Clinical trials: Multi-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
Muscular Atrophy, Spinal
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants
Multi-center, randomized, double-blind, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of Risdiplam in adult and pediatric participants with Type 2 and Type 3 SMA.
- Trial name
- SUNFISH (NCT02908685)
- Testing
- Risdiplam
- Where
- 45 sites in United States, Belgium and 14 more
- Volunteers
- 231 people
- Sponsor
- Hoffmann-La Roche
Multiple System Atrophy
Lithium in Multiple System Atrophy
The purpose of this study is to determine safety and tolerability of the treatment with lithium in Multiple System Atrophy. Moreover, clinical symptoms, neuronal loss, quality of life and depressive symptoms, will be considered…
- Trial name
- LAMU (NCT00997672)
- Testing
- Lithium Carbonate
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- Federico II 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 (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.
Multiple System Atrophy
Study of Rifampicin in Multiple System Atrophy
The purpose of this study was to determine whether Rifampicin was effective in slowing or reversing the progression of multiple system atrophy (MSA).
- Trial name
- MSA (NCT01287221)
- Testing
- Rifampicin
- Where
- 10 sites in United States
- Volunteers
- 100 people
- Sponsor
- Phillip Low
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
DM1, Myotonic Dystrophy 1
Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
AOC 1001-CS1 is a randomized, double-blind, placebo-controlled, Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple-doses of AOC 1001 Administered Intravenously to…
- Conditions
- DM1, Myotonic Dystrophy 1, Myotonic Dystrophy, Myotonic Dystrophy Type 1 (DM1)
- Trial name
- MARINA (NCT05027269)
- Testing
- AOC 1001
- Where
- 8 sites in United States
- Volunteers
- 39 people
- Sponsor
- Avidity Biosciences, Inc.
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
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
Hypertension, Pure Autonomic Failure
Nebivolol in the Supine Hypertension of Autonomic Failure
The purpose of this study is to evaluate the effect of the antihypertensive drug, nebivolol (Bystolic), compared to metoprolol (Lopressor) and sildenafil (Viagra) on blood pressure in patients with autonomic failure and supine…
- Conditions
- Hypertension, Pure Autonomic Failure, Multiple System Atrophy
- Trial name
- NCT01044693
- Testing
- Nebivolol 5 mg, metoprolol tartrate 50 mg, Sildenafil25 mg
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Vanderbilt University
Multiple System Atrophy
Progression Rate of MSA Under EGCG Supplementation as Anti-Aggregation-Approach
MSA is a rapidly progressive disorder with an average survival time of about 7 years after the first clinical manifestation. No potent symptomatic treatment is currently available.
- Trial name
- PROMESA (NCT02008721)
- Testing
- EGCG as putative neuroprotective agent
- Where
- 12 sites in Germany
- Volunteers
- 92 people
- Sponsor
- Dr. Johannes Levin
Symptomatic Neurogenic Orthostatic Hypotension
Sustained Effect of Droxidopa in Symptomatic Neurogenic Orthostatic Hypotension
To evaluate the time to treatment intervention in patients with Parkinson's Disease (PD), Multiple System Atrophy (MSA), Pure Autonomic Failure (PAF), Non-Diabetic Autonomic Neuropathy (NDAN) or Dopamine Beta Hydroxylase (DBH)…
- Trial name
- RESTORE (NCT02586623)
- Testing
- Droxidopa capsules
- Where
- 168 sites in United States
- Volunteers
- 453 people
- Sponsor
- H. Lundbeck A/S
Symptomatic Neurogenic Orthostatic Hypotension, Parkinson's Disease
A Clinical Study of Patients With Symptomatic NOH to Assess Sustained Effects of Droxidopa Therapy
Evaluate the clinical efficacy and safety of droxidopa versus placebo over a 17 week (maximum) treatment period in patients with symptomatic NOH.
- Conditions
- Symptomatic Neurogenic Orthostatic Hypotension, Parkinson's Disease, Multiple Systems Atrophy, Pure Autonomic Failure
- Trial name
- NCT01927055
- Testing
- Droxidopa
- Where
- 3 sites in United States
- Volunteers
- 61 people
- Sponsor
- Chelsea Therapeutics
Hypertension, Pure Autonomic Failure
Local Heat Stress in Autonomic Failure Patients With Supine Hypertension
Patients with autonomic failure are characterized by disabling orthostatic hypotension (low blood pressure on standing), and at least half of them also have high blood pressure while lying down (supine hypertension).
- Conditions
- Hypertension, Pure Autonomic Failure, Multiple System Atrophy, Autonomic Failure
- Trial name
- NCT02417415
- Testing
- Passive heat stress, Control (non-heating)
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Vanderbilt University
Multiple System Atrophy
A Futility Trial of Sirolimus in Multiple System Atrophy
Single-center, randomized, placebo-controlled, phase-II, futility clinical trial to determine if oral sirolimus is of sufficient promise to slow disease progression in MSA, prior to embarking on a large-scale and costly phase III…
- Trial name
- NCT03589976
- Testing
- Sirolimus 2 MG
- Where
- United States
- Volunteers
- 47 people
- Sponsor
- NYU Langone Health
Symptomatic Neurogenic Orthostatic Hypotension (NOH), Non-diabetic Neuropathy
A Clinical Study for Patients With Neurogenic Orthostatic Hypotension (NOH) Using Droxidopa
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
- NOH301 (NCT00782340)
- Testing
- Droxidopa
- Where
- 21 sites in United States, Canada
- Volunteers
- 263 people
- Sponsor
- Chelsea Therapeutics
Duchenne Muscular Dystrophy
Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients
This study is designed to assess the efficacy, safety, tolerability, and pharmacokinetics (PK) of AVI-4658 (eteplirsen) in both 50.0 mg/kg and 30.0 mg/kg doses administered over 24 weeks in subjects diagnosed with Duchenne…
- Trial name
- NCT01396239
- Testing
- AVI-4658 (Eteplirsen)
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Sarepta Therapeutics, Inc.
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.
Symptomatic Neurogenic Orthostatic Hypotension, MSA
Phase 3 Clinical Effect Durability of TD-9855 for Treating Symptomatic nOH in Subjects With Primary Autonomic Failure
A Phase 3, 22-week, Multi-center, Randomized Withdrawal Study of ampreloxetine in Treating Symptomatic Neurogenic Orthostatic Hypotension in Subjects with Primary Autonomic Failure
- Conditions
- Symptomatic Neurogenic Orthostatic Hypotension, MSA, Parkinson's Disease (PD), Pure Autonomic Failure (PAF)
- Trial name
- REDWOOD (NCT03829657)
- Testing
- ampreloxetine
- Where
- 82 sites in United States, Australia and 17 more
- Volunteers
- 203 people
- Sponsor
- Theravance Biopharma