Duchenne Muscular Dystrophy
Condition Description
An inherited disorder of progressive muscular weakness, typically in boys.
Many people with muscular dystrophy have Duchenne syndrome. Girls can be carriers and mildly affected, but the disease typically affects boys.
Symptoms include frequent falls, trouble getting up or running, waddling gait, big calves, and learning disabilities.
Active Trials
-
TRIAL TITLE: A Phase 1/2, Randomized, Placebo-controlled, Double-blind, Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects of Single and Multiple Ascending Doses of AOC 1044 Administered Intravenously to Healthy Adult Volunteers and Participants with DMD Mutations Amenable to Exon 44 Skipping
DESCRIPTION: AOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants with DMD mutations amenable to exon 44 skipping.
Part A is a single dose design with multiple cohorts (dose levels) in healthy adult volunteers.
Part B is a multiple-ascending dose design with 3 cohorts (dose levels) in participants with Duchenne.
SPONSOR: Avidity Biosciences
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: Explore 44 AOC 1044-CS1
PHASE: Part B
STATUS: Active
RECRUITING PATIENTS: No
-
TRIAL TITLE: A Phase 2, Open-label Study to Evaluate the Pharmacodynamics and Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants with Mutations Amenable to Exon 44 Skipping.
DESCRIPTION: AOC 1044-CS2 (EXPLORE44-OLE) is an open label, extension study to Part B of AOC 1044-CS1 (EXPLORE44). AOC 1044-CS2 is designed to evaluate the pharmacodynamic effect of AOC 1044 on dystrophin protein production in skeletal muscle as well as the long-term safety, tolerability, pharmacokinetics, and exploratory efficacy of AOC 1044.
Participants from AOC 1044-CS1 Part B are eligible to enroll in AOC 1044-CS2 if they have satisfactorily completed AOC 1044-CS1.
SPONSOR: Avidity Biosciences
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: AOC 1044-CS2
PHASE: 2
STATUS: Active
RECRUITING PATIENTS: No
-
TRIAL TITLE: A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-3) (HOPE-3)
DESCRIPTION: HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function. Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during the first 12-months of the study. All participants will be eligible to receive 4 doses of CAP-1002 for an additional 12 months as part of an open-label extended assessment period.
SPONSOR: Capricor Inc.
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: CAP-1002-DMD-04
PHASE: 3
STATUS: Active
RECRUITING PATIENTS: No
Inquire About This Trial → -
TRIAL TITLE: CureDuchenne Link®: A Resource to Support Research Studies in Duchenne and Becker Muscular Dystrophy (DMD/BMD).
DESCRIPTION: CureDuchenne link is a data hub comprised of integrated biospecimens, clinical data, and self- and/or caregiver-reported information from participants. Anyone over 4 weeks old who has been diagnosed with DMD or BMD or who is a carrier of DMD or BMD can join. Parents or legal guardians can sign up their child(ren).
SPONSOR: CureDuchenne
INDICATION: Duchenne Muscular Dystrophy/ Becker Muscular Dystrophy
STUDY PROTOCOL: CD-2021-01
PHASE: NA
STATUS: Active
RECRUITING PATIENTS: Yes
-
TRIAL TITLE: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping (DELIVER)
DESCRIPTION: The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension period (96 weeks).
SPONSOR: Dyne Therapeutics
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: DYNE251-DMD-201
PHASE: 1 & 2
STATUS: Active
RECRUITING PATIENTS: No
-
TRIAL TITLE: Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
DESCRIPTION: 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.
SPONSOR: Italfarmaco
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: DSC 14/2357/51
PHASE: LT OL
STATUS: Active
RECRUITING PATIENTS: No
-
TRIAL TITLE: A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)
DESCRIPTION: The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, placebo-controlled part A, followed by an open-label part B.
SPONSOR: Edgewise Therapeutics, Inc.
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: EDG-5506-210
PHASE: 2
STATUS: Active
RECRUITING PATIENTS: Yes
-
TRIAL TITLE: A Phase 2 Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NS-089/NCNP-02 in Boys with Duchenne Muscular Dystrophy (DMD)
DESCRIPTION: This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to <15 years with DMD due to mutations amenable to exon 44 skipping. Participants will receive a selected dose of NS-089/NCNP-02 administered once weekly.
The study consists of 2 parts: Part 1 and Part 2. Six participants (Cohort 1) will participate in both Part 1 and Part 2, and 14 participants (Cohort 2) will be added for Part 2.
SPONSOR: NS Pharma, Inc.
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: NS-089/NCNP-02-201
PHASE: 2
STATUS: Active
RECRUITING PATIENTS: Yes
-
TRIAL TITLE: Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
DESCRIPTION: The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or treatment plan, and siblings of those participants (provided those participants have completed the placebo-controlled portion of the trial).
The treatment will continue under this protocol until consent withdrawal by participants, withdrawal due to worsen condition after initiating ataluren treatment, withdrawal by investigator, withdrawal due to participant unable to tolerate ataluren, participant is eligible to participate in another ataluren nmDBMD clinical trial program initiated by sponsor, study is discontinued by the relevant regulatory authority and/or sponsor, or until ataluren becomes commercially available.
SPONSOR: PTC Therapeutics
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: PTC124-GD-016 DMD
PHASE: 3
STATUS: Active
RECRUITING PATIENTS: No
-
TRIAL TITLE: Anti-AAV8 Antibody Assessment Study of Boys with Duchenne Muscular Dystrophy Aged 0 to <12 Years
DESCRIPTION: This is a noninterventional screening study; no investigational product is administered in this study. Information collected in this study may be used to identify potential participants for DMD investigational gene therapy clinical trials. This study consists of a single phone/video interview, a single home health visit, and a single phone/video call for communication of AAV8 antibody test results.
SPONSOR: REGENXBIO Inc.
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: RGX-202-0101
PHASE: N/A
STATUS: Active
RECRUITING PATIENTS: Yes
-
TRIAL TITLE: Two-Part Study for Dose Determination of SRP-5051 (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (MOMENTUM)
DESCRIPTION: This study will be comprised of 2 parts: 1) Part A (Multiple Ascending Dose [MAD]) will be conducted to evaluate the safety and tolerability of SRP-5051 at MAD levels to determine doses to be administered in Part B, and 2) Part B will be conducted to further evaluate the SRP-5051 doses selected in Part A. Participants enrolling in Part B will be those who completed Part A or Study 5051-102 and meet applicable eligibility criteria for Part B, as well as additional participants who meet applicable eligibility criteria for enrollment at the beginning of Part B.
SPONSOR: Sarepta Therapeutics, Inc.
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: Momentum 5051-201
PHASE: 2
STATUS: Active
RECRUITING PATIENTS: No
Inquire About This Trial → -
TRIAL TITLE: A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
DESCRIPTION: This study will be comprised of 2 parts: Part 1 (dose escalation) will be conducted to evaluate the safety and tolerability of 2 doses (high dose level 1 and high dose level 2) of eteplirsen in approximately 10 participants with DMD; Part 2 (dose finding and dose comparison) will be conducted for the selection of a high dose (high dose level 1 versus high dose level 2) and its comparison with the 30 milligrams/kilogram (mg/kg) dose of eteplirsen, in approximately 144 participants with genetically confirmed deletion mutations amenable to treatment by skipping exon 51.
SPONSOR: Sarepta Therapeutics, Inc.
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: MIS51ON 4658-402
PHASE: 3b
STATUS: Active
RECRUITING PATIENTS: No
Inquire About This Trial →
Upcoming Trials
Past Trials
-
TRIAL TITLE: A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-2)
DESCRIPTION: HOPE-2 is a double-blind clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD). Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during a 12-month period.
SPONSOR: Capricor Inc.
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: CAP-1002-DMD-02
PHASE: 2
STATUS: Completed
-
TRIAL TITLE: Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (PolarisDMD)
DESCRIPTION: "The PolarisDMD study is a Phase 3, global study to evaluate the efficacy and safety of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD. Male patients from 4-7 years of age (up to 8th birthday) will be enrolled.
Edasalonexent is an orally administered small molecule that inhibits NF-kB, which is the key link between loss of dystrophin and disease pathology and plays a fundamental role in the initiation and progression of skeletal and cardiac muscle disease in DMD."
SPONSOR: Catabasis Pharmaceuticals
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: CAT-1004-301
PHASE: 3
STATUS: Completed
-
TRIAL TITLE: An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (GalaxyDMD)
DESCRIPTION: "The GalaxyDMD study is a global Phase 3, open-label, treatment extension study to evaluate the safety, tolerability, and durability of effect in long-term dosing of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD. Patients who completed CAT-1004-201 or CAT-1004-301 or siblings of these boys from 4-12 years of age (up to 13th birthday) will be enrolled.
Edasalonexent is an orally administered small molecule that inhibits NF-kB, which is a key link between loss of dystrophin and disease pathology and plays a fundamental role in the initiation and progression of skeletal and cardiac muscle disease in DMD."
SPONSOR: Catabasis Pharmaceuticals
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: CAT-1004-302
PHASE: 3
STATUS: Terminated
-
TRIAL TITLE: Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
DESCRIPTION: This is a Phase 2, open-label, single arm trial of pamrevlumab (FG-3019) to estimate pamrevlumab's safety and efficacy in non-ambulatory participants with DMD.
SPONSOR: FibroGen
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: FGCL-3019-079
PHASE: 2
STATUS: Closed
RECRUITING PATIENTS: No
-
TRIAL TITLE: A Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids, in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD)
DESCRIPTION: To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids in subjects with non-ambulatory Duchenne muscular dystrophy (age 12 years and older).
SPONSOR: FibroGen
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: FGCL-3019-093
PHASE: 3 DB
STATUS: Closed
RECRUITING PATIENTS: No
-
TRIAL TITLE: A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in ambulatory subjects with Duchenne Muscular Dystrophy (DMD).
DESCRIPTION: This is a global, randomized, double-blind, trial of pamrevlumab or placebo in combination with systemic corticosteroids in participants with DMD, aged 6 to <12 years (ambulatory participants only). Approximately 70 participants will be randomized at a 1:1 ratio to Arm A (pamrevlumab + systemic deflazacort or equivalent potency of corticosteroids administered orally) or Arm B (placebo+ systemic deflazacort or equivalent potency of corticosteroids administered orally), respectively.
SPONSOR: FibroGen, Inc.
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: FGCL-3019-094
PHASE: 3
STATUS: Completed
RECRUITING PATIENTS: No
-
TRIAL TITLE: Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
DESCRIPTION: "it is a randomised, double blind, parallel group, placebo controlled study. A total of 179 male ambulant subjects will be randomised 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."
SPONSOR: Italfarmaco
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: DSC 14/2357/48
PHASE: 3 DB
STATUS: Completed
-
TRIAL TITLE: A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy (BRAVE)
DESCRIPTION: This is a multicenter, double blind, placebo controlled, multiple dose study to examine the safety and efficacy of MNK-1411 in male subjects 4 to 8 years of age (inclusive) with Duchenne Muscular Dystrophy (DMD).
SPONSOR: Mallinckrodt ARD LLC
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: MNK-14112096
PHASE: 2
STATUS: Terminated
-
TRIAL TITLE: A phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of PF-06939926 for the treatment of Duchenne Muscular Dystrophy.
DESCRIPTION: The purpose of the study is to demonstrate the safety and efficacy of PF-06939926 treatment in participants with DMD.
SPONSOR: Pfizer
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: C3391003
PHASE: 3
STATUS: Completed
-
TRIAL TITLE: 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)
DESCRIPTION: This study is designed to evaluate safety, tolerability, and pharmacokinetics (PK) in male children with nmDMD aged ≥6 months to <2 years treated daily for 24 weeks with orally administered ataluren 10, 10, and 20 milligrams/kilogram (mg/kg) (morning, mid-day, and evening dose, respectively).
SPONSOR: PTC Therapeutics
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: PTC124-GD-048-DMD
PHASE: 2
STATUS: Closed
RECRUITING PATIENTS: No
-
TRIAL TITLE: An Open-Label Study to Evaluate the Safety of Golodirsen in Non-Ambulant Patients with Duchenne Muscular Dystrophy
DESCRIPTION: This is an open-label study to evaluate the safety and tolerability of golodirsen injection in non-ambulant DMD patients with confirmed genetic mutations amenable to treatment by exon 53 skipping.
SPONSOR: Rare Disease Research, LLC
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: WVE-DMDX51-003
PHASE: 4
STATUS: Completed
-
TRIAL TITLE: Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
DESCRIPTION: This is a multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory boys with Duchenne Muscular Dystrophy (DMD).
SPONSOR: Hoffmann-La Roche
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: Roche WN40227
PHASE: 2 & 3
STATUS: Completed
-
TRIAL TITLE: A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids (SIDEROS)
DESCRIPTION: The purpose of the study is to assess the efficacy of idebenone in delaying the loss of respiratory function in patients with DMD receiving concomitant glucocorticoid steroids
SPONSOR: Santhera Pharmaceuticals
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: SNT-III-012
PHASE: 3
STATUS: Terminated
-
TRIAL TITLE: An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051
DESCRIPTION: 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.
SPONSOR: Sarepta Therapeutics, Inc.
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: SRP-5051-102
PHASE: 1 & 2 OL
STATUS: Terminated
-
TRIAL TITLE: Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E) (SIDEROS-E)
DESCRIPTION: The purpose of the study is to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study.
SPONSOR: Santhera Pharmaceuticals
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: SNT-III-012-E
PHASE: 3 OL
STATUS: Terminated
-
TRIAL TITLE: A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy (DMD)
DESCRIPTION: 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.
SPONSOR: Sarepta Therapeutics, Inc.
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: SRP-5051-101
PHASE: 1
STATUS: Completed
-
TRIAL TITLE: Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
DESCRIPTION: This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
SPONSOR: Sarepta Therapeutics, Inc.
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: SRP-4658-203
PHASE: 2 OL
STATUS: Completed
-
TRIAL TITLE: Study of Eteplirsen in DMD Patients (PROMOVI)
DESCRIPTION: The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51. Additional objectives include evaluation of safety, biomarkers and the long-term effects of eteplirsen up to 96 weeks, followed by a safety extension (not to exceed 48 weeks).
SPONSOR: Sarepta Therapeutics, Inc.
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: SRP-4658-301
PHASE: 3 OL
STATUS: Completed
-
TRIAL TITLE: Study of SRP-4045 and SRP-4053 in Participants With Duchenne Muscular Dystrophy (DMD) (ESSENCE)
DESCRIPTION: The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053 compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively.
SPONSOR: Sarepta Therapeutics, Inc.
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: SRP-4045-301
PHASE: 3
STATUS: Completed
-
TRIAL TITLE: An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
DESCRIPTION: The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).
SPONSOR: Sarepta Therapeutics, Inc.
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: Essence 4045-302
PHASE: 3 OL
STATUS: Completed
-
TRIAL TITLE: Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy
DESCRIPTION: This is a Phase 1, double-blind, placebo-controlled, single ascending dose cohort study to evaluate the safety, tolerability, and plasma concentrations of WVE-210201 in ambulatory and non-ambulatory male pediatric patients with DMD amenable to exon 51 skipping intervention.
SPONSOR: Wave Life Sciences Ltd.
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: WVE-DMDX51-001
PHASE: 1
STATUS: Completed
-
TRIAL TITLE: A Multicenter, Open-Label Extension Study of WVE-210201 in Patients previously enrolled in WVE-DMDX51-001
DESCRIPTION: This is an open-label extension study (OLE) to evaluate the safety, tolerability, pharmacodynamics, pharmacokinetics and exploratory functional effects of multiple doses of WVE-210201 in male pediatric patients with DMD amenable to exon 51 skipping intervention.
SPONSOR: Wave Life Sciences Ltd.
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: WVE-DMDX51-002
PHASE: Open label extension
STATUS: Completed
-
TRIAL TITLE: Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy (DYSTANCE 51)
DESCRIPTION: This is a Phase 2/3, multicenter, randomized, double-blind, placebo-controlled study with an open-label extension period to evaluate the safety and efficacy of WVE-210201 (suvodirsen) in ambulatory male pediatric patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping intervention (DYSTANCE 51)
SPONSOR: Wave Life Sciences Ltd.
INDICATION: Duchenne Muscular Dystrophy
STUDY PROTOCOL: WVE-DMDX51-003
PHASE: 2 & 3
STATUS: Completed