Clinical trials: Multi-System-Atrophy
Fragile X Syndrome, Angelman Syndrome
Parent and Infant Inter(X)Action Intervention (PIXI)
The objective is to develop and test, through an iterative process, an intervention to address and support the development of infants with a confirmed diagnosis of a neurogenetic disorder with associated developmental delays or…
- Conditions
- Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome
- Trial name
- NCT03836300
- Testing
- Parent-Infant Inter(X)action Intervention (PIXI)
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- RTI International
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
Pure Autonomic Failure, Multiple System Atrophy
Autonomic Failure Patients for RNA Blood Sampling
The purpose of this study is to identify 15 patients with autonomic failure and obtain blood samples for RNA from those participants and 15 control subjects within the same age range.
- Conditions
- Pure Autonomic Failure, Multiple System Atrophy, Parkinson's Plus Syndromes
- Trial name
- NCT00742586
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Vanderbilt University
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
Primary Ciliary Dyskinesia (PCD)
Comparison of Individuals With Primary Ciliary Dyskinesia and Healthy Controls
To date, the literature has not yet elucidated the status of malnutrition and sarcopenia in children and adolescents with PCD compared to healthy controls.
- Trial name
- NCT07740551
- Testing
- Physical Evaluations in Children with Primary Ciliary Dyskinesia, Physical Evaluations in Healthy Children
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Izmir Democracy University
Multiple System Atrophy - Cerebellar Subtype (MSA-C), Multiple System Atrophy - Parkinsonian Subtype (MSA-P)
TSPO PET in the Evaluation of Neuroinflammation in Patients with Multiple System Atrophy
The aim of this study was to apply PET/MR techniques targeting TSPO to explore the temporal and spatial alterations characterizing neuroinflammation in MSA patients, and to assess its value as a biomarker for diagnostic typing…
- Trial name
- NCT06890390
- Where
- China
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- Min Zhao,MD
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
Parkinson Disease, Dementia With Lewy Bodies
[18F]-MFBG Versus [123I]-MIBG and [18F]-PE2I in PD vs. MSA and DLB vs. AD
Study goal: The goal of this prospective head to head comparison is to evaluate the effectiveness of [18F]-MFBG PET in assessing cardiac innervation, comparing it with [123I]-MIBG SPECT The study's primary focus is on…
- Conditions
- Parkinson Disease, Dementia With Lewy Bodies, MSA - Multiple System Atrophy, Alzheimer Disease
- Trial name
- NCT06120049
- Testing
- [18F]-MFBG PET CT, [18F]-FE-PE2I PET CT or PET MRI, [123I]-MIBG SPECT CT
- Where
- 2 sites in Belgium
- Volunteers
- 113 people, healthy volunteers welcome
- Sponsor
- prof. dr. Koen Van Laere
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
Physical Disability
Multi-ingredient Nutrition Supplement in Older Adults
The aims of the present study are: i. To conduct a population study of body composition, muscle function and ability to undertake activities of daily living in elderly Irish individuals ii.
- Trial name
- NCT02001831
- Testing
- Supplement
- Where
- Ireland
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University College Dublin
Parkinson Disease, Multiple System Atrophy
Retinal Abnormalities as Biomarker of Disease Progression and Early Diagnosis of Parkinson Disease
To determine whether retinal abnormalities, as measured by high definition optical coherence tomography (HD-OCT) and visual electrophysiology techniques can be used as a clinical biomarker to monitor disease progression overtime…
- Conditions
- Parkinson Disease, Multiple System Atrophy, REM Sleep Behavior Disorder, Pure Autonomic Failure
- Trial name
- NCT02640339
- Where
- United States
- Volunteers
- 166 people, healthy volunteers welcome
- Sponsor
- NYU Langone Health
Autonomic Failure, Pure Autonomic Failure
[18F]F-DOPA Imaging in Patients With Autonomic Failure
Alpha-synucleinopathies refer to age-related neurodegenerative and dementing disorders, characterized by the accumulation of alpha-synuclein in neurons and/or glia.
- Conditions
- Autonomic Failure, Pure Autonomic Failure, Parkinson Disease, Multiple System Atrophy
- Trial name
- NCT04246437
- Testing
- [18F]FDOPA, Carbidopa 200mg oral dose, Entacapone 400mg oral dose
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Daniel Claassen
Parkinson's Disease, Multiple System Atrophy
PET Imaging Evaluation of [11C]SY08
The overall goal of the proposed research is to evaluate the use of [11C]SY08 as a PET radiotracer for aggregated alpha synuclein (αS) in individuals with Parkinson's disease (PD), Multiple system atrophy (MSA), Dementia with…
- Conditions
- Parkinson's Disease, Multiple System Atrophy, Dementia With Lewy Bodies, Healthy Controls
- Trial name
- NCT06098612
- Testing
- C11-SY08
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
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
Parkinson's Disease, Parkinsonism
VisMemoPD Protocol: an Observational Pilot Study on Spatial and Topographical Memory in Patients With Parkinson's Disease and Parkinsonism
VISMEMO-PD is an observational pilot study aimed at investigating visuospatial memory in reaching space and topographical memory in navigational space in patients with Parkinson's disease and Parkinsonism.
- Conditions
- Parkinson's Disease, Parkinsonism, Multiple System Atrophy, Progressive Supranuclear Palsy
- Trial name
- VISMEMO-PD (NCT07827963)
- Testing
- Multimodal visuospatial and topographical memory assessment
- Where
- 3 sites in Italy
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- IRCCS San Raffaele Roma
Type 1 Diabetes, Diabetes Insipidus
Tracking Neurodegeneration in Early Wolfram Syndrome
The goal of this study is to determine the pattern of early neurodegenerative changes in WFS (Wolfram Syndrome). The investigator will perform cross-sectional and longitudinal assessments of youth with WFS, targeting sensitive…
- Conditions
- Type 1 Diabetes, Diabetes Insipidus, Diabetes Mellitus, Wolfram Syndrome
- Trial name
- TRACK (NCT02455414)
- Where
- United States
- Volunteers
- 101 people, healthy volunteers welcome
- Sponsor
- Washington University School of Medicine
Alpha-Synucleinopathy, Parkinson's Disease
Study to Evaluate a Potential PET Radioligand for Imaging Alpha-synuclein Deposits in the Brain
The overall goal of this protocol is to evaluate [18F]MNI-1216 (also known as [18F]ACI-12589) as an α-synuclein targeted radiopharmaceutical in 3 parts as follows: Part 1: first in human (FiH) imaging Optional Part 2: expansion…
- Conditions
- Alpha-Synucleinopathy, Parkinson's Disease, Dementia With Lewy Bodies, Multisystem Atrophy
- Trial name
- NCT06445465
- Testing
- [18F]MNI-1216
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Invicro
Parkinson Disease, Multiple System Atrophy
A Pilot Biomarker Study Assessing Alpha-synuclein Aggregates Across Biofluid Reservoirs in Patients With Synucleinopathies
This will be an observational study looking at clinical and biomarker characteristics in patients with Parkinson's Disease (PD), Multiple System Atrophy (MSA), Rapid Eye Movement Sleep Behavior Disorder (RBD), Normal Pressure…
- Conditions
- Parkinson Disease, Multiple System Atrophy, Rapid Eye Movement Sleep Behavior Disorder, Normal Pressure Hydrocephalus
- Trial name
- NCT04020198
- Testing
- Biomarker assay
- Where
- United States
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Stony Brook University
Duchenne Muscular Dystrophy, Exon 44
Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping
AOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants…
- Trial name
- EXPLORE44 (NCT05670730)
- Testing
- AOC 1044
- Where
- 10 sites in United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Avidity Biosciences, Inc.