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Results for “Neurodegenerative Disease, Hereditary”

2,233 trials

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Showing 20 of 2,233 results

Large-scale testing (Phase 3)Study completedNCT05156320
What this trial is testing

Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

Who this might be right for
Spinal Muscular AtrophySpinal Muscular Atrophy Type 3Spinal Muscular Atrophy Type 2+7 more
Scholar Rock, Inc. 188
Not applicableLooking for participantsNCT07385443
What this trial is testing

The Spanish National Registry for Myotonic Dystrophy Type 1

Who this might be right for
Myotonic Dystrophy 1DM1Myotonic Dystrophy Type 1+2 more
Fundació Institut Germans Trias i Pujol 3,000
Not applicableStudy completedNCT04805385
What this trial is testing

Effects of Lactobacillus Plantarum PS128 in Patients With Tourette's Syndrome

Who this might be right for
Tourette's Syndrome
China Medical University Hospital 121
Early research (Phase 1)Ended earlyNCT02292225
What this trial is testing

Duvelisib With Obinutuzumab in Patients With CLL/SLL Previously Treated With a BTKi (SYNCHRONY)

Who this might be right for
Lymphocytic Leukemia, ChronicLymphoma, Small Lymphocytic
SecuraBio 3
Not applicableStudy completedNCT02990676
What this trial is testing

Exploring Computerised Cognitive Training for People With Huntington's Disease

Who this might be right for
Huntington's Disease
Cardiff University 30
Testing effectiveness (Phase 2)Looking for participantsNCT05104983
What this trial is testing

Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study

Who this might be right for
Tuberous Sclerosis ComplexEpilepsy
Darcy Krueger 64
Early research (Phase 1)Looking for participantsNCT07246941
What this trial is testing

Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in Huntington's Disease

Who this might be right for
Huntington's Disease
Hoffmann-La Roche 40
Large-scale testing (Phase 3)Study completedNCT01994889
What this trial is testing

Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy

Who this might be right for
Transthyretin (TTR) Amyloid Cardiomyopathy
Pfizer 441
Not applicableStudy completedNCT04141891
What this trial is testing

Advancing Understanding of Transportation Options

Who this might be right for
Diabetic RetinopathyMacular DegenerationGlaucoma+29 more
University of Colorado, Denver 529
Not applicableUnknownNCT05701397
What this trial is testing

Prevalence of Cardiac Amyloidosis in Patients Undergoing Surgery for Lumbar Spinal Stenosis.

Who this might be right for
Cardiac AmyloidosisSpinal Stenosis
Steen Hvitfeldt Poulsen 200
Not applicableUnknownNCT03942341
What this trial is testing

Sleep in Adults With Down Syndrome and Alzheimer's Disease

Who this might be right for
Alzheimer's DiseaseObstructive Sleep ApneaSleep Disturbances
Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau 90
Not applicableUnknownNCT01777451
What this trial is testing

Whole Body Magnetic Resonance Imaging With Diffusion Weighted Imaging : Potential Role in Neurofibromatosis

Who this might be right for
Whole Body ImagingMagnetic Resonance ImagingNeurofibromatosis 1+2 more
Universitaire Ziekenhuizen KU Leuven 30
Not applicableLooking for participantsNCT06539169
What this trial is testing

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Who this might be right for
Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more
xCures 1,000
Not applicableNo Longer AvailableNCT03317366
What this trial is testing

Expanded Access to Provide ARQ 092 for the Treatment of Overgrowth Diseases and/or Vascular Anomalies

Who this might be right for
Proteus SyndromePIK3CA-Related Overgrowth Spectrum (PROS)Growth Disorders
ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
Early research (Phase 1)Ended earlyNCT04800822
What this trial is testing

PF-07284892 in Participants With Advanced Solid Tumors

Who this might be right for
Solid Tumor
Pfizer 53
Testing effectiveness (Phase 2)WithdrawnNCT02112253
What this trial is testing

Optimising Anterior Pallidal Deep Brain Stimulation for Tourette's Syndrome

Who this might be right for
Tourette's Syndrome
The University of Western Australia
Testing effectiveness (Phase 2)Study completedNCT00846430
What this trial is testing

Medical Treatment of "High-Risk" Neurofibromas

Who this might be right for
Neurofibromatosis 1
Corewell Health West 9
Not applicableStudy completedNCT05795400
What this trial is testing

Administration of the SGLT-2 Inhibitor Dapagliflozin in the Patients With Amyloid Cardiomyopathy

Who this might be right for
Heart Failure With Reduced Ejection FractionHeart Failure With Preserved Ejection FractionHeart Failure With Mid Range Ejection Fraction+1 more
National Medical Research Center for Cardiology, Ministry of Health of Russian Federation 50
Testing effectiveness (Phase 2)Study completedNCT01733407
What this trial is testing

L-Serine Supplementation in Hereditary Sensory Neuropathy Type 1

Who this might be right for
Hereditary Sensory and Autonomic Neuropathy Type I
Massachusetts General Hospital 18
Not applicableLooking for participantsNCT06866912
What this trial is testing

Observational Epidemiological Study of Patients Suffering From Systemic Amyloidosis

Who this might be right for
Cardiomyopathy
IRCCS Azienda Ospedaliero-Universitaria di Bologna 750
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