Clinical trials: Multi-System-Atrophy
Ventilator Induced Diaphragm Dysfunction
A Protocol Comparing Temporary Transvenous Diaphragm Pacing to Standard of Care for Weaning From Mechanical Ventilation
This clinical investigation is an open-label, multi-center RCT to demonstrate the safety and effective performance of the Lungpacer DPTS (plus standard of care) as compared to Control (standard of care only) in patients aged 18…
- Trial name
- RESCUE 3 (NCT03783884)
- Testing
- Lungpacer Diaphragm Pacing Therapy
- Where
- 33 sites in United States, France and 1 more
- Volunteers
- 223 people
- Sponsor
- Lungpacer Medical Inc.
Multiple System Atrophy
Trial to Evaluate the Efficacy and Safety of KM-819 Treatment to Slow the Progression of Multiple System Atrophy (MSA)
This trial will the efficacy of KM-819 compared to placebo in subjects with MSA for slowing the progression of MSA.
- Trial name
- NCT05695378
- Testing
- KM-819
- Where
- South Korea
- Volunteers
- 68 people
- Sponsor
- Kainos Medicine Inc.
Multiple System Atrophy
A Study of TAK-341 in Treatment of Multiple System Atrophy
The main aim is to see how TAK-341 works after 52 weeks in participants with multiple system atrophy as measured by the Unified Multiple System Atrophy Rating Scale Part I (UMSARS).
- Trial name
- NCT05526391
- Testing
- TAK-341
- Where
- 41 sites in United States, Austria and 8 more
- Volunteers
- 158 people
- Sponsor
- Takeda
Symptomatic Neurogenic Orthostatic Hypotension
Phase 3 Open-Label Extension Study of TD-9855 for Treating Symptomatic nOH in Subjects With Primary Autonomic Failure
A Phase 3, multi-center, open-label study to evaluate the safety and tolerability of ampreloxetine in subjects with primary autonomic failures (MSA, PD, and PAF) and symptomatic nOH over 182 weeks.
- Trial name
- OAK (NCT04095793)
- Testing
- ampreloxetine
- Where
- 55 sites in United States, Australia and 16 more
- Volunteers
- 110 people
- Sponsor
- Theravance Biopharma
Duchenne Muscular Dystrophy (DMD)
Study of Eteplirsen in DMD Patients
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.
- Trial name
- PROMOVI (NCT02255552)
- Testing
- eteplirsen
- Where
- 37 sites in United States
- Volunteers
- 109 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy
A Multi-center, Randomized, Double-blind, Placebo-controlled, Phase 1/2 Trial to Evaluate the Efficacy and Safety of EN001 in Patients with Duchenne Muscular Dystrophy
- Trial name
- NCT06328725
- Testing
- EN001
- Where
- 2 sites in South Korea
- Volunteers
- 88 people
- Sponsor
- ENCell
Muscular Dystrophy, Duchenne
Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
The primary objective of this study is to explore safety and tolerability of eteplirsen in participants with advanced stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
- Trial name
- NCT02286947
- Testing
- Eteplirsen
- Where
- 9 sites in United States
- Volunteers
- 24 people
- Sponsor
- Sarepta Therapeutics, Inc.
Spinal Muscular Atrophy
An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)
An open-label, multi-part, first-in-human study of oral branaplam in infants with Type 1 spinal muscular atrophy. The purpose of this study was to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD)…
- Trial name
- NCT02268552
- Testing
- branaplam
- Where
- 14 sites in Belgium, Bulgaria and 5 more
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
Duchenne Muscular Dystrophy, Exon 44
Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping
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…
- Trial name
- EXPLORE44 (NCT05670730)
- Testing
- AOC 1044
- Where
- 10 sites in United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Avidity Biosciences, Inc.
Duchenne Muscular Dystrophy
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
The purpose of this study is to test the safety and tolerability of BMN 351 in participants with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping.
- Trial name
- NCT06280209
- Testing
- BMN 351
- Where
- 7 sites in Italy, Netherlands and 3 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical
Multiple System Atrophy
Study of ATH434 in Participants with Multiple System Atrophy
This study will assess the safety and efficacy of ATH434 in participants with Multiple System Atrophy
- Trial name
- NCT05109091
- Testing
- ATH434 dose level 1, ATH434 dose level 2
- Where
- 23 sites in United States, Australia and 4 more
- Volunteers
- 77 people
- Sponsor
- Alterity Therapeutics
Symptomatic Neurogenic Orthostatic Hypotension (NOH), Non-diabetic Neuropathy
Clinical Study of Droxidopa in Patients With Neurogenic Orthostatic Hypotension (NOH)
The purpose of this study is to see whether droxidopa is effective in treating symptoms of neurogenic orthostatic hypotension in patients with Primary Autonomic Failure (Pure Autonomic Failure, Multiple System Atrophy…
- Conditions
- Symptomatic Neurogenic Orthostatic Hypotension (NOH), Non-diabetic Neuropathy, Primary Autonomic Failure, Dopamine Beta Hydroxylase Deficiency
- Trial name
- NOH302 (NCT00633880)
- Testing
- Droxidopa
- Where
- 54 sites in United States, Australia and 2 more
- Volunteers
- 181 people
- Sponsor
- Chelsea Therapeutics
Spinal Muscular Atrophy
An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy
This study will test the safety, tolerability, and pharmacokinetics of escalating doses of nusinersen (ISIS 396443) administered into the spinal fluid either two or three times over the duration of the trial, in participants with…
- Trial name
- NCT01703988
- Testing
- Nusinersen
- Where
- 4 sites in United States
- Volunteers
- 34 people
- Sponsor
- Biogen
Symptomatic Neurogenic Orthostatic Hypotension
Clinical Effect of Ampreloxetine (TD-9855) for Treating Symptomatic nOH in Subjects With Primary Autonomic Failure
A Phase 3 study to evaluate efficacy, safety, and tolerability of ampreloxetine (TD-9855) in subjects with primary autonomic failures (MSA, PD, or PAF) and symptomatic nOH with up to 4 weeks of treatment.
- Trial name
- SEQUOIA (NCT03750552)
- Testing
- ampreloxetine
- Where
- 125 sites in United States, Australia and 17 more
- Volunteers
- 195 people
- Sponsor
- Theravance Biopharma
Autoimmune Diseases of the Nervous System, Multiple Sclerosis
Study of Tcelna (Imilecleucel-T) in Secondary Progressive Multiple Sclerosis
The purpose of this study is to determine whether Tcelna (imilecleucel-T, autologous T-Cell Immunotherapy) is effective in the treatment of secondary progressive multiple sclerosis (SPMS).
- Conditions
- Autoimmune Diseases of the Nervous System, Multiple Sclerosis, Secondary Progressive Multiple Sclerosis, Disease Progression
- Trial name
- Abili-T (NCT01684761)
- Testing
- Tcelna
- Where
- 36 sites in United States, Canada
- Volunteers
- 183 people
- Sponsor
- Opexa Therapeutics, Inc.
MSA - Multiple System Atrophy, MSA
Autologous Stem Cell Therapy in Patients With Multiple System Atrophy
This study investigates the safety and efficacy of autologous stem cell therapy in patients with Multiple System Atrophy (MSA), a rare and progressive neurodegenerative disorder characterized by autonomic failure, parkinsonism…
- Trial name
- NCT07465198
- Testing
- MSCs
- Where
- Poland
- Volunteers
- 50 people
- Sponsor
- Biocells Medical
Duchenne Muscular Dystrophy
Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
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 vesleteplirsen at MAD levels to determine the maximum tolerated dose (MTD), and 2)…
- Trial name
- MOMENTUM (NCT04004065)
- Testing
- Vesleteplirsen
- Where
- 25 sites in United States, Belgium and 6 more
- Volunteers
- 62 people
- Sponsor
- Sarepta Therapeutics, Inc.