Duchenne Muscular Dystrophy

Josh & Kingston

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 (EMBARK)

A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Patients With Duchenne Muscular Dystrophy (EMBARK)

About: The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.

Ages Eligible for Study: 4 Years to 7 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.


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:  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 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.


Italfarmico 51: Givinostat in Duchenne’s Muscular Dystrophy Long-term Safety and Tolerability Study

Open Label, Long-term Safety, Tolerability, and Efficacy Study of GIVINOSTAT in All DMD Patients Who Have Been Previously Treated in One of the GIVINOSTAT Studies

About: This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne’s muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies. GIVINOSTAT oral suspension (10 mg/mL) has to be administered orally as 2 oral doses daily while the subject is in a fed state. The starting dose of GIVINOSTAT in the present long term study will be the same that the subject was receiving at the end of the previous DMD GIVINOSTAT study.

Ages Eligible for Study: 7 years and older (Child, Adult, Older 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 Samantha Norman, Clinical Research Coordinator.