Duchenne Muscular Dystrophy

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

UPDATE: CAMBRIDGE, Mass., Nov. 13, 2018 “We are pleased with the progress we have made on IGNITE DMD, dosing two additional patients with SGT-001 since the study resumed,” said Jorge Quiroz, M.D., Chief Medical Officer of Solid Biosciences. “Six patients have now been randomized in IGNITE DMD, three to the active treatment group, all of whom are doing well, and three to the delayed treatment control group. Continuing to enroll IGNITE DMD is a top priority, and we look forward to understanding the potential of SGT-001 in the clinic. As always, we remain grateful to the patients and families participating in IGNITE DMD and the team at the University of Florida.”

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 either:

Stephanie Salabarria
Clinical Research Coordinator 

 

Sarepta 203: Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy

An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early-Stage Duchenne Muscular Dystrophy 

About: The Sarepta-203 study was designed for male participants with Duchenne Muscular Dystrophy who are between the ages of 4-6 that have an out-of-frame deletion that can be corrected by exon 51 skipping through weekly Eteplirsen infusions.

Ages Eligible for Study:    4 Years to 6 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 either:

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 either:

Norane Shehab
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 has not yet started recruiting.

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 either:

Norane Shehab
Clinical Research Coordinator 

 

5051-101: A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy (DMD)

A Phase 1 Trial to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Treatment

About: The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK) of 5 escalating doses of SRP-5051 administered as a single dose to patients with DMD amenable to exon 51 skipping treatment.

Ages Eligible for Study:    12 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:    Male

Status:  Actively 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 the study please contact:

Norane Shehab
Clinical Research Coordinator 

 

5051-102: An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051

An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051

About:The purpose of this extension study is to evaluate the safety, tolerability, and pharmacokinetics of repeat administrations of SRP-5051 in patients with Duchenne muscular dystrophy (DMD) who participated in studies of SRP-5051.

Ages Eligible for Study:    12 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:    Male

Status: This study has not yet started recruiting.

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:

Norane Shehab
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

 

HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) (HOPE)

A Randomized, Open-label Study of the Safety and Efficacy of Multi- Vessel Intracoronary Delivery of Allogeneic Cardiosphere-Derived Cells in Patients With Cardiomyopathy Secondary to Duchenne Muscular Dystrophy

About: Male subjects with cardiomyopathy secondary to Duchenne muscular dystrophy (DMD) meeting all inclusion and no exclusion criteria will be randomized. All subjects will be at least 12 years of age. They will be randomized in a 1:1 manner to either intracoronary infusion of CAP-1002 in three coronary arteries supplying the three major cardiac territories of the left ventricle of the heart (anterior, lateral, inferior/posterior) or usual care. In the active treatment arm, all three major cardiac territories will be treated (infused) during a single procedure in an open label fashion.

Ages Eligible for Study:    12 Years and older   (Child, Adult, Senior)
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 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

 

Roche WN40226 (BMS CN001006): Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD

A Multi-site, Randomized, Placebo-controlled, Double-blind, Multiple Ascending Subcutaneous Dose Study to Evaluate the Safety Tolerability and Pharmacokinetics of RO7239361 (BMS-986089) in Ambulatory Boys With Duchenne Muscular Dystrophy

About: The purpose of this study is to determine the safety and tolerability of RO7239361 (BMS-986089) in boys with Duchenne Muscular Dystrophy with any genetic mutation.

Ages Eligible for Study:    5 Years to 10 Years   (Child)
Sexes Eligible for Study:    Male

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

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

 

Roche WN40227 (BMS CN001016): Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 (BMS-986089) in Ambulatory Boys With Duchenne Muscular Dystrophy

A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 (BMS-986089) in Ambulatory Boys with Duchenne Muscular Dystrophy

About: The purpose of this study is to assess the efficacy, safety, and tolerability of RO7239361 (BMS-986089) in boys with Duchenne Muscular Dystrophy with any genetic mutation.

Ages Eligible for Study:    6 Years to 11 Years   (Child)
Sexes Eligible for Study:    Male

Status: Actively recruiting and enrolling participants.

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

 

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:

Cristina Liberati
Clinical Research Coordinator