Duchenne Muscular Dystrophy

Josh & Kingston

Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (IGNITE DMD)

A Randomized, Controlled, Open-label, Single-ascending Dose, Phase I/II Study to Investigate the Safety and Tolerability, and Efficacy of Intravenous SGT-001 in Male Adolescents and Children With Duchenne Muscular Dystrophy

About: This is a randomized, controlled, open-label, single-ascending dose study to evaluate the safety, tolerability and efficacy of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Eligible patients will be randomized to an active treatment group or an untreated control group. Patients in the active treatment group will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 2 years. Patients in the untreated control group who continue to meet treatment criteria will receive SGT-001 after 1 year on study.

Ages Eligible for Study: 4 Years to 17 Years (Child)

Sexes Eligible for Study: Male

Status:  Actively Recruiting Participants

For more information about this study, including eligibility criteria, please go to the study’s ClinicalTrials.gov page. If you are interested in participating in this study or have questions about research, please contact Stephanie Salabarria, Clinical Research Coordinator.


Sarepta 301: Confirmatory Study of Eteplirsen in DMD Patients

An Open-Label, Multi-Center, 48-Week Study with a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy 

About: The Sarepta-301 study was designed for male participants with Duchenne Muscular Dystrophy who are between the ages of 7-16 that have an out-of-frame deletion that can be corrected by exon 51 skipping through weekly Eteplirsen infusions.

Ages Eligible for Study: 7 Years to 16 Years (Child)

Sexes Eligible for Study: Male

Status: This study is ongoing, but not recruiting participants. Accepting transfers.

For more information about this study, including eligibility criteria, please go to the study’s ClinicalTrials.gov page. If you are interested in participating in this study or have questions about research, please contact Stephanie Salabarria, Clinical Research Coordinator.


ESSENCE 301: Study of SRP-4045 and SRP-4053 in Duchenne Muscular Dystrophy Patients 

A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy

About: The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP- 4053 compared to placebo in Duchenne muscular dystrophy (DMD) patients with out of- frame deletion mutations amenable to skipping exon 45 and exon 53 respectively. Additional objectives include evaluation of safety, pharmacokinetics and biomarkers.

Ages Eligible for Study: 7 Years to 13 Years (Child)

Sexes Eligible for Study: Male

Status:  This study is ongoing, but not recruiting participants. Accepting transfers.

For more information about this study, including eligibility criteria, please go to the study’s ClinicalTrials.gov page. If you are interested in participating in this study or have questions about research, please contact Stephanie Salabarria, Clinical Research Coordinator.


ESSENCE 302: An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy

Long-term, Open-label Extension Study for Patients With Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen

About:The main objective of this study is to evaluate the safety and tolerability of long-term treatment with 30 mg/kg of casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).

Ages Eligible for Study: 7 Years to 21 Years (Child, Adult)

Sexes Eligible for Study: Male

Status:  This study is ongoing, but not recruiting participants. Accepting transfers.

For more information about this study, including eligibility criteria, please go to the study’s ClinicalTrials.gov page. If you are interested in participating in this study or have questions about research, please contact Stephanie Salabarria, Clinical Research Coordinator.


CPI Study: Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy 

About: The purpose of this study is to monitor long term changes in heart function and breathing muscles in children with Duchenne muscular dystrophy and to develop better imaging techniques to evaluate these muscles.

Inclusion criteria is boys with Duchenne who are between the ages of 5-15 years old. Participation includes magnetic resonance imaging (MRI) of the heart and breathing muscles, breathing tests (called pulmonary function tests), exercise tests (using a stationary bike) and possibly an echocardiogram (ultrasound of the heart). This is a four year study with annual visits. Travel reimbursement is available based on the availability of charitable funding and the needs of the subject.

Ages Eligible for Study: 5 Years to 15 Years (Child)

Sexes Eligible for Study: Male   

Status: This study is ongoing, but not recruiting participants.

For more information about this study, including eligibility criteria, please go to the study’s ClinicalTrials.gov page. If you are interested in participating in this study or have questions about the study please contact Tina Cousins, Clinical Research Coordinator.


Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy

Randomised, Double Blind, Placebo Controlled, Multicentre Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy

About: It is a randomized, double blind, parallel group, placebo controlled study. A total of 213 male ambulant subjects will be randomized 2:1 (Givinostat: placebo). Subjects will be stratified for their concomitant use of steroids in 4 strata:

  1. Deflazacort daily regimen
  2. Deflazacort intermittent regimen
  3. Other steroids daily regimen
  4. Other steroids intermittent regimen. The study duration is planned for 19 months.

Ages Eligible for Study: 6 Years to 17 Years (Child)

Sexes Eligible for Study: Male

Status: Actively Recruiting Participants. Accepting transfers.

For more information about this study, including eligibility criteria, please go to the studies ClinicalTrials.gov page. If you are interested in participating in this study or have questions about research, please contact Gee Kim, Clinical Research Coordinator.


ImagingDMD

ImagingDMD

The study, known as “ImagingDMD” is a joint effort between researchers at the University of Florida, The Children’s Hospital of Philadelphia, Oregon Health and Science University, and Shriners Hospital for Children-Portland to investigate the potential of magnetic resonance imaging (MRI) to non-invasively monitor disease progression in Duchenne muscular dystrophy. Families interested in learning more about the study are encouraged to contact the ImagingDMD Team.