Clinical trials: Multiple System Atrophy
Duchenne Muscular Dystrophy
DART Electrical Impedance Myography (EIM) Trial in Duchenne Muscular Dystrophy (DMD) and Healthy Controls
Physicians seek a method to assess neuromuscular disease that is both non-invasive and quantifiable. Many patients do not tolerate standard current day assessment tools (such as needle electromyogram), and Electrical Impedance…
- Trial name
- NCT01523964
- Testing
- Testing with EIM
- Where
- 6 sites in United States
- Volunteers
- 92 people, healthy volunteers welcome
- Sponsor
- Dart Therapeutics. LLC
Duchenne Muscular Dystrophy
Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
The main objective of this study is to evaluate the efficacy of Viltolarsen compared to placebo in Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping.
- Trial name
- NCT04060199
- Testing
- Viltolarsen
- Where
- 40 sites in United States, Australia and 17 more
- Volunteers
- 77 people
- Sponsor
- NS Pharma, Inc.
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.
Muscular Dystrophy (DMD)
Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD
The purpose of this study is to determine the safety and tolerability of RO7239361 in boys with Duchenne Muscular Dystrophy with any genetic mutation.
- Trial name
- NCT02515669
- Testing
- RO7239361
- Where
- 12 sites in United States, Canada
- Volunteers
- 43 people
- Sponsor
- Hoffmann-La Roche
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
Duchenne Muscular Dystrophy (DMD)
Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
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.
- Trial name
- NCT02420379
- Testing
- eteplirsen
- Where
- 13 sites in United States
- Volunteers
- 33 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)
This Phase II study is an open-label, multiple dose study to evaluate the safety, tolerability, PK, PD, clinical efficacy, behavior and neuropsychology, and physical functioning vamorolone over a treatment period of 12 weeks in…
- Trial name
- NCT05185622
- Testing
- Vamorolone
- Where
- 5 sites in Canada
- Volunteers
- 54 people
- Sponsor
- Santhera Pharmaceuticals
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
Finding the Optimum Regimen for Duchenne Muscular Dystrophy
The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine which of the three ways increases…
- Trial name
- FOR-DMD (NCT01603407)
- Testing
- Prednisone, Deflazacort
- Where
- 32 sites in United States, Canada and 3 more
- Volunteers
- 196 people
- Sponsor
- University of Rochester
Muscular Dystrophy, Duchenne
Rimeporide in Patients With Duchenne Muscular Dystrophy
In Duchenne Muscular Dystrophy (DMD) there is an imbalance between the levels of calcium and sodium in the muscles cells which is thought to be important in the damage which occurs overtime.
- Trial name
- RIM4DMD (NCT02710591)
- Testing
- Rimeporide
- Where
- 4 sites in France, Italy and 2 more
- Volunteers
- 20 people
- Sponsor
- EspeRare Foundation
Spinal Muscular Atrophy
A Study of CK-2127107 in Patients With Spinal Muscular Atrophy
This study will evaluate the pharmacodynamic (PD) effect of CK-2127107 (hereafter referred to as reldesemtiv) versus placebo on measures of skeletal muscle function or fatigability in patients with Type II, III, or IV spinal…
- Trial name
- NCT02644668
- Testing
- Reldesemtiv 150 mg, Reldesemtiv 450 mg
- Where
- 19 sites in United States, Canada
- Volunteers
- 70 people
- Sponsor
- Cytokinetics
Duchenne Muscular Dystrophy (DMD)
Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy
The primary objective of this study is to assess the ongoing efficacy, safety, and tolerability of an additional 212 weeks of treatment with eteplirsen injection in Duchenne muscular dystrophy (DMD) subjects who have successfully…
- Trial name
- NCT01540409
- Testing
- AVI-4658 (Eteplirsen)
- Where
- 11 sites in United States
- Volunteers
- 12 people
- Sponsor
- Sarepta Therapeutics, Inc.
Leukodystrophy, White Matter Disease
LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
Leukodystrophies, and other heritable disorders of the white matter of the brain, were previously resistant to genetic characterization, largely due to the extreme genetic heterogeneity of molecular causes.
- Conditions
- Leukodystrophy, White Matter Disease, 4H Syndrome, Adrenoleukodystrophy
- Trial name
- NCT02699190
- Where
- United States
- Volunteers
- 236 people
- Sponsor
- Children's Hospital of Philadelphia
Spinal Muscular Atrophy
Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy
This is a multi-center trial to assess safety and efficacy of a combined regimen of oral valproic acid (VPA) and carnitine in patients with Spinal Muscular Atrophy (SMA) 2 to 17 years of age.
- Trial name
- NCT00227266
- Testing
- Valproic Acid and Levocarnitine
- Where
- 6 sites in United States, Canada
- Volunteers
- 94 people
- Sponsor
- University of Utah
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.
Spinal Muscular Atrophy
Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam
This was a Phase IIIb open-label, single arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101B in participants with SMA aged 2 to <18 years after the discontinuation of treatment with nusinersen or…
- Trial name
- STRENGTH (NCT05386680)
- Testing
- OAV101
- Where
- 13 sites in United States, Australia and 7 more
- Volunteers
- 27 people
- Sponsor
- Novartis Pharmaceuticals
Leber's Congenital Amaurosis
Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
The purpose of this study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection in subjects with LCA due to the CEP290 p.Cys998X mutation.
- Trial name
- NCT03140969
- Testing
- QR-110
- Where
- 3 sites in United States, Belgium
- Volunteers
- 11 people
- Sponsor
- Laboratoires Thea
REM Behavior Disorder, Parkinsonism
Efficacy and Tolerability of Ramelteon in Patients With Rapid Eye Movement (REM) Behavior Disorder and Parkinsonism
Parkinson's disease (PD) is the second most common neurodegenerative disorder of the elderly that affects a million patients in US. Sleep dysfunction impacts up to 90% of PD patients.
- Trial name
- NCT00745030
- Testing
- Rozerem
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Northwestern University
Muscular Dystrophy, Duchenne
A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy
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).
- Trial name
- BRAVE (NCT03400852)
- Testing
- MNK-1411
- Where
- 16 sites in United States, Bulgaria and 6 more
- Volunteers
- 44 people
- Sponsor
- Mallinckrodt ARD LLC
Duchenne Muscular Dystrophy
Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to see whether PRO044 is safe and effective to use as medication for DMD patients with a mutation around location 44 in the DNA for the dystrophin protein.
- Trial name
- NCT01037309
- Testing
- PRO044 SC, PRO044 IV
- Where
- 4 sites in Belgium, Italy and 2 more
- Volunteers
- 18 people
- Sponsor
- BioMarin Pharmaceutical