| Sponsor |
Study Name |
Drug Name |
Drug Type |
Phase |
Route of Administration |
Exons Eligible |
Status |
Age |
Steroid Regimen Requirement |
Ambulation Status |
Genders Eligible |
Location |
ClinicalTrials.gov Identifier |
| Avidity Biosciences |
Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants with Mutations Amenable to Exon44 Skipping (EXPLORE44OLE) |
AOC 1044 |
Exon 44 Skipping |
2 |
IV Infusion |
Parent Project Muscular Dystrophy – Duchenne Deletion Tool
Duchenne Population Potentially Amenable to Exon Skipping
|
Active, not recruiting |
7 Years to 27 Years (Child, Adult ) |
If on corticosteroids, stable dose for 30 days before screening and throughout the study |
Ambulatory and Non-Ambulatory |
Male |
US |
NCT06244082 |
| Avidity Biosciences |
Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping (EXPLORE44) |
AOC 1044 |
Exon 44 Skipping |
1/2 |
IV Infusion |
Parent Project Muscular Dystrophy – Duchenne Deletion Tool
Duchenne Population Potentially Amenable to Exon Skipping
|
Completed |
7-55 yrs |
If on corticosteroids, stable dose for 30 days before screening and throughout the study |
Ambulatory and Non-Ambulatory |
Male |
US |
NCT05670730 |
| Avidity Biosciences |
Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping |
delpacibart zotadirsen |
Exon 44 Skipping |
Expanded Access |
IV Infusion |
Refer to exon skipping tool for more information on if you or your child may be eligible for this drug. |
Available |
≥ 6 yrs |
None Noted |
Ambulatory and Non-Ambulatory |
Male |
US |
NCT07250737 |
| BioMarin Pharmaceutical |
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy |
BMN 351 |
Exon 51 Skipping |
1/2 |
IV Infusion |
Parent Project Muscular Dystrophy – Duchenne Deletion Tool
Duchenne Population Potentially Amenable to Exon Skipping
|
Recruiting |
4-10 yrs |
Currently on consistent doses of steroid treatment for the last 12 weeks |
Ambulatory |
Male |
EX-US |
NCT06280209 |
| Capricor Inc. |
Open-label Extension of the HOPE-2 Trial (HOPE-2-OLE)* |
CAP-1002 |
Cardiosphere-Derived Cells (CDCs) |
2 |
IV Infusion |
Mutation Agnostic – All eligible |
Active, not Recruiting |
≥10 yrs |
None Noted |
Ambulatory and Non-Ambulatory |
All |
US |
NCT04428476 |
| Capricor Inc. |
A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-3) |
CAP-1002 |
Cardiosphere-Derived Cells (CDCs) |
3 |
IV Infusion |
Mutation Agnostic – All eligible |
Active, not recruiting |
≥10 yrs |
Treatment with systemic glucocorticoids for at least 12 months and at a stable dose at least 6 months prior to study participation, except for either weight-based dose adjustment or a decrease in steroid dose of ≤ 10% for toxicity. |
Ambulatory and Non-Ambulatory |
Male |
US |
NCT05126758 |
| Catalyst Pharmaceuticals, Inc. |
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients with Duchenne Muscular Dystrophy. |
Vamorolone |
Corticosteroid |
N/A |
Oral |
Mutation Agnostic – All eligible |
Recruiting |
≥ 2 yrs |
Currently on treatment with AGAMREE®. |
Ambulatory and Non-Ambulatory |
Male |
US |
NCT06564974 |
| Children’s National Research Institute |
Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy |
ILARIS |
anti-interleukin antibody injection |
1/2 |
Subcutaneous Injection |
Mutation Agnostic – All eligible |
Active, not Recruiting |
≥ 2 yrs and < 6 yrs |
Subject is naïve to treatment with glucocorticoids for DMD. |
Ambulatory |
Male |
US |
NCT03936894 |
| Constant Therapeutics LLC |
TXA127 in Non-Ambulant Patients With DMD Cardiomyopathy |
TXA127 |
Synthetic Peptide |
2 |
subcutaneous injection |
Mutation Agnostic – All eligible |
Recruiting |
16 Years and older (Child, Adult, Older Adult ) |
Taking systemic glucocorticoids for at least six months prior to screening |
Non-Ambulatory |
Male |
EX-US |
NCT06013839 |
| Cumberland Pharmaceuticals |
Oral Ifetroban in Subjects with Duchenne Muscular Dystrophy (DMD) |
Ifetroban |
Selective Thromboxane Receptor Antagonist |
2 |
Oral |
Mutation Agnostic – All eligible |
Active, not recruiting |
≥7 yrs |
Stable dose of oral corticosteroids for at least 8 weeks or has not received corticosteroids for at least 30 days. |
Ambulatory and Non-Ambulatory |
Male |
US |
NCT03340675 |
| Dyne Therapeutics |
Safety, Tolerability, Pharmodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (DELIVER) |
DYNE-251 |
Exon Skipping |
1/2 |
IV Infusion |
Parent Project Muscular Dystrophy – Duchenne Deletion Tool
Duchenne Population Potentially Amenable to Exon Skipping
|
Active, not recruiting |
4-16 yrs old |
Receiving a stable dosage of glucocorticoids for at least 12 weeks prior to the start of study drug administration, with the expectation of maintaining a stable dose during the Placebo-Controlled and Open-Label Periods of the study |
Ambulatory and Non-Ambulatory |
Male |
US/EX-US |
NCT05524883 |
| Edgewise |
A Study of EDG-5506 in Children with Duchenne Muscular Dystrophy (LYNX) |
EDG-5506 |
Oral Small Molecule Muscle Stabilizer |
2 |
Oral |
Mutation Agnostic – All eligible |
Active, not Recruiting |
4-9 yrs old |
For Cohorts 1, 2, 3, 4 and 5: Aged 4-9 years on a stable dose of corticosteroids for a minimum of 6 months prior to the Baseline visit.
For Cohort 2 Non-Steroid (Cohort 2NS): Aged 4-7 years not on corticosteroids within 6 months prior to the Baseline visit.
|
Ambulatory |
Male |
US |
NCT05540860 |
| Edgewise |
A Study of EDG-5506 in Adult Males with Becker Muscular Dystrophy (CANYON) |
EDG-5506 |
Oral Small Molecule Muscle Stabilizer |
1b |
Oral |
Mutation Agnostic – All eligible |
Completed |
18-55 yrs old |
May NOT have received oral corticosteroids for >5 days in the previous 6 months at a dose of >5 mg equivalent per day. |
Ambulatory |
Male |
US |
NCT05160415 |
| Edgewise |
Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy (MESA) |
EDG-5506 |
Oral Small Molecule Muscle Stabilizer |
2 |
Oral |
Mutation Agnostic – All eligible |
Enrolling by invitation |
(Child, Adult, Older Adult ) |
In Key Exclusion Criteria: Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months. |
Ambulatory |
Male |
US |
NCT06066580 |
| Edgewise |
Phase 2 Study of EDG-5506 in Children and Adolescents with Duchenne Muscular Dystrophy Previously Treated with Gene Therapy (FOX) |
EDG-5506 |
Oral Small Molecule Muscle Stabilizer |
2 |
Oral |
Mutation Agnostic – All eligible |
Active, not Recruiting |
6 yrs to 17 yrs |
Treatment with a stable dose of corticosteroids for a minimum of 6 months prior to the Baseline visit. |
Ambulatory |
Male |
US |
NCT06100887 |
| Edgewise |
Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON) |
EDG-5506 |
Oral Small Molecule Muscle Stabilizer |
2 |
Oral |
Mutation Agnostic – All eligible |
Active, not Recruiting |
12-50 yrs old |
May NOT have received oral corticosteroids for the treatment of BMD in the previous 6 months. |
Ambulatory |
Male |
US/EX-US |
NCT05291091 |
| ENCell |
Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy (DMD) |
EN001 |
Stem Cells |
1 |
IV Infusion |
Mutation Agnostic – All eligible |
Completed |
2-18 yrs old |
Use of systemic corticosteroids at a stable dose for 24 weeks prior to screening and are expected to maintain the constant dose throughout the study period. |
Ambulatory and Non-Ambulatory |
Male |
EX-US |
NCT05338099 |
| ENCell |
Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy |
EN001 |
Stem Cells |
1/2 |
IV Infusion |
Mutation Agnostic – All eligible |
Not yet recruiting |
6 – 11 yrs old |
Stable dose of glucocorticoids for at least 12 weeks prior to screening, with treatment maintained. Dosage adjustments for body weight changes are allowed. |
Ambulatory |
Male |
EX-US only |
NCT06328725 |
| Entrada |
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44 (ELEVATE-44) |
ENTR-601-44 |
Exon Skipping 44 |
1/2 |
IV Infusion |
Refer to exon skipping tool for more information on if you or your child may be eligible for this drug. |
Recruiting |
4 – 20 yrs old |
None Noted |
Ambulatory |
Male |
EX-US only |
NCT07037862 |
| Entrada |
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45 (ELEVATE-45) |
ENTR-601-45 |
Exon Skipping 45 |
1/2 |
IV Infusion |
Refer to exon skipping tool for more information on if you or your child may be eligible for this drug. |
Recruiting |
4 – 20 yrs old |
None Noted |
Ambulatory |
Male |
EX-US only |
NCT07038824 |
| Hoffmann-La Roche |
A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) (SHIELD DMD) |
Satralizumab |
interleukin-6 (IL-6) receptor inhibitors |
2 |
Subcutaneous Injection |
Mutation Agnostic – All eligible |
Recruiting |
8 – 17 yrs old |
Daily oral corticosteroids. |
Ambulatory and Non-Ambulatory |
Male |
US/EX-US |
NCT06450639 |
| Hoffmann-La Roche |
A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) (ENVOL) |
delandistrogene moxeparvovec |
Gene Therapy |
2 |
IV Infusion |
A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive) |
Recruiting |
<3 yrs old |
May NOT be receiving regular oral corticosteroids as a treatment for DMD or planning to receive oral corticosteroids as a treatment for DMD within 1 year of baseline. |
Ambulatory |
Male |
EX-US |
NCT06128564 |
| Larry W. Markham, Indiana University |
Sodium/Glucose Cotransporter-2 Inhibitors (SGLT2i) Therapy in Duchenne Cardiomyopathy (CHERISH) |
SGLT2 inhibitor |
|
1 |
Oral |
None noted |
Not yet recruiting |
8 – 18 yrs old |
None Noted |
|
Male |
US |
NCT07172971 |
| Insmed Gene Therapy LLC |
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD) (ASCEND) |
INS1201 |
Gene Therapy |
1 |
Intrathecal Injection |
Genetic report must describe a frameshift deletion, frameshift duplication, premature stop (“nonsense”), canonical splice site mutation, or other pathogenic variant in the DMD gene fully contained between exons 18 to 58 (inclusive) that is expected to lead to absence of a functional dystrophin protein. |
Recruiting |
2-4 years of age |
None noted |
Ambulatory |
Male |
US |
NCT06817382 |
| Italfarmaco |
Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients with DMD |
Givinostat |
HDAC Inhibitor |
3 |
Oral |
Mutation Agnostic – All eligible |
Completed |
6-17 yrs old |
Have used systemic corticosteroids for a minimum of 6 months immediately prior to the start of study treatment, with no significant change in corticosteroids type or dosage or dosing regimen (excluding changes related to body weight change) for a minimum of 6 months immediately prior to start of study treatment and a reasonable expectation that dosage and dosing regimen will not change significantly for the duration of the study. |
Ambulatory |
Male |
US/EX-US |
NCT02851797 |
| Italfarmaco |
Givinostat in Duchenne’s Muscular Dystrophy Long-term Safety and Tolerability Study |
Givinostat |
HDAC Inhibitor |
2/3 |
Oral |
Mutation Agnostic – All eligible |
Active, not Recruiting |
≥7 yrs old |
None noted |
Ambulatory and Non-Ambulatory |
Male |
US/EX-US |
NCT03373968 |
| Italfarmaco |
Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old |
Givinostat |
HDAC Inhibitor |
2 |
Oral |
Mutation Agnostic – All eligible |
Recruiting |
2-6 yrs old |
Subjects who start study on steroids must remain on stable dose (no increases) within 3 months of starting treatment.
Subjects not on steroids may not start steroids prior to starting treatment. |
None noted |
None noted |
EX-US |
NCT06769633 |
| Masonic Cancer Center, University of Minnesota |
Trial of Cell Based Therapy for DMD |
Tacrolimus |
CD54+ allogeneic muscle progenitor cell product |
1 |
Injection |
Mutation Agnostic – All eligible |
Recruiting |
18 Years and older (Adult, Older Adult ) |
None Noted |
Non-Ambulatory |
All |
US |
NCT06692426 |
| Medical University of Gdansk |
The Efficacy and Safety of Metoprolol as add-on Treatment to Standard of Care in Preventing Cardiomyopathy in Patients With DMD (MeDMD) |
Metoprolol Succinate |
beta-blocker |
3 |
Oral |
Complete dystrophin deficiency; out-of-frame mutation; or nonsense mutation, deletion/duplication leading to a downstream stop codon |
Recruiting |
≥8 and <17 yrs old |
None Noted |
Ambulatory and Non-Ambulatory |
Male |
Poland |
NCT05066633 |
| Nationwide Children’s Hospital |
Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy |
Biological: rAAVrh74.MCK.GALGT2 |
Gene Therapy |
1/2a |
IV Infusion |
Confirmed mutations in the DMD gene using a clinical accepted technique that completely defines the mutation 1,2 |
Completed |
≥4 yrs old |
Stable daily dose of corticosteroid therapy (including either prednisone, prednisolone, deflazacort or their generic forms) for 12 weeks prior to gene transfer. |
Ambulatory |
Male |
US |
NCT03333590 |
| Nationwide Children’s Hospital |
AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications |
Biological: scAAV9.U7.ACCA |
Gene Therapy |
1/2 |
IV Infusion |
Confirmed duplication of exon 2 in the DMD gene using a clinically accepted technique that completely defines the mutation |
Completed |
6 Months – 13 yrs old |
Stable dose and regimen of corticosteroid therapy (prednisone, deflazacort, or their generic forms) for at least 12 weeks prior to gene transfer. |
Pre-Ambulatory and Ambulatory |
Male |
US |
NCT04240314 |
| Nationwide Children’s Hospital |
Once Weekly Infant Corticosteroid Trial for DMD |
Prednisolone |
Corticosteroid |
4 |
Oral |
Mutation Agnostic – All eligible |
Recruiting |
1 month – 30 months |
Trial testing corticorsteroid regimen. |
Pre-Ambulatory and Ambulatory |
Male |
US |
NCT05412394 |
| NS Pharma |
NS-089/NCNP-02-201 in Boys with Duchenne Muscular Dystrophy (DMD) |
NS-089/NCNP-02
(Brogidirsen) |
Exon Skipping 44 |
2 |
IV Infusion |
Parent Project Muscular Dystrophy – Duchenne Deletion Tool
Duchenne Population Potentially Amenable to Exon Skipping
|
Recruiting |
4-14 yrs old |
Stable dose of glucocorticoid for at least 3 months and the dose is expected to remain on a stable dose for the duration of the study. |
Ambulatory |
Male |
US/EX-US |
NCT05996003 |
| NS Pharma |
NS-050/NCNP-03 in Boys With DMD (Meteor50) |
NS-050/NCNP-03 |
Exon Skipping 50 |
1/2 |
IV Infusion |
Parent Project Muscular Dystrophy – Duchenne Deletion Tool
Duchenne Population Potentially Amenable to Exon Skipping
|
Recruiting |
4-14 yrs old |
Stable dose of glucocorticoid for at least 3 months and the dose is expected to remain on a stable dose for the duration of the study. |
Ambulatory |
Male |
US/EX-US |
NCT06053814 |
| NS Pharma |
Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with DMD (RACER 53-X) |
Viltolarsen |
Exon Skipping 53 |
3 |
IV Infusion |
Parent Project Muscular Dystrophy – Duchenne Deletion Tool
Duchenne Population Potentially Amenable to Exon Skipping
|
Active, not recruiting |
Child, Adult, Older Adult |
None Noted |
Ambulatory |
Male |
EX-US |
NCT04768062 |
| NS Pharma |
Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502) |
Viltolarsen |
Exon Skipping 53 |
4 |
IV Infusion |
Duchenne Population Potentially Amenable to Exon Skipping |
Active, not recruiting |
Child, Adult, Older Adult |
None Noted |
Ambulatory and Non-Ambulatory |
Male |
US/EX-US |
NCT04687020 |
| NS Pharma |
Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with DMD (RACER53) |
Viltolarsen |
Exon Skipping 53 |
3 |
IV Infusion |
Parent Project Muscular Dystrophy – Duchenne Deletion Tool
Duchenne Population Potentially Amenable to Exon Skipping
|
Completed |
4-7 yrs old |
Stable dose of glucocorticoid (GC) for at least 3 months prior to study entry and is expected to remain on stable dose of GC treatment for the duration of the study. |
Ambulatory |
Male |
US/EX-US |
NCT04060199 |
| NS Pharma |
Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant Boys and Non-Ambulant Boys with DMD (Galactic53) |
Viltolarsen |
Exon Skipping 53 |
2 |
IV Infusion |
Parent Project Muscular Dystrophy – Duchenne Deletion Tool
Duchenne Population Potentially Amenable to Exon Skipping
|
Completed |
≥8 yrs old |
Must be on a stable dose of glucocorticoid (GC) or not treated with GC for at least 3 months prior to the first dose of IP and is expected to remain on stable dose of GC treatment or off GC for the duration of the study. |
Ambulatory and Non-Ambulatory |
Male |
US/EX-US |
NCT04956289 |
| NS Pharma |
The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation |
Viltolarsen |
Exon Skipping 53 |
Expanded Access |
IV Infusion |
Refer to exon skipping tool for more information on if you or your child may be eligible for this drug. |
Approved for marketing |
3-12 yrs old |
|
Ambulatory |
Male |
|
NCT04337112 |
| Peking Union Medical College Hospital |
A Study to Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular Dystrophy. (GEN6050XIIT) |
GEN6050XIIT |
Gene Therapy |
1 |
intravenous injection |
Patients with DMD gene exon deletion types confirmed by molecular diagnosis: 8-49, 20-49, 22-49, 51, 51-53, 51-55, 51-57, 51-59, 51-60, 51-67, 51-69, 51-75 or 51-78 and other mutations amenable to exon 50 skipping. |
Recruiting |
4 Years to 10 Years (Child ) |
Receipt of glucocorticoids for 6 months and a stable daily dose for at least 12 weeks prior to study entry |
Ambulatory |
Male |
EX-US |
NCT06392724 |
| Peking Union Medical College Hospital |
A Study to Evaluate the Safety and Tolerability of RAG-18 in Pediatric Patients With Duchenne Muscular Dystrophy |
RAG-18 |
Gene Therapy |
Early 1 |
intravenous injection |
None noted |
Not yet recruiting |
4-15 yrs old |
None noted |
Ambulatory |
Male |
EX-US |
NCT07282652 |
| Pfizer |
A Phase 3 Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy |
Fordadistrogene Movaparvovec |
Gene Therapy |
3 |
IV Infusion |
All except any mutation (exon deletion, exon duplication, insertion, or point mutation) affecting any exon between exon 9 and exon 13, inclusive; OR
A deletion that affects both exon 29 and exon 30;OR
A deletion that affects any exons between 56-71, inclusive. |
Active, not recruiting |
4-7 yrs old |
Receiving a stable daily dose (at least 0.5 mg/kg/day prednisone or prednisolone, or at least 0.75 mg/kg/day deflazacort) for at least 3 months prior to Screening. |
Ambulatory |
Male |
US/EX-US |
NCT04281485 |
| Precision BioSciences, Inc. |
PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD) |
PBGENE-DMD |
Gene Therapy |
1/2a |
IV Infusion |
Patients with DMD harboring mutations amenable to excision of exons 45-55 |
Not yet recruiting |
2-7 yrs old |
None Noted |
Ambulatory |
Male |
No location data |
NCT07429240 |
| PTC Therapeutics |
Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy |
deflazacort |
Corticosteroid |
|
Oral |
|
Approved for marketing |
≥5 yrs old |
None Noted |
Ambulatory and Non-Ambulatory |
Male |
US |
NCT02592941 |
| PTC Therapeutics |
A Study to Assess Dystrophin Levels in Participants with Nonsense Mutation DMD (nmDMD) |
Ataluren |
Nonsense Mutations Inhibitor |
2 |
Oral |
Documentation of the presence of a nonsense point mutation in the dystrophin gene as determined by gene sequencing. |
Completed |
2-7 yrs old |
None Noted |
N/A |
Male |
US |
NCT03648827 |
| PTC Therapeutics |
A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) |
Ataluren |
Nonsense Mutations Inhibitor |
2 |
Oral |
Documentation of the presence of a nonsense point mutation in the dystrophin gene as determined by gene sequencing. |
Completed |
6 months – 2 years old |
None Noted |
Ambulatory |
Male |
US |
NCT04336826 |
| PTC Therapeutics |
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy |
Ataluren |
Nonsense Mutations Inhibitor |
3 |
Oral |
Documentation of the presence of a nonsense point mutation in the dystrophin gene as determined by gene sequencing. |
Completed |
7-16 yrs old |
Use of systemic corticosteroids (prednisone/prednisolone or deflazacort)for a minimum of 12 months immediately prior to start of study treatment, with no significant change in dosage or dosing regimen for a minimum of 3 months immediately prior to start of study treatment. |
Ambulatory |
Male |
US/EX-US |
NCT03179631 |
| PTC Therapeutics |
A Study to Assess Dystrophin Levels in Participants with Nonsense Mutation DMD (nmDMD) Who Have Been Treated with Ataluren |
Ataluren |
Nonsense Mutations Inhibitor |
2 |
Oral |
Documentation of the presence of a nonsense point mutation in the dystrophin gene as determined by gene sequencing. |
Completed |
None |
None Noted |
Ambulatory |
Male |
US |
NCT03796637 |
| REGENXBIO |
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) |
RGX-202 |
Gene Therapy |
1/2/3 |
IV Infusion |
Any mutation except for those with deletions or point mutations in exons 8, 9 and/or 10. |
Recruiting |
1 year and older |
For boys ages 1 to <4 years old: Either off corticosteroids or on a stable dose of corticosteroids for prior 12 weeks
For boys 4 years of age or older: On a stable dose of corticosteroids for prior 12 weeks |
Ambulatory |
Male |
US/EX-US |
NCT05693142 |
| ReveraGen Biopharma Inc. |
A Study to Assess the Efficacy and Safety of Vamorolone in Boys with DMD |
Vamorolone |
Corticosteroid |
2 |
Oral |
Mutation Agnostic – All eligible |
Completed |
4-7 yrs old |
May NOT currently be treated or have received previous treatment with oral glucocorticoids or other immunosuppressive agents [Notes: Past transient use of oral glucocorticoids or other oral immunosuppressive agents for no longer than 1 month cumulative, with last use at least 3 months prior to first dose of study medication, will be considered for eligibility on a case-by-case basis, unless discontinued for intolerance. |
Ambulatory |
Male |
US/EX-US |
NCT03439670 |
| ReveraGen Biopharma Inc. |
A Study to Assess Vamorolone in Becker Muscular Dystrophy |
Vamorolone |
Corticosteroid |
2 |
Oral |
Mutation Agnostic – All eligible |
Completed |
18-64 yrs old |
May NOT have received oral glucocorticoids or other oral immunosuppressive agents for at least 3 months prior to first administration of study medication. |
Ambulatory and Non-Ambulatory |
Male |
US |
NCT05166109 |
| Santhera Pharmaceuticals |
A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD) |
Vamorolone |
Corticosteroid |
2 |
Oral |
Mutation Agnostic – All eligible |
Active, not Recruiting |
2-17 yrs old |
If 7 to <18 years of age and currently taking standard of care glucocorticoids for treatment of DMD, subject has been taking standard of care glucocorticoids at stable dose for at least 3 months prior to enrollment in the study, and will continue the same stable dose regimen through the date of the Baseline Day -1 Visit.
If 7 to <18 years of age, and not currently glucocorticoid-treated, subject has not received oral glucocorticoids or other oral immunosuppressive agents for at least 3 months prior to enrollment.
|
Ambulatory and Non-Ambulatory |
Male |
EX-US |
NCT05185622 |
| Sardocor Corp |
Modulation of SERCA2a of Intra-Myocytic Calcium Trafficking in Cardiomyopathy Secondary to Duchenne Muscular Dystrophy (MUSIC-DMD) |
SRD-001 |
Gene therapy-cardiac |
1 |
Intracoronary Infusion |
Mutation Agnostic – All eligible |
Recruiting |
≥18 yrs old |
Individualized, optimized cardiac medical therapy and glucocorticoid treatment for at least 12 months prior to enrollment |
Non-Ambulatory |
Male |
US |
NCT06224660 |
| ReveraGen Biopharma Inc. |
A Study to Assess Vamorolone in Becker Muscular Dystrophy |
Vamorolone |
Corticosteroid |
2 |
Oral |
Mutation Agnostic – All eligible |
Recruiting |
18-64 yrs old |
May NOT have received oral glucocorticoids or other oral immunosuppressive agents for at least 3 months prior to first administration of study medication. |
Ambulatory and Non-Ambulatory |
Male |
US |
NCT05166109 |
| Santhera Pharmaceuticals |
A Study on Safety and Effectiveness of Long-term Treatment With Vamorolone in Boys With Duchenne Muscular Dystrophy (GUARDIAN) |
Vamorolone |
Corticosteroid |
4 |
Oral |
Mutation Agnostic – All eligible |
Recruiting |
Child, Adult, Older Adult |
Must be on vamorolone on day of enrollment. |
Ambulatory and Non-Ambulatory |
Male |
EX-US |
NCT06713135 |
| Sarepta |
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD) |
SRP-5051 |
Exon Skipping 51 |
1 |
IV Infusion |
Refer to exon skipping tool for more information on if you or your child may be eligible for this drug. |
Completed |
≥12 yrs old |
Have been on a stable dose or dose equivalent of oral corticosteroids for at least 12 weeks prior to randomization and the dose is expected to remain constant (except for modifications to accommodate changes in weight and stress-related needs as per the recently published guidelines throughout the study. |
Ambulatory |
Male |
US/EX-US |
NCT03375255 |
| Sarepta |
Study of SRP-4045 and SRP-4053 in DMD Patients (ESSENCE) |
SPR-4045 & SRP-4043 |
Exon Skipping 45/53 |
3 |
IV Infusion |
Parent Project Muscular Dystrophy – Duchenne Deletion Tool
Duchenne Population Potentially Amenable to Exon Skipping
|
Completed |
6-13 yrs old |
Stable dose of oral corticosteroids for at least 24 weeks prior to Week 1, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight). |
Ambulatory |
Male |
US/EX-US |
NCT02500381 |
| Sarepta |
A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD) |
SRP-9001 |
Gene Therapy |
1/2 |
IV Infusion |
A frameshift mutation contained between exons 18 and 58 (inclusive). |
Completed |
4-7 yrs old |
Stable dose equivalent of oral corticosteroids for at least 12 weeks. |
Ambulatory |
Male |
US |
NCT03769116 |
| Sarepta |
A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Duchenne Muscylar Dystrophy (DMD) Patients (MIS51ON) |
Eteplirsen |
Exon Skipping 51 |
3 |
IV Infusion |
Parent Project Muscular Dystrophy – Duchenne Deletion Tool
Duchenne Population Potentially Amenable to Exon Skipping
|
Active, not recruiting |
4-13 yrs old |
Have been on a stable dose or dose equivalent of oral corticosteroids for at least 12 weeks prior to randomization and the dose is expected to remain constant (except for modifications to accommodate changes in weight and stress-related needs as per the recently published guidelines throughout the study. |
Ambulatory |
Male |
US/EX-US |
NCT03992430 |
| Sarepta |
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 in Participants with DMD (EMBARK) |
SRP-9001 |
Gene Therapy |
3 |
IV Infusion |
A pathogenic frameshift mutation or premature stop codon contained between exons 18 and 79 (inclusive), with the exception of mutation fully contained within exon 45. |
Completed |
4-7 yrs old |
Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight). |
Ambulatory |
Male |
US/EX-US |
NCT05096221 |
| Sarepta |
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) (ENDEAVOR) |
SRP-9001 |
Gene Therapy |
1 |
IV Infusion |
Genetic mutation inclusion criteria vary by cohort |
Recruiting |
≥2 yrs old |
Cohorts 1, 2, 3, 5, 7 and 8 only: Stable dose equivalent of oral glucocorticoids for at least 12 weeks before screening and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the first year of the study.
Cohorts 4 and 6: Do not yet require use of chronic steroids for treatment of their DMD, in the opinion of the Investigator, and are not receiving steroids at the time of Screening.
|
Ambulatory and Non-Ambulatory. Ambulation criteria vary by cohort. Cohort 8: Non-ambulatory per protocol-specified criteria at the time of Screening |
Male |
US |
NCT04626674 |
| Sarepta |
A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) |
SRP-9001 |
Gene Therapy |
1/2 |
IV Infusion |
Cohorts A & B: A frameshift mutation contained between exons 18 and 58 (inclusive) |
Completed |
3 months-7 yrs old |
Cohort A participants: No previous treatment with corticosteroids. Cohort B participants: Stable dose equivalent of oral corticosteroids for at least 12 weeks prior to screening and the dose is expected to remain constant (except for potential modifications to accommodate changes in weight) throughout the first year of the study.
|
Ambulatory |
Male |
US |
NCT03375164 |
| Sarepta |
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) (ENVISION) |
SRP-9001 |
Gene Therapy |
3 |
IV Infusion |
A pathogenic frameshift mutation or premature stop codon in the DMD gene, except for any deletion mutations in exon 8 and/or 9. |
Active, not recruiting |
Child, Adult, Older Adult |
Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight). |
Ambulatory and Non-Ambulatory |
Male |
US/EX-US |
NCT05881408 |
| Sarepta |
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study (EXPEDITION) |
SRP-9001 |
Gene Therapy |
3 |
IV Infusion |
Genetic mutations vary |
Enrolling by invitation |
(Child, Adult, Older Adult ) |
None Noted |
Ambulatory and Non-Ambulatory |
Male |
US/EX-US |
NCT05967351 |
| Satellos Bioscience, Inc. |
First in Human SAD/MAD Safety and PK Study with Adult DMD Safety and PK Cohort |
SAT-3247 |
small molecule inhibitor |
1 |
Oral |
Mutation Agnostic – All eligible |
Completed |
18 Years to 40 Years (Adult ) |
Stable dose of systemic glucocorticosteroids, heart medications, and/or other supportive medications, vitamins and supplements according to the standard of care for DMD for 3 months prior to the Screening visit and for the duration of the study |
Ambulatory and Non-Ambulatory |
Male |
EX-US |
NCT06565208 |
| Satellos Bioscience, Inc. |
A Long-term Follow-up Study for Participants That Completed the SAT-3247-CL-101 Study |
SAT-3248 |
Small molecule inhibitor |
2 |
Oral |
Mutation Agnostic – All eligible |
Enrolling by invitation |
18Years to 40 Years (Adult ) |
Previously participated in the SAT-3247-CL-101 parent trial. Continued status of stable steroid dose or no steroid dose from parent trial. Continued stable doses of prescription meds and over the counter meds and/or herbal supplements from parent trial |
Ambulatory and Non-Ambulatory |
Male |
EX-US |
NCT06867107 |
| Satellos Bioscience, Inc. |
Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients (BASECAMP) |
SAT-3247 |
Small molecule inhibitor |
2 |
Oral |
Mutation Agnostic – All eligible |
Recruiting |
7-9 yrs old |
Stable dose of systemic glucocorticoids (i.e., prednisolone, deflazacort, or vamorolone) according to the standard of care for ≥ 3 months prior to the Screening Visit and for the duration of the trial. Patients who are not receiving glucocorticosteroids are also eligible if stopped ≥ 3 months prior to the Screening Visit. |
Ambulatory |
Male |
US/EX-US |
NCT07287189 |
| Sebastiano Lava |
Repurposing Empagliflozin for DMD-associated Cardiomyopathy in Children 6-18 Years of Age (REDMeD) |
Empagliflozin Tablets |
Enzyme inhibtor |
2 |
Oral |
Mutation Agnostic – All eligible |
Not yet recruiting |
6 Years to 18 Years (Child, Adult ) |
None Noted |
Ambulatory and Non-Ambulatory |
All |
EX-US |
NCT06643442 |
| Shanghai Jiao Tong University School of Medicine |
Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients With Duchenne Muscular Dystrophy |
BBM-D101 |
Gene Therapy |
1 |
Injection |
Mutation Agnostic – All eligible |
Recruiting |
4 Years to 8 Years (Child) |
None Noted |
Ambulatory |
Male |
EX-US |
NCT06641895 |
| Shanghai Jiao Tong University School of Medicine |
Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy |
LE051 |
Exon skipping |
Early 1 |
IV Infusion |
Exon51 |
Recruiting |
5 Years to 8 Years (Child) |
None Noted |
Ambulatory |
Male |
EX-US |
NCT06900049 |
| Shanghai Siponuoyin Biotechnology Co Ltd |
Safety and Dystrophin Expression of SPOT-mRNA03 in Duchenne Muscular Dystrophy (DMD) Patients |
SPOT-mRNA03 |
Gene Therapy |
Early 1 |
IV Infusion |
Mutation Agnostic – All eligible |
Recruiting |
2-6 yrs old |
A total of 6 eligible subjects will participate in the study with 3 subjects in each dose cohort [No previous treatment with corticosteroids]. |
Ambulatory |
Male |
EX-US |
NCT07188012 |
| Solid Biosciences |
Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (IGNITE DMD) |
SGT-001 |
Gene Therapy |
1/2 |
IV Infusion |
Mutation Agnostic – All eligible |
Active, not recruiting |
4-17 yrs old |
Stable daily dose (or equivalent) of oral corticosteroids ≥ 12 wks. |
Ambulatory and Non-Ambulatory |
Male |
US |
NCT03368742 |
| Solid Biosciences |
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) |
SGT-003 |
Gene Therapy |
1/2 |
IV Infusion |
Exclusion Criteria: Established clinical diagnosis of DMD that is associated with any deletion variant or variant predicted not to express exons 1 to 11, exons 42 to 45, or exons 57 to 69, inclusive of the DMD gene as documented by a genetic report. |
Recruiting |
0-17 yrs old |
On a stable dose of at least 0.5 mg/kg/day of oral daily prednisone or 0.75 mg/kg/day deflazacort for ≥12 weeks prior to entering the study. |
Ambulatory and Non-Ambulatory |
Male |
US/EX-US |
NCT06138639 |
| Solid Biosciences |
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE) |
SGT-003 |
Gene Therapy |
3 |
IV Infusion |
Exclusion Criteria: Established clinical diagnosis of DMD that is associated with any deletion variant or variant predicted not to express exons 1 to 11, exons 42 to 45, or exons 57 to 69, inclusive of the DMD gene as documented by a genetic report. |
Recruiting |
7-11 yrs old |
On a stable daily oral regimen of at least 0.5 mg/kg/day prednisone or 0.75 milligrams per kilogram per day (mg/kg/day) deflazacort for at least 6 months prior to entering the study, allowing for weight-based dose modifications in accordance with clinical practice. |
Ambulatory |
Male |
EX-US |
NCT07160634 |
| Sqy Therapeutics |
Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular Dystrophy (AVANCE1) |
SQY51 |
Exon Skipping 51 |
1/2 |
IV Infusion |
Parent Project Muscular Dystrophy – Duchenne Deletion Tool
Duchenne Population Potentially Amenable to Exon Skipping
|
Recruiting |
≥6 yrs old |
None Noted |
Ambulatory and Non-Ambulatory |
Male |
EX-US |
NCT05753462 |
| Taiho Pharmaceutical Co., Ltd. |
A Phase 3 Study of TAS-205 in Patients with Duchenne Muscular Dystrophy |
TAS-205 |
Selective hematopoietic prostaglantin D synthase (HPGDS) inhibitor |
3 |
Oral |
Mutation Agnostic – All eligible |
Active, not recruiting |
≥5 yrs old |
If taking oral glucocorticoids no significant change in the total daily or dosing 90 days prior to obtaining consent, or not taking oral glucocorticoids for more than 90 days prior to obtaining consent and whose symptoms are stable. |
Ambulatory |
Male |
EX-US |
NCT04587908 |
| University Hospital, Basel, Switzerland |
Tamoxifen in Duchenne Muscular Dystrophy: A Multicenter, Randomised, Double-blind, Placebo-controlled, Phase 3 Safety and Efficacy 48-Week Trial |
Tamoxifen |
Selective estrogren receptor modulator (SERM) |
3 |
Oral |
Mutation Agnostic – All eligible |
Completed |
78 months-16 yrs old |
Stable treatment with glucocorticoids >6 months (no significant change in dosage (>0.2mg/kg)) at screening; dosing adaptations according to weight change are allowed. |
Ambulatory and Non-Ambulatory |
Male |
EX-US |
NCT03354039 |
| University of Florida |
Vasodilator and Exercise Study for DMD (VASO-REx) |
Tadalafil |
Phosphodiesterase 5 (PDE5) inhibitor |
2 |
Oral |
Mutation Agnostic – All eligible |
Recruiting |
6 Years and older (Child, Adult, Older Adult ) |
On stable glucocorticoid regimen (for > 3 months). |
Ambulatory |
Male |
US |
NCT06290713 |
| University of Florida |
Tadalafil as Adjuvant Therapy for DMD |
Tadalafil |
Phosphodiesterase 5 (PDE5) inhibitor |
2/3 |
Oral |
Mutation Agnostic – All eligible |
Active, not recruiting |
7 – 13 yrs old |
None Noted |
Ambulatory |
Male |
US |
NCT05195775 |
| Wave Life Sciences |
Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy |
WVE-N531 |
Exon Skipping 53 |
2 |
IV Infusion |
Refer to exon skipping tool for more information on if you or your child may be eligible for this drug. |
Not yet recruiting |
(Child, Adult, Older Adult ) |
None Noted |
Ambulatory and Non-Ambulatory |
Male |
EX-US |
NCT07209332 |
| Wave Life Sciences |
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53) |
WVE-N531 |
Exon Skipping 53 |
1/2 |
IV Infusion |
Parent Project Muscular Dystrophy – Duchenne Deletion Tool
Duchenne Population Potentially Amenable to Exon Skipping
|
Recruiting |
4-18 yrs old |
Currently on a stable corticosteroid therapy regimen, defined as initiation of systemic corticosteroid therapy that occurred ≥6 months prior to Screening and no changes in dose ≤3 months prior to Screening visit. |
Ambulatory |
Male |
US/EX-US |
NCT04906460 |
| West China Hospital |
A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of Single Intravenous Infusion of JWK007 in Patients With Duchenne Muscular Dystrophy (DMD) |
JWK007 |
Gene Therapy |
1 |
Injection |
frameshift mutation (deletion or duplication) or a premature stop codon mutation in the DMD gene between exons 18 to 58. |
Active, not recruiting |
5 – 10 yrs old |
Participants must have been taking a stable dose of oral corticosteroids for at least 12 weeks prior to screening, and the expected dose should remain constant throughout the study, except for adjustments related to changes in body weight. |
Ambulatory |
Male |
EX-US |
NCT06114056 |
