Clinical trials: Multiple System Atrophy
Multiple System Atrophy, MSA - Multiple System Atrophy
Study to Evaluate Investigational Allogeneic Cell Therapy Product hOMSC300 for Treatment of Early to Moderate Stage Multiple System Atrophy (MSA)
Purpose of this phase 1 study is to assess the safety of intrathecal administration of allogeneic human oral mucosa stem cells (hOMSCs) in patients suffering from early to moderate stage Multiple System Atrophy (MSA)
- Trial name
- NCT05698017
- Testing
- hOMSC300
- Where
- Israel
- Volunteers
- 10 people
- Sponsor
- Cytora Ltd.
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.
All Diagnosed Health Conditions, ADD/ADHD
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
We are the missing link in clinical trials, connecting patients and researchers seamlessly and conveniently using a mobile health platform to advance medical research.
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis
- Trial name
- NCT01931644
- Where
- United States
- Volunteers
- 17,667 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
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
Healthy Adult
Evaluation of the Pharmacokinetics, Biodistribution and Radiation Dosimetry of [18F]MODAG-009 Positron Emission Tomography (PET) Radiotracers in Adult Healthy Volunteers
This is an open-label, single-center, phase 1 study to further characterize [18F]MODAG-009 in Healthy Volunteers (HV). Approximately 6 participants will be enrolled in this study.
- Trial name
- NCT07617688
- Testing
- [¹⁸F]MODAG-009 PET Imaging
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- MODAG GmbH
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
Muscular Atrophy
A Safety, Tolerability, Pharmacokinetic and Pharmacodynamic Study of ACE-031 in Healthy Postmenopausal Women
The purpose of this study is to establish safe dose levels of ACE-031 in healthy postmenopausal women following multiple dose administration. This study will also evaluate if ACE-031 has an effect on muscle.
- Trial name
- NCT00952887
- Testing
- ACE-031
- Where
- 2 sites in Canada
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Spinal Muscular Atrophy (SMA), Carrier Screening
Multi-disease Carrier Screening Test Validation
The purpose of this study is to collect blood samples to enable validation of genetic testing for diseases within a multi-disease carrier screening panel.
- Conditions
- Spinal Muscular Atrophy (SMA), Carrier Screening, Genetic Testing
- Trial name
- NCT01663584
- Testing
- Blood draw
- Where
- United States
- Sponsor
- Natera, Inc.
Multiple System Atrophy
A Phase 2 Study of YA-101 in Patients With Multiple System Atrophy
This is a Phase 2, double-blind, placebo-controlled, multi-center, Phase II, dose escalation study to evaluate the safety, tolerability, pharmacokinetics and efficacy of Ya-101 in subjects with multiple system atrophy.
- Trial name
- NCT06848231
- Testing
- YA-101
- Where
- 9 sites in United States, Japan and 1 more
- Volunteers
- 75 people
- Sponsor
- Dasher Neuroscience Inc.
Multiple System Atrophy
A Trial to Test if TEV-56286 is Effective for Treatment of Participants With Multiple System Atrophy
The primary objective of the study is to evaluate the efficacy of TEV-56286 administered orally for the treatment of adult participants with Multiple System Atrophy (MSA).
- Trial name
- TOPAS-MSA (NCT06568237)
- Testing
- TEV-56286
- Where
- 61 sites in United States, France and 6 more
- Volunteers
- 349 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D LLC
Healthy Volunteer, Becker Muscular Dystrophy
A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults
EDG-5506 is an investigational product intended to protect and improve function of dystrophic muscle fibers. This Phase 1 study of EDG-5506 will assess the safety, tolerability, and pharmacokinetics (PK) and of EDG-5506 in adult…
- Trial name
- NCT04585464
- Testing
- EDG-5506
- Where
- United States
- Volunteers
- 127 people, healthy volunteers welcome
- Sponsor
- Edgewise Therapeutics, Inc.
Spinal Muscular Atrophy Type 3
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients
The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 3 (SMA 3) patients.
- Trial name
- NCT06421831
- Testing
- GC101
- Where
- China
- Volunteers
- 21 people
- Sponsor
- GeneCradle Inc
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
Multiple System Atrophy
Open-Label Access to ATH434 for Patients Who Completed Study ATH434-201 in France
This multicenter open-label extension study conducted in France is designed to provide ATH434 orally BID to eligible patients who completed the Phase 2 study ATH434-201, and who may benefit from this treatment according to the…
- Trial name
- NCT07729852
- Testing
- ATH434
- Where
- 4 sites in France
- Volunteers
- 7 people
- Sponsor
- Alterity Therapeutics
Duchenne Muscular Dystrophy
A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
Study to characterize the single-state and steady-state dosing of oral deflazacort in pediatric and adolescents subjects.
- Trial name
- NCT02251600
- Testing
- Deflazacort
- Where
- 4 sites in United States
- Volunteers
- 24 people
- Sponsor
- PTC Therapeutics
Multiple System Atrophy, Neurodegenerative Diseases
Study Assessing Safety and Therapeutic Activity of AFFITOPE® PD01A and PD03A in Patients With Early MSA
This is a randomized controlled parallel Group phase I study to investigate the safety and immunological/ therapeutic activity of two new vaccines, AFFITOPE® PD01A and AFFITOPE® PD03A, given to patients with early Multiple System…
- Trial name
- AFF009 (NCT02270489)
- Testing
- AFFITOPE® PD01A + Adjuvant, AFFITOPE® PD03A + Adjuvant, Adjuvant without active component
- Where
- 2 sites in France
- Volunteers
- 30 people
- Sponsor
- Affiris AG
Multiple System Atrophy (MSA), Parkinson's Disease (PD)
A Study to Evaluate Satety, Tolerability, Biodistribution, Radiation Dosimetry, and Pharmacokinetics of SST001 in Healthy Volunteers, Patients With PD and Patients With MSA
The non-randomized, open-label phase I study aims to evaluate the safety, tolerability, biodistribution, radiation dosimetry, and pharmacokinetics of SST001 in healthy volunteers, patients with MSA and patients with PD.
- Trial name
- NCT07604116
- Testing
- SST001
- Where
- 2 sites in China
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Synusight Biotech (Shanghai) Co., Ltd.
Healthy Volunteer, Muscular Atrophy, Spinal
A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec
In this study, researchers will learn about a study drug called BIIB115 in healthy adult male volunteers and in participants with spinal muscular atrophy (SMA). This study will focus on children with SMA.
- Trial name
- NCT05575011
- Testing
- BIIB115
- Where
- 16 sites in Belgium, Canada and 7 more
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- Biogen
Duchenne Muscular Dystrophy
A Study of TAS-205 for Duchenne Muscular Dystrophy
The objective of this study is to evaluate the safety and pharmacokinetic of TAS-205 in patients with Duchenne Muscular Dystrophy.
- Trial name
- NCT02246478
- Testing
- TAS-205
- Where
- Japan
- Volunteers
- 23 people
- Sponsor
- Taiho Pharmaceutical Co., Ltd.