Clinical trials: Multiple System Atrophy
Neurogenic Orthostatic Hypotension, Multiple System Atrophy (MSA) With Orthostatic Hypotension
TD-9855 Phase 2 in Neurogenic Orthostatic Hypotension (nOH)
This multiple-center, 3-part, single-blind dose escalation (Part A), randomized, double-blind (Part B), and open-label multiple dose extension (Part C) study will be conducted in male and female subjects with neurogenic…
- Conditions
- Neurogenic Orthostatic Hypotension, Multiple System Atrophy (MSA) With Orthostatic Hypotension, Pure Autonomic Failure, Parkinson Disease
- Trial name
- NCT02705755
- Testing
- TD-9855
- Where
- 6 sites in United States
- Volunteers
- 34 people
- Sponsor
- Theravance Biopharma
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
Parkinson Disease, Multiple System Atrophy
Augmenting Effects of L-DOPS With Carbidopa and Entacapone
An experimental drug called L-DOPS increases production in the body of a messenger chemical called norepinephrine. Cells in the brain that make norepinephrine are often gone in Parkinson disease.
- Conditions
- Parkinson Disease, Multiple System Atrophy, Autonomic Nervous System Diseases
- Trial name
- NCT00547911
- Testing
- Droxidopa, Carbidopa, Entacapone
- Where
- United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
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
Parkinson Disease, Multiple System Atrophy
Treatment of Parkinson Disease and Multiple System Atrophy Using Intranasal Insulin.
Parkinson disease (PD) and multiple system atrophy (MSA) are progressive neurodegenerative disorders characterized by abnormal accumulation of α-synuclein.
- Trial name
- NCT02064166
- Testing
- Intranasal Insulin
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Peter Novak
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
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
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
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
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
Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2
To evaluate the safety and efficacy of intravenous onasemnogene abeparvovec-xioi in pre-symptomatic patients with SMA and 2 or 3 copies SMN2
- Trial name
- SPR1NT (NCT03505099)
- Testing
- onasemnogene abeparvovec-xioi
- Where
- 16 sites in United States, Australia and 4 more
- Volunteers
- 30 people
- Sponsor
- Novartis Gene Therapies
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
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
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
Spinal Muscular Atrophy
A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
The primary objective of the study is to examine the efficacy of multiple doses of Nusinersen administered intrathecally in preventing or delaying the need for respiratory intervention or death in infants with genetically…
- Trial name
- NURTURE (NCT02386553)
- Testing
- Nusinersen
- Where
- 21 sites in United States, Australia and 5 more
- Volunteers
- 25 people
- Sponsor
- Biogen
Multiple System Atrophy
Safinamide for Multiple System Atrophy (MSA)
The study is a placebo controlled study, with two parallel arms, in which participants will be randomly assigned in a 2:1 ratio to receive either active (200 mg safinamide) or placebo in a double blind manner.
- Trial name
- NCT03753763
- Testing
- Safinamide Methanesulfonate
- Where
- 19 sites in Italy, Spain
- Volunteers
- 49 people
- Sponsor
- Zambon SpA
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.
Multiple System Atrophy
Clinical Trial to Assess Efficacy, Safety, and Tolerability of Rasagiline Mesylate 1 mg in Patients With Multiple System Atrophy of the Parkinsonian Subtype (MSA-P)
To test the clinical effect of rasagiline on subjects with MSA of the parkinsonian subtype.
- Trial name
- NCT00977665
- Testing
- rasagiline mesylate
- Where
- 47 sites in United States, Austria and 10 more
- Volunteers
- 174 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
Diabetic Macular Edema
Multiple Dose Safety and Efficacy of LKA651 in Patients With Diabetic Macular Edema
The primary objectives of this study were to evaluate the safety and efficacy of LKA651 in patients with macular edema from diabetic macular edema (DME),
- Trial name
- NCT03927690
- Testing
- LKA651, Lucentis
- Where
- 22 sites in United States, Germany and 3 more
- Volunteers
- 91 people
- Sponsor
- Novartis Pharmaceuticals
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