Clinical trials: Multi-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
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
Multiple System Atrophy
A Phase 2a Study of Foralumab Nasal in Patients With Multiple System Atrophy (MSA)
A Phase 2a Study of Foralumab Nasal in Patients with Multiple System Atrophy (MSA)
- Trial name
- NCT06868628
- Testing
- Foralumab Nasal
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Tiziana Life Sciences LTD
the Treatment of Multiple System Atrophy Parkinsonian Variant (MSA-P)
The Safety, Tolerability, and Efficacy of Dopaminergic Neural Progenitor Cells (XS411CN Cell Injection) for the Treatment of Multiple System Atrophy Parkinsonian Variant (MSA-P).
A Phase I Study to Assess the Safety, Tolerability and Efficacy of dopaminergic neural progenitor cells (XS411CN cell injection) for the treatment of multiple system atrophy Parkinsonian variant (MSA-P).
- Trial name
- NCT07617012
- Testing
- XS411CN Cell Injection
- Volunteers
- 3 people, healthy volunteers welcome
- Sponsor
- XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd.
Myotonic Dystrophy Type 1 and Type 2
Study to Investigate the Efficacy and Safety of Mexiletine in Patients With Myotonic Dystrophy Type 1 and Type 2
A Randomized, Double-blind, Placebo-controlled, Multi-center Study to Investigate the Efficacy and Safety of Mexiletine During 26 Weeks of Treatment in Patients with Myotonic Dystrophy Type 1 and Type 2 [The MIND Study]
- Trial name
- MIND (NCT04700046)
- Testing
- Mexiletine 167 mg
- Sponsor
- Lupin Ltd.
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.
Duchenne Muscular Dystrophy (DMD)
A Trial to Investigate the Safety and Pharmacokinetics of GRT6019 in Healthy Male Participants
The purpose of this trial is to assess the safety, tolerability, and PK of 3 doses of GRT6019 in healthy male participants. This Phase I trial will be a multiple dose trial in healthy male participants with administration of…
- Trial name
- NCT07347548
- Testing
- GRT6019
- Where
- France
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Grünenthal GmbH
Relapsing Remitting Multiple Sclerosis
Assessment Study of Steroid Effect in Relapsing Multiple Sclerosis Subjects Treated With Glatiramer Acetate
This is a study evaluating the effect on brain volume of daily glatiramer acetate (GA) and add-on pulse steroids.
- Trial name
- ASSERT (NCT00203047)
- Testing
- Glatiramer Acetate, Prednisone
- Volunteers
- 414 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
Muscular Dystrophy, Duchenne
Safety Study of BLS-M22 in Healthy Volunteers
BLS-M22 is being developed as an anti-myostatin agent for the treatment of Duchenne Muscular Dystrophy (Muscular Dystrophy). A total of 37 subjects participated in this study to confirm the safety of BLS-M22.
- Trial name
- NCT03789734
- Testing
- BLS-M22
- Where
- South Korea
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- BioLeaders Corporation
Sarcopenia
Evaluate the Efficacy and Safety of EN001 in Patients With Sarcopenia
A Multi-center, Randomized, Double-blind, Placebo-controlled, Phase 1/2 Clinical Trial to Evaluate the Efficacy and Safety of EN001 in Patients with Sarcopenia
- Trial name
- NCT07808905
- Testing
- EN001
- Where
- South Korea
- Volunteers
- 132 people
- Sponsor
- ENCell
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
NS-050/NCNP-03 in Boys With DMD (Meteor50)
This is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD…
- Trial name
- NCT06053814
- Testing
- NS-050/NCNP-03
- Where
- 19 sites in United States, Canada and 3 more
- Volunteers
- 20 people
- Sponsor
- NS Pharma, Inc.
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.
Facioscapulohumeral Muscular Dystrophy (FSHD)
Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular Dystrophy
The purpose of this study is to assess the safety and tolerability profile of ATYR1940 in the treatment of adult participants with molecularly defined genetic muscular dystrophies
- Trial name
- NCT02239224
- Testing
- ATYR1940
- Where
- 5 sites in United States, France and 2 more
- Volunteers
- 20 people
- Sponsor
- aTyr Pharma, Inc.
Duchenne Muscular Dystrophy
A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy
This is a Phase 2 randomized, 2-period, double-blind, placebo-controlled, multiple ascending dose study to evaluate the safety, efficacy, PK and PD of PF-06252616 administered to ambulatory boys diagnosed with Duchenne Muscular…
- Trial name
- NCT02310763
- Testing
- PF-06252616
- Where
- 66 sites in United States, Australia and 6 more
- Volunteers
- 121 people
- Sponsor
- Pfizer
Cognition, Brain Volume Loss
Effect of Fingolimod on Neurodegeneration
This was a 24-month, open-label, multicenter study with a single treatment arm design. Primary objective of this study was: -To investigate the effects of Fingolimod on cognitive performance in highly active relapsing remitting…
- Trial name
- NCT02575365
- Testing
- 0,5 mg Fingolimod
- Where
- 3 sites in Turkey (Türkiye)
- Volunteers
- 4 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
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
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 Sclerosis
Fingolimod Versus Interferon Beta 1b in Cognitive Symptoms
The purpose of this pilot study is to evaluate, by means of a specific cognitive test battery (Brief Repeatable Battery and Delis-Kaplan Executive Function System scale), the slowing/reduction of cognitive dysfunction progression…
- Trial name
- Cognition (NCT01333501)
- Testing
- Fingolimod, Interferon beta 1b
- Where
- 26 sites in Germany, Italy
- Volunteers
- 151 people
- Sponsor
- Novartis Pharmaceuticals
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