| Sponsor |
Study Name |
Drug Name |
Drug Type |
Phase |
Route of Administration |
Exons Eligible |
Status/Age |
Age |
Ambulation Status |
Location |
ClinicalTrials.gov Identifier |
|
|
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
|
Please see Exon Deletion Tool or Chart for more information*
|
Recruiting
|
7-55 yrs
|
Ambulatory and Non-Ambulatory
|
US
|
NCT05670730
|
|
|
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
|
Ambulatory and Non-Ambulatory
|
US
|
NCT04428476
|
|
|
Capricor Inc.
|
A Study of 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
|
Recruiting
|
≥10 yrs
|
Ambulatory and Non-Ambulatory
|
US
|
NCT05126758
|
|
|
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
|
Ambulatory
|
US
|
NCT03936894
|
|
|
Cumberland Pharmaceuticals
|
Oral Ifetroban in Subjects with Duchenne Muscular Dystrophy (DMD)
|
Ifetroban
|
Selective Thromboxane Receptor Antagonist
|
2
|
Oral
|
Mutation Agnostic – All eligible
|
Recruiting
|
≥7 yrs
|
Ambulatory and Non-Ambulatory
|
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
|
Please see Exon Deletion Tool or Chart for more information*
|
Recruiting
|
4-16 yrs old
|
Ambulatory and Non-Ambulatory
|
US/AU/EU/CA
|
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
|
Recruiting
|
4-9 yrs old
|
Ambulatory
|
US
|
NCT05540860
|
|
|
Edgewise
|
A Study of EDG-5506 in Adult Males with Becker Muscular Dystrophy (ARCH)
|
EDG-5506
|
Oral Small Molecule Muscle Stabilizer
|
1b
|
Oral
|
Mutation Agnostic – All eligible
|
Active, not Recruiting
|
18-55 yrs old
|
Ambulatory
|
US
|
NCT05160415
|
|
|
Edgewise
|
A Phase 2 study of EDG-5506 in Adolescents and Adult Males living with Becker Muscular Dystrophy (CANYON)
|
EDG-5506
|
Oral Small Molecule Muscle Stabilizer
|
2
|
Oral
|
Mutation Agnostic – All eligible
|
Active, not Recruiting
|
12-50 yrs old
|
Ambulatory
|
US/EX-US
|
NCT05291091
|
|
|
Edgewise
|
A Phase 2, pivotal cohort to study EDG-5506 in Adult Males living with Becker Muscular Dystrophy (GRAND CANYON)
|
EDG-5506
|
Oral Small Molecule Muscle Stabilizer
|
2
|
Oral
|
Mutation Agnostic – All eligible
|
Recruiting
|
18-50 yrs old
|
Ambulatory
|
US/EX-US
|
NCT05291091
|
|
|
ENCell
|
Phase 1 Clinical Trial to 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
|
Ambulatory and Non-Ambulatory
|
EX-US
|
NCT05338099
|
|
|
FibroGen
|
Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD (LELANTOS-2)
|
Pamrevlumab
|
mAb to CTGF
|
3
|
IV Infusion
|
Medical history includes diagnosis of DMD and confirmed Duchenne mutation, including status of exon 44 using a validated genetic test.
|
Active, not recruiting
|
6 – 11 yrs
|
Ambulatory
|
US/EX-US
|
NCT04632940
|
|
|
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
|
Active, not recruiting
|
6-17 yrs old
|
Ambulatory
|
US/EX-US
|
NCT02851797
|
|
|
Italfarmaco
|
Givinostat in Duchenne Muscular Dystrophy Long Term Safety and Tolerabillity Study
|
Givinostat
|
HDAC Inhibitor
|
2/3
|
Oral
|
Mutation Agnostic – All eligible
|
Completed
|
≥7 yrs old
|
Patients previously treated in Givinostat study
|
US/EX-US
|
NCT03373968
|
|
|
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
|
Active, not recruiting
|
≥4 yrs old
|
Ambulatory
|
US
|
NCT03333590
|
|
|
Nationwide Children’s Hospital
|
Phase 1/2a Systemic Gene Delivery Clinical Trial of scAAV9.U7.AACA for Exon 2 Duplication-Associated Duchenne Muscular Dystrophy
|
Biological: scAAV9.U7.ACCA
|
Gene Therapy
|
1/2a
|
IV Infusion
|
Confirmed duplication of exon 2 in the DMD gene using a clinically accepted technique that completely defines the mutation
|
Active, not recruiting
|
6 Months – 13 yrs old
|
Pre-Ambulatory and Ambulatory
|
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
|
Pre-Ambulatory and Ambulatory
|
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
|
Please see Exon Deletion Tool or Chart for more information*
|
Recruiting
|
4-14 yrs old
|
Ambulatory
|
US/Japan
|
NCT05996003
|
|
|
NS Pharma
|
A Phase 1/2 of NS-050/NCNP-03 in Boys with DMD (Meteor50)
|
NS-050/NCNP-03
|
Exon Skipping 50
|
1/2
|
IV Infusion
|
Please see Exon Deletion Tool or Chart for more information*
|
Not yet recruiting
|
4-14 yrs old
|
Ambulatory
|
US/Japan
|
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
|
Please see Exon Deletion Tool or Chart for more information*
|
Active, not recruiting
|
Child, Adult, Older Adult
|
Ambulatory
|
US/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
|
Please see Exon Deletion Tool or Chart for more information*
|
Active, not recruiting
|
Child, Adult, Older Adult
|
Ambulatory and Non-Ambulatory
|
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
|
Please see Exon Deletion Tool or Chart for more information*
|
Completed
|
4-7 yrs old
|
Ambulatory
|
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
|
Please see Exon Deletion Tool or Chart for more information*
|
Completed
|
≥8 yrs old
|
Ambulatory and Non-Ambulatory
|
US/EX-US
|
NCT04956289
|
|
|
PepGen
|
A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (CONNECT1-EDO51)
|
PGN-EDO51
|
Exon Skipping 51
|
2
|
IV Infusion
|
“Parent Project Muscular Dystrophy – Duchenne Deletion Tool
|
Recruiting
|
8 Years and older (Child, Adult, Older Adult )
|
Ambulatory and Non-Ambulatory
|
EX-US
|
NCT06079736
|
|
|
Percheron Therapeutics
|
A Multicentre, Randomised, Double-blind, Placebo-controlled and Open Label Extension Study to Assess the Efficacy, Safety, and Pharmacokinetic Profile of of ATL1102 in Non-ambulatory Participants With Duchenne Muscular Dystrophy
|
ATL1102
|
Anti-Infammatory
|
2b
|
Subcutaneous Injection
|
Mutation Agnostic – All eligible
|
Recruiting
|
10-17 years of age
|
Non-Ambulatory
|
EX-US
|
NCT05938023
|
|
|
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
|
Ambulatory
|
US/EX-US
|
NCT04281485
|
|
|
Pfizer
|
A Study to Evaluate the Safety and tolerability of PF-06939926 Gene Therapy in DMD
|
Fordadistrogene Movaparvovec
|
Gene Therapy
|
1b
|
IV Infusion
|
All except any 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.
|
Active, not recruiting
|
≥4 yrs old
|
Ambulatory and Non-Ambulatory
|
US
|
NCT03362502
|
|
|
Pfizer
|
A Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy
|
Fordadistrogene Movaparvovec
|
Gene Therapy
|
2
|
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
|
2-3 yrs old
|
Ambulatory
|
US/EX-US
|
NCT05429372
|
|
|
Pfizer
|
A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for 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.
|
Recruiting
|
≥0 years old
|
Ambulatory and Non-Ambulatory
|
US/EX-US
|
NCT05689164
|
|
|
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
|
N/A
|
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
|
Ambulatory
|
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
|
Ambulatory
|
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
|
Ambulatory
|
US
|
NCT03796637
|
|
|
RegenxBio
|
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
|
RGX-202
|
Gene Therapy
|
1 & 2
|
IV Infusion
|
DMD gene mutation in exons 18 and above, and a clinical picture consistent with typical DMD.
|
Recruiting
|
4-11 yrs old
|
Ambulatory
|
US
|
NCT05693142
|
|
|
RegenxBio
|
Anti-AAV8 Antibody Assessment Study of Boys with DMD
|
N/A
|
Observational Screening Study
|
N/A
|
N/A
|
Mutation Agnostic – All eligible
|
Recruiting
|
0 to <12 years of age
|
Ambulatory and Non-Ambulatory
|
US
|
NCT05683379
|
|
|
ReveraGen Biopharma and Santhera Pharmaceuticals
|
A Study to Assess the Efficacy and Safety of Vamorolone in Boys with DMD
|
Vamorolone
|
Anti-Infammatory
|
2b
|
Oral
|
Mutation Agnostic – All eligible
|
Completed
|
4-7 yrs old
|
Ambulatory
|
US/EX-US
|
NCT03439670
|
|
|
ReveraGen Biopharma and 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
|
Anti-Infammatory
|
2
|
Oral
|
Mutation Agnostic – All eligible
|
Recruiting
|
2-17 yrs old
|
Ambulatory and Non-Ambulatory
|
EX-US only
|
NCT05185622
|
|
|
ReveraGen Biopharma and Santhera Pharmaceuticals
|
A Study to Assess Vamorolone in Becker Muscular Dystrophy
|
Vamorolone
|
Anti-Infammatory
|
2
|
Oral
|
Mutation Agnostic – All eligible
|
Recruiting
|
18-64 yrs old
|
Ambulatory and Non-Ambulatory
|
US
|
NCT05166109
|
|
|
Sarepta
|
Study of SRP-4045 and SRP-4053 in DMD Patients (ESSENCE)
|
SPR-4045 & SRP-4043
|
Exon Skipping 45/53
|
3
|
IV Infusion
|
Please see Exon Deletion Tool or Chart for more information*
|
Recruiting
|
6-13 yrs old
|
Ambulatory
|
US/EX-US (only recruiting EX-US)
|
NCT02500381
|
|
|
Sarepta
|
A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD)
|
SRP-9001
|
Gene Therapy
|
2
|
IV Infusion
|
A frameshift mutation contained between exons 18 and 58 (inclusive).
|
Active, not Recruiting
|
4-7 yrs old
|
Ambulatory
|
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
|
Please see Exon Deletion Tool or Chart for more information*
|
Recruiting
|
4-13 yrs old
|
Ambulatory
|
US/EX-US
|
NCT03992430
|
|
|
Sarepta
|
Two-Part Study for Dose Determination of SRP-5051 (Part A), Then Dose Expansion (Part B) in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (MOMENTUM)
|
SRP-5051
|
Exon Skipping 51
|
2
|
IV Infusion
|
Please see Exon Deletion Tool or Chart for more information*
|
Recruiting
|
7-21 yrs old
|
Ambulatory and Non-Ambulatory
|
US/EX-US
|
NCT04004065
|
|
|
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.
|
Active, not recruiting
|
4-7 yrs old
|
Ambulatory
|
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
|
Mutation Agnostic – All eligible
|
Enrolling by Invitation
|
≥3 yrs old
|
Ambulatory and Non-Ambulatory
|
US
|
NCT04626674
|
|
|
Sarepta
|
A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9001 in Participants With Duchenne Muscular Dystrophy (DMD)
|
SRP-9001
|
Gene Therapy
|
1/2
|
IV Infusion
|
Mutation Agnostic – All eligible
|
Completed
|
3 months-7 yrs old
|
Ambulatory
|
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 contained between exons 18 and 79 (inclusive).
|
Recruiting
|
≥8 to <18 yrs
|
Ambulatory and Non-Ambulatory
|
US
|
NCT05881408
|
|
|
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
|
Completed
|
4-17 yrs old
|
Ambulatory and Non-Ambulatory
|
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
|
All mutations except established clinical diagnosis of DMD that is associated with any deletion mutation in exons 1 to 11 or 42 to 45, inclusive.
|
Not yet recruiting
|
4-7 yrs old
|
Ambulatory
|
US
|
NCT06138639
|
|
|
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
|
Recruiting
|
≥5 yrs old
|
Ambulatory
|
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
|
Ambulatory and Non-Ambulatory
|
EX-US
|
NCT03354039
|
|
|
University of Florida
|
Tadalafil as an Adjuvant to Therapy for Duchenne Muscular Dystrophy
|
Tadalafil
|
Phosphodiesterase 5 (PDE5) inhibitor
|
2/3
|
Oral
|
Mutation Agnostic – All eligible
|
Recruiting
|
7-13 yrs old
|
Ambulatory
|
US
|
NCT05195775
|
|
|
Wave
|
An Open-label Phase 1b/2a Study of WVE-N531 in Patients with Duchenne Muscular Dystrophy
|
WVE-N531
|
Exon Skipping 53
|
1/2
|
IV Infusion
|
Please see Exon Deletion Tool or Chart for more information*
|
Active, not Recruiting
|
5-18 yrs old
|
Ambulatory and Non-Ambulatory
|
EX-US
|
NCT04906460
|
|
