Clinical trials: Multi-System-Atrophy
Oculopharyngeal Muscular Dystrophy
Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients
The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.
- Trial name
- HOPEMD (NCT02015481)
- Testing
- Cabaletta
- Where
- 4 sites in United States, Canada and 1 more
- Volunteers
- 25 people
- Sponsor
- Bioblast Pharma Ltd.
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
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
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.
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
Parkinsonian Syndrome, Parkinson Disease(PD)
A Study to Evaluate the Diagnostic Efficacy of DaTSCAN™ Ioflupane (123I) Injection in Single Photon Emission Computed Tomography (SPECT) for the Diagnosis of Parkinsonian Syndrome (PS) in Chinese Patients
This is a multicenter, open-label, non-controlled, non-randomized, phase 3 clinical study to compare the SPECT findings after a single IV administration of DaTSCAN™ ioflupane (123I) injection for patients with a clinical…
- Conditions
- Parkinsonian Syndrome, Parkinson Disease(PD), Multiple System Atrophy (MSA), Progressive Supranuclear Palsy (PSP)
- Trial name
- NCT04193527
- Testing
- DaTSCAN™ Ioflupane (123I) Injection
- Where
- 10 sites in China
- Volunteers
- 172 people, healthy volunteers welcome
- Sponsor
- GE Healthcare
Duchenne Muscular Dystrophy
Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
The purpose of this study is to determine if ACE-031 is safe and well-tolerated in boys with Duchenne Muscular Dystrophy (DMD) and to select the optimal doses of ACE-031 in terms of safety and pharmacodynamic (PD) activity for…
- Trial name
- NCT01099761
- Testing
- ACE-031 0.5 mg/kg q4wk, ACE-031 1.0 mg/kg q2wk
- Where
- 5 sites in Canada
- Volunteers
- 24 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
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.
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
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
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
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
A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants
This will be a Phase I, 2-part, open-label, non-randomized study to investigate the safety, tolerability, and pharmacokinetics (PK) of a multiple-dosing regimen of risdiplam (Part 1) and the effect of risdiplam on the PK of…
- Trial name
- NCT03988907
- Testing
- Risdiplam, Midazolam
- Where
- United States
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Hoffmann-La Roche
Neovascular Age-related Macular Degeneration (nAMD)
Study to Evaluate the Safety and Effectiveness of Intravitreal Injections (IVI) of Brolucizumab in Patients With Neovascular Age-related Macular Degeneration (nAMD)
The purpose of this study was to generate additional safety and effectiveness data in Indian neovascular age-related macular degeneration (nAMD) patients that more closely resemble the real-world population intended to be treated…
- Trial name
- NCT05269966
- Testing
- Injection Brolucizumab
- Where
- 10 sites in India
- Volunteers
- 105 people
- Sponsor
- Novartis Pharmaceuticals
Duchenne Muscular Dystrophy
A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to determine whether a new medication called vamorolone is safe and well-tolerated by boys with Duchenne muscular dystrophy (DMD) ages ≥ 4 and < 7 years old.
- Trial name
- NCT02760264
- Testing
- Vamorolone 0.25 mg/kg/day, Vamorolone 0.75 mg/kg/day, Vamorolone 2.0 mg/kg/day
- Where
- 12 sites in United States, Australia and 4 more
- Volunteers
- 48 people
- Sponsor
- ReveraGen BioPharma, Inc.
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
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
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
Chronic Obstructive Pulmonary Disease (COPD) With Cachexia
BYM338 in Chronic Obstructive Pulmonary Disease (COPD) Patients With Cachexia
This study will assess the pharmacodynamics, pharmacokinetics, safety and tolerability of BYM338 in patients with COPD and cachexia. The primary outcome will be a change in thigh muscle volume compared to placebo.
- Trial name
- NCT01669174
- Testing
- BYM338
- Where
- 9 sites in United States, Netherlands and 1 more
- Volunteers
- 67 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
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