavis.edu 

See more information on ClinicalTrials.gov

Enrollment Open

AveXis

Study Population: Patients with genetically confirmed SMA on or after 24 May 2018

Diagnosis: Spinal Muscular Atrophy (SMA)

This study collects relevant data from regular doctor’s appointments of enrolled patients every six months, as well as collecting biannual questionnaires that will help AveXis determine if the treatments for SMA will change the quality of life. The data will be collected from medical records for a period of fifteen years.

Contact Information
Rachel Baek
916-734-2439
jrbaek@ucdavis.edu

See more information on ClinicalTrials.gov

Enrollment Open

No current trials are open at this time, but please check back at a later date. 

Enrolling Soon

Radicava

Target Population: Participants who have Sporadic or Familial Amyotrophic Lateral Sclerosis (ALS) who are at least 18 years old

Diagnosis: Sporadic or Familial Amyotrophic Lateral Sclerosis (ALS)

This research study is designed to investigate a select panel of biomarkers in patients with ALS, treated with Edaravone. Biomarkers of oxidative stress, inflammation and neurodegeneration will be explored. The primary objective of this study is to identify putative biomarker(s) to serve as quantifiable biological non-clinical measure(s) of Edaravone’s pharmacodynamic effect in ALS. The secondary objective is to evaluate the effectiveness of Edaravone in participants with ALS, in real-world settings. The intervention is limited to the collection of blood and urine samples for biomarker testing.

Contact Information
Erica Goude
916-734-0384
ggfortugno@ucdavis.edu

See more information on ClinicalTrials.gov

Enrollment Closed

Cytokinetics Expanded Access

Target Population: Patients who previously participated in Cytokinectics studies

Diagnosis: Amyotrophic Lateral Sclerosis (ALS)

This clinical study is a multi-national, double-blind, randomized, placebo-controlled, clinical study of drug called Tirasemtiv in patients with ALS. Following a two week open label phase of this study, patients will be randomized in placed in one of the three different target dose levels of Tirasemtiv. The primary objective of this study is to make Tirasemtic available to a limited group of patients who suffer from ALS and those who participated in previous Cytokinetic studies.

Contact Information
Alina Nicorici
916-734-0968
arnicorici@ucdavis.edu

Enrollment Open

ESTEEM

Study Population: Patients between the ages of 30 and 75 years old

Diagnosis: Chronic Kidney Disease (CKD) and those without CKD

The purpose of this study is to conduct a randomized controlled trial to test the impact of combined aerobic and resistance exercise training on skeletal muscle dysfunction in CKD. A nested cross-sectional investigation will compare the skeletal muscle function of patients with kidney disease to controls without kidney disease and to patients with muscular dystrophinopathies without kidney disease.

Contact Information
Colleen Anthonisen
916-734-4307
canthonisen@ucdavis.edu