Clinical trials: Neuromuscular Disease
Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne
Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18…
- Conditions
- Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne, Muscular Dystrophy (DMD), DMD
- Trial name
- FORZETTO (NCT07608432)
- Testing
- Zeleciment Rostudirsen (DYNE-251)
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Dyne Therapeutics
Neuromuscular Disease, Neuromuscular Diseases (NMD)
A Phase 3 Study of NTLA-2001 in ATTRv-PN
This study will be conducted to evaluate the efficacy and safety of a single dose of nexiguran ziclumeran (NTLA-2001) compared to placebo in participants with ATTRv-PN.
- Conditions
- Neuromuscular Disease, Neuromuscular Diseases (NMD), Neurodegenerative Disease, Neurodegenerative Disease, Hereditary
- Trial name
- NCT06672237
- Testing
- nexiguran ziclumeran
- Where
- 14 sites in Argentina, Brazil and 4 more
- Volunteers
- 60 people
- Sponsor
- Intellia Therapeutics
Amyotrophic Lateral Sclerosis, Neuromuscular Disease
Connect-One: Early Feasibility Study of Connexus® Brain-Computer Interface (BCI)
The Connect-One Study is an early feasibility study to obtain preliminary device safety information for the Connexus Brain-Computer Interface (BCI).
- Conditions
- Amyotrophic Lateral Sclerosis, Neuromuscular Disease, Stroke, Tetraplegia/Tetraparesis
- Trial name
- CONNECT-ONE (NCT07357428)
- Testing
- Connexus Brain-Computer Interface
- Where
- 3 sites in United States
- Volunteers
- 2 people
- Sponsor
- Paradromics
Myotonic Dystrophy 1
An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)
The purpose of this study is to learn about the long-term safety and tolerability of PGN-EDODM1 in participants with myotonic dystrophy type 1 (DM1) who have completed a prior study with PGN-EDODM1.
- Trial name
- NCT07220603
- Testing
- PGN-EDODM1
- Where
- 3 sites in Canada
- Volunteers
- 48 people
- Sponsor
- PepGen Inc
Muscular Dystrophy, Duchenne, Muscular Dystrophies
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT03179631
- Testing
- Ataluren
- Where
- 64 sites in United States, Australia and 16 more
- Volunteers
- 360 people
- Sponsor
- PTC Therapeutics
Myotonic Dystrophy 1
Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1
The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1).
- Trial name
- FREEDOM-DM1 (NCT06204809)
- Testing
- PGN-EDODM1 for infusion
- Where
- 12 sites in United States, Canada and 1 more
- Volunteers
- 24 people
- Sponsor
- PepGen Inc
Obstructive Sleep Apnea, Obesity Hypoventilation Syndrome (OHS)
Evaluation of Auto-EPAP for Management of Upper Airway Obstruction During Non-Invasive Ventilation
To evaluate the safety and effectiveness of the Automatic Expiratory Positive Airway Pressure (Auto-EPAP) feature versus manual expiratory positive airway pressure (EPAP) in the Vivo 45 LS Ventilator.
- Conditions
- Obstructive Sleep Apnea, Obesity Hypoventilation Syndrome (OHS), Chronic Obstructive Pulmonary Disease (COPD), Neuromuscular Diseases (NMD)
- Trial name
- Vivo NOVA (NCT06560411)
- Testing
- Non-Invasive Ventilation
- Where
- 4 sites in United States
- Volunteers
- 23 people
- Sponsor
- Breas Medical, Inc.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
This Phase 3 trial (Study SRK-015-003) was conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) (i.e., Type 2 and Type 3 SMA) and were receiving an approved…
- Conditions
- Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA
- Trial name
- SAPPHIRE (NCT05156320)
- Testing
- Apitegromab
- Where
- 50 sites in United States, Belgium and 7 more
- Volunteers
- 188 people
- Sponsor
- Scholar Rock, Inc.
Facioscapulohumeral Muscular Dystrophy 1
Evaluation of Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments of Losmapimod for FSHD1 With Extension
This clinical trial is a study to evaluate the safety, tolerability, and changes in biomarker and clinical outcome assessments of Losmapimod for patients with Facioscapulohumeral Muscular Dystrophy 1 (FSHD1) with an open-label…
- Trial name
- FSHD (NCT04004000)
- Testing
- Losmapimod
- Where
- Netherlands
- Volunteers
- 14 people
- Sponsor
- Fulcrum Therapeutics
Duchenne Muscular Dystrophy
A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment
The study consists of 3 periods: A Screening Period (up to 45 days), a Multiple Ascending Dose (MAD) Period (16 weeks), and a Long-Term Extension (LTE) Period (108 weeks).
- Trial name
- CONNECT1-EDO51 (NCT06079736)
- Testing
- PGN-EDO51
- Where
- 5 sites in Canada
- Volunteers
- 7 people
- Sponsor
- PepGen Inc
Facioscapulohumeral Muscular Dystrophy (FSHD)
Efficacy and Safety of Losmapimod in Treating Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) (REACH)
This is a study to evaluate the safety and efficacy of losmapimod in treating participants with Facioscapulohumeral Muscular Dystrophy (FSHD).
- Trial name
- NCT05397470
- Testing
- Losmapimod
- Where
- 33 sites in United States, Canada and 7 more
- Volunteers
- 260 people
- Sponsor
- Fulcrum Therapeutics
MuSK Myasthenia Gravis
Open-label Study to Evaluate the Safety of Various Dosing Regimens of MuSK-CAART for MuSK Myasthenia Gravis
Muscle-specific tyrosine kinase (MuSK) myasthenia gravis (MG) is a rare but potentially severe disease, in which patients develop pathogenic autoantibodies that specifically target the MuSK protein in the neuromuscular junction.
- Trial name
- NCT05451212
- Testing
- MuSK-CAART
- Where
- 5 sites in United States
- Volunteers
- 7 people
- Sponsor
- Cabaletta Bio
Facioscapulohumeral Muscular Dystrophy (FSHD)
Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE)
This study is an open-label extension to evaluate the safety and tolerability of long-term dosing of Losmapimod in patients with FSHD1 who participated in the ReDux4 study.
- Trial name
- NCT04264442
- Testing
- Losmapimod
- Where
- 17 sites in United States, Canada and 2 more
- Volunteers
- 76 people
- Sponsor
- Fulcrum Therapeutics
Neuromuscular Disorders, Neurodevelopmental Disorders
Efficacy and Usability of the EXPLORER Exoskeleton in Children With Neurodevelopmental Disorders in Natural Environments
Neurodevelopmental disorders often lead to abnormal development of the Central Nervous System (CNS), frequently causing motor dysfunctions such as an inability to stand and walk.
- Trial name
- NCT06765070
- Testing
- EXPLORER
- Where
- 6 sites in Spain
- Volunteers
- 15 people
- Sponsor
- MarsiBionics
Neuromuscular Disorders, Neurodevelopmental Disorders
Safety, Usability, and Effectiveness of a Gait Exoskeleton for Children and Adolescents With Neurodevelopmental Disorders.
Neurodevelopmental disorders often result in abnormal development of the Central Nervous System (CNS), frequently causing motor dysfunctions such as the inability to stand and walk.
- Trial name
- NCT07159360
- Testing
- CLINICAL EXPLORER
- Where
- 2 sites in Mexico, Spain
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- MarsiBionics
Neuromuscular Diseases
Oscillation and Lung Expansion (OLE) Therapy for Treatment of Neuromuscular Disease (NMD) Patients
The study will be a non-randomized open label pilot study comparing a retrospective control period to an active treatment period with oscillation and lung expansion (OLE) therapy.
- Trial name
- NCT05366010
- Testing
- Oscillation and Lung Expansion (OLE) therapy
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- Baxter Healthcare Corporation
Amyotrophic Lateral Sclerosis, Motor Neuron Disease
Safety of Intrathecal Riluzole in Patients With Amyotrophic Lateral Sclerosis
The purpose of this study is to investigate the safety and tolerability of intrathecal riluzole in adults with amyotrophic lateral sclerosis.
- Trial name
- NCT07093268
- Testing
- Intrathecal Riluzole
- Where
- 2 sites in Australia
- Volunteers
- 10 people
- Sponsor
- Brain Trust Bio
Lumbosacral Radiculopathy
Safety and Efficacy Study on AFA-281 for the Treatment of Low Back Pain
The goal of this clinical trial is to evaluate if the drug candidate AFA-281 works to treat chronic low back and leg pain caused by painful lumbosacral radiculopathy (PLSR) in adults.
- Trial name
- NCT06649747
- Testing
- AFA-281
- Volunteers
- 408 people
- Sponsor
- Afasci Inc
Duchenne Muscular Dystrophy (DMD)
A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
The study consists of 3 periods: A Screening Period (up to 45 days), a double-blind, placebo-controlled Multiple Ascending Dose (MAD) Period (28 weeks), and a Long-Term Extension (LTE) Period (108 weeks).
- Trial name
- CONNECT2-EDO51 (NCT06833931)
- Testing
- IV infusion
- Sponsor
- PepGen Inc
Neuromuscular Disorders
Safety and Usability of the EXPLORER Exoskeleton in Adults With Neuromuscular Diseases
Neurodevelopmental disorders frequently result in abnormal development of the Central Nervous System (CNS), often leading to motor impairments such as difficulty in standing and walking.
- Trial name
- NCT06894160
- Testing
- Adults EXPLORER
- Where
- Spain
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- MarsiBionics