Clinical trials: Multi-System-Atrophy
Multiple System Atrophy (MSA)
Expanded Access Protocol of Verdiperstat in Patients With Multiple System Atrophy (MSA)
The purpose of this expanded access program is to provide access to the investigational drug verdiperstat in patients with Multiple System Atrophy (MSA).
- Trial name
- NCT05086094
- Testing
- BHV3241, verdiperstat
- Where
- United States
- Sponsor
- Biohaven Pharmaceuticals, Inc.
Multiple System Atrophy
GDNF Gene Therapy for Multiple System Atrophy
The objective of this randomized, double-blinded, placebo-controlled Phase 1 investigation is to evaluate the safety and potential clinical effect of AAV2-GDNF delivered to the putamen in subjects with either a possible or…
- Trial name
- NCT04680065
- Testing
- AAV2-GDNF gene therapy
- Where
- 7 sites in United States
- Volunteers
- 9 people
- Sponsor
- Brain Neurotherapy Bio, Inc.
Multiple System Atrophy
Long-term Follow-up of Phase 1 Clinical Trial of CS10BR05(CS10BR05-MSA101)
The study purpose is conducting follow-up surveillance for the incidence of adverse events and efficacy of subjects participated in phase 1 trial to evaluate the safety and tolerability of autologous bone marrow-derived…
- Trial name
- NCT04495582
- Where
- South Korea
- Volunteers
- 8 people
- Sponsor
- Corestemchemon, Inc.
Parkinson Disease, Progressive Supranuclear Palsy(PSP)
Parkinson Atypical Rating of Oculometric Patterns Evaluated Routinely
This is an observational longitudinal study in 4 cohorts of patients with Parkinsonian syndromes, who are visiting the Movement Disorders outpatient clinics.
- Conditions
- Parkinson Disease, Progressive Supranuclear Palsy(PSP), Multiple System Atrophy
- Trial name
- PARATROOPER (NCT06597071)
- Testing
- NeuraLight PD, NeuraLight PSP, NeuraLight MSA
- Where
- Spain
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- NeuraLight
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.
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
Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD
PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
The purpose of this Phase 1/2a trial is to evaluate the safety, tolerability, and preliminary efficacy of PBGENE-DMD in patients with DMD harboring mutations amenable to excision of exons 45-55.
- Trial name
- NCT07429240
- Testing
- PBGENE-DMD (IV)
- Where
- 2 sites in United States
- Volunteers
- 18 people
- Sponsor
- Precision BioSciences, Inc.
Duchenne Muscular Dystrophy
A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to determine whether increasing doses of SMT C1100 are safe, well tolerated and achieve appropriate blood levels in patients with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT02056808
- Testing
- SMT C1100
- Where
- 4 sites in United Kingdom
- Volunteers
- 12 people
- Sponsor
- Summit Therapeutics
Sarcopenia
Study of the Safety and Efficacy of REGN1033 (SAR391786) in Patients With Sarcopenia
This is a randomized, double-blind, placebo-controlled, multicenter phase 2 study of the safety and efficacy of 3-month subcutaneous (SC) REGN1033 (SAR391786) treatment in patients with sarcopenia.
- Trial name
- NCT01963598
- Testing
- REGN1033 (SAR391786)
- Where
- 43 sites in United States, France and 2 more
- Volunteers
- 253 people
- Sponsor
- Regeneron Pharmaceuticals
Duchenne Muscular Dystrophy
Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)
The aim of this study is to provide continuing access to BMN 044 treatment for subjects previously treated with BMN 044. The information gained from this study is expected to further characterize the efficacy and safety of BMN…
- Trial name
- NCT02958202
- Testing
- BMN 044 IV 6 mg/kg, BMN 044 IV 9 mg/kg, BMN 044 SC 6 mg/kg
- Where
- 5 sites in Belgium, Italy and 2 more
- Volunteers
- 7 people
- Sponsor
- BioMarin Pharmaceutical
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
Spinal Muscle Atrophy
A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)
This multicenter, non-interventional, prospective study with a retrospective component aims to evaluate the real-world clinical outcomes, safety profile, and motor function improvements of Risdiplam treatment in participants with…
- Trial name
- NCT07531719
- Where
- 2 sites in Egypt
- Volunteers
- 30 people
- Sponsor
- Hoffmann-La Roche
Duchenne Muscular Dystrophy
First in Human SAD/MAD Safety and PK Study With Adult DMD Safety and PK Cohort
This is a first-in-human (FIH), Phase 1 study of orally administered SAT-3247 in healthy adult volunteers (HVs) and adult participants with DMD to determine safety, tolerability, pharmacokinetics and pharmacodynamics.
- Trial name
- NCT06565208
- Testing
- SAT-3247
- Where
- 2 sites in Australia
- Volunteers
- 77 people, healthy volunteers welcome
- Sponsor
- Satellos Bioscience, Inc.
Parkinson's Disease, Multiple System Atrophy, Parkinson Variant
AB-1005 Long-Term Follow-up Study
This is an observational, long term, follow-up study for participants with Parkinson's disease (PD) or Multiple System Atrophy-Parkinsonian subtype (MSA-P) who participated in prior interventional studies with AB-1005.
- Trial name
- NCT07081841
- Testing
- Observation
- Where
- 2 sites in United States
- Volunteers
- 132 people
- Sponsor
- AskBio Inc
Duchenne Muscular Dystrophy (DMD)
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy…
- Trial name
- DELIVER (NCT05524883)
- Testing
- DYNE-251
- Where
- 30 sites in United States, Australia and 7 more
- Volunteers
- 86 people
- Sponsor
- Dyne Therapeutics
Spinal Muscular Atrophy (SMA)
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
Risdiplam works by helping the body produce more survival motor neuron (SMN) protein throughout the body. This means fewer motor neurons - nerve cells that pass impulses from nerves to muscles to cause movement - are lost, which…
- Trial name
- MANATEE (NCT05115110)
- Testing
- RO7204239, Risdiplam
- Where
- 30 sites in United States, Australia and 10 more
- Volunteers
- 97 people
- Sponsor
- Hoffmann-La Roche
Menopause
The Effects of a Novel Probiotic Supplement on Menopausal Symptoms and Bone Health
This Phase II study is designed to evaluate the potential effects of a novel probiotic supplement on the severity of global menopause symptoms (e.g., psychological, somatic, urogenital symptoms) and on bone health in…
- Trial name
- MPS (NCT04001088)
- Testing
- Probiotic
- Where
- 5 sites in United States
- Volunteers
- 144 people, healthy volunteers welcome
- Sponsor
- Lallemand Health Solutions
Multiple System Atrophy
An Extension Trial to Test if TEV-56286 is Effective in Relieving Multiple System Atrophy
The primary objective of the trial is to describe the long-term safety and tolerability of TEV-56286 administered orally for the treatment of adult participants with Multiple System Atrophy (MSA).
- Trial name
- NCT07197866
- Testing
- TEV-56286
- Where
- 41 sites in United States, France and 5 more
- Volunteers
- 200 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D LLC
Parkinson's Disease (PD), Multiple System Atrophy (MSA)
[18F]ACI-15916 PET in α-synucleinopathies
The goal of this clinical trial is to test whether we can reliably and safely measure the accumulation of pathological protein α-synuclein [involved in some diseases such as Parkinson's disease, Lewy body dementia and Multiple…
- Conditions
- Parkinson's Disease (PD), Multiple System Atrophy (MSA), Dementia With Lewy Bodies (DLB)
- Trial name
- NCT06891703
- Testing
- [18F]ACI-15916
- Where
- Sweden
- Volunteers
- 46 people, healthy volunteers welcome
- Sponsor
- AC Immune SA
Symptomatic Neurogenic Orthostatic Hypotension (NOH), Parkinson's Disease
Fipamezole in Neurogenic Orthostatic Hypotension
The purpose of this study is to determine whether Fipamezole is effective in the treatment of orthostatic hypotension and related symptoms in multiple system atrophy and Parkinson's disease.
- Conditions
- Symptomatic Neurogenic Orthostatic Hypotension (NOH), Parkinson's Disease, Multiple System Atrophy
- Trial name
- Foehn (NCT00758849)
- Testing
- Fipamezole
- Where
- 4 sites in France, Portugal
- Volunteers
- 24 people
- Sponsor
- Juvantia Pharma Ltd